81 FR 26803 - Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01)
DEPARTMENT OF HEALTH AND HUMAN SERVICES
Food and Drug Administration Federal Register Volume 81, Issue 86 (May 4, 2016)
Food and Drug Administration
Federal Register Volume 81, Issue 86 (May 4, 2016)
| Page Range | 26803-26804 | |
| FR Document | 2016-10398 |
[Federal Register Volume 81, Number 86 (Wednesday, May 4, 2016)] [Notices] [Pages 26803-26804] From the Federal Register Online [www.thefederalregister.org] [FR Doc No: 2016-10398] ----------------------------------------------------------------------- DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA-2016-N-0012] Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01) AGENCY: Food and Drug Administration, HHS. ACTION: Notice. ----------------------------------------------------------------------- SUMMARY: The Food and Drug Administration (FDA) is announcing the availability of grant funds for the support of FDA's Office of Orphan Products Development (OOPD) Natural History Grants Program. The goal of the Orphan Products Natural History Grants Program is to support studies that advance rare disease medical product development through characterization of the natural history of rare diseases/conditions, identification of genotypic and phenotypic subpopulations, and development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics. The ultimate goal of these natural history studies is to support clinical development of products for use in rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. FDA provides grants for natural history studies that will either assist or substantially contribute to market approval of these products. Applicants must include in the application's Background and Significance section documentation to support that the estimated prevalence of the orphan disease or condition in the United States is less than 200,000 (or in the case of a vaccine or diagnostic, information to support that the product will be administered to fewer than 200,000 people in the United States per year), and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development. DATES: Important dates are as follows: 1. The application due dates are October 14, 2016 and October 15, 2018. 2. The anticipated start dates are March 2017 and March 2019. 3. The opening dates are August 15, 2016 and August 15, 2018. 4. The expiration date is October 16, 2018. ADDRESSES: Submit electronic applications to: http://www.grants.gov. For more information, see section III of the SUPPLEMENTARY INFORMATION section of this notice. FOR FURTHER INFORMATION AND ADDITIONAL REQUIREMENTS CONTACT: Katherine Needleman, Office of Orphan Products Development, Food and Drug Administration, 10903 New Hampshire Ave., Bldg. 32, Rm. 5295, Silver Spring, MD 20993-0002, 301-796-8660, email: [email protected]; or Daniel Lukash, Office of Acquisitions and Grant Services, 5630 Fishers Lane, Rockville, MD 20857, 240-402-7596, email: [email protected]. For more information on this funding opportunity announcement (FOA) and to obtain detailed requirements, please refer to the full FOA located at http://grants.nih.gov/grants/guide (select the ``Request for Applications'' link), http://www.grants.gov (see ``For Applicants'' section), and http://www.fda.gov/orphan. [[Page 26804]] SUPPLEMENTARY INFORMATION: I. Funding Opportunity Description RFA-FD-16-043 93.103 A. Background The OOPD was created to identify and promote the development of orphan products. Orphan products are drugs, biologics, medical devices, and medical foods that are indicated for a rare disease or condition. The term ``rare disease or condition'' is defined in section 528 of the Federal Food, Drug, and Cosmetic Act (21 U.S.C. 360ee). FDA generally considers drugs, devices, and medical foods potentially eligible for grants under the OOPD grant program if they are indicated for a disease or condition that has a prevalence, not incidence, of fewer than 200,000 people in the United States. Diagnostics and vaccines are considered potentially eligible for such grants only if the U.S. population to whom they will be administered is fewer than 200,000 people in the United States per year. The natural history of a disease is the natural course of a disease from the time immediately prior to its inception, progressing through its pre-symptomatic phase and different clinical stages to the point where the disease has ended without external intervention. Natural history studies track the course of disease over time, identifying demographic, genetic, environmental, and other variables that correlate with its development and outcomes in the absence of treatment. Thorough understanding of disease natural history is the foundation upon which