Regen BioPharma Inc (RGBP)

[JMS814]

FDA
Orphan Products Grants Program

FAQ Concerning the Orphan Products Grants

http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/WhomtoContactaboutOrphanProductDevelopment/ucm224956.htm


Who may apply for an Orphan Product Grant?

Orphan products 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 that are not part of the Department of Health and Human Services may also apply. For-profit entities must commit to excluding fees or profit in their request for support to receive grant awards. Organizations that engage in lobbying activities, as described in section 501(c)(4) of the Internal Revenue Code of 1968, are not eligible to receive grant awards.

What types of products qualify?
Products that qualify for this grant program are drugs, biologics, medical devices, and foods for medical purposes that are indicated (used) for a disease or condition that affects fewer than 200,000 people in the United States. Diagnostics and vaccines will qualify for the program only if the United States population to whom they will be administered is fewer than 200,000 people per year.

Must a product hold orphan drug designation in order to be eligible for the grant program?
A drug or biologic product does NOT have to hold orphan drug designation to be eligible for the grant program. The Background and Significance section of the application must contain information documenting the prevalence, not incidence, of the population to be served by the product is fewer than 200,000 individuals in the United States. The applicant should include a detailed explanation supplemented by authoritative references in support of the prevalence figure. Diagnostic tests and vaccines will qualify only if the population to whom they will be administered is fewer than 200,000 individuals in the United States per year.

The orphan drug designation process is the mechanism by which sponsors of drugs and biologics for rare diseases may qualify for incentives of the Orphan Drug Act such as tax credits and marketing exclusivity. Orphan designation is encouraged especially if the indication is questionable whether it would apply for orphan drug status.

What studies qualify for orphan product grants?
Only clinical studies qualify for consideration. Each application should propose one discrete clinical study designed to facilitate FDA approval of the product for use in a rare disease or condition. The study may address an unapproved new product or an unapproved new use for a product already on the market.

The goal of FDA's OPD grant program is to support the 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 clinical studies on safety and/or effectiveness that will either result in, or substantially contribute to, market approval of these products.

How much money is available for orphan product grants?
Of the estimated FY funding ($14.1 million), approximately $10 million will fund noncompeting continuation awards, and approximately $4.1 million will fund 5 to 10 new awards, subject to availability of funds.

Phase 1 studies are eligible for up to $250,000 per year for up to 3 years. Phase 2 and 3 studies are eligible for up to $500,000 per year for up to 4 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 $250,000 or $500,000 total cost limit, whichever is applicable.

How are applications reviewed? How are awards made?
FDA grants management and program staff will review all applications. To be responsive, an application must be submitted in accordance with the requirements of the Request for Applications (RFA). Applications found to be non-responsive will be returned to the applicant without further consideration.

Responsive applications will be reviewed and evaluated for scientific and technical merit by an ad hoc panel of experts in the subject field of the specific application. Consultation with the proper FDA review division may also occur during this phase of the review to determine whether the proposed study will provide acceptable data that could contribute to product approval. Responsive applications will be subject to a second review by the National Cancer Institute, National Cancer Advisory Board (NCAB) for concurrence with the recommendations made by the first-level reviewers, and funding decisions will be made by the Commissioner of Food and Drugs or his designee.

A score will be assigned to each application based on the scientific/technical review criteria. The review panel may advise the program staff about the appropriateness of the proposal to the goals of the OPD grant program.

Applications submitted will compete for available funds with all other recommended applications submitted in response to the RFA. The following will be considered in making funding decisions:

Scientific merit of the proposed project as determined by peer review.
Availability of funds.
Relevance of the proposed project to program priorities.
Do I need an Investigational New Drug Application (IND) or an Investigational Device Exemption (IDE) in order to qualify for an OOPD grant?
Yes, an IND/IDE is needed except for medical foods that do not need premarket approval and medical devices that are classified as non-significant risk (NSR). Applicants studying an NSR device should provide a letter in the application from FDA's Center for Devices and Radiological Health indicating the device is an NSR device.

Protocols involving an approved drug that would otherwise be eligible for an exemption from the IND regulations must be conducted under an IND in order to qualify for funding under this program. The proposed clinical protocol should be submitted to the applicable FDA IND/IDE review division a minimum of 30 days before the grant application deadline. Be sure to indicate in the IND cover letter that you are applying for an orphan product grant. In order to qualify for programmatic/scientific review, the study protocol proposed in the grant application must be under an active IND or IDE (i.e., not on clinical hold).

