Regulatory Focus™ > News Articles > FDA Schedules HIV Meeting as Basic Framework of Patient-Focused Drug Development Takes Shape

FDA Schedules HIV Meeting as Basic Framework of Patient-Focused Drug Development Takes Shape

Posted 20 May 2013 | By Alexander Gaffney, RAC 

The US Food and Drug Administration (FDA) has announced that it will soon hold the second of at least 20 meetings focused on ways to drive pharmaceutical development in ways that best meet the needs of patients, this time focusing on human immunodeficiency virus (HIV) infections.

Background

Under the Food and Drug Administration Safety and Innovation Act of 2012, FDA was charged with setting up a "Patient-Focused Drug Development Initiative," which is set to seek out opinions from patients in 20 different disease areas over the next five years.

"The human drug and biologic review process could benefit from a more systematic and expansive approach to obtaining input from patients who experience a particular disease or condition," FDA explained in a Federal Register posting announcing the program in September 2012.

Practically, the program means FDA will start to assess the benefit:risk paradigms that each patient group is willing to accept in return for a treatment. A patient group suffering from an under-treated but otherwise medically benign condition-trichotillomania, for example-might be less willing to accept high levels of risk in return for an effective treatment. Conversely, those suffering from Chronic Fatigue Syndrome (CFS)-the focus of FDA's first patient-focused meeting-might be more willing than most to accept a drug that might either be less effective or more dangerous that FDA's benefit:risk paradigm ordinarily would permit.

The point, however, is for FDA to determine this framework in tandem with patients. The agency recently launched a new Patient Network website aimed in part at educating patients about FDA's regulatory process so that they can better engage with regulators at these meetings and at other venues.

"We really wanted to provide a resource for any patient or patient advocate that is at all interested in FDA-regulated medical products or issues to have a home for getting educated about these products, how they're regulated and how they can be a part of that regulatory process," James Valentine, program analyst at FDA's Office of Health and Constituent Affairs, told Focus in an interview in May 2013. Valentine added that he hopes these kinds of patient initiatives can "grow their influence" and allow them to participate more regularly and productively.

A Focus on Patients

That participation is predicated upon some fairly specific questions. At the CFS meeting, for example, FDA said it wanted specific input regarding the significance of symptoms experienced by patients, the negative impacts of those symptoms, the effects of the condition on daily living, the treatments patients currently take, and the positive and negative attributes of their prescribed medications.

But whereas CFS is currently operating in something of a treatment vacuum, owing perhaps to the fact that as recently as a few years ago, HIV patients have had the benefit of approximately two decades of treatment, though all stop short of a cure.

Still, FDA's treatment questions are quite similar to those asked of CFS patients, and could represent a sort of framework of questions to be asked of the remaining 18 patient populations.

Patient Perspective on Current Approaches to Treatment

HIV

CFS

Of all the symptoms that you experience because of your condition or because of your therapy or treatment, provide one to three symptoms that have the most significant impact on your life?

What are the most significant symptoms that you experience resulting from your condition? What are the most negative impacts on your daily life that result from your condition and its symptoms?

How does the condition affect your daily life on the best days and worst days?

What are you currently doing to help manage your HIV and any symptoms you experience because of your condition or other therapies?

What changes have you had to make in your life because of your condition?

What treatments are you currently using to help treat your condition or its symptoms?

What specific symptoms do your therapies or treatments address?

What specific symptoms do your treatments address?

How long have you been on treatment and how has your treatment regimen changed over time?

How has your treatment regimen changed over time and why?

How well does your current treatment regimen treat any significant symptoms of your condition?

How well does your current treatment regimen treat the most significant symptoms of your disease?

Are there specific activities that are important to you but that you cannot do at all or as fully as you would like because of your condition?

Have these treatments improved your daily life (for example, improving your ability to do specific activities)?

How well have these treatments worked for you as your condition has changed over time?

How well have these treatments worked for you as your condition has changed over time?

What are the most significant downsides to your current therapies or treatments, and how do they affect your daily life?

What are the most significant downsides of these treatments (for example, specific side effects)?

Are there symptoms that your current regimen does not address at all, or does not treat as well as you would like?

Assuming there is currently no complete cure for your condition, what specific things would you look for in an ideal therapy or treatment to manage your condition?


But FDA also asks HIV patients a second set of questions, one more narrowly tailored to their views of and on research opportunities.

Patients' Perspectives on HIV Cure Research

What do you believe are the benefits of participating in an HIV cure research study?

What would motivate you to participate or to not participate in an HIV cure research study?

What risks would you find unacceptable for participating in an HIV cure research study, and why?

In certain HIV cure research studies, you would be asked to stop any other HIV medications that you are currently taking. How would this affect your decision whether to participate in an HIV cure research study?

The process of informed consent is an important way for the researchers to communicate the purpose of an HIV research study, as well as its expected benefits and potential risks, so that people can make an informed decision whether to participate in the study.

How should the informed consent clearly communicate to you the purpose of an HIV cure research study, particularly when a study is designed only to provide scientific information that could guide future research and development of treatments?

How should the informed consent clearly communicate to you the potential benefits of an HIV cure research study? In particular, how should the informed consent describe benefit when we do not think that participants in the study may gain any direct health benefits? 

How should informed consent communicate clearly to you the potential risks of participating in an HIV cure research study? In particular, how should the informed consent describe a study if there is very limited understanding about how the medications or interventions may affect participants or what are the potential risks of those interventions or medications?

Is there any other information that you would find helpful when deciding whether to enter an HIV cure research study?

What else do you want FDA to know about HIV Cure Research from your perspective?


These questions were not asked of CFS patients, presumably due to a lack of familiarity with the clinical trials process as a patient population. What remains to be seen is whether this second set of questions will be asked of all future patient populations, or will be a selectively asked of some-but not all-patients.

The meeting will be held on 14 June 2013 at FDA's White Oak Campus in Silver Spring, MD.


Federal Register: HIV Patient-Focused Drug Development Meeting


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