a clinical development program for drugs, biologics, medical foods or medical devices is built. Rare diseases, as defined in the United States Orphan Drug Act (ODA) (Pub. L. 97-414), are diseases or conditions with a prevalence of fewer than 200,000 persons in the United States. Though individually rare, together there are approximately 30 million Americans affected by 7,000 known rare diseases. Unlike common diseases, there is little existing knowledge on the natural history of most rare diseases, which makes natural history studies of particular importance for rare diseases product development. In January 2014, the FDA organized a Public Workshop on Complex Issues in Developing Drugs for Rare Diseases. During the workshop, the lack of natural history studies was reconfirmed by all stakeholders (patients, industry, researchers and the FDA) as one of the most common and urgent issues that hinder treatment development for rare diseases. The need for natural history studies was also emphasized in the recently published (August 17, 2015) draft FDA Guidance for Industry, ``Rare Diseases: Common Issues in Drug Development,'' available at http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM458485.pdf. B. Research Objectives The objective of FDA's Orphan Products Natural History Grants Program is to support studies that characterize the natural history of rare diseases/conditions, identify genotypic and phenotypic subpopulations, and develop and/or validate clinical outcome measures, biomarkers and/or companion diagnostics. The ultimate goal of these natural history studies is to support clinical development of products for use in serious rare diseases or conditions where no current therapy exists or where the proposed product will be superior to the existing therapy. FDA provides grants for natural history studies that will either assist or substantially contribute to market approval of these products. Applicants must include in the application's Background and Significance section documentation to support that the estimated prevalence of the orphan disease or condition in the United States is less than 200,000 (or in the case of a vaccine or diagnostic, information to support that the product will be administered to fewer than 200,000 people in the United States per year), and an explanation of how the proposed study will either help support product approval or provide essential data needed for product development. C. Eligibility Information The grants are available to any foreign or domestic, public or private, for-profit or nonprofit entity (including State and local units of government). Federal Agencies may not apply. II. Award Information/Funds Available A. Award Amount Of the estimated FY 2017 funding ($17.7 million), approximately $2 million will fund 2 to 5 new awards, subject to availability of funds. Prospective Natural History Studies are eligible for grants of up to $400,000 per year for up to 5 years. Retrospective Natural History Studies or Surveys are eligible for grants of up to $150,000 per year for up to 2 years. Please note that the dollar limitation will apply to total costs (direct plus indirect). Budgets for each year of requested support may not exceed the $150,000 or $400,000 total cost limit, whichever is applicable. B. Length of Support The length of support will depend on the nature of the study. For those studies with an expected duration of more than 1 year, all future years of noncompetitive continuation of support will depend on the following factors: (1) Performance during the preceding year; (2) compliance with regulatory requirements as applicable; and (3) availability of Federal funds. III. Electronic Application, Registration, and Submission Only electronic applications will be accepted. To submit an electronic application in response to this FOA, applicants should first review the full announcement located at http://grants.nih.gov/grants/guide. For all electronically submitted applications, the following steps are required.Step 1: Obtain a Dun and Bradstreet (DUNS) Number Step 2: Register With System for Award Management (SAM) (formerly Central Contractor Registration (CCR)) Step 3: Obtain Username & Password on Grants.gov Step 4: Authorized Organization Representative (AOR) Authorization Step 5: Track AOR Status Step 6: Register With Electronic Research Administration (eRA) Commons Steps 1 through 5, in detail, can be found at http://www07.grants.gov/applicants/organization_registration.jsp. Step 6, in detail, can be found at https://commons.era.nih.gov/commons/registration/registrationInstructions.jsp. After you have followed these steps, submit electronic applications to: http://www.grants.gov. Dated: April 28, 2016. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2016-10398 Filed 5-3-16; 8:45 am] BILLING CODE 4164-01-P
