Rare Disease Clinical Outcome Assessment Consortium (U01 Clinical Trial Not Allowed)

A rare disease is defined by the Orphan Drug Act as a disease that affects less than 200,000 people in the US.

As described in FDA draft Guidance, "Rare Diseases:
Common Issues in Drug Development Guidance for Industry" (https://www.fda.gov/ucm/groups/fdagov-public/@fdagov-drugs-gen/documents/document/ucm45848

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5. pdf ), fit-for-purpose clinical endpoints for many rare diseases are not available.

Selection or development of clinical outcome assessments for use to support efficacy of a treatment in a rare disease can be challenging due to the small sample size of possible participants for participation in instrument development and clinical trials and heterogeneity of the target patient population (e.g., phenotypic or genotypic variations, age, clinical manifestations, variations in patient experience, and rate of disease progression).

However, many rare diseases share similar clinical characteristics such as decline in cognition and physical function, which offers an opportunity to explore clinical outcome assessments that may cover a spectrum of rare diseases.

This cooperative agreement will provide funding to establish a rare disease consortium focusing on clinical outcome assessments appropriate for use in drug development to demonstrate clinical benefit.

Many rare diseases include neurodegenerative decline resulting in loss of ability to perform daily activities.

Therefore, domains of interest include, but are not limited to, cognition, adaptive behavior, physical function (e.g., fine motor function, ambulation, speech, swallowing) and assessments of patients' ability to perform activities of daily living.

Special considerations that should be addressed include the fact that many rare diseases affect children as well as adults and trials are often multiregional, which necessitates attention to cross-cultural considerations.

The final outcome would be the creation of a common resource describing publicly available fit-for-purpose clinical outcome assessments as well as accompanying information, such as the populations for use and the strengths and limitations of each tool.

Work in the pre-competitive setting of a consortium where data is shared can enhance the development of this resource and, thus would help to address unmet measurement needs in rare disease drug development.

Related Programs

Food and Drug Administration Research

Department of Health and Human Services

Agency: Department of Health and Human Services

Office: Food and Drug Administration

Estimated Funding: $300,000

Who's Eligible

Relevant Nonprofit Program Categories

Obtain Full Opportunity Text:
Office of Postsecondary Education: Developing Hispanic-Serving Institutions (HSI) Program CFDA Number 84.031S-Individual Development Grants: Notice Inviting Applications for New Awards for Fiscal Year (FY) 2010:

Additional Information of Eligibility:
Eligible Applicants: Institutions of higher education (IHEs) that qualify as eligible HSIs are eligible to apply for new Individual Development Grants and Cooperative Arrangement Development Grants under the HSI Program.

To be an eligible HSI, an IHE must-- (1) Be accredited or preaccredited by a nationally recognized accrediting agency or association that the Secretary has determined to be a reliable authority as to the quality of education or training offered; (2) Be legally authorized by the State in which it is located to be a junior college or to provide an educational program for which it awards a bachelor's degree; (3) Be designated as an ``eligible institution'' by demonstrating that it: (A) Has an enrollment of needy students as described in 34 CFR 606.3; and (B) has low average educational and general expenditures per full-time equivalent (FTE) undergraduate student as described in 34 CFR 606.4; and (4) Have an enrollment of undergraduate FTE students that is at least 25 percent Hispanic students at the end of the award year immediately preceding the date of application.

The HSI eligibility requirements are in 34 CFR 606.2 through 606.5 and can be accessed from the following Web site: http:// www.access.gpo.gov/nara/cfr/waisidx_01/34cfr606_01.html.

These regulations do not reflect the changes made to the HSI Program requirements by the Third Higher Education Extension Act of 2006 or the HEOA.

Section 502(a) of the Higher Education Act of 1965, as amended (HEA), requires institutions applying for an HSI grant to report their undergraduate Hispanic FTE percentage at the end of the award year immediately preceding the date of application.

Funds for the HSI Program are awarded each fiscal year; thus, for this program, the end of the award year refers to the end of the fiscal year prior to the application due date.

The end of the fiscal year occurs on September 30 for any given year.

Therefore, for purposes of making the determination described in paragraph (4), IHEs must report their undergraduate Hispanic FTE percent based on the student enrollment count closest to, but not after, September 30, 2009.

Note: The Higher Education Opportunity Act of 2008 (HEOA) amended section 503(b) of the HEA to include, among the authorized activities under the HSI Program, activities to improve student services, including innovative and customized instruction courses designed to help retain students and move the students into core courses; articulation agreements and student support programs designed to facilitate the transfer of students from 2-year to 4-year institutions; and providing education, counseling services, and financial information designed to improve the financial and economic literacy of students or their families.

The list of authorized activities in section 503(b) was also amended to use the term ``distance education technologies'' in place of ``distance learning academic instruction capabilities.'' Applicants may include these activities in their proposals.

For this competition, the Notice Inviting Applications for Designation as Eligible Institutions for FY 2010 was published in the Federal Register on December 7, 2009 (74 FR 64059) and the deadline for application was January 6, 2010.

Only institutions that submitted the required application and received designation through that process are eligible to submit an application for this competition.

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