Fit-for-Purpose Rare Disease Framework Initiative; Public Meeting; Request for Information and Comments

Issuing agencies

Abstract

The Food and Drug Administration (FDA, the Agency, or we) is announcing a public meeting entitled "Fit-for-Purpose Rare Disease Framework Initiative". Convened by the Reagan-Udall Foundation for, and in collaboration with FDA, the meeting will focus on developing a fit-for-purpose framework for the development and regulatory review of rare disease therapies. There will be an opportunity to request to provide public comments at the meeting. In addition, FDA is establishing a public docket to collect feedback on and input related to its Rare Disease Framework Initiative.

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<title>Federal Register, Volume 91 Issue 199 (Friday, October 16, 2026)</title>
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[Federal Register Volume 91, Number 199 (Friday, October 16, 2026)]
[Notices]
[Pages 65750-65752]
From the Federal Register Online via the Government Publishing Office [<a href="http://www.gpo.gov">www.gpo.gov</a>]
[FR Doc No: 2026-21142]


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DEPARTMENT OF HEALTH AND HUMAN SERVICES

Food and Drug Administration

[Docket No. FDA-2026-N-12214]


Fit-for-Purpose Rare Disease Framework Initiative; Public 
Meeting; Request for Information and Comments

AGENCY: Food and Drug Administration, HHS.

ACTION: Notice of public meeting; request for information and comments.

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SUMMARY: The Food and Drug Administration (FDA, the Agency, or we) is 
announcing a public meeting entitled ``Fit-for-Purpose Rare Disease 
Framework Initiative''. Convened by the Reagan-Udall Foundation for, 
and in collaboration with FDA, the meeting will focus on developing a 
fit-for-purpose framework for the development and regulatory review of 
rare disease therapies. There will be an opportunity to request to 
provide public comments at the meeting. In addition, FDA is 
establishing a public docket to collect feedback on and input related 
to its Rare Disease Framework Initiative.

DATES: The hybrid public meeting will be held on October 28, 2026, from 
1 to 5 p.m. Eastern Time, and will take place in-person and virtually. 
Either electronic or written comments must be submitted by December 11, 
2026. See the SUPPLEMENTARY INFORMATION section for registration 
information.

ADDRESSES: The hybrid public meeting will be held in-person at the FDA 
White Oak Campus, 10903 New Hampshire Ave., White Oak Conference 
Center, Bldg. 2, Rm. 2047, Silver Spring, MD 20993-0002, and virtually 
using the Zoom platform. Entrance for the public meeting participants 
(non-FDA employees) is through Building 1 where routine security check 
procedures will be performed. For security and parking information, 
please refer to <a href="https://www.fda.gov/about-fda/visitor-information">https://www.fda.gov/about-fda/visitor-information</a> and 
<a href="https://www.fda.gov/about-fda/visitor-information/visitor-parking-and-campus-map">https://www.fda.gov/about-fda/visitor-information/visitor-parking-and-campus-map</a>.
    You may submit comments as follows. Please note that late, untimely 
filed comments will not be considered. The <a href="https://www.regulations.gov">https://www.regulations.gov</a> 
electronic filing system will accept comments until 11:59 p.m. Eastern 
Time at the end of December 11, 2026. Comments received by mail/hand 
delivery/courier (for written/paper submissions) will be considered 
timely if they are received on or before that date.

Electronic Submissions

    Submit electronic comments in the following way:
    <bullet> Federal eRulemaking Portal: <a href="https://www.regulations.gov">https://www.regulations.gov</a>. 
Follow the instructions for submitting comments. Comments submitted 
electronically, including attachments, to <a href="https://www.regulations.gov">https://www.regulations.gov</a> 
will be posted to the docket unchanged. Because your comment will be 
made public, you are solely responsible for ensuring that your comment 
does not include any confidential information that you or a third party 
may not wish to be posted, such as medical information, your or anyone 
else's Social Security number, or confidential business information, 
such as a manufacturing process. Please note that if you include your 
name, contact information, or other information that identifies you in 
the body of your comments, that information will be posted on <a href="https://www.regulations.gov">https://www.regulations.gov</a>.
    <bullet> If you want to submit a comment with confidential 
information that you do not wish to be made available to the public, 
submit the comment as a written/paper submission and in the manner 
detailed (see ``Written/Paper Submissions'' and ``Instructions'').