![Federal Register / Vol. 81, No. 86 / Wednesday, May 4, 2016 / Notices 26803
the 1976 amendments (May 28, 1976) 510(k) is necessary to provide Program is to support studies that
(generally referred to as preamendments reasonable assurance of the safety and advance rare disease medical product
devices) have been classified by FDA effectiveness of a class II device. These development through characterization
under the procedures set forth in section factors are discussed in the guidance the of the natural history of rare diseases/
513(c) and (d) of the FD&C Act through Agency issued on February 19, 1998, conditions, identification of genotypic
the issuance of classification regulations entitled ‘‘Procedures for Class II Device and phenotypic subpopulations, and
into one of these three regulatory Exemptions from Premarket development and/or validation of
classes. Devices introduced into Notification, Guidance for Industry and clinical outcome measures, biomarkers
interstate commerce for the first time on CDRH Staff’’ (Ref. 1). and/or companion diagnostics. The
or after May 28, 1976 (generally referred ultimate goal of these natural history
III. Proposed Class II Device
to as postamendments devices), are studies is to support clinical
Exemptions
classified through the premarket development of products for use in rare
notification process under section FDA has received the following diseases or conditions where no current
510(k) of the FD&C Act (21 U.S.C. petition requesting an exemption from therapy exists or where the proposed
360(k)). Section 510(k) of the FD&C Act premarket notification for a class II product will be superior to the existing
and the implementing regulations, 21 device: Martin O’Connor, Germaine therapy. FDA provides grants for natural
CFR part 807, require persons who Laboratories, Inc., 11030 Wye Dr., San history studies that will either assist or
intend to market a new device to submit Antonio, TX 78217, for its Method, substantially contribute to market
a premarket notification (510(k)) Metallic Reduction, Glucose (urinary, approval of these products. Applicants
containing information that allows FDA non-quantitative) classified under 21 must include in the application’s
to determine whether the new device is CFR 862.1340. Background and Significance section
‘‘substantially equivalent’’ within the IV. Reference documentation to support that the
meaning of section 513(i) of the FD&C estimated prevalence of the orphan
Act to a legally marketed device that The following reference is on display
disease or condition in the United States
does not require premarket approval. in the Division of Dockets Management
is less than 200,000 (or in the case of a
On November 21, 1997, the President (see ADDRESSES) and is available for
vaccine or diagnostic, information to
signed into law FDAMA (Pub. L. 105– viewing by interested persons between
support that the product will be
115). Section 206 of FDAMA, in part, 9 a.m. and 4 p.m., Monday through
administered to fewer than 200,000
added a new section, 510(m), to the Friday; it is also available electronically
people in the United States per year),
FD&C Act. Section 510(m)(1) of the at http://www.regulations.gov. FDA has
and an explanation of how the proposed
FD&C Act requires FDA, within 60 days verified the Web site address, as of the
study will either help support product
after enactment of FDAMA, to publish date this document publishes in the
approval or provide essential data
in the Federal Register a list of each Federal Register, but Web sites are
needed for product development.
type of class II device that does not subject to change over time.
DATES: Important dates are as follows:
require a report under section 510(k) of 1. ‘‘Procedures for Class II Device 1. The application due dates are
the FD&C Act to provide reasonable Exemptions from Premarket Notification,
October 14, 2016 and October 15, 2018.
assurance of safety and effectiveness. Guidance for Industry and CDRH Staff,’’
February 1998, (http://www.fda.gov/
2. The anticipated start dates are
Section 510(m) of the FD&C Act further
downloads/MedicalDevices/Device March 2017 and March 2019.
provides that a 510(k) will no longer be
RegulationandGuidance/Guidance 3. The opening dates are August 15,
required for these devices upon the date
Documents/UCM080199.pdf). 2016 and August 15, 2018.
of publication of the list in the Federal 4. The expiration date is October 16,
Register. FDA published that list in the Dated: April 26, 2016. 2018.
Federal Register of January 21, 1998 (63 Leslie Kux, ADDRESSES: Submit electronic
FR 3142). Associate Commissioner for Policy.