I have an IND/IDE for the product for another use - is this sufficient?
No. The clinical protocol that is being submitted in the grant application must be submitted to the appropriate FDA review division a minimum of 30 days before the grant application deadline.

Can the proposed study include research being performed at foreign sites?
Can all or part of the funding be used to support foreign clinical study sites?
Yes. 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 that are not part of the Department of Health and Human Services (HHS) may apply. Agencies that are part of HHS may not apply. For-profit entities must commit to excluding fees or profit in their request for support to receive grant awards. Organizations that engage in lobbying activities, as described in section 501(c)(4) of the Internal Revenue Code of 1968, are not eligible to receive grant awards.

Foreign applications must indicate how the proposed project has specific relevance to the mission and objectives of FDA and has the potential for significantly advancing sciences in the United States.

Do I need a Federal Wide Assurance (FWA) for all foreign and domestic study sites?
Yes. All institutions engaged in human subject research financially supported by HHS must file an assurance of protection for human subjects with the Office of Human Research Protections (OHRP) (45 CFR part 46). Applicants are advised to visit the OHRP Web site for guidance on human subject protection issues. Federal regulations (45 CFR 46) require that applications and proposals involving human subjects must be evaluated with reference to the risks to the subjects, the adequacy of protection against these risks, the potential benefits of the research to the subjects and others, and the importance of the knowledge gained or to be gained.

The requirement to file an assurance applies to both awardee and collaborating performance site institutions. Awardee institutions are automatically considered to be engaged in human subject research whenever they receive a direct HHS award to support such research, even where all activities involving human subjects are carried out by a subcontractor or collaborator. In such cases, the awardee institution bears the responsibility for protecting human subjects under the award.

The awardee institution is also responsible for, among other things, ensuring that all collaborating performance site institutions engaged in the research hold an approved assurance prior to their initiation of the research. No awardee or performance site institution may spend funds on human subject research or enroll subjects without the approved and applicable assurance(s) on file with OHRP. An awardee institution must, therefore, have its own IRB of record and assurance. The IRB of record may be an IRB already being used by one of the performance sites, but it must specifically be registered as the IRB of record with OHRP.

For further information, applicants should review the section on human subjects in the application instructions as posted on the Grants.gov application Web site. The clinical protocol should comply with ICHE6 Good Clinical Practice Consolidated Guidance which sets an international ethical and scientific quality standard for designing, conducting, recording, and reporting trials that involve the participation of human subjects. All human subject research regulated by FDA is also subject to FDA's regulations regarding the protection of human subjects (21 CFR parts 50 and 56). Applicants are encouraged to review the regulations, guidance, and information sheets on human subject protection and good clinical practice available on the Internet at Running Clinical Trials.

Do I need Institutional Review Board (IRB) approval at time of submission of grant application?
IRB approval is not required at the time of the submission of grant application although documentation of IRB approval for the IRB of record must be on file with the FDA grants management office before an award to fund the study will be made.

Should the protocol and informed consent forms be included in the grant application?
The protocol should be submitted in the application as an appendix. Informed consent and assent forms should be provided as an appendix as well. A draft form of the informed consent and assent documents are acceptable.

Who can I contact for questions concerning the budget of my potential grant application?
Questions regarding financial aspects of a proposed application should be addressed to Vieda Hubbard, Office of Acquisitions & Grant Services, 5630 Fishers Lane, (HFA-500), Room 2141, Rockville, MD 20857, 301-827-7177, e-mail: [e-mail].

Who can I contact for questions regarding the research/clinical protocol sections of my potential grant application?
Scientific and Research questions should be addressed to Katherine Needleman, Office of Orphan Products Development, Food and Drug Administration 10903 New Hampshire Avenue, WO32-5271, Silver Spring, MD 20993-0002, 301-796-8660, e-mail: [e-mail]

How and where do I submit my application?
All applications must be submitted electronically through Grants.gov. The applications must be prepared using the SF424 (R&R) application forms along with the SF424 (R&R) Application Guide for this FOA through http://www.grants.gov/applicants/apply_for_grants.