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Consensus Document of the New Jersey Working Group to Improve Outcomes in Cancer Patients

Background

New Jersey suffers with one of the nation’s highest incidence rates and death rates from cancer, ranking near the top of the list for all of the major malignancies. In 1998, it is estimated that 40,500 New Jerseyans will learn they have cancer and 18,600 will die from this disease. Improvement in the outcomes for cancer patients is possible only through the administration of carefully planned, scientifically valid, clinical trials. Cancer clinical trials are designed to evaluate the efficacy and safety of a new therapy for treatment, palliation, or prevention of cancer. Thus, they are crucial to improve outcomes. However, only about three percent of patients with cancer throughout the nation and less than two percent of cancer patients in New Jersey enrolled onto clinical trials.

Cancer clinical trials provide the tools we need for tomorrow’s better care and offer patients today a chance at life-saving new therapies. Research and development is the lifeblood of the American healthcare industry, especially in oncology. Patient care has improved because of clinical research, but despite our best available treatments, approximately half of all adults and 20% of children diagnosed with cancer will die from this disease. This is an unacceptable number of deaths. The mechanism by which detection, treatment, and prevention of cancer is improved is through well-designed, scientifically valid, clinical trials; and, indeed, improved survival, better quality of life, and lower costs can only be achieved through clinical research. Without clinical trials, we cannot improve upon the therapeutic options we offer patients. The parties to this document recognize that health plan members currently participate in clinical trials, many times on a haphazard basis. Additionally, gaining access to clinical trials, due to the lack of a systematic process, is time consuming and inefficient. In order to facilitate timely and easy access to clinical trials, this consensus document proposes to establish a methodology for participation in clinical trials.

When new techniques or therapies are introduced, clinical trials are essential to provide valid and convincing evidence that such techniques are beneficial. Adoption of therapies which do not improve outcomes is unfair to patients and costly to society. For example, stem cell transplant is frequently used in the treatment of breast cancer, although it has not been proven to be efficacious. The costly use of this unproven procedure became standardized when a third-party payor denied approval of payment for stem cell transplant within the context of a clinical trial. This resulted in a law suit and a settlement for the claimant, with the interpretation that the procedure should be covered, regardless of whether the patient is enrolled onto a valid scientific trial. Thus, while still unproven, this procedure consumes tremendous healthcare resources.

Another example of the value of clinical trials involves lung cancer therapy, which is the leading cancer killer of both men and women. When lung cancer recurs following surgery and/or radiation therapy, it is an entirely fatal disease. Standard chemotherapy is noncurative, and only an optimistic reading of the literature suggests that chemotherapy for lung cancer meaningfully prolongs life or palliates symptoms. Yet, treatment with “standard chemotherapy” is paid for by almost every insurer. In contrast, if a new treatment becomes available which shows promising results in pre-clinical trials and has shown to be safe in early clinical trials, insurers frequently will not pay for the care associated with receiving this new promising treatment because it is only available through a clinical trial. This is true even though the costs of the new treatment may be similar or even less than standard therapy. Therefore, payors are commonly reimbursing for therapies that do not work, instead of paying for new therapies that may or may not work, but which have potential.

An example of a cancer clinical trial improving the cost/benefit ratio of therapy is a trial conducted in patients with metastatic testicular cancer which compared three versus four cycles of chemotherapy administered with curative intent. It was determined by analysis of the results of the trial that for patients meeting the definition of minimal or moderate disease, three cycles of systemic chemotherapy resulted in the same cure rate as did four cycles. Three cycles of therapy is associated with a significant decrease in both short- and long-term toxicities, and thus an improvement in the clinical outcome. Simultaneously, administration of three cycles rather than four cycles results in both a decrease in the cost of therapy and a decrease in the cost of added care due to toxicities associated with the additional cycle of therapy.

Clinical trials are the lifeblood of modern cancer treatments. Only through clinical trials will we be able to find more cost-effective and clinically effective treatments which will ultimately lower premiums. Increasing access to clinical trials holds the potential to improve patient outcomes and foster development of cost-effective treatments. Clinical research has found ways to significantly reduce the costs of treatments for ovarian cancer and non-Hodgkin’s lymphoma. New prevention technologies may have even greater benefits, with low-cost interventions preventing high-risk patients from ever needing treatment. At the same time, it is understood that palliative care is an option that should be discussed with each patient.

Those New Jerseyans who are involved in healthcare either as insurers, providers, or patients have a compelling interest in facilitating improvement in the methods of cancer detection, treatment, and prevention and recognize that clinical trials are necessary for continued improvement in the outcomes of cancer patients. Thus we wish to reach consensus regarding reimbursement of cancer clinical trials in order to improve outcome in cancer patients.

The cost of clinical trials should not be borne solely by the insurers, nor should it be borne solely by providers. Rather, a fair and equitable mechanism must be established by which the pharmaceutical companies, the insurers, and the providers join together in cooperation for the benefit of cancer patients.

The opportunity to assemble a synergistic model in New Jersey consisting of the medical profession and institutions, the pharmaceutical companies, and the insurers aimed at providing continuing quality improvement in the approach to cancer is a realistic goal. To this end, the New Jersey Working Group to Improve Outcomes in Cancer Patients shall work to form consensus regarding the process by which New Jersey can become a leader in improving outcomes in cancer patients.

Purpose

The intent and purpose of the New Jersey Working Group to Improve Outcomes in Cancer Patients is to reach consensus between insurers, healthcare providers, and patients regarding reimbursement of routine patient-care expenses of approved cancer clinical trials by insurers and other healthcare payors to promote the health and welfare of the people of New Jersey. Sound healthcare practices, such as approved cancer clinical trials which meet the requirements of this document, should be available to all of the residents of this state, regardless of the practices and the discretion of insurers and healthcare providers. The New Jersey Working Group to Improve Outcomes in Cancer Patients, through this consensus document, seeks to curtail the cost of medical treatments that are of unproven benefit, while seeking to pursue treatments or interventions which experts believe have a reasonable expectation of being effective. Cancer clinical trials which meet the requirements of this document are those which have been reviewed at a national level for scientific validity and thereby meet the definition of having a reasonable expectation of effectiveness, regardless of the phase of the study.

Overall Goal

To provide standards for insurance reimbursement of routine patient-care costs when an insured enrollee or subscriber participates in approved cancer clinical trials.

Specific Aims

1. To reach consensus regarding the definition of approved clinical trials.
2. To reach consensus regarding which costs should be fairly reimbursed by payors and which should be borne elsewhere.
3. To establish a methodology for timely and easy access to clinical trials.
4. To establish a mechanism for ongoing review and discussion of issues affecting access to clinical trials.