Section 510(m)(2) of the FD&C Act applications to: http://www.grants.gov.
provides that 1 day after date of [FR Doc. 2016–10388 Filed 5–3–16; 8:45 am] For more information, see section III of
publication of the list under section BILLING CODE 4164–01–P the SUPPLEMENTARY INFORMATION section
510(m)(1), FDA may exempt a device on of this notice.
its own initiative or upon petition of an FOR FURTHER INFORMATION AND
DEPARTMENT OF HEALTH AND ADDITIONAL REQUIREMENTS CONTACT:
interested person if FDA determines
HUMAN SERVICES Katherine Needleman, Office of Orphan
that a 510(k) is not necessary to provide
reasonable assurance of the safety and Products Development, Food and Drug
Food and Drug Administration
effectiveness of the device. This section Administration, 10903 New Hampshire
requires FDA to publish in the Federal [Docket No. FDA–2016–N–0012] Ave., Bldg. 32, Rm. 5295, Silver Spring,
Register a notice of intent to exempt a MD 20993–0002, 301–796–8660, email:
Natural History Studies for Rare katherine.needleman@fda.hhs.gov; or
device, or of the petition, and to provide
Disease Product Development: Orphan Daniel Lukash, Office of Acquisitions
a 30-day comment period. Within 120
Products Research Project Grant (R01) and Grant Services, 5630 Fishers Lane,
days of publication of this document,
FDA must publish in the Federal AGENCY: Food and Drug Administration, Rockville, MD 20857, 240–402–7596,
Register its final determination HHS. email: daniel.lukash@fda.hhs.gov.
For more information on this funding
asabaliauskas on DSK3SPTVN1PROD with NOTICES
regarding the exemption of the device ACTION: Notice.
that was the subject of the notice. If FDA opportunity announcement (FOA) and
fails to respond to a petition under this SUMMARY: The Food and Drug to obtain detailed requirements, please
section within 180 days of receiving it, Administration (FDA) is announcing the refer to the full FOA located at http://
the petition shall be deemed granted. availability of grant funds for the grants.nih.gov/grants/guide (select the
support of FDA’s Office of Orphan ‘‘Request for Applications’’ link),
II. Criteria for Exemption Products Development (OOPD) Natural http://www.grants.gov (see ‘‘For
There are a number of factors FDA History Grants Program. The goal of the Applicants’’ section), and http://
may consider to determine whether a Orphan Products Natural History Grants www.fda.gov/orphan.
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![26804 Federal Register / Vol. 81, No. 86 / Wednesday, May 4, 2016 / Notices
SUPPLEMENTARY INFORMATION: diseases. The need for natural history exceed the $150,000 or $400,000 total
studies was also emphasized in the cost limit, whichever is applicable.
I. Funding Opportunity Description
recently published (August 17, 2015)
RFA–FD–16–043 B. Length of Support
draft FDA Guidance for Industry, ‘‘Rare
93.103 Diseases: Common Issues in Drug The length of support will depend on
Development,’’ available at http:// the nature of the study. For those
A. Background
www.fda.gov/downloads/Drugs/ studies with an expected duration of
The OOPD was created to identify and GuidanceComplianceRegulatory more than 1 year, all future years of
promote the development of orphan Information/Guidances/ noncompetitive continuation of support
products. Orphan products are drugs, UCM458485.pdf. will depend on the following factors: (1)
biologics, medical devices, and medical Performance during the preceding year;
foods that are indicated for a rare B. Research Objectives (2) compliance with regulatory
disease or condition. The term ‘‘rare The objective of FDA’s Orphan requirements as applicable; and (3)
disease or condition’’ is defined in Products Natural History Grants availability of Federal funds.
section 528 of the Federal Food, Drug, Program is to support studies that
and Cosmetic Act (21 U.S.C. 360ee). III. Electronic Application,
characterize the natural history of rare
FDA generally considers drugs, devices, Registration, and Submission
diseases/conditions, identify genotypic
and medical foods potentially eligible and phenotypic subpopulations, and Only electronic applications will be
for grants under the OOPD grant develop and/or validate clinical accepted. To submit an electronic
program if they are indicated for a outcome measures, biomarkers and/or application in response to this FOA,
disease or condition that has a companion diagnostics. The ultimate applicants should first review the full
prevalence, not incidence, of fewer than goal of these natural history studies is announcement located at http://
200,000 people in the United States. to support clinical development of grants.nih.gov/grants/guide. For all
Diagnostics and vaccines are considered products for use in serious rare diseases electronically submitted applications,
potentially eligible for such grants only or conditions where no current therapy the following steps are required.