[[Page 65751]]

Written/Paper Submissions

    Submit written/paper submissions as follows:
    <bullet> Mail/Hand Delivery/Courier (for written/paper 
submissions): Dockets Management Staff (HFA-305), Food and Drug 
Administration, 5630 Fishers Lane, Rm. 1061, Rockville, MD 20852.
    <bullet> For written/paper comments submitted to the Dockets 
Management Staff, FDA will post your comment, as well as any 
attachments, except for information submitted, marked and identified, 
as confidential, if submitted as detailed in ``Instructions.''
    Instructions: All submissions received must include the Docket No. 
FDA-2026-N-12214 for ``Fit-For-Purpose Rare Disease Framework; Public 
Meeting; Request for Information and Comments.'' Received comments, 
those filed in a timely manner (see ADDRESSES), will be placed in the 
docket and, except for those submitted as ``Confidential Submissions,'' 
publicly viewable at <a href="https://www.regulations.gov">https://www.regulations.gov</a> or at the Dockets 
Management Staff between 9 a.m. and 4 p.m., Monday through Friday, 240-
402-7500.
    <bullet> Confidential Submissions--To submit a comment with 
confidential information that you do not wish to be made publicly 
available, submit your comments only as a written/paper submission. You 
should submit two copies total. One copy will include the information 
you claim to be confidential with a heading or cover note that states 
``THIS DOCUMENT CONTAINS CONFIDENTIAL INFORMATION.'' The Agency will 
review this copy, including the claimed confidential information, in 
its consideration of comments. The second copy, which will have the 
claimed confidential information redacted/blacked out, will be 
available for public viewing and posted on <a href="https://www.regulations.gov">https://www.regulations.gov</a>. 
Submit both copies to the Dockets Management Staff. If you do not wish 
your name and contact information to be made publicly available, you 
can provide this information on the cover sheet and not in the body of 
your comments and you must identify this information as 
``confidential.'' Any information marked as ``confidential'' will not 
be disclosed except in accordance with 21 CFR 10.20 and other 
applicable disclosure law. For more information about FDA's posting of 
comments to public dockets, see 80 FR 56469, September 18, 2015, or 
access the information at: <a href="https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf">https://www.govinfo.gov/content/pkg/FR-2015-09-18/pdf/2015-23389.pdf</a>.
    Docket: For access to the docket to read background documents or 
the electronic and written/paper comments received, go to <a href="https://www.regulations.gov">https://www.regulations.gov</a> and insert the docket number, found in brackets in 
the heading of this document, into the ``Search'' box and follow the 
prompts and/or go to the Dockets Management Staff, 5630 Fishers Lane, 
Rm. 1061, Rockville, MD 20852, 240-402-7500.

FOR FURTHER INFORMATION CONTACT: Philipa Friedman, Rare Disease 
Innovation Hub, 202-940-6508, <a href="/cdn-cgi/l/email-protection#7200161b1c1c1d0413061b1d1c1a0710321416135c1a1a015c151d04"><span class="__cf_email__" data-cfemail="b5c7d1dcdbdbdac3d4c1dcdadbddc0d7f5d3d1d49bddddc69bd2dac3">[email&#160;protected]</span></a>.

SUPPLEMENTARY INFORMATION:

