Shared Benefits of Clinical Research Should Come with Shared Responsibility

Yu-Ning Wong, MD, MSCE

	Yu-Ning Wong, MD, MSCE

One of the most frustrating aspects of clinical research is when you learn that your patient, who is both interested and eligible for a study, doesn’t “have coverage for clinical trials.” It is difficult to understand what incentive insurance companies have for including such a clause, since the investigational agent is typically provided by the sponsor, and “routine” costs will accumulate even if the patient isn’t on study.

Unfortunately, all of these arguments will fall on deaf ears if the patient’s insurance plan contains language that explicitly excludes coverage for clinical trials. Although there is always the prospect of appeal, this is often not practical for a patient who needs to start treatment quickly.

This is inherently unfair for several reasons. The benefits of clinical research are shared with patients and insurers; therefore, the costs should be as well. Clinical research benefi ts society as a whole, not just the individual participants. The progress made in pediatric cancers would not have occurred without the commitment of the pediatric oncology community, which enrolls more than 70% of children with cancer on trials. The knowledge from these trials is also shared; treatments for adult leukemias were developed from pediatric regimens.¹ Clinical trials also play an equally important role in clarifying the toxicity profi le of new treatments. If a novel treatment is found to be more toxic or less effective than an existing one, society benefits when it falls out of favor because patients are no longer being harmed and payers are no longer bearing the higher cost of managing side effects and complications of treatment.

Fortunately, Medicare has already acknowledged the importance of clinical trials. In 2000, President Clinton directed the Department of Health and Human Services (DHHS) to “explicitly authorize [Medicare] payment for routine patient care costs...and costs due to medical complications associated with participation in clinical trials.” Following this Medicare coverage determination, enrollment of older patients in Southwest Oncology Group studies increased.² However, this regulation has no bearing on the 85% of the population not covered by Medicare.

Increasing Confusion: Patchwork of Existing State Laws and Insurance Plans

Although many insurers also recognize the value of clinical research and provide coverage for routine costs, this is hardly consistent. There are many factors that go into whether a patient has clinical trial coverage. Thirty states and the District of Columbia have enacted laws requiring coverage for routine costs associated with clinical trials. Another six states have agreement contracts between states and insurance companies. However, these policies are inconsistent and vary from state to state. Some require coverage for phase I to IV studies, while others specifi cally exclude phase I studies, and some require that the trial be conducted in state.³

Significant confusion also occurs at the insurance plan level. Many employers (or individuals) pay premiums to an insurer who then assumes the risk of paying for the member’s health care. This is distinctly different from employers who choose to “self-insure” with their own resources and pay the claims themselves. These self-insured plans may choose not to cover clinical trials and are exempt from state laws under the federal Employee Retirement Income Security Act of 1974 (ERISA). However, self-insured employers often contract with large insurers to administer the plan. Therefore, it is often difficult for clinicians and their offi ce staff to determine patients’ coverage at the time they are discussing potential clinical trials.

Reduced Access: Patients without Specific Protections

State mandates have been shown to have a modest effect on clinical trial enrollment, suggesting that insurance is only one of many factors that influence a patient’s decision.⁴ However, the lack of insurance coverage is an insurmountable barrier for individual patients who might otherwise be interested in participating.

A Johns Hopkins study found that 13.6% of insured patients with cancer who consented to therapeutic trials were denied enrollment because they did not have clinical trial coverage. More than 80% of the patients in the sample were from Maryland, Pennsylvania, and Virginia. Patients from Pennsylvania were more likely to be denied enrollment than patients from the other two states.⁵ Unlike Maryland or Virginia, Pennsylvania is among the 15 states that does not have any laws or agreements requiring insurers to cover clinical trials.

These data suggest that without specific mandates, certain payers can and will continue to restrict their patients’ access to clinical trials. They may cite the increased cost and their responsibility to control premiums. However, there is no data supporting the notion that this care is substantially more expensive. Analyses of clinical trial participants from the 1990s suggest that this may be 6% to 10% higher than routine care, with those who died or received bone marrow transplant or early-phase drugs accruing the highest incremental costs.^6,7 Because the cost of standard therapies has risen in the past two decades, it is unlikely that this gap has widened. Furthermore, because less than 5% of patients with cancer enroll in clinical trials, the overall cost to a specific insurer is very low and should not be used as an excuse to “opt out” of this shared responsibility.