if the U.S. population to whom they will exists or where the proposed product • Step 1: Obtain a Dun and Bradstreet
be administered is fewer than 200,000 will be superior to the existing therapy. (DUNS) Number
people in the United States per year. FDA provides grants for natural history • Step 2: Register With System for
The natural history of a disease is the Award Management (SAM) (formerly
studies that will either assist or
natural course of a disease from the time Central Contractor Registration (CCR))
substantially contribute to market
immediately prior to its inception, • Step 3: Obtain Username & Password
approval of these products. Applicants
progressing through its pre-symptomatic on Grants.gov
must include in the application’s
phase and different clinical stages to the • Step 4: Authorized Organization
Background and Significance section
point where the disease has ended Representative (AOR) Authorization
documentation to support that the
without external intervention. Natural • Step 5: Track AOR Status
estimated prevalence of the orphan
history studies track the course of • Step 6: Register With Electronic
disease or condition in the United States
disease over time, identifying Research Administration (eRA)
is less than 200,000 (or in the case of a
demographic, genetic, environmental, Commons
and other variables that correlate with vaccine or diagnostic, information to
its development and outcomes in the support that the product will be Steps 1 through 5, in detail, can be
absence of treatment. Thorough administered to fewer than 200,000 found at http://www07.grants.gov/
understanding of disease natural history people in the United States per year), applicants/organization_
is the foundation upon which a clinical and an explanation of how the proposed registration.jsp. Step 6, in detail, can be
development program for drugs, study will either help support product found at https://commons.era.nih.gov/
biologics, medical foods or medical approval or provide essential data commons/registration/
devices is built. needed for product development. registrationInstructions.jsp. After you
Rare diseases, as defined in the C. Eligibility Information have followed these steps, submit
United States Orphan Drug Act (ODA) electronic applications to: http://
(Pub. L. 97–414), are diseases or The grants are available to any foreign www.grants.gov.
conditions with a prevalence of fewer or domestic, public or private, for-profit
Dated: April 28, 2016.
than 200,000 persons in the United or nonprofit entity (including State and
local units of government). Federal Leslie Kux,
States. Though individually rare, Associate Commissioner for Policy.
together there are approximately 30 Agencies may not apply.
[FR Doc. 2016–10398 Filed 5–3–16; 8:45 am]
million Americans affected by 7,000 II. Award Information/Funds Available
BILLING CODE 4164–01–P
known rare diseases. Unlike common
diseases, there is little existing A. Award Amount
knowledge on the natural history of Of the estimated FY 2017 funding DEPARTMENT OF HEALTH AND
most rare diseases, which makes natural ($17.7 million), approximately $2 HUMAN SERVICES
history studies of particular importance million will fund 2 to 5 new awards,
for rare diseases product development. subject to availability of funds. Food and Drug Administration
In January 2014, the FDA organized a Prospective Natural History Studies are
asabaliauskas on DSK3SPTVN1PROD with NOTICES
Public Workshop on Complex Issues in eligible for grants of up to $400,000 per [Docket No. FDA–2016–N–0001]
Developing Drugs for Rare Diseases. year for up to 5 years. Retrospective Quantitative Assessment of
During the workshop, the lack of natural Natural History Studies or Surveys are Assumptions To Support Extrapolation
history studies was reconfirmed by all eligible for grants of up to $150,000 per of Efficacy in Pediatrics; Public
stakeholders (patients, industry, year for up to 2 years. Please note that Workshop
researchers and the FDA) as one of the the dollar limitation will apply to total
most common and urgent issues that costs (direct plus indirect). Budgets for AGENCY: Food and Drug Administration,
hinder treatment development for rare each year of requested support may not HHS.
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Document Created: 2016-05-04 01:23:01
Document Modified: 2016-05-04 01:23:01
| Category | Regulatory Information | |
| Collection | Federal Register | |
| sudoc Class | AE 2.7: GS 4.107: AE 2.106: | |
| Publisher | Office of the Federal Register, National Archives and Records Administration | |
| Section | Notices | |
| Action | Notice. | |
| Dates | Important dates are as follows: | |
| FR Citation | 81 FR 26803 |
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