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FAQs for Orphan Products Grant Applicants Home
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          FAQs for Orphan Products Grant ApplicantsFor Industry FAQs for Orphan Products Grant Applicants
  	
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Orphan Product Grants FAQs

Please view the appropriate program sections to see the most commonly asked questions with further details on the application process and grant requirements.

Clinical Trials
	General Information, Eligibility, and Requirements
	Application Review Process and Timelines
	Post-Award Responsibilities
	Contact Information
	Natural History Studies
	General Information, Eligibility, and Requirements
	Application Review Process and Timelines
	Post-Award Responsibilities
	Contact InformationClinical Trials

General Information, Eligibility, and Requirements

What is the application submission deadline for the Clinical Trials Grant?
	See Funding opportunities for rare disease research for upcoming receipt dates.

Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date. Applicants should be aware that on-time submission means that an application is submitted error free (of both Grants.gov and eRA Commons errors) **by 11:59 PM Eastern Time on the application submission due date**.

Is the Letter of Intent Required?
	A Letter of Intent is not required, is non-binding, and does not enter into the review of a subsequent application. However, the information that it contains allows FDA staff to estimate the potential review workload and plan the review. No responsiveness decision will be made based on the letter of intent.

Prospective applicants are asked to submit the letter of intent with information contained in the RFA by 11:59 PM Eastern Time at least 30 days before the application submission deadline.

Who may apply for an Orphan Products Clinical Trials Grant?
	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.

How much money is available?
	Of the estimated fiscal year funding for clinical trial grants (approximately $15 million), approximately $10 million will fund noncompeting continuation (e.g., ongoing) awards, and approximately $5 million will fund new awards, subject to availability of funds.

Application budgets are limited to $650,000 per year in total costs (direct and indirect costs) for up to a maximum of four years of support. Detailed budgets for each requested year need to reflect the actual needs of the proposed project including both direct and indirect costs.

Applicants may request additional funding over the above listed maximum for innovative and efficient trial approaches. The additional funding request shall not exceed an additional $250,000 total costs per year (to a maximum total award cost of $900,000 per year) for up to four years. Justification for the additional funding request must be reflected in the budget request and will be reviewed annually by the program. Applications requesting additional funding for innovative and efficient trial approaches must submit a clear description and justification as an appendix (limited to three pages).

How many years of funding are available?
	The scope of the proposed project should determine the project period. The maximum project period is 4 years. However, the length of support will depend on the nature of the study.

For those studies with an expected duration of more than 1 year, a second, third, or fourth year of noncompetitive continuation of support will depend on the following factors: (1) Performance during the preceding year; (2) compliance with regulatory requirements of investigational new drug (IND)/investigational device exemption (IDE), if applicable; and (3) availability of Federal funds.

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. (Please Note: Applications may be considered for the use of a product in an orphan subset of a non-rare disease or condition when the applicant can explain based on a characteristic or feature of the product (e.g., mechanism of action, toxicity profile, prior clinical experience) why the product will be limited to use in the subset of question. An orphan subset is not based on an unmet need, or how a sponsor may wish to study or indicate a product. The explanation for the orphan subset must make it clear to FDA that the product would not be appropriate in the disease or condition outside of the subset). For studies proposing assessing multiple rare diseases, supportive prevalence data for each rare disease is required.

Must a product hold orphan drug designation to be eligible for the grant program?
	No. A drug or biologic product does NOT have to hold orphan drug designation to be eligible for the grant program. However, 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 drug designation is encouraged, especially if there is a question as to whether the proposed disease or condition would be eligible for orphan drug status or in cases where the estimated prevalence of the population is close to 200,000.

The Rationale Section of the Research Strategy should include documentation to support that the estimated prevalence of the orphan disease or condition in the United States is less than 200,000. In the case of an acute disease (i.e., less than 1 year duration), the annual incidence of the disease must be less than 200,000 per year; 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). For studies proposing assessing multiple rare diseases, supportive prevalence data for each rare disease is required. *If a designation by the Office of Orphan Products Development has been received by the institution submitting the grant for the drug for the disease subject to the grant, the designation number and date of designation should be provided in this section.*

Applications may be considered for the use of a product in an orphan subset of a non-rare disease or condition when the applicant can explain based on a characteristic or feature of the product (e.g., mechanism of action, toxicity profile, prior clinical experience) why the product will be limited to use in the subset of question. An orphan subset is not based on an unmet need, or how a sponsor may wish to study or indicate a product. The explanation for the orphan subset must make it clear to FDA that the product would not be appropriate in the disease or condition outside of the subset, including pediatric subpopulations.

