82 FR 42100 - Food and Drug Administration, Center for Drug Evaluation and Research Rare Diseases Public Workshop: Strategies, Tools, and Best Practices for Effective Advocacy in Rare Diseases Drug Development

DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration

Federal Register Volume 82, Issue 171 (September 6, 2017)

The Food and Drug Administration (FDA), Center for Drug Evaluation and Research (CDER), is sponsoring a public workshop entitled ``CDER Rare Diseases Public Workshop: Strategies, Tools, and Best Practices for Effective Advocacy in Rare Diseases Drug Development.'' This public workshop builds upon previous CDER patient advocacy public workshops and is primarily for the rare disease community to help them effectively understand what FDA needs to enhance drug development. This effort is consistent with FDA's efforts to support the integration of patient experience in drug development programs, including through implementation of the ``Patient-Focused Drug Development'' provisions of the 21st Century Cures Act (Cures Act). This public workshop will include case studies demonstrating the beneficial overlap of effective advocacy techniques and FDA regulations in rare disease drug development.

[Federal Register Volume 82, Number 171 (Wednesday, September 6, 2017)]
[Notices]
[Page 42100]
From the Federal Register Online  [www.thefederalregister.org]
[FR Doc No: 2017-18810]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2017-N-0001]


Food and Drug Administration, Center for Drug Evaluation and 
Research Rare Diseases Public Workshop: Strategies, Tools, and Best 
Practices for Effective Advocacy in Rare Diseases Drug Development

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public workshop.

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SUMMARY: The Food and Drug Administration (FDA), Center for Drug 
Evaluation and Research (CDER), is sponsoring a public workshop 
entitled ``CDER Rare Diseases Public Workshop: Strategies, Tools, and 
Best Practices for Effective Advocacy in Rare Diseases Drug 
Development.'' This public workshop builds upon previous CDER patient 
advocacy public workshops and is primarily for the rare disease 
community to help them effectively understand what FDA needs to enhance 
drug development. This effort is consistent with FDA's efforts to 
support the integration of patient experience in drug development 
programs, including through implementation of the ``Patient-Focused 
Drug Development'' provisions of the 21st Century Cures Act (Cures 
Act). This public workshop will include case studies demonstrating the 
beneficial overlap of effective advocacy techniques and FDA regulations 
in rare disease drug development.

DATES: The public workshop will be held on October 30, 2017, from 8 
a.m. to 5 p.m.

ADDRESSES: The public workshop will be held at FDA White Oak Campus, 
10903 New Hampshire Ave., Bldg. 31 (the Great Room), Silver Spring, MD 
20993. Entrance for the public workshop participants (non-FDA 
employees) is through Building 1 where routine security check 
procedures will be performed. For parking and security information, 
please refer to https://www.fda.gov/AboutFDA/WorkingatFDA/BuildingsandFacilities/WhiteOakCampusInformation/ucm241740.htm.

FOR FURTHER INFORMATION CONTACT: Francis Kalush, Center for Drug 
Evaluation and Research, Food and Drug Administration, 10903 New 
Hampshire Ave., Silver Spring, MD 20993-0002, 301-796-5429, [email protected]

SUPPLEMENTARY INFORMATION: FDA is announcing a public workshop entitled 
``CDER Rare Diseases Public Workshop: Strategies, Tools, and Best 
Practices for Effective Advocacy in Rare Diseases Drug Development.'' 
The purpose of the public workshop, consistent with FDA's broad effort 
to more comprehensively include patients' perspectives and experiences 
with a disease or condition in the drug development process, including 
through implementation of the ``Patient-Focused Drug Development'' 
provisions of the Cures Act, is to aid in bridging the gap between rare 
disease patients' stories and data needed to support drug development. 
This public workshop will include presentations on strategies, tools, 
and best practices on key aspects of rare diseases drug development and 
engaging with FDA. There will be an opportunity for questions and 
answers following each presentation.
    Registration: There is no registration fee to attend the public 
workshop. Early registration is recommended because seating is limited, 
and registration will be on a first-come, first-served basis. There 
will be no onsite registration. Persons interested in attending this 
public workshop must register online at https://www.fda.gov/Drugs/NewsEvents/ucm565398.htm before September 30, 2017. For those without 
internet access, please contact Francis Kalush (see FOR FURTHER 
INFORMATION CONTACT) to register.
    If you need special accommodations due to a disability, please 
contact Francis Kalush (see FOR FURTHER INFORMATION CONTACT) no later 
than October 23, 2017.
    Transcripts: A transcript of the public workshop will be available 
for review at the Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852, and 
on the internet at https://www.regulations.gov approximately 30 days 
after the public workshop. Transcripts will also be available in either 
hard copy or on CD-ROM, after submission of a Freedom of Information 
request. The Freedom of Information office address is available on the 
Agency's Web site at https://www.fda.gov.

    Dated: August 30, 2017.
Anna K. Abram,
Deputy Commissioner for Policy, Planning, Legislation, and Analysis.
[FR Doc. 2017-18810 Filed 9-5-17; 8:45 am]
 BILLING CODE 4164-01-P