   Formation of the New Jersey Working Group to Improve Outcomes in Cancer Patients: The New Jersey Working Group to Improve Outcomes in Cancer Patients (hereafter referred to as the Working Group) shall be formed and shall have fair and equitable representation among all stakeholders, including the medical/scientific community, indemnity and managed care providers, the pharmaceutical/biotechnology industry, advocacy/patient groups, and appropriate government entities.

   Two subcommittees shall be organized. A scientific/technical subcommittee shall be created whose voting members hold appropriate scientific, medical, or ethical expertise. The purpose of this subcommittee shall be to review clinical trials not fitting the current working definitions of “Approved Cancer Clinical Trials,” consider evidence for the scientific validity, and make recommendations about such trials. A policy oversight subcommittee shall be created with appropriate stakeholder representatives to review and implement recommendations regarding reimbursement issues. The purpose of this subcommittee shall be to serve as the administrative arm of the Working Group, and shall review competing interests regarding reimbursement issues and assist in developing reporting procedures.

5. The Scientific/Technical Subcommittee (STSC):

Membership shall include

1. A physician licensed to practice medicine and surgery in New Jersey who specializes in oncology and is a member of a community oncology practice and who is not on the staff of a comprehensive or clinical cancer center designated by the National Cancer Institute (NCI).
2. A physician or scientist who specializes in oncology and is on the staff of a New Jersey NCI-designated comprehensive or clinical cancer center.
3. A physician licensed to practice medicine and surgery in New Jersey who specializes in oncology and who is a principal investigator on a clinical research program designated by the NCI, such as a Community Clinical Oncology Program.
4. A medical ethicist recognized for his or her expertise in evaluating ethical implications of healthcare practices and procedures.
5. Two physicians licensed to practice medicine and surgery in New Jersey who are appointed by a health maintenance organization (HMO).
6. A physician licensed to practice medicine and surgery in New Jersey who is appointed by a nonprofit health plan.
7. A physician licensed to practice medicine and surgery in New Jersey who is appointed by an indemnity insurance carrier.
8. A physician or scientist who represents a pharmaceutical or biotechnology company located in New Jersey.
9. A cancer patient advocate who participates regularly in advocacy activities and in a nationally recognized cancer organization.

Charges shall include

1. The STSC shall meet as needed at the call of the chair or three other members.
2. The STSC shall elect from its membership and by majority vote, a chairperson to oversee the STSC. The chairperson shall be a physician.
3. The STSC shall limit its discussion to technical matters of science and medicine only.
4. The STSC shall review and assess any trial not in strict conformance with the working definition of an approved cancer clinical trial, as defined in this document.
5. The STSC shall advise the Policy and Oversight Subcommittee (POSC) upon request.
6. The STSC shall make recommendations for a methodology for identifying the routine medical costs associated with participation in approved cancer clinical trials.

The Policy Oversight Subcommittee

1. Membership shall include
   • A representative from a pharmaceutical or biotechnology company located in New Jersey
   • A representative from an indemnity insurance company located in New Jersey
   • A representative from an NCI-approved cancer center located in New Jersey
   • An individual representing a community hospital appointed by the New Jersey Hospital Association
   • A representative from the New Jersey Association of Health Plans
   • A cancer patient advocate who participates regularly in advocacy activities and in a nationally recognized advocacy group
   • A representative from the New Jersey Commission on Cancer Research, within the New Jersey Department of Health and Senior Services
   • A representative from the American Cancer Society, Eastern Division
   • A representative from the Oncology Society of New Jersey
   • The chairperson of the STSC
2. The charges of the POSC shall be:
   • The POSC shall meet as needed at the call of the Chair or three other members
   • The POSC shall elect a chairperson from its membership by a majority vote to oversee the POSC
   • The POSC shall examine policy issues which arise from the implementation of this document, including but not limited to: eligibility reimbursement issues arising from participating (PAR) and nonparticipating (non-PAR) provider services rendered to patients during the course of an approved cancer clinical trial
   • The POSC shall, upon receipt of a recommendation from the STSC, evaluate said recommendation and develop procedures for its implementation should it be adopted

Officers & Meetings

1. The two subcommittee chairs shall serve as Co-chairs of the New Jersey Working Group to Improve Outcomes in Cancer Patients (hereafter referred to as the Working Group). The chair of each subcommittee is responsible for communicating to the chair of the other subcommittee.
2. Joint meetings of the subcommittees shall meet on an as-needed basis at the call of either chair or at the call of three other members of the Working Group.
3. The Working Group shall meet as needed at the call of the chair of either subcommittee but no less than annually.

Working Definitions

1. Approved Cancer Clinical Trial: A scientific study of a new therapy or intervention for the treatment, palliation, or prevention of cancer in human beings that meets the following requirements:
   • The treatment or intervention is provided pursuant to an approved cancer clinical trial that has been authorized or approved by one of the following:
     • The National Institutes of Health. (phase I, II, and III)
     • The United States Food and Drug Administration, in the form of an investigational new drug (IND) exemption. (phase I, II, and III)
     • The United States Department of Defense
     • The United States Department of Veterans Affairs
   • The proposed therapy has been reviewed and approved by the applicable qualified Institutional Review Board
   • The available clinical or preclinical data indicate that the treatment or intervention provide pursuant to the approved cancer clinical trial will be at least as effective as standard therapy, if such therapy exists, and is anticipated to constitute an improvement in effectiveness for treatment, prevention, or palliation of cancer
   • The facility and personnel providing the treatment are capable of doing so by virtue of their experience and training
   • The trial consists of a scientific plan of treatment that includes specified goals, a rationale and background for the plan, criteria for patient selection, specific directions for administering therapy and monitoring patients, a definition of quantitative measures for determining treatment response, and methods for documenting and treating adverse reactions. All such trials must have undergone a review for scientific content and validity, as evidenced by approval by one of the federal entities identified in items la-ld. A cost/benefit analysis of clinical trials will be performed when such an evaluation can be included with a reasonable expectation of sound assessment
2. Routine Patient Care Costs
   • Physician fees, laboratory expenses and expenses associated with the hospitalization, administering of treatment and evaluation of the patient during the course of treatment or a condition associated with a complication of the underlying disease or treatment, which are consistent with usual and customary patterns and standards of care incurred whenever a patient receives medical care associated with an approved cancer clinical trial, and which would be covered if such items and services were provided other than in connection with an approved cancer clinical trial. Such costs shall not include the costs of the investigational drugs or devices themselves, the costs of any non-health services that might be required for a person to receive the treatment or intervention, or the costs of managing the research, or costs which would not be covered under the policy for non-investigational treatments

New Jersey Association of Health Plans official signatures for the revised final clinical trial consensus document (includes phases I and II) November 12, 1999

Parties to the Consensus Document

In recognition of the importance of improving the outcomes of cancer patients and in an effort to establish a voluntary process to further participation in clinical trials, the organizations signing below agree to abide by the provisions of this consensus document, to make a good faith effort to facilitate participation of their members in approved cancer clinical trials, to use the “Summary Report and Recommendations on Health Insurance Coverage for Cancer Patients in New Jersey” and the appendix attached hereto in guiding coverage decisions regarding cancer clinical trials, and to support the effort of the New Jersey Working Group to Improve Outcomes of Cancer Patients.

Applicability to Medicaid Contracts

As a plan offering coverage to members who are solely Medicaid beneficiaries, we support the efforts of the New Jersey Working Group to Improve Outcomes of Cancer Patients. We are a party to this consensus document because we believe in the principles and goals of the group. We sign the agreement with the stipulation that any coverage which we authorize in accordance with the group’s activity is subject to the approval of the Division of Medical Assistance and Health, New Jersey Department of Human Services, pursuant to its contract for coverage of its beneficiaries.

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Report and Recommendations on Inclusion of Phase I Clinical Trials

Introduction

In February 1999, Governor Christine Todd Whitman and Assemblywomen Rose Heck (R., D. 38) and Charlotte Vandervalk, (R., D 39) announced the formation of the New Jersey Working Group to Improve Clinical Outcomes in Cancer Patients. A voluntary, statewide coalition of health insurers, healthcare providers, pharmaceutical companies, cancer researchers, ethicists, and patient consumers, this Working Group is charged with the development of mechanisms for insured patients to receive routine costs of care when enrolled in approved cancer clinical trials. As a first step in this process, the Scientific and Technical Committee of the Working Group was created to study the inclusion of health insurance coverage for the costs of routine patient care surrounding phase I and phase II clinical cancer trials in the program. This report highlights the critical concerns and issues raised within this effort. It includes basic information about clinical trials, definitions of the costs associated with clinical trials, and a discussion of the advantages and disadvantages of clinical trials for patients with cancer compared to the care delivered outside of these clinical trials. To complete our work, we considered pertinent ethical questions that the committee raised, and carefully thought about clinical trials in the context of the fiduciary responsibility of the health plan. These include the reasons why insurers should consider reimbursement for routine patient care surrounding phase I and II trials; what costs and benefits are involved; how fiduciary responsibilities might be handled; how ethical questions may be resolved; what predictability exists and what misperceptions and misunderstandings need to be changed. After consideration of the science, the advantages and disadvantages of clinical trials for members and insurers, the Group concluded that health insurers should be willing to provide coverage for the routine patient care surrounding both phase I and phase II trials.

Cancer Clinical Trials

Within the past 20 years, major clinical advances in cancer treatment have emerged as the result of thorough, carefully designed clinical trials. Clinical cancer trials are organized studies conducted to evaluate new, promising treatments for cancer patients. After careful laboratory testing for safety and effectiveness, new therapies are evaluated in people during three phases. Phase I trials determine toxicities through a continuum of modest dosing to determine safe levels in humans (See Appendix 2, ASCO Statement). Phase II trials begin to evaluate the effectiveness of the treatment. Phase III trials compare the new regimen to standard care to evaluate relative efficacy and therapeutic value. It is important to recognize that clinical research is a process that involves incremental steps that are scientifically based, and as such, may offer potential benefits at any point along the continuum. Therefore, cancer clinical trials offer patients the opportunity to obtain new treatments and may be the only therapeutic option available for seriously ill cancer patients. Funding for clinical trials traditionally comes from a variety of sources, including pharmaceutical companies, research institutions, government agencies, and health insurance companies.

According to the New Jersey Commission on Cancer Research (NJCCR), only 4.5% of cancer patients in New Jersey are currently enrolled in clinical trials. Nationally the figure is between 3% and 5%. The American Cancer Society estimates that 42,800 people from New Jersey will be diagnosed with cancer this year. New Jersey ranks in the top ten among all states for overall cancer deaths.

The NJCCR estimates that 41 hospitals in New Jersey offer some form of phase III trials to cancer patients. In addition, 80% of these same hospitals offer phase II trials, but less than 20% offer phase I trials. Sixty-three percent are involved in at least one drug or pharmaceutical trial. The most common affiliations with national cooperative groups are National Surgical Adjuvant Breast and Bowel Project (NSABP), Eastern Cooperative Oncology Group (ECOG), Radiation Therapy Oncology Group (RTOG), Cancer and Leukemia Group B (CALGB), Gynecologic Oncology Group (GOG), and Southwest Oncology Group (SWOG). There are ten pediatric cancer centers in New Jersey with affiliations with the Pediatric Oncology Group (POG) or the Children’s Cancer Group (CCG). (NJCCR, 1999, based upon survey responses of all NJ acute care hospitals.)

This report provides an overview of the critical issues and concerns that require responses if agreement among the health insurers, research and medical community, the pharmaceutical/biotechnology industry, and patient consumers on health insurance coverage for the routine patient care surrounding phase I and II trials is to be resolved.

Essential Insights

1. No therapy comes to a phase I clinical trial for cancer without proven efficacy and safety in small and large animal models. This scientific basis is not a guarantee for efficacy in humans, but is equally not a shot in the dark to discover a new treatment.
2. Eligible patients who do not enroll in clinical trials for cancer still receive “standard” therapy, often with no benefit, ie, refractoriness.
3. Patients considering enrollment in a phase I or II clinical trial for cancer must be provided with information in language commensurate with their educational level that is culturally and language sensitive to enable them to make a decision as prudent laypersons (Appendix 3).
4. Clinical trials eligible for approval must address important scientific questions, ultimately have the potential to benefit standard care, and must undergo external scientific review and an independent Investigative Review Board (IRB) review. This control defines dosing ranges, side-effect reporting and, continuation/stopping points prior to trial initiation.
5. The rigorous control and oversight in cancer clinical trials include a well-defined independent safety and monitoring board that reviews interim data regularly as well as at least two levels of protocol adherence monitoring on a predefined regular schedule regardless of the source of sponsorship. These requisites in place for cancer clinical trials are in sharp contrast to the descriptions in recent lay publications about lax monitoring of non-cancer-related trials.
6. Single-institution sponsored, directed, and managed clinical trials for cancer that are not approved by NCI, DOD, FDA or the VA systems are precluded from consideration under this Working Group’s agreement (see original working agreement document.)
7. The realities of a benefit for most cancer clinical trials are a 20% to 25% response rate, ie, phase I moves on to phase II with toxicity related to the study drug in approximately 5% or less.
8. The realities of current care are that patients with cancer who are refractory to standard therapy and who would be eligible for enrollment in phase I and phase II clinical trials now receive multiple rounds of third, fourth, fifth and more line therapy.
9. This care is often delivered in an uncontrolled and unaudited environment and often results in more toxicity and morbidity for the patient because monitoring controls are not in place. This care is considered “routine,” but often has little scientific basis, and yet, is supported by insurers.

