The principles of evidence-based medicine increasingly govern healthcare policy and practice in the United States. The hallmark of the evidence-based approach is research data generated through clinical trials, and particularly through “gold standard” randomized controlled trials (RCTs). Healthcare policy increasingly relies on evidence furnished by RCTs. It is therefore of paramount importance that investigators are able to execute RCTs, and that those trials include a fair representation of the general population, as well as of the specific populations of relevance to topics being studied. Currently, valid concerns surround low rates of participation in clinical trials and disparities in clinical trial participation. Various factors might influence patients’ interest and willingness to participate in clinical trials. Financial repercussions, which depend upon the payment policies of third-party insurers, may be an important element in patients’ decisions regarding whether or not to participate in a clinical trial, as well as whether patients stay in the trial once initiated. Payment policies may exert an influence on clinical trials in other ways, impacting not only recruitment and retention, but also conduct of the trial and the subsequent quality of the evidence base.
The Duke Center for Clinical Health Policy Research and Duke Cancer Care Research Program conducted this study, supported by a contract with the Agency for Healthcare Research and Quality (AHRQ), to ascertain whether, and to what extent, payment policies may be influencing participant recruitment to clinical trials, rates of participation, and retention in clinical trials. A further objective was to gather input on the issue of whether or not payment policies, through influencing clinical trial participation, may have a deleterious effect on the resulting evidence base. This report will help to inform the Centers for Medicare and Medicaid Services (CMS) if there is causal relationship between the timing of initiating coverage for new therapeutic technologies and beneficiary participation in clinical trials to provide evidence of effectiveness of these new technologies in the elderly and disabled Medicare population.
We employed a variety of strategies to gather data and experiences relevant to the topic. These strategies were: (1) a nationally selected Advisory Panel to provide expert input; (2) a systematic literature review of MEDLINE and ClinicalTrials.gov; (3) a Public Forum held on the CMS campus in Baltimore, MD, to gather public input; and, (4) a series of teleconferences with “key informants” representing diverse stakeholders including government, industry sponsors, third-party insurers, clinical trials investigators and staff, and patient advocates. Flexibility in the study design permitted iterative expansion of the inquiry based on information and insights gathered during the exploratory process.
Published data are virtually non-existent to quantify the difficulties encountered by trials with recruitment and retention, as it pertains to third party payment policies. However, in practice several large-scale clinical trials have encountered substantial difficulties due to the deterrent effect of payment policy on participation. Medical device trials have been more affected by these issues than have drug trials. Lack of a common understanding of which costs should be assumed by which party (sponsor, site, third-party payer), and lack of common definitions of “standard of care” versus “research-related” costs complicate payment policy and likely impact enrollment. To ensure that trials get completed, investigators are developing creative solutions to assure participants’ coverage on-trial. To ensure that payment policy does not result in a financial loss, sites are analyzing financial impact and may decide not to initiate trials if the financial prospects are negative. Poor coordination among government agencies, industry, third party payers, patients, and researchers is contributing to the difficulties.
Payment policy does bear an impact on clinical trial participation, though this impact is difficult to quantify and unevenly felt across different types of studies, stage of trials, and study populations. The issue of payment policy is closely related to issues of access to care and disparities in care. Payment policies do affect evidence development, in that their impact on clinical trial enrollment results in slower accrual, longer time to completion of studies, and sometimes early termination of studies due to lack of sufficient sample size. Better coordination among government agencies, and between government, third-party payers, sponsors, and sites is necessary. Presuming that participation in clinical trials is a good thing for individual patients and the public at large, a coherent strategy that stipulates when coverage should be initiated, specifies which costs should be covered, and assigns responsibility for those costs to specific payers, coordinated to maximize clinical trials enrollment and retention, could help to (1) rationalize the process of reimbursement when patients are enrolled in clinical trials, (2) ensure equal access to clinical trials for patients interested in participating, and (3) facilitate the generation of high-quality evidence to support future policy-making and clinical practice.