The applicant should include a detailed explanation supplemented by authoritative references in support of the prevalence figure. However, if the calculations submitted in the application were not adequate and FDA has questions that are not easily resolved during the initial review of the application, FDA may recommend an official Orphan Drug Designation application to resolve the issues prior to review of the grant application.

What studies qualify for orphan products clinical trials grants?
	Only clinical trials of products evaluating efficacy and/or safety in support of a new indication or change in labeling of products to address unmet needs in rare diseases or conditions will qualify.

This funding opportunity announcement  is intended to support clinical trials of orphan products in all phases of product development (phase 1, 2 and/or 3) for rare diseases. Depending on the phase of development, these trials may need to include an appropriate comparator, such as a placebo, a concurrent external control, or a historical control.  Additionally, to address the remaining unmet need and the lack of treatments for the majority of rare diseases, FDA is focusing their funding efforts to facilitate and move new therapies along in drug development in safe yet efficient means by encouraging the use of established infrastructure and resources (e.g., clinical trial networks and data standardization, analytics, and sharing platforms), collaborative efforts between stakeholders (e.g., industry/academia/patient organizations), and early and ongoing patient engagement in trial design (e.g., study feasibility, assessment of important clinical outcomes).

To facilitate efficient product development, the use of shared, established infrastructure and resources and collaborative efforts between stakeholders in industry, academia and patient organizations are highly encouraged. Additionally, patients living with a rare disease or caregivers, have experiences and knowledge that contribute to important considerations in product development, such as with trial feasibility, thus early and ongoing patient engagement is also highly encouraged.

Through the funding of collaborative, efficient, and/or innovative clinical trials, FDA expects to increase the number of treatments for rare diseases with an unmet medical need and exert a broad and positive impact on rare disease drug development.

Do I need an Investigational New Drug Application (IND) or an Investigational Device Exemption (IDE) to qualify for an Orphan Products Clinical Trials grant?
	Yes, the purpose of this RFA is to fund clinical trials in support of a new indication or change in labeling of products to address unmet needs in rare diseases or conditions. The study protocol proposed in the grant application (including studies of already approved products evaluating new orphan indications) must comply with the applicable regulations in 21 CFR 312 for drugs and biologics and 21 CFR 812 for devices, with the exception noted above. An active (e.g., non-exempt and non-hold) IND/IDE is required, 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 need to provide a letter in the grant application from FDA's Center for Devices and Radiological Health indicating the device is an NSR device.

The final version of the protocol that is submitted with the grant application must be the protocol that was submitted to the applicable FDA IND/IDE review division. This protocol should have been submitted to the FDA review divisions **a minimum of 30 days**before the grant application deadline. Be sure to indicate in the IND cover letter to the review division your intent of applying for an FDA Office of Orphan Products Development Clinical Trials grant and how the study will be used for future marketing approval and product development. This will allow the review division to assess the IND. To qualify for programmatic/scientific review, the study protocol proposed in the grant application must be deemed as safe to proceed under an active IND or IDE (i.e., not on clinical hold and not exempt).

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 for the product for the disease/condition being studied through the proposed trial must be submitted to the appropriate FDA review division a minimum of 30 days before the grant application deadline.

Do I have to be the sponsor of the IND/IDE?
	If you are not the sponsor on the IND/IDE, then you must have a formal collaborative agreement with the company or individual who is the official sponsor of the IND and should include documentation of such a relationship in your grant application.

Can the proposed study include research being performed at foreign 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).
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 science 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 theOffice 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 for multi-site research must, therefore establish a single IRB of record and assurance. The single IRB of record may be an IRB already being used by one of the performance sites, but it must specifically be registered as the single 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 part 50 and 21 CFR part 56). Applicants are encouraged to review the regulations, guidance, and information sheets on human subject protection and good clinical practice. For further information, applicants should review the section on human subjects in the application instructions of the RFA.

Do I need Institutional Review Board (IRB) approval at time of submission of grant application?
	No. IRB approval is not required at the time of the submission of grant application. However, documentation of IRB approval for the single 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?
	Yes. The protocol should be submitted in the application as an appendix. Informed consent and assent forms should be provided as an appendix as well.

How, when 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 funding opportunity as well as any program-specific instructions. Please see the current RFAfor receipt dates and information on how to apply.

The earliest submission date for the RFA is 60 days prior to the application submission deadline. A letter of intent is due (not required) a minimum of 30 days prior to the application due date. Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date.

Applicants should be aware that on-time submission means that an application is submitted error free (of both Grants.gov and eRA Commons errors) by 11:59 PM Eastern Time on the application due date. Late applications will not be accepted for this funding opportunity.

