Since the passage of the Food and Drug Administration Safety and Innovation Act (FDASIA) in 2012, the US Food and Drug Administration (FDA) has been publicly meeting with patients who suffer from debilitating conditions without adequate treatment options in the hopes of better understanding their wants and needs in the drug development process.
Now the agency is gearing up for its ninth patient-focused drug development meeting, this time focusing on the views of patients with idiopathic pulmonary fibrosis (IPF), a debilitating and often deadly disease which progressively attacks a patient's lungs.
The patient-centered drug development program was the brainchild of disease advocate groups, which convinced legislators to include it in FDASIA. Under Section X of the act, FDA is instructed to assess its risk-benefit decision making as it relates to disease areas where treatment options are less than optimal. Patients may be willing to accept more risk if the alternative to treatment is worse, proponents of the section hypothesized, and FDA was ordered to facilitate a program that assesses that tolerance for risk on a disease-wide level, instead of doing so on a piecemeal basis for individual products.
"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 noted in its Federal Register posting announcing the program.
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 might be less willing to accept high levels of risk in return for an effective treatment. Conversely, those suffering from a disease with few or even no recognized treatment options might be more willing than most to accept a drug that might either be less effective or more dangerous than 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 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.
New Meeting, Familiar Questions
Previously, FDA has announced meetings around eight different disease areas: chronic fatigue syndrome (CFS), HIV, lung cancer, narcolepsy, fibromyalgia, sickle cell disease (SCD), pulmonary arterial hypertension (PAH) and neurologic manifestations of inborn errors of metabolism.
The agency's ninth meeting, on IPF, reflects the severity of the disease, FDA said in a statement posted in the Federal Register.
"Many people with idiopathic pulmonary fibrosis survive only 3 to 5 years from the time of diagnosis," the agency explained. "There is no cure for IPF.”
As with other diseases under FDA's patient-focused drug development program, the agency has listed a number of questions it intends to ask of patients at its meeting.
But for the first time ever, all of the questions FDA says it will ask of IPF patients are substantially similar (or identical) to ones asked of patients affected by other diseases subject to prior patient-focused drug development meetings.
|FDA's Questions for IPF Patients|
|Of all the symptoms that you experience because of your condition, which one to three symptoms have the most significant impact on your life?|
|What are you currently doing to help treat your condition or its symptoms?|
|How do your symptoms and their negative impacts affect your daily life on the best days? |
|How do your symptoms and their negative impacts affect your daily life on the worst days?|
|What are the most significant downsides to your current treatments and how do they affect your daily life?|
|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?|
|How have your condition and its symptoms changed over time?|
|Because there is no complete cure for your condition, what specific things would you look for in an ideal treatment for your condition?|
The list of questions is also notable for its brevity. FDA is asking IPF patients just eight questions—the second fewest thus far, and just half the number asked of HIV and sickle cell disease (SCD) patients.
|Questions Asked of Each Group by FDA|
|CFS||PAH||Narcolepsy||Lung Cancer||Fibromyalgia||SCD||HIV||Inborn Errors of Metabolism||IPF|
The recent trend toward fewer questions asked at each meeting could be an aberration, but might also be the start of a trend in which FDA focuses more heavily on a core group of important questions for the purposes of its meeting.
That question will be put to the test over the next few years, as the agency has already announced it plans to hold eight more patient-focused drug development meetings focused on the following diseases:
- Alpha-1 antitrypsin deficiency
- Breast cancer
- Chronic Chagas disease
- Female sexual dysfunction
- Hemophilia A, Hemophilia B, von Willebrand disease, and other heritable bleeding disorders
- Irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors
- Neurological manifestations of inborn errors of metabolism
- Parkinson’s disease and Huntington’s disease
FDA's IPF meeting will take place on 26 September 2014 at FDA's White Oak Campus in Silver Spring, MD. Registration for the meeting closes on 10 September 2014.
Federal Register notice.