Natural History Studies for Rare Disease Product Development: Orphan Products Research Project Grant (R01), 26803-26804 [2016-10398]

Download as PDF asabaliauskas on DSK3SPTVN1PROD with NOTICES Federal Register / Vol. 81, No. 86 / Wednesday, May 4, 2016 / Notices the 1976 amendments (May 28, 1976) (generally referred to as preamendments devices) have been classified by FDA under the procedures set forth in section 513(c) and (d) of the FD&C Act through the issuance of classification regulations into one of these three regulatory classes. Devices introduced into interstate commerce for the first time on or after May 28, 1976 (generally referred to as postamendments devices), are classified through the premarket notification process under section 510(k) of the FD&C Act (21 U.S.C. 360(k)). Section 510(k) of the FD&C Act and the implementing regulations, 21 CFR part 807, require persons who intend to market a new device to submit a premarket notification (510(k)) containing information that allows FDA to determine whether the new device is ‘‘substantially equivalent’’ within the meaning of section 513(i) of the FD&C Act to a legally marketed device that does not require premarket approval. On November 21, 1997, the President signed into law FDAMA (Pub. L. 105– 115). Section 206 of FDAMA, in part, added a new section, 510(m), to the FD&C Act. Section 510(m)(1) of the FD&C Act requires FDA, within 60 days after enactment of FDAMA, to publish in the Federal Register a list of each type of class II device that does not require a report under section 510(k) of the FD&C Act to provide reasonable assurance of safety and effectiveness. Section 510(m) of the FD&C Act further provides that a 510(k) will no longer be required for these devices upon the date of publication of the list in the Federal Register. FDA published that list in the Federal Register of January 21, 1998 (63 FR 3142). Section 510(m)(2) of the FD&C Act provides that 1 day after date of publication of the list under section 510(m)(1), FDA may exempt a device on its own initiative or upon petition of an interested person if FDA determines that a 510(k) is not necessary to provide reasonable assurance of the safety and effectiveness of the device. This section requires FDA to publish in the Federal Register a notice of intent to exempt a device, or of the petition, and to provide a 30-day comment period. Within 120 days of publication of this document, FDA must publish in the Federal Register its final determination regarding the exemption of the device that was the subject of the notice. If FDA fails to respond to a petition under this section within 180 days of receiving it, the petition shall be deemed granted. II. Criteria for Exemption There are a number of factors FDA may consider to determine whether a VerDate Sep<11>2014 18:44 May 03, 2016 Jkt 238001 510(k) is necessary to provide reasonable assurance of the safety and effectiveness of a class II device. These factors are discussed in the guidance the Agency issued on February 19, 1998, entitled ‘‘Procedures for Class II Device Exemptions from Premarket Notification, Guidance for Industry and CDRH Staff’’ (Ref. 1). III. Proposed Class II Device Exemptions FDA has received the following petition requesting an exemption from premarket notification for a class II device: Martin O’Connor, Germaine Laboratories, Inc., 11030 Wye Dr., San Antonio, TX 78217, for its Method, Metallic Reduction, Glucose (urinary, non-quantitative) classified under 21 CFR 862.1340. IV. Reference The following reference is on display in the Division of Dockets Management (see ADDRESSES) and is available for viewing by interested persons between 9 a.m. and 4 p.m., Monday through Friday; it is also available electronically at https://www.regulations.gov. FDA has verified the Web site address, as of the date this document publishes in the Federal Register, but Web sites are subject to change over time. 1. ‘‘Procedures for Class II Device Exemptions from Premarket Notification, Guidance for Industry and CDRH Staff,’’ February 1998, (https://www.fda.gov/ downloads/MedicalDevices/Device RegulationandGuidance/Guidance Documents/UCM080199.pdf). Dated: April 26, 2016. Leslie Kux, Associate Commissioner for Policy. [FR Doc. 2016–10388 Filed 5–3–16; 8:45 am] BILLING CODE 4164–01–P 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. 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 SUMMARY: PO 00000 Frm 00031 Fmt 4703 Sfmt 4703 26803 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: https://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: katherine.needleman@fda.hhs.gov; or Daniel Lukash, Office of Acquisitions and Grant Services, 5630 Fishers Lane, Rockville, MD 20857, 240–402–7596, email: daniel.lukash@fda.hhs.gov. For more information on this funding opportunity announcement (FOA) and to obtain detailed requirements, please refer to the full FOA located at https:// grants.nih.gov/grants/guide (select the ‘‘Request for Applications’’ link), https://www.grants.gov (see ‘‘For Applicants’’ section), and https:// www.fda.gov/orphan. E:\FR\FM\04MYN1.SGM 04MYN1 26804 Federal Register / Vol. 81, No. 86 / Wednesday, May 4, 2016 / Notices SUPPLEMENTARY INFORMATION: asabaliauskas on DSK3SPTVN1PROD with NOTICES 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 VerDate Sep<11>2014 18:44 May 03, 2016 Jkt 238001 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 https:// www.fda.gov/downloads/Drugs/ GuidanceComplianceRegulatory Information/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 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 https:// 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 https://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: https:// 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 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 PO 00000 Frm 00032 Fmt 4703 Sfmt 4703 DEPARTMENT OF HEALTH AND HUMAN SERVICES Food and Drug Administration [Docket No. FDA–2016–N–0001] Quantitative Assessment of Assumptions To Support Extrapolation of Efficacy in Pediatrics; Public Workshop AGENCY: Food and Drug Administration, HHS. E:\FR\FM\04MYN1.SGM 04MYN1

Agencies

[Federal Register Volume 81, Number 86 (Wednesday, May 4, 2016)]
[Notices]
[Pages 26803-26804]
From the Federal Register Online via the Government Publishing Office [www.gpo.gov]
[FR Doc No: 2016-10398]

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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: https://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:
katherine.needleman@fda.hhs.gov; or Daniel Lukash, Office of
Acquisitions and Grant Services, 5630 Fishers Lane, Rockville, MD
20857, 240-402-7596, email: daniel.lukash@fda.hhs.gov.
For more information on this funding opportunity announcement (FOA)
and to obtain detailed requirements, please refer to the full FOA
located at https://grants.nih.gov/grants/guide (select the ``Request for
Applications'' link), https://www.grants.gov (see ``For Applicants''
section), and https://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 https://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 https://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 https://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: https://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

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