**Application Review Process and Timelines**

How are applications reviewed?
	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 RFA. Applications found to be non-responsive will be returned to the applicant without further consideration.

Responsive applications will be peer reviewed and evaluated for scientific and technical merit by a panel of experts in the subject field of the specific application. Consultation with the relevant 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. 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 grant program.

What criteria are considered for the application review?
	Reviewers will provide an overall impact score to reflect their assessment of the likelihood for the project to exert a sustained, powerful influence on the research field(s) involved, in consideration of the following review criteria and additional review criteria (as applicable for the project proposed).

Reviewers will consider each of the review criteria below in the determination of scientific merit as described in the RFA.

1. Rationale

2. Study Design

3. Inclusion of Patient Input

4. Investigator(s), Infrastructure and Financial Resources

5. Ability to Advance the Current Field

In addition, reviewers will evaluate the following additional items while determining scientific and technical merit:

Budget and Period of Support
	Protections for Human Subject
	Inclusion of Women, Minorities, and Individuals Across the Lifespan
	Resource Sharing Plan
	Foreign Organizations
	BiohazardsSee the RFA for further information.

When will I be notified of the review results?
	Applicants will usually be notified of the review results via a summary statement approximately 6 months from the application deadline.

How and when are grant funds distributed?
	Once the grant applications are reviewed and scored by the ad hoc panel of experts, a cut-off score for funding will be established. Those applications within the anticipated fundable range will be ranked in order of their score with the earliest possible funding start date as specified in the RFA based on availability of funds.

**Post-Award Responsibilities**

What happens after the grant review is completed?
	All responsive applications will be issued scores and summary statements following the review of the applications. If your grant application received a favorable/competitive score you will be sent a cover memo and a Pre-Funding Certification Form (PCF) along with a copy of the Summary Statement. You will be required to complete and return the PCF and accompanying information (including your responses to the Summary Statement critiques, the current IRB approval letter, Federal Wide Assurance documentation and verification of product availability) by the date specified in the cover memo. This is not a guarantee that you will receive funding.

Who will be my main contact within the Office of Orphan Products Development (OOPD) when I receive grant funding?
	If you receive grant funding, you will receive a formal Notice of Grant Award. Your grant will be assigned an OOPD grant Project Officer (PO) who will be your main contact. You will be required to keep the PO informed throughout the grant of any issues and changes including request for approval of protocol changes by OOPD and the appropriate FDA Review Division under which the IND or IDE for the study is held, adverse events, changes in key study personnel, etc. If you have any questions or concerns about the grant or the study, you may contact the PO for assistance. Additionally, if you experience any difficulties in patient enrollment, OOPD may be able to assist or suggest options.

After I receive grant funding, what can I expect as a grantee?
	You will receive a congratulatory letter from the PO outlining your roles, responsibilities, and requirements as a grantee. You will be required to follow and be in compliance with Good Clinical Practices and Current Good Manufacturing Practices. You will be required to maintain regulatory requirements such as IRB approvals, up to date FWA, IND requirements including submission of IND annual reports and appropriate adverse event reporting, up to date clinicaltrials.gov information (for applicable or voluntarily registered trials), etc.

The program project officer will oversee grantees' activities periodically through telephone conversations, e-mails, or written correspondences. To assist in monitoring your grant, your PO will establish enrollment and progress goals for each funding year with you upon initial funding. You will also be required to submit Quarterly Reports and Annual Reports to FDA’s Office of Orphan Products Development (OOPD) on information such as study progress, enrollment, problems, changes in protocol, and study oversight activities. There will be at least one grant evaluation with officials of the grantee organization during the lifetime of your grant to ensure extramural funded studies, which involve human subjects, are consistent with grant agreement terms and human subjects’ protection requirements. To ensure that funded studies support the long-term goal of product approval, regulatory milestone meetings will be initiated as needed. OOPD should be contacted before any protocol changes are made (including Key Personnel and Performance Site changes). Publication of study results is encouraged.

In addition, OOPD may hold periodic interactive webinars as part of the Orphan Grantees Unite Initiative to connect grantees and provide a forum to share useful information.

What reports will be required once my grant is funded?
	**Quarterly Reports**will be required typically every three months from the date the grant was issued. Information including, but not limited to, information regarding study progress, enrollment, problems, adverse events, changes in protocol, study monitoring activities, new collaborations, publications, financial and data leveraging, and changes in clinical guidelines based on the project will be requested. FDA may request information related to the impact of this study on future approvals and other outcomes such as publications or data leveraging. For “applicable clinical trials” and voluntarily registered trials, updates on the ClinicalTrials.gov entry for the study will be requested.