Improving the Process

Section 2709 of the Patient Protection and Affordable Care Act (PPACA) “Coverage for Individuals Participating in Approved Clinical Trials” is the first federal law mandating private insurers to cover the routine patient costs associated with participation in clinical trials. It establishes a national minimum coverage standard, but state laws that impose broader protections will still apply.

To qualify, patients must be eligible for an “approved” clinical trial (Table 1). “Routine patient costs” include all items and services the payer would cover for a patient not enrolled in a clinical trial. This goes into effect January 1, 2014.

One complicated aspect of the PPACA is that “grandfathered” plans are exempt for certain provisions, including Section 2709. A grandfathered plan is one that existed on March 23, 2010 (the day the PPACA was signed) but will lose this status if it makes certain signifi cant changes that reduce benefi ts or increase costs to consumers.

It is difficult to estimate the number of patients who will remain in grandfathered plans when Section 2709 is enacted. The DHHS estimates that by 2013, 36% to 66% of patients covered by large employers will remain grandfathered. Because they are often more sensitive to cost, a smaller number (20%-51%) of patients covered by small employers will likely still be grandfathered.⁸ These estimates may change in 2014 as health insurance exchanges become available and nongrandfathered plans in the individual market are no longer able to incorporate preexisting medical conditions to determine premiums.

Realizing the Law’s Full Potential

There are many questions about the actual implementation of Section 2709. ASCO and other cancer advocacy groups have recommended to DHHS that upcoming regulations include certain safeguards that support the spirit of the law and maintain access to clinical trials. A link to the full text of the recommendations can be found in the January 20 article, “ASCO Seeks Better Access to Clinical Trials for Cancer Patients” at ascoaction.asco.org.

In addition, attention must be paid to the existing coverage for clinical trials under the grandfathered plans. Although it is possible that many grandfathered plans will cover clinical trials, it is still not clear how many may still be without coverage. This might seem counterintuitive, but it is important to understand the political need to allow plans to remain grandfathered. Policymakers have learned that many Americans may be resistant to health care reform because they are already satisfi ed with their health care coverage and do not want it to change. It will be interesting to know if patients elect to stay with their grandfathered plans as newer plans with broader protections become available.

As the ongoing presidential campaign and court cases challenging it show, there is still signifi cant debate surrounding the PPACA. Despite this, I believe that the cancer community should see Section 2709 as a step forward for our patients. Support for clinical trials should be seen as a shared responsibility for all insurers, as the benefi ts to society are undoubtedly shared. The current outdated system of private plans, state laws, and ERISA exemptions has demonstrated that we cannot rely on the private insurance market and state laws to guarantee support for this important shared, national resource.

Dr. Wong is a paid consultant to Independence Blue Cross.

About the Author: Dr. Wong is an assistant professor of medical oncology at Fox Chase Cancer Center. She is a health services researcher and studies patient decision making and the utilization of novel therapies. She has been an ASCO member since 2003. She serves on the Cancer Research Committee and was the co-chair of the Clinical Trials Subgroup of the 2011 ASCO Provider-Payor Initiative.

References:

1. Unguru Y. The successful integration of research and care: How pediatric oncology became the subspecialty in which research defi nes the standard of care. Pediatr Blood Cancer. 2011;56:1019-1025.
2. Unger JM, Coltman CA, Crowley JJ, et al. Impact of the year 2000 medicare policy change on older patient enrollment to cancer clinical trials. J Clin Oncol. 2006;24:141-144.
3. Kircher SM, Benson AB 3rd, Farber M, et al. Effect of the accountable care act of 2010 on clinical trial insurance coverage. J Clin Oncol. 2012;30:548-553.
4. Gross CP, Murthy V, Li Y, et al. Cancer trial enrollment after state-mandated reimbursement. J Natl Cancer Inst. 2004;96:1063-1069.
5. Klamerus JF, Bruinooge SS, Ye X, et al. The impact of insurance on access to cancer clinical trials at a comprehensive cancer center. Clin Cancer Res. 2010;16:5997-6003.
6. Fireman BH, Fehrenbacher L, Gruskin EP, et al. Cost of care for patients in cancer clinical trials. J Natl Cancer Inst. 2000;92:136-142.
7. Goldman DP, Berry SH, McCabe MS, et al. Incremental treatment costs in national cancer institute-sponsored clinical trials. JAMA. 2003;289:2970-2977.
8. Healthcare.gov. “Keeping the Health Plan You Have: The Affordable Care Act and ‘Grandfathered’ Health Plans.” http://www.healthcare.gov/news/factsheets/2010/06/keepingthe-health-plan-you-have-gradfathered.html. June 14, 2010.