Why should health insurance consider reimbursement for routine patient care surrounding phase I and II studies?

Many of the major achievements in cancer treatment in the past twenty years were accomplished through controlled clinical trials. Some examples are management of acute leukemia, increased cure of testicular cancer, use of adjuvant chemotherapy in breast cancer, increased cure of certain pediatric solid tumors, and the use of combination modalities for GI and lung cancers. It is logical that if more patients participated, clinical trials would be completed in a more timely fashion, and critical scientific and treatment questions would be answered. This would result in improved care for all patients.

Health insurers want evidence-based medicine that reduces variation, eliminates unnecessary treatments, and reduces waste. Patients want to have high quality and affordable healthcare. Well-designed clinical trials meet all of these needs.

Clinical trials in all phases have more accountability, control, and monitoring than most “standard care,” including the reporting and analysis of adverse events. The criteria and standards to which these activities are held exceed any existing community or practical guidelines. Clinical trials must adhere to specific guidelines and procedures which establish in advance all the parameters of the trial, including specific objectives, eligibility, endpoints, details of treatment, and removal from the study. This structure ultimately provides more predictability of costs.

Clinical trials are designed from sound scientific hypotheses. They are based on animal studies that provide tested information on safety and efficacy. They are not random, unfounded attempts at trying to find something that may work.

Clinical trials must be reviewed by a qualified investigative review board, which assures safety and protection against any undue risks to the patient. Clinical trials are also reviewed by institutional scientific committees, which review the scientific validity of assumptions. In addition to these local committees, trials of investigational agents must meet the regulatory guidelines established by the FDA, further ensuring the protection of patients involved.

Clinical trials are carefully monitored during administration by specifically trained personnel. For both the National Cancer Institute (NCI) and the pharmaceutical industry, clinical investigative sites are not approved unless the staff is fully trained and qualified to administer such trials.

During a trial, the NCI, the cooperative groups, the cancer centers, and industry demand onsite reviews of the conduct of the trials by independent reviewers. These reviewers compare research records with documents, which validate interactions, and review adherence to protocol requirements and reporting all adverse events. Investigators that deviate from defined guidelines are subject to sanctions. These inherent controls foster a high level of investigator and sponsor accountability, control and monitoring of the trial.

What are the costs associated with clinical trials?

There are three components of costs involved in the conduct of Phase I and Phase II trials.

1. The administrative costs of the study, which include data gathering, statistical study, meetings and travel. These costs are borne by the sponsoring organizations. Results of a survey by the American Society of Clinical Oncology (ASCO) of 9000 oncologists indicated that reimbursement rates by NCI were less than 25% of actual research costs. At the same time, 80% of oncologists have been involved in performing clinical trials in the past three years. (ASCO, May 14, 1999.)
2. The usual and customary cost for the treatment of the patient. These costs occur regardless of participation in the clinical trial. These costs have historically been borne by third-party insurers.
3. The extra cost of patient care generated specifically by the cancer clinical trial. Examples are costs for additional medication, laboratory studies, or diagnostic imaging. These costs are also borne by the trial sponsor. For example: if during treatment, the study requires more frequent evaluation for response, eg, monthly as opposed to the more common every two or three monthly courses, then the additional evaluations would be the responsibility of the sponsor. Similarly, in follow-up, if the study calls for restaging every three months as opposed to every six months (such as ovarian cancer or lung cancer), then the added tests, (imaging, markers, etc) would be the responsibility of the sponsor. Another example: if a special laboratory test, such as gene sequencing, is required by the study but is not a usual component of routine care, the additional cost of this test will be reimbursed by the study grant. In other words, there is a general consensus that research costs such as those for experimental drugs, extra tests, and data management are the responsibility of the clinical trial sponsor, with health insurers responsible for the costs of routine care costs. Recent studies of the cost differential between clinical trials and standard care indicate that cancer chemotherapy trials do not add substantially to the cost of care. A case-controlled study of 61 patients in a highly regimented institution compared the costs of participation in clinical trials with matched “control” patients and found a difference of $38 per month over a 5-year period (Chute et al, JNCI, Vol 91, No 10, May 19, 1999: Appendix.)
4. A second study announced at the ASCO annual meeting by researchers from Kaiser-Permanente supported these findings with similar results. As reported in this study, costs for one year of participation in clinical research were $17,003, whereas one-year costs in control cases were $16,516. When bone marrow transplantation trials were removed from the analysis, clinical trials were actually less expensive (ASCO, May 15, 1999: Appendix 5.)

Therefore, any incremental costs in clinical studies appear minimal, but the opportunity for predictability, greater.

How are the costs of the side effects of treatment reimbursed?

Adverse events during treatment can be divided into those that reflect the natural history of the disease, or its progression, and those that are unique to the experimental treatment. The former would be the responsibility of the insurer, while the latter would be the responsibility of the sponsor. Toxicities from clinical trials can be classified as those that are expected (eg, from the natural history of the disease), those that are common in the routine care of the disease (eg, myelosuppression, mucositis, neurotoxicity) as might occur during regular or standard treatment and those that are unexpected or unique to the investigational agent. Again, the former would be the responsibility of the insurer, the latter the responsibility of the sponsor. In phase I or II studies, drug-related toxicities are usually carefully defined in the pre-experimental models and are the subject of extreme vigilance. Therefore, the number of patients experiencing these toxicities are limited by monitoring and reporting procedures (estimated at 5%), resulting in a certain predictability and control in the process. Moreover, the number of participants in Phase I clinical studies is estimated to be only around 2000 nationally. (Also See: Clinical Trials for Cancer Patients in NJ Who Are Members of Managed Care Organizations [MCO], #4)

What are some benefits of clinical trials for health plans?

The pros and cons (ie, barriers to coverage) of each of the succeeding issues were discussed in detail by the committee. Statements are presented as a consensus of the discussions that began with the question, why shouldn’t health plans cover the routine patient care surrounding phase I and II clinical trials? The in-depth discussion acknowledged and focused upon the realities faced by insurers, including concerns about added costs, determination of routine costs, selection of approved trials, and “out of network” issues.

