Life Science Compliance Update

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April 25, 2013

FDA: Enhancing Benefit-Risk Assessment in Regulatory Decision Making

Last summer, Congress enacted the Food and Drug Administration Safety and Innovation Act (FDASIA), which included the fifth authorization of the Prescription Drug User Fee Act (PDUFA V).  Title I of FDASIA reauthorizes the Prescription Drug User Fee Act (PDUFA), which provides FDA with the necessary user fee resources to maintain an efficient review process for human drug and biologic products.  The reauthorization of PDUFA includes performance goals and procedures that represent FDA’s commitments during FY 2013-2017.  These commitments are referred to in section 101 of FDASIA.

Section X of these commitments relates to enhancing benefit-risk assessment in regulatory decision-making.  A key part of regulatory decision-making is establishing the context in which the particular decision is made.  For purposes of drug marketing approval, this includes an understanding of the severity of the treated condition and the adequacy of the available therapies.  Patients who live with a disease have a direct stake in the outcome of FDA's decisions and are in a unique position to contribute to the understanding of their disease.

FDA has committed to obtain the patient perspective on 20 disease areas during the course of PDUFA V.  For each disease area, the Agency will conduct a public meeting to discuss

  • The disease and its impact on patients' daily lives,
  • The types of treatment benefit that matter most to patients, and
  • Patients' perspectives on the adequacy of available therapies.  

These meetings will include participation of FDA review divisions, the relevant patient community, and other interested stakeholders.

In early April of this year, FDA announced the selection of the first 20 diseases.  FDA selected these disease areas based on a set of selection criteria, the perspectives of the reviewing divisions at FDA, and the public input received on a preliminary set of disease areas published in the Federal Register on September 24, 2012.  Almost 4,500 comments addressing over 90 disease areas were submitted by patients, patient advocates and advocacy groups, caregivers, healthcare providers, professional societies, scientific and academic experts, pharmaceutical companies, and others.

The extent of public comment for specific disease areas was one of many factors used to select the disease areas for Patient-Focused Drug Development during FY 2013-2015.  In selecting the disease areas of focus, FDA carefully considered the public comments received, the perspectives of reviewing divisions at FDA, and the following selection criteria, which were published in the September 24, 2012, Federal Register notice:

Disease areas that are chronic, symptomatic, or affect functioning and activities of daily living;

disease areas for which aspects of the disease are not formally captured in clinical trials; and

disease areas for which there are currently no therapies or very few therapies, or the available therapies do not directly affect how a patient feels or functions.

FDA’s selection also reflects the Agency’s desire to include a diverse set of disease areas that represent the wide range of diseases the Agency encounters in its regulatory decision-making.  These criteria, also published in the September 24, 2012, Federal Register notice, were overarching considerations that the Agency took into account in selecting the set of disease areas:

Disease areas that reflect a range of severity, from diseases that are life-threatening to those that are mild and symptomatic;

Disease areas that have a severe impact on identifiable subpopulations, such as children or the elderly; and

Disease areas that represent a broad range in terms of size of the affected population, including common conditions experienced by large numbers of patients and rare diseases that affect much smaller patient populations.

Patient-Focused Drug Development was conceived as a mechanism to learn more from patients where their perspectives could be helpful to drug development and FDA's review of applications for new drugs in certain disease areas. For FDA's review divisions, this kind of input is most helpful when the impact of a disease on patients is not well understood or endpoints for studying drugs for a disease are not clearly defined or established. The potential to fill these information gaps by hearing from patients was also a key consideration in identifying the initial 12 disease areas.

FDA selected the following diseases to be addressed in FY 2013-2015 (meeting date):

  • Alpha-1 antitrypsin deficiency;
  • breast cancer;
  • chronic Chagas disease;
  • female sexual dysfunction;
  • fibromyalgia;
  • hemophilia A, hemophilia B, von Willebrand disease, and other heritable bleeding disorders;
  • HIV (June 14, 2013)
  • idiopathic pulmonary fibrosis;
  • irritable bowel syndrome, gastroparesis, and gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors;
  • lung cancer (June 28, 2013)
  • myalgic encephalomyelitis/chronic fatigue syndrome (April 25, 2013)
  • narcolepsy (TBD, approx. September 2013)
  • neurological manifestations of inborn errors of metabolism;
  • Parkinson's disease and Huntington's disease;
  • pulmonary arterial hypertension; and
  • sickle cell disease.

A schedule of the meetings planned for each year can be found at the FDA Patient-Focused Drug Development Web site described in the following section of this notice.

FDA will initiate a second public process to determine the list of disease areas for FY 2016-2017.  FDA recognized that there are many more disease areas than can be addressed in the planned FDA meetings under PDUFA V, and it will seek other opportunities to gather public input on disease areas not addressed through this PDUFA V commitment.

FDA also encouraged stakeholders to identify and organize patient-focused collaborations to generate public input on other disease areas with regard to the types of questions addressed through this PDUFA commitment, using the process established through Patient-Focused Drug Development as a model.  More information on other opportunities for gathering patient input can be found on the Patient-Focused Drug Development Web site.

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Risk assessment affects any industry and training is important.

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