[This summary was posted on the AHRQ or CMS website, along with a study plan document, to provide the public with a lay description of the project and potential items on which the public may want to comment.]

Funded by the Agency for Healthcare Research and Quality (AHRQ), this project is examining what impact, if any, changes in insurance coverage for new treatments – such as drugs and procedures – have on patients’ interest in participating in clinical research studies evaluating those treatments. Patients often participate in a research study in order to gain access to a new treatment option that is not available outside of the study. Sometimes the study provides an opportunity to have the new treatment paid for, and other times it does not. Many research studies are “randomized”, meaning that the participant has an equal random chance of receiving the new treatment under study or the current standard of care, and cannot specify which treatment is received.

Sometimes, either before or during the course of the research study, health funders such as Medicare or private insurance will decide to cover the treatment for patients who are not involved in the research study – but will not reimburse patients who are on the study. In these situations, it is possible that the change in payment policy will affect patients’ willingness to enroll in, or to stay in, the research study. As a result, it is possible that there will never be adequate descriptive information about benefits or harms of the new treatment, and how it compares to older treatments.

Together with the Center for Medicare and Medicaid Services (CMS) and the AHRQ, we are conducting this public policy project to understand how decisions regarding healthcare payment for these new treatments impact participation in clinical research studies about the new treatments. We will convene in small groups opinion leaders from CMS, the National Institutes of Health, the Food and Drug Administration, industry, other healthcare funders, and patient advocacy groups, as well as clinical researchers and clinical research staff.

Your input can help us shape these discussions, and therefore the outcome of this policy project. We seek your input on the extent to which insurance coverage for a treatment would affect your interest in participating in a clinical research study. We also need to know what factors should be considered as we develop this report. We ask for your input on the following questions:

1. Would you be willing to participate in a clinical research study if that were the only way to gain access to a new, and possibly more effective, treatment?
2. Would you be willing to participate in a clinical research study of a new drug or procedure, if you would have to pay out-of-pocket for the drug/procedure when on study?
3. What if you could receive reimbursement for it off-trial, how would that influence your decision? Why or why not?
4. If you decided to enroll in a clinical research study comparing two treatments, and one of the two treatments would cost more than the other, would you be willing to be assigned to a group receiving either of the treatments?
5. If you might be assigned to a study group where you do not receive the new drug or procedure (but instead receive the traditional “standard care” treatment), would you still be willing to participate in a clinical research study? Would your decision be different if you knew that you might get the new treatment for free by being in the trial?
6. If you have been involved in the conduct of a clinical trial (e.g., as an investigator, coordinator, manager, or administrator), have you felt that patients’ decisions to enroll or continue participation in the trial were influenced by payment policy considerations? If so, please explain.
7. If you are a person who relies on the output from clinical trials to make decisions regarding healthcare (e.g., as a part of evidence-based decision making), have you felt that the output from clinical trials was influenced by payment policy considerations? If so, please explain.
8. Our research plan is included for public comment. Are there other things that we should consider when conducting this project?