1. Participation in clinical trials allows plan enrollees to have access to new drugs and emerging therapies at no cost to the plan.
2. Investigational therapy may actually result in lower costs by reducing uncontrollable end-of-life, futile, and unproven care. It should be noted here that the aforementioned Mayo studies were confined to a single, highly regarded institution and did not include the less-regimented care that may be found in other institutions.
3. Investigational therapy may result in lower morbidity and mortality.
4. Clinical trials provide outcomes data necessary to assess medical practice and build an evidence-based, value-driven healthcare system.
5. The public values research, and organizations committed to the well-being of their enrollees will have a positive market image.
6. Allowing participation in clinical trials helps avoid costly (financial, emotional, and image) litigation that may result from denying coverage and access.
7. Explicit criteria for involvement in high quality trials approved by federal agencies increases control of medical care through the reliance upon specific treatment protocols.
8. A pro-active approach offers opportunities for open communication and resolution of differences, reducing reliance on regulation or legislation mandating insurers to cover participation in clinical trials.

What are some benefits of clinical trials for members?

A comprehensive discussion of the pros and cons of the benefits and costs for patients of phase I and II trials was undertaken. Again, it is recognized that barriers to participation do exist and will need to be addressed as part of the Working Group’s responsibilities. However, consensus agreement on the following benefits for patients was reached:

• Increases ability to make informed decisions, and expands choices of medical care
• Provides an alternative to unproven treatment
• Provides a scientific-based treatment with expectation of benefit where no effective therapy exists
• Provides objective, independent safety monitoring and increased accountability in treatment as a result of IRB, governmental, and sponsor oversight
• Benefits society and promotes the advancement of science
• Offers a model approach to other life-threatening diseases

The theoretical disadvantages to participation in phase I and II trials include the risks of adverse events and an efficacy rate of 20% to 25%. The role of the informed-consent process in patient decision making is recognized as critical.

How does providing coverage for clinical trials maintain the fiduciary responsibilities of the health plan?

Under most fully insured programs, the insurance company or HMO typically takes full responsibility for determining the appropriateness of coverage, including coverage for the routine patient care surrounding phase I and II clinical trials. The key determination is whether the trials can be determined to be medically necessary under the terms of the policy documents. Historically, those insurers that have decided to provide coverage have not found it to be an issue with respect to any fiduciary responsibility. A key consideration must be a consistent application of the standards utilized.

With respect to self-funded coverage, the plan sponsor oftentimes maintains fiduciary responsibility. In those instances, the decision to cover would ultimately be that of the plan sponsor who has that decision-making authority.

Clinical Trials and Insurance Coverage: An Ethics Critique

The objections of insurance companies to covering clinical trials are based on the following assertions:

Insurance companies exist to provide coverage for proven (effective and safe) treatments; what transpires in clinical trials cannot be considered treatment strictly speaking because the interventions remain to be demonstrated to be effective and safe. While it may be true that these interventions are experimental, they are nonetheless treatments and designed as such. A number of physicians who are opposed to controlled clinical trials base this opposition precisely on the grounds that if treatment is good enough for the patient within a trial, it has to be good enough for patients who want to receive it outside a trial. It is also the case that certain “standard” treatments that have never been subjected to rigorous testing for efficacy and safety nevertheless generally qualify for insurance coverage.

Under these conditions, insurance companies argue, it is unethical to facilitate (by paying for clinical trial participation) the exposure of their clients to something that may be harmful.

We have at one end, treatments of proven efficacy and safety that have the potential to save the lives of patients; at the other, there are questionable (quack) treatments (unproven efficacy and safety) without any scientific justification. Neither of these extremes poses difficulty in assessing their essential ethical merit and therefore the merits of a patient’s claim to receive one or the other. If that were all that was at stake, the ethical problem would be relatively small. However, we have to recognize that midway between the two extremes, there are those promising but unproven treatments, which bring into possible conflict the interests of the totality of insured members and those of the individual insured patient. The characterization of treatment as promising means that there are reasonable grounds for believing in the efficacy and safety of the treatment which, however, remain to be demonstrated scientifically. It also means that there are reasonable ethical grounds for both the patient and his/her physician to accept the possible risks of the unproven treatment precisely because the expectation of benefits from the treatment is proportionately reasonable. In cases of last-chance treatments in particular, the claims of the individual insured patient may presumably take precedence over those of the totality of the insured population so that there would be an ethical obligation to provide coverage for the individual insured patient in this extreme situation. This obligation also takes precedence over the claim of insurance companies to protect their enrollees from potential harm incurred by participation in clinical trials.

These circumstances mean a reduced role for the insurance company as the one responsible for the stewardship of the resources held in common by the totality of the insured population. The ethical justification, derived from the principle of justice, is found in the critical nature of the patient’s clinical condition. Given that condition, noticeable for its clinical uncertainties and the extreme need of patients whose treatment options are becoming less, it is ethically justified to give the patient and his/her physician the greatest amount of control over their shared decision making. (This is a synopsis of a fuller discourse on this topic that may be found as Appendix 6.)

Clinical Trials for Cancer Patients in NJ Who Are Members of Managed Care Organizations (MCO)

In the context of the following assumptions, predictability for all parties can be assured and a stable system can be created because:

Assumptions:

1. The member meets eligibility requirements for a clinical trial (approved under the auspices of the Working Group’s memorandum of agreement) for cancer therapy by virtue of
   • There are no curative standard therapy options
   • The trial has been approved under the auspices of the Working Group’s Memorandum of Agreement
   • The member meets medical criteria for trial
   • The member agrees to receive care at an approved cancer facility in New Jersey
2. The member, after being fully informed of the risks and benefits of the trial, agrees to be a participant.
   • The organization(s) and associated professional(s) responsible for the trial have a contractual relationship with the MCO(s) for the trials. This also defines responsibility of costs and side effects costs allocated between insurers and sponsors. MCOs agree to cover the costs of customary and usual care for patients enrolled in clinical trials. Physicians, health professionals, and researchers will need to be more willing to work within the managed care system and be open to joining as participating (PAR) members, especially in the provision of services related to clinical studies.

Issues or questions related to symptoms and side effects of the study drug (eg, manifestations of toxicity, conflicts over coverage responsibility) shall be defined as part of the scientific/technical evaluation of phase I protocols prior to approval and patient participation.

