[Federal Register Volume 78, Number 70 (Thursday, April 11, 2013)]
[Notices]
[Pages 21613-21614]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2013-08441]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2012-N-0967]


Prescription Drug User Fee Act Patient-Focused Drug Development; 
Announcement of Disease Areas for Meetings Conducted in Fiscal Years 
2013-2015

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of Availability.

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SUMMARY: The Food and Drug Administration (FDA) is announcing the 
selection of disease areas to be addressed during the first 3 years of 
Patient-Focused Drug Development. This 5-year initiative is being 
conducted to fulfill FDA's performance commitments made as part of the 
fifth authorization of the Prescription Drug User Fee Act (PDUFA V). It 
provides a more systematic approach for the Agency to obtain patients' 
input on specific disease areas, including their perspectives on their 
condition, its impact on daily life, and available therapies. 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.

ADDRESSES: The general schedule of fiscal years (FY) 2013-2015 meetings 
concerning Patient-Focused Drug Development, information on how 
stakeholders can prepare for them, and information on how stakeholders 
may leverage Patient-Focused Drug Development to generate input on 
disease areas that are not addressed through the PDUFA V commitments 
can be found at the Web site for Patient-Focused Drug Development: 
http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm.

FOR FURTHER INFORMATION CONTACT: Graham Thompson, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Bldg. 51, rm. 1199, Silver Spring, MD 20993, 301-796-
5003, FAX: 301-847-8443, Email: [email protected]; or Stephen 
Ripley, Center for Biologics Evaluation and Research (HFM-17), Food and 
Drug Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 
20852-1448, 301-827-6210.

SUPPLEMENTARY INFORMATION:

I. Background

    On July 9, 2012, the President signed into law the Food and Drug 
Administration Safety and Innovation Act (FDASIA) (Pub. L. 112-144). 
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 and are available 
on the FDA Web site at http://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM270412.pdf.
    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.

II. Disease Area Selection

    On September 24, 2012, FDA published a Federal Register notice (77 
FR 58849) that announced an opportunity for public comment on potential 
disease areas to be addressed throughout PDUFA V. In that notice, based 
on several criteria listed therein, FDA identified 39 disease areas as 
potential candidates for 20 public meetings and invited public comment 
on the preliminary list and on disease areas that were not listed. The 
Agency obtained public comment through a docket and a public meeting 
convened on October 25, 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 
majority of comments were submitted by individual patients. The 
comments generally focused on one or more of the following: Nominations 
of support for individual disease areas or groups of disease areas, 
general suggestions for Patient-Focused Drug Development, and topics 
outside the scope of the program. Many comments discussed the impact of 
the disease on daily life and the symptoms that were most concerning to 
patients. Others addressed lack of treatment options or the nature of 
specific treatments. Over half of the comments received concerned lung 
cancer, narcolepsy, and interstitial lung disease. Other disease areas 
also received a significant number of comments, including migraine, 
pulmonary fibrosis, amyloidosis, myalgic encephalomyelitis/chronic 
fatigue syndrome, amyotrophic lateral sclerosis, chronic obstructive 
pulmonary disease, lysosomal storage disorders, peripheral neuropathy, 
dystonia, and fibromyalgia. Comments were received for numerous other 
disease areas not listed in this notice. Individual comments may be 
viewed at http://www.regulations.gov/#!docketDetail;D=FDA-2012-N-0967, 
or by visiting FDA Dockets Management at 5630 Fishers Lane, rm. 1061, 
HFA-305, Rockville, MD 20852.
    Input from the public was particularly helpful for FDA in better 
understanding the aspects of diseases that are not formally measured in 
clinical trials as

[[Page 21614]]

well as cases where available therapies do not directly impact the 
aspects of disease that matter most to patients. 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 has selected the following diseases to be addressed in FY 2013-
2015:
     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;
     idiopathic pulmonary fibrosis;
     irritable bowel syndrome, gastroparesis, and 
gastroesophageal reflux disease with persistent regurgitation symptoms 
on proton-pump inhibitors;
     lung cancer;
     myalgic encephalomyelitis/chronic fatigue syndrome;
     narcolepsy;
     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. The Agency recognizes that there are 
many more disease areas than can be addressed in the planned FDA 
meetings under PDUFA V, and FDA will seek other opportunities to gather 
public input on disease areas not addressed through this PDUFA V 
commitment. FDA also encourages 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.

III. Patient-Focused Drug Development Web site

    FDA has a Web site on Patient-Focused Drug Development: http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm326192.htm. 
This Web site contains the general schedule of upcoming meetings for FY 
2013-2015, information on how stakeholders can prepare for upcoming 
meetings, and information on how stakeholders may leverage Patient-
Focused Drug Development to generate input on disease areas not 
addressed through the Patient-Focused Drug Development PDUFA V 
commitment. The Web site will be updated as new information becomes 
available.

    Dated: April 5, 2013.
Leslie Kux,
Assistant Commissioner for Policy.
[FR Doc. 2013-08441 Filed 4-10-13; 8:45 am]
BILLING CODE 4160-01-P