I. Background

    FDA is announcing the launch of its Rare Disease Framework 
Initiative (Framework Initiative) to develop a regulatory framework 
purpose-built for rare disease therapies. Roughly 1 in 10 Americans 
lives with a rare disease, and only approximately 5 percent of rare 
conditions have an FDA-approved treatment. Developing therapies for 
rare diseases can present scientific and practical challenges, 
including small patient populations, limited understanding of certain 
diseases, and disease heterogeneity. FDA's Framework Initiative will 
respond to these challenges via a holistic approach that approaches 
regulatory review according to the particularities of each product 
based on specific disease state, product type, patient population, and 
attainable evidence.
    The Framework Initiative--spearheaded by the Rare Disease 
Innovation Hub, the Office of the Commissioner, the Center for 
Biologics Evaluation and Research (CBER), and the Center for Drug 
Evaluation and Research (CDER)--will result in a rare disease drug 
development and review process that:
    1. Provides greater clarity, consistency, and predictability for 
drug developers, patients, disease advocates, and researchers regarding 
rare disease regulatory decision-making through tailored guidance and 
transparent communication based on the latest science.
    2. Embeds the patient voice both early and at clearly defined 
milestones in the development and review process for rare disease 
therapies such that both sponsors and reviewers can benefit from 
patient perspectives on disease burden, benefit-risk priorities, and 
meaningful measures of progress.
    3. Further leverages external expertise to support a tailored 
approach in regulatory review, especially for rare conditions with very 
small patient populations and limited understanding of natural history, 
as a structural feature to direct FDA reviewers in customizing their 
decision-making to each specific development program, product, and 
disease state.
    4. Builds shared resources, robust training, and institutional 
knowledge on rare disease drug development and review to ensure 
consistency in drug development and review.
    5. Applies risk-based approaches, particularly to chemistry, 
manufacturing, and controls review and pre-approval inspections, to 
support earlier patient access without compromising rigorous quality, 
safety, and effectiveness standards.
    6. Champions the use of cutting-edge science, novel methods and 
clinical trial designs, and innovative statistical approaches in the 
development of rare disease therapies via guidance, transparent 
communication, and programming that reflects FDA's commitment to 
partnering with rare disease innovators to accelerate patient access to 
rare disease therapies.

II. Issues for Consideration and Request for Information

    FDA is seeking feedback from the public--including rare disease 
drug developers, patients living with rare diseases and their 
caregivers, disease advocates, and researchers--on persistent 
challenges for the development of rare disease therapies, as well as 
input on how a tailored approach to regulatory review might mitigate 
these challenges. Feedback will inform development of the framework and 
potential implementation actions, which may include new or revised 
guidance documents related to rare disease drug development and 
regulatory review, revisions to FDA's regulations, training for FDA 
review staff, and organizational enhancements.
    This Request for Information and Comments is being published in 
conjunction with a public meeting on October 28, 2026, to solicit 
feedback on the Rare Disease Framework Initiative. Interested parties 
may provide feedback on FDA's Rare Disease Framework Initiative by 
requesting to provide public comments at the public meeting, providing 
comments in the public docket that will be open from October 28 through 
December 11, 2026, or both. Registration information for the October 
28, 2026, free public meeting, including information about how to 
request to provide public comments, can be found at the following web 
address: <a href="https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fda-rare-disease-framework-initiative-public-meeting-10282026">https://www.fda.gov/news-events/fda-meetings-conferences-and-workshops/fda-rare-disease-framework-initiative-public-meeting-10282026</a>. In-person attendance is based on space availability, with 
priority given to early registrants. Early

[[Page 65752]]

registration is recommended because seating is limited; therefore, FDA 
may limit the number of participants from each organization. 
Registrants will receive confirmation when they have been accepted. If 
you need special accommodations due to a disability, please email 
<a href="/cdn-cgi/l/email-protection#c0b2a4a9aeaeafb6a1b4a9afaea8b5a280a6a4a1eea8a8b3eea7afb6"><span class="__cf_email__" data-cfemail="e89a8c818686879e899c818786809d8aa88e8c89c680809bc68f879e">[email&#160;protected]</span></a> no later than October 26, 2026, 11:59 p.m. 
Eastern Time.
    FDA welcomes suggestions, ideas, and comments regarding CDER and 
CBER's roles in rare disease drug development and review. Below are 
four broad focus areas outlined in the Framework Initiative. We seek 
suggestions and comments within these topic areas and ask that you be 
as specific as possible in your feedback or recommendation. To help FDA 
review comments efficiently, please identify the topic area to which 
you are responding by its associated number. If you are responding to 
more than one topic area, please identify each topic area to which you 
are responding:
    1. Fit-for-purpose review of rare disease therapies based on 
attainable evidence, particular disease state, and population.
    2. Consistency and predictability of FDA's rare disease drug 
development and review regulatory requirements.
    3. FDA engagements with external parties--including patients, 
disease experts, and researchers--to inform review decision-making for 
rare disease therapies.
    4. Considerations for chemistry, manufacturing, and controls (CMC) 
specific to rare disease therapies.

Grace R. Graham,
Deputy Commissioner for Policy, Legislation, and International Affairs.
[FR Doc. 2026-21142 Filed 10-14-26; 11:15 am]
BILLING CODE 4164-01-P


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