Misperceptions, Myths, and Misunderstanding

If understanding and communication among various stakeholders about this critical issue are to be reached, certain misperceptions and myths will need to be corrected. A review of the literature surrounding the debate about insurance coverage and clinical trials indicated a number of differences in perceptions of definitions and working concepts. These include

1. Purpose of clinical research—Researchers and physicians view enrollment in clinical trials as having definite therapeutic benefit for their patients. Given the extensive preclinical evaluation now required prior to human studies, even phase I trials are viewed as having real potential value for patients. The assumption that a clinical trial is the scientific pursuit of unproven treatment approaches and of no medical benefit, especially in early phases, is not consistent with these beliefs. However, phase I clinical trials are not a guarantee of success for patients with cancer, and there are risks involved in participation.
2. Medical appropriateness—Are clinical research trials at the phase I and early phase II levels less medically appropriate than phase III and phase IV clinical trials? Insurers historically have viewed the distinctions between phases as strict boundaries. Researchers, in contrast, argue that clinical research trials are a continuum in which incremental steps are taken in a carefully controlled process. Most researchers already extend coverage for the patient care surrounding clinical research in phase III trials on a case-by-case basis without compromising fiduciary responsibility or the ability to uphold denials of care based upon experimental language in filings and certificates of coverage.
3. Health insurers do not cover clinical trials—Several surveys of oncology investigators indicated that most clinical research trials are currently being covered by insurers. The rate of payment denials has not increased with the penetration of managed care into the health settings. However, increased hassles and time-consuming paperwork have grown substantially as appeals have become more commonplace. Researchers report that appeals are usually successful and denials are often reversed.

Policy Issues and Concerns

While the Scientific & Technical Committee has addressed a number of important issues, it recognizes that the Policy Committee will need to deal with specific concerns involving the development of

1. A mechanism for long-term funding and organizational development of the Working Group aimed at increased efficiencies and improved communications.
2. A comprehensive database that allows access to current clinical trial information on a continuing basis.
3. A system to increase physician participation as participating (PAR) providers for managed care in clinical trials and procedures for handling PAR vs nonparticipating (NON-PAR) providers.
4. Broad-based education programs about the importance of clinical research trials that reach healthcare professionals, patients, and the public.

Conclusions

Approved clinical trials are carefully designed treatment protocols that require external scientific oversight and Institutional Review Board (IRB) approval. Quite separate from the continuum of clinical trials, there is ad hoc treatment that is uncontrolled, single-patient oriented and based on the chance occurrence that a given regimen will work. By not covering clinical trials, there is unwitting support for ad hoc care that may have no value or may be futile. This construct results in great expense and provides no answers to pressing enigmas regarding treatment for patients whose cancer has failed to respond to standard therapy or for which no effective standard therapy exists at the moment.

While regulatory and legislative proposals have been promulgated in a number of states, New Jersey is seeking instead to address these issues through a cooperative effort. Accordingly, it has created the New Jersey Working Group to Improve Outcomes in Cancer Patients. This is a voluntary group consisting of leaders from health insurers, healthcare providers, the research community, the pharmaceutical industry, and patient consumers. The purpose is to develop a fair and equitable mechanism for insurance reimbursement of routine patient care costs when an insured enrollee or subscriber participates in approved clinical trials. The Working Group and the state wish to improve the quality of care and provide much greater access for New Jersey citizens to clinical trials in general, decrease the overall cost of care, increase patient participation in cancer trials in particular, and improve the research climate in New Jersey.

Our joint goal is to make coverage available through third-party insurers for New Jersey citizens with cancer who are eligible for scientifically approved clinical trials.

The New Jersey Association of Health Plans, whose individual insurers provide coverage for more than 2.1 million New Jersey residents, has already agreed to cover the routine costs of care in phase III cancer trials. The Working Group has conducted a comprehensive review of the costs and benefits of phase I and phase II trials as summarized in this report, and we are in agreement to amend the original Memorandum of Agreement to include coverage for the routine patient care surrounding phase I and phase II clinical trials.

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Report and Recommendations on High Technology and Transplantation Clinical Trials Under Working Agreement

Task Force on Transplantation & High Technology Clinical Trials of the New Jersey Working Group to Improve Outcomes in Cancer Patients Report & Recommendations

Membership

Frederick B. Cohen, MD, Chair, New Jersey Commission on Cancer Research; John Barrett, MD, Chief of Bone Marrow Transplantation Unit, National Institute of Heart, Lung and Blood; Ephriam Casper, MD, Director, Memorial Sloan Kettering @ St. Clair’s Hospital; T. Patrick Hill, Ethicist; Selina Luger, MD, Oncology Transplantation Program, University of Pennsylvania; Donald Stangler, MD, Senior Medical Director, Oxford Health Plans; Donald Stavis, MD, Medical Director, Horizon Blue Cross Blue Shield of New Jersey; Ann Marie Hill, Staff Support, Executive Director, New Jersey Commission on Cancer Research.

Background & Significance

The New Jersey Working Group to Improve Outcomes in Cancer Patients has been functioning since December 1999. The Working Group was responsible for a first of its kind consensus agreement in which New Jersey’s major health insurers agreed to voluntarily cover the routine costs of all phases of cancer clinical trials. Since its inception, the agreement has been progressing well. However, questions about how to deal with transplantation and trials involving high-technology procedures have been raised by researchers, insurers, healthcare providers, and patients alike. The Working Group has dealt with these issues on an ad hoc basis, but feels that a consensus policy that is fair to all parties is warranted. For this reason, it has created a blue ribbon advisory group to provide an objective evaluation of the questions raised and make policy recommendations to the Working Group.

Bone marrow transplantation (BMT) and peripheral blood stem cell transplantation (PBSCT) are procedures that restore stem cells that have been destroyed by high doses of chemotherapy or radiation therapy. BMT and PBSCT are most commonly used in treatment of leukemia and lymphoma. They are also used in treatment of certain childhood cancers, including neuroblastoma and brain tumors. The procedures are being evaluated in a number of clinical trials, including cancers of the breast and ovary, multiple myeloma, and Wilm’s tumors. In addition, mini-transplants or a type of allergenic transplant that uses lower, less toxic doses of chemotherapy or full-body radiation and produces graft-versus-tumor effects are being studied. Cancers under investigation with mini-transplants include leukemia, lymphoma, multiple myeloma, melanoma, and kidney cancer.

While improvements in treatment methods have reduced the costs of transplantation, the procedures are complicated and expensive. Insurance coverage for transplantation may vary from contract to contract. Under the New Jersey agreement, insurers have generally covered the costs of transplantation when an approved clinical trial is recommended and appropriate enrollment criteria met. However, concerns about the scientific validity of some trials, the medical appropriateness of procedures for certain patients, inadequate IRB oversight and informed-consent procedures, accrual issues, and an unclear verification process are concerns that have been raised by NJ insurers. On the other hand, researchers have felt that approvals for these high technology procedures are a constant struggle, waste valuable time, and create hardship for patients and their families whose lives are at risk.