**Research Performance Progress Report (RPPR)s** are required yearly for grants more than 1 year in length. These reports will include items such as progress made, budget information, updates on any revisions made, accomplishments, future goals, publications/presentations resulting from the award, and personnel updates.

A**Final Report**will be due within 90 days after the end date of the project period. Items that are needed include a Final Financial Status Report, Final Invention Statement Certification, and Final Performance/Progress report.

**Contact Information**

Who can I contact for questions concerning the budget of my potential grant application or funded grant?
	Questions regarding financial aspects of a proposed application and a funded grant should be addressed to:

Shashi Malhotra, FDA/Office of Acquisitions & Grant Services

Telephone: 240-402-7592

E-mail: Shashi.Malhotra@fda.hhs.gov

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, FDA/Office of Orphan Products Development

Telephone: 301-796-8660

E-mail: Katherine Needleman

Who can I contact for questions regarding technical issues with submitting the grant application?
	Technical questions regarding submitting through grants.gov should be submitted to:

eRA Service Desk (Questions regarding ASSIST, eRA Commons registration, submitting and tracking an application, documenting system problems that threaten submission by the due date, post submission issues)

o   Finding Help Online: Need Help? | eRA (nih.gov)

o   Telephone: 301-402-7469 or 866-504-9552 (Toll Free)

Grants.gov Customer Support (Questions regarding Grants.gov registration and submission, downloading forms and application packages)

o   Contact Center Telephone: 800-518-4726

o   Email: support@grants.gov

Natural History Studies

General Information, Eligibility, and Requirements

What is the application submission deadline for the Natural History Grant?
	See Funding opportunities for rare disease research for upcoming receipt dates.

Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date. **Applicants should be aware that on-time submission means that an application is submitted error free** (of both Grants.gov and eRA Commons errors)**by 11:59 PM Eastern Time on the application submission due date**.

Is the Letter of Intent Required?
	A Letter of Intent is not required, is non-binding, and does not enter into the review of a subsequent application. However, the information that it contains allows FDA staff to estimate the potential review workload and plan the review. No responsiveness decision will be made based on the letter of intent.
Prospective applicants are asked to submit the letter of intent with information contained in the RFA by 11:59 PM Eastern Time at least 30 days before the application submission deadline.

Who may apply for an Orphan Products Natural History Grant?
	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.

How much money is available for the Orphan Products Grants Program Natural History Grants?
	Funding is dependent on the availability of federal funds. FDA intends to commit approximately $2 million to natural history grants per fiscal year.

Application budgets need to reflect the actual needs of the proposed project and should not exceed the following in maximum total costs (direct and indirect) and maximum years of support.

For prospectiveNatural History Studies application budgets are limited to $400,000 per year in total costs (direct and indirect costs) for up to a maximum of four years of support.

For retrospectiveNatural History Studies application budgets are limited to $400,000 per year in total costs (direct and indirect costs) for up to a maximum of two years of support.

How many years of funding are available?
	The scope of the proposed project should determine the project period. For prospective Natural History Studies the maximum project period is four (4) years. For retrospective Natural History Studies the maximum project period is two(2)years.

For those studies with an expected duration of more than 1 year, a second, third, or fourth year of noncompetitive continuation of support will depend on the following factors: (1) Performance during the preceding year; (2) compliance with regulatory requirements of investigational new drug (IND)/investigational device exemption (IDE), if applicable; and (3) availability of Federal funds.

What types of studies qualify?
	This funding opportunity announcement (FOA) is intended to support efficient and innovative prospective and retrospective natural history studies that advance medical product development in rare diseases/conditions with unmet needs. Through the support of natural history studies with high quality and interpretable data elements, FDA expects to address critical knowledge gaps, remove major barriers to progress in the field, exert a significant and broad impact on a specific rare disease or multiple rare diseases with similar pathophysiology, and facilitate rare disease product development.

Areas of interest for the purpose of this FOA) as outlined in more detail in the RFA are the ability to address unmet needs pertinent to stage of product development, inclusion of high quality and interpretability of the data, use of data standards, use of existing infrastructure and resources and collaboration among stakeholders in industry, academia and patient organizations, inclusion of patient engagement, ability to exert a broad impact in advancing multiple rare diseases sharing a similar pathophysiology, inclusion of future plans for data dissemination and collaborative use of data.  FDA encourages applicants to refer to the "Rare Diseases: Natural History Studies for Drug Development, Guidance for Industry" for guidance on the conduct of a natural history study, considerations to enhance interpretability of study results, and discussion on data collection standards and data quality and integrity.

The Rationale Section of the Research Strategy should also include a subsection with the heading “Rare Disease Prