FDA to Include Advocates in Planning of Phase III Trials

ROCKVILLE, Md—The Food and Drug Administration (FDA) is expanding the role of patient advocates in the approval process of cancer drugs. For some years, patient representatives have participated in meetings of the Oncologic Drugs Advisory Committee (ODAC). FDA’s new corps of patient consultants will initially participate in discussions of the design of phase III clinical trial protocols, as well as ODAC proceedings.

Psychologist JoAnn Minor, director of FDA’s Patient Consultant Program, explains the new role for patient advocates and the reasoning behind it in this interview with Patrick Young, Oncology News Inernational’s Washington Bureau Chief.

Oncology News International: What do you expect the patient consultants to contribute to the drug development process?

Ms. Minor: Patient consultants will provide the patient perspective—a voice at the table focused on patient issues. In this new program, FDA is asking the patient consultant to be a part of the process as we work with drug companies to develop protocols for phase III trials.

Our focus is going to be on involving patient consultants in the end-of-phase-II meetings when FDA sits down with a company to discuss their phase III clinical trial protocol.

Patient consultants can help with questions that particularly need the patient perspective such as: How can we best recruit patients for the trial? Is the toxicity such that patients will be able to manage and tolerate it? Should the company consider an expanded access program? FDA is trying to provide a way for advocates to understand and contribute to the drug review process early on.

ONI: I can understand using a cancer survivor as a patient consult, but you also opened the program up to parents, other family, and friends of cancer patients.

Ms. Minor: In the end, the choices were cancer survivors, parents of cancer patients, caretakers, and oncology professionals. The 24 consultants that we selected, and the vast majority of the 109 applicants, were cancer survivors or a parent of a cancer survivor.

ONI: Another of the criteria was knowledge of cancer. How did you determine that?

Ms. Minor: In their resumes, applicants described how they were active in the advocacy community and some of the activities they were involved in as advocates. One example was Project Lead. This is an intensive workshop conducted over several days on what clinical trials are and how to look critically at clinical trials and research.

Some applicants taught or had helped teach workshops about clinical trials. Some lectured at medical schools, talking to new medical students about patient issues and how to interact with patients, especially when working with patients with a life-threatening disease. So the patient advocates presented to us varied backgrounds of experiences that spoke to what they knew about their cancer and drug research.

ONI:Will the consultants work only on the cancer they had or that their child had?

Ms. Minor: Correct. Patient consultants are very specifically targeted for their area of cancer expertise. Advocates are, I think, unique among individuals whose lives are touched by a serious disease. Advocates are highly motivated to learn everything about their disease. Usually, they jump in and learn all they can before deciding on their treatment. They sit down and talk to the doctor. They ask tough questions, and, as their therapy goes on, they often end up being patients who have a continuing dialogue with their physician and work together with their physician in their therapy.

ONI: What kind of training will patient consultants receive?

Ms. Minor: The training will be an overview of the drug review process. FDA has scheduled a 2-day workshop in October. We are also setting up a mentoring program. The mentors will be other patient consultants or patients reps who have already had experience at FDA. This will give new consultants the perspective of someone who has had experience within FDA. They can call and talk with their mentors about the process and how things work

ONI: What role will the consultants play in the ODAC meetings?

Ms. Minor: If we had a patient consultant at the end-of-phase-II meeting, and the drug comes up before ODAC, the consultant would be asked to serve as the patient rep at that ODAC meeting.

ONI: Currently, you have a consumer rep and a patient rep when ODAC reviews a drug. Will there now be a third, the patient consultant?

Ms. Minor: No, the patient consultant will then put on the hat of the patient rep. I don’t think of the programs as two distinct programs. We have added the dimension of the patient consultant to the overall program and incorporated the patient perspective earlier into the drug review process.

ONI:Will patient consultants on ODAC have a vote?

Ms. Minor: They have the vote now.

ONI:ODAC is getting more deeply involved in advising FDA on trial procedures and conduct. Will patient consultants join in these discussions as well?

Ms. Minor: FDA will continue to have the patient voice at the table for all of these discussions, including ODAC subcommittee meetings. The pediatric oncology subcommittee has two patients at the table. So whether we choose from the pool of patient consultants or the pool of patient reps, the patient perspective will be represented at ODAC.

ONI: Does FDA plan to expand the program to other advisory committees?

Ms. Minor: FDA has patient reps on lots of committees in three centers. Right now, the patient consultant program is focused on cancer. We will continue to fine tune the program and certainly hope that we can expand it to other life-threatening diseases.

As we develop the program, I am sure other ideas will come to the forefront. We will work toward incorporating the patient consultant into some of the meetings other than end of phase II. For, example, when a company submits a new drug application, there is a meeting between the company and FDA to discuss the application review process. It would be good to have a patient consultant as a part of that meeting.

ONI:What safeguards are in place to prevent conflicts of interest?

Ms. Minor: Every time a patient consultant meets with the company and FDA, he or she completes a conflict of interest review. They are very aware that all the laws that apply to federal employees also apply to them when they are acting in the role of patient consultant.

ONI:Does FDA plan to name additional patient consultants?

Ms. Minor: FDA looks at this program as a beginning. We hope to recruit each year so that there will be a growing pool of patient consultants. Anyone interested in finding out more about the patient consultant program can email me at jminor@oc.fda.gov.