To address these issues, the Working Group has charged its blue ribbon panel with reviewing the following issues and providing recommendations that will serve as a general policy for transplantation and other emerging high technology trials. Major questions include

• How can the relevant distinction between standard care and experimental procedures in transplantation and other high technology trials be determined to clarify processing of requests for coverage?
• Are the definitions agreed upon in the Working Group agreement reasonable for transplantation and other high technology trials?
• How can a fair system be established to determine what is routine care in trials that involve high technology procedures?
• Should the “medical appropriateness” of high technology procedures in experimental settings for specific patients be factored into decision making for coverage by insurers?
• How can IRB oversight and informed consent procedures be improved?
• How should disagreements between insurers and investigators involving transplantation or high technology procedures be handled?

Scientific review and monitoring by national agencies, such as the National Cancer Institute, the United States Food and Drug Administration, the United States Department of Defense, and the United States Department of Veterans Affairs, remain critical for all approved clinical trials under the working agreement.

The routine costs of high technology trials are not different from regular trials. While expensive, it is better to pay for these costs in the setting of a clinical trial than in a system based upon care by demand. Questions of medical effectiveness will be answered more directly and waste will be reduced (eg, breast cancer and bone marrow transplantation).

As an essential aspect of their scientific integrity, methodological quality is a primary requirement of ethics in clinical trials.

How can the relevant distinction between standard care and experimental procedures in transplantation and other high technology trials be determined to clarify processing of requests for coverage?

While the rescue of patients with hematologic malignancies, such as lymphomas and leukemias, has become accepted practice, many research questions surrounding high-dose chemotherapy, graft-verses-host disease, and full-body radiation that rely upon stem cell, autologous and allogeneic transplantation for rescue, remain experimental. For example, high-dose chemotherapeutic or biologic regimens that may be proven in lymphomas are now being tested in a number of other malignancies using stem cell rescue to see if similar effects can be obtained in those diseases. While the procedure is standard care in lymphoma, it may be experimental in multiple myeloma. If the case is considered standard treatment then insurers cover costs using their contract procedures. If experimental, then insurers may require evidence that the trial fits the approval criteria established under the working agreement. Problems arise when differences in opinion occur about what is standard care and what is experimental. This situation results in additional paperwork, prolonged arguments, and tension between parties. Because the state of the art in this arena is in flux, determining what is routine or experimental has become a source of disagreement between researchers and insurers. Traditionally, reports of well-designed investigations published in peer-reviewed journals and verified by a second study from a different research group provide some criteria for drawing the line between standard and experimental treatments. Meta-analysis has also proven to be useful in areas where controversy arises. The use of practice guidelines may offer a helpful system for evaluating standard care and the NCCN has agreed to provide their guidelines to the Working Group for this purpose. It is suggested that these guidelines may offer a “reasonable” baseline, or starting point, for distinguishing between standard care and investigational protocols. It should be recognized, however, that medical practice can change rapidly and some flexibility should exist in reaching agreements. Where substantial disagreement exists, review may be required. In the end, there is no simple way to determine when a protocol moves from investigational to standard care, and such efforts will require a case-by-case evaluation of the study.

While there are gray areas in research, the scientific scrutiny undertaken by scientific review boards was created to assure that clinical trials are scientifically valid and monitored appropriately. Because the working agreement includes such oversight, insurers should feel confident that assurances are in place to limit frivolous research under the working agreement.

Are the “approved” definitions under the working agreement appropriate for transplantation and other high tech trials?

The critical component of the approved definitions under the working agreement is the scientific review, monitoring, and evaluation provided by the national entities cited within the agreement. This oversight assures scientific validity and patient safety at the highest level possible at this time. While this is a stringent requirement, the federal standards provide assurances and a reasonableness that is fair to all parties within the agreement. Under this system, investigators should provide evidence that their studies fit the approved definitions within the agreement. NCI approval numbers, cooperative group trial numbers, and NCI letters approving trials by designated cancer centers must be provided by investigators either to the Working Group or insurers. There is a difference between, on the one hand, participating as a co-investigator in an approved study under the consensus agreement, and on the other hand, following a protocol on a “like” study basis. In this situation, physicians use approved protocols, but are not working under the direct scrutiny of a scientific review board or safety/monitoring board. For multi-institutional trials where an NCI center has provided a scientific review, a letter stating this fact, including NCI approval numbers and monitoring assurances, should be provided to the Working Group or the insurer. Single-institutional trials that do not have this scientific oversight and continuous monitoring in place should either seek to collaborate with an NCI-designated center (that is providing the review and monitoring for the institution), cooperative group or work within an FDA IND application. Otherwise, protocols fall outside the working agreement and insurers are within their rights to not provide coverage or exceed their normal contractual requirements. Given these standards, insurers should have confidence that the highest scrutiny is being applied to the approved trials within the agreement and continuous review should not be required. (See Appendix II)

What is a fair system of cost reimbursement for these trials?

The costs of high technology include both the research components and the routine medical costs related to patient care and recovery. In most cases, the research costs are not significant components of overall costs. Costs of experimental drugs, additional tests/procedures and data management are usually covered by sponsoring agencies or provided free by pharmaceutical companies. Therefore, most of the costs of high technology trials will involve the routine costs incurred in the medical care of patients. While admittedly expensive, it is much more desirable to pay for these costs within a clinical trial than through legal and legislative mandates. Research will provide objective evaluations of the quality of these studies more effectively. Because the definitions of approved clinical trials under the working agreement are stringent, insurers can be assured that a strong scientific basis exists for the trial.

It is up to the individual insurers to negotiate payment schedules for the routine costs of procedures within trials with their providers through standard contract practices. The Working Group does not get involved in any rate setting or cost negotiations between parties. However, it is the researchers best interests to keep costs as low as possible so that patients are not forced to face unreasonable hardships or burdens.

Should the medical appropriateness of high technology procedures in experimental settings for specific patients be factored into decision making by insurers?

Insurers argue that researchers exist within a competitive environment and that pressures to accrue patients to their clinical trials are high. Therefore, it is argued that part of insurance review should include the medical appropriateness of the recommended clinical trial for the specific patient. For example, should a woman that is HER-2NEU positive be enrolled on a bone marrow transplantation trial for metastatic breast cancer prior to being treated with Herceptin? Researchers and patients argue that this decision should be made within the physician-patient relationship and not by insurers. Insurers argue that compelling reasons exist to use medical appropriateness in the decision-making process. Practice guidelines may help clarify most situations.

In other instances, conditional regimens are tested in a wide range of malignancies. Scientific evidence may exist to validate the study for some cancers, but very wide criteria are used that include almost any type of cancer. Concerns about such umbrella approaches have been raised. In these cases, patient numbers for specific cancers may be so limited that scientific questions will not be answered, especially in single-institution trials. In these circumstances, insurers feel that it is legitimate to question the validity of these trials.

While there are gray areas in research, the scientific scrutiny undertaken by scientific review boards was created to assure that clinical trials are scientifically valid and monitored appropriately. Because the working agreement includes such oversight, insurers should feel confident that assurances are in place to limit frivolous research under the working agreement.

It is recognized that ethics plays a substantial role in any answer to this question. Generally, securing the best interests of the patients must be the overriding obligation and should be the central consideration of any discussion of medical appropriateness. A full discussion of the ethical implications of this question is provided in the appendix.

How can informed consent and human protection assurances be strengthened for high technology trials?

While federal policies on conducting research on humans exist, insurers have raised concerns about variations in the quality of IRB review and informed-consent process, especially in clinical trials that do not involve cooperative groups or NCI-designated cancer centers. The Office of Human Research Protections (OHRP) and the FDA have stringent protections in place for any research involving federally funded studies or INDs. Under the working agreement, all approved studies are subject to these protections and failure to comply with policies results in severe penalties. Furthermore, the HHS through the OHRP, has released revised policies and procedures designed to improve the quality of human subjects protection. These procedures should improve the overall performance of IRBs and the quality of the informed consent process (May 2000). At a state level, the New Jersey Commission on Cancer Research should initiate education programs aimed at improving understanding of human protection and safety policies for New Jersey researchers, IRB members, and research administrators. OHRP requires that all principal investigators and key personnel be educated about the regulations, policies, and ethical standards governing the protection of human subjects. However, more intensive courses could offer greater understanding of the informed consent requirements, ethical requirements, and risk benefit analysis. In addition, specialized training for IRB members, hospital administrators, and research personnel on such issues as risk/benefit analysis, informed consent requirements, monitoring and compliance, privacy and confidentiality, and continuing review should be provided on an ongoing basis.

How should disagreements between insurers and investigators involving transplantation or high technology procedures be handled?

The Working Group has access to an independent external reviewer process to help provide objective, scientifically based evaluations of a protocol where the principal investigator of the study disputes a determination that the clinical trial does not fall under the definition of an approved trial within the Working Group agreement. In such instances, it is the responsibility of the principal investigator to provide written evidence and documentation that the protocol fits the definition of an approved trial. The purpose of this review is not to evaluate a specific patient case but the protocol itself. It should not overlap or conflict with the full internal review that is provided by the insurer under the standard appeal procedures.

Conclusions & Recommendations

The approved criteria under the working agreement offers stringent oversight and a framework to assure that only carefully designed clinical trials involving transplantation and high technologies are covered. However, investigators, hospitals and physicians should be educated about the requirements for approval under the working agreement to prevent submission of unauthorized protocols. Appropriate verification information must be included in all requests for insurance coverage and routine cost-analysis grids provided for all phase I and noncooperative group phase II trials. On the other hand, NCI-designated cancer centers and cooperative groups should consider mechanisms for outside scientists to submit their trials through scientific review and monitoring boards so that innovative ideas might prosper. Efforts should be made to improve the informed consent process and institutional review board training provided. Finally, a system of objective scientific review as proposed by the Working Group should be offered in situations when disputes appear irresolvable using standard internal and external review processes.

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APPENDIX I

Ethical Considerations

Under normal circumstances and in light of the very considerable risks incurred in a bone marrow transplantation trial for metastatic breast cancer, the ethical assumption must be that available and known-to-be-effective treatment, in this case, Herceptin, takes precedence over an experimental treatment. There are two ethical issues here: securing the best interests of the patient and avoiding harm to the patient. Medical appropriateness relates directly to the first ethical issue. Securing the best interests of the patient is the overriding obligation in any medical treatment situation, whether we are speaking of standard or experimental treatment. Proportionate reason relates directly to the second. Clinical trials are the occasion of what ethicists call nonmoral harms—a range of physical and psychological discomforts unavoidable in the course of a trial. In the absence of proportionate reason justifying incurring these discomforts, what are nonmoral harms become moral harms. That is, the action taken, in this case the clinical trial, undermines the very value it was intended to secure. It is disproportionate to the clinical outcome sought. The role of ethics is to guard against the transition from nonmoral harms to moral harms, and proportionate reason is the measure by which to determine an appropriate balance between the nonmoral harms incurred by and the benefits to follow from participation in the trial. Where the balance is such that the accruing benefits outweigh the incurred harms, these unavoidable harms remain nonmoral.

This brings us to the matter of who should decide on medical appropriateness, the patient’s physician or the insurance company? Ethically, it is not a simple matter. There are two sets of interests here that need to be protected and reconciled: the medical interests of the patient and the financial interests of all the enrollees in any given insurance program. Again, it must be the ethical assumption that the medical interests of the patient are best protected within the confines of the patient-physician relationship where indispensable personal clinical knowledge on the part of the physician and informed consent on the part of the patient can most realistically be said to obtain. Accordingly, judgments about the appropriateness of a specific treatment for a specific patient ought to be made within the confines of the patient-physician relationship. As stewards of the financial resources of an insurance company available as a matter of justice to all enrollees because of their contributions, the appropriate personnel of an insurance company have the responsibility of disbursing these resources in the best interests of enrollees. It must be the ethical assumption that these personnel are responsible for making judgments of medical appropriateness of individual treatments in themselves based on standard practice and research findings, or clinical trials approved by such agencies as the National Cancer Institute, the Food and Drug Administration, and the Department of Defense—for the purpose of determining the most equitable disbursement of available resources. Thus, in a case where Herceptin, despite appropriate clinical conditions, has been bypassed in favor of a bone marrow transplantation trial for metastatic breast cancer, it is ethical, relative to the demands of justice and the equitable disbursement of resources, to judge Herceptin medically appropriate and the trial for the time being as medically inappropriate. Under these circumstances, it would be incumbent on the physician to justify the choice of the trial over Herceptin. As a matter of standard application, the principle of distributive justice requires that patients in similar clinical conditions are to be treated similarly, unless, as the Belmont Report indicates, there are grounds for making an exception.
There is a third ethical issue that relates, though less directly, to medical appropriateness. Placing a patient in a clinical trial primarily for purposes of meeting accrual requirements for the trial is ethically unacceptable on the grounds that it is the equivalent of regarding a person as a means only. Even in a phase I trial where treatment is not the goal, the trial should be open to that possibility. Otherwise, the presence of the patient in the trial serves primarily the purposes of the trial, not the needs of the patient. This would violate the integrity of the person, which is also the ethical basis for insisting on securing the best interests of, and avoiding harm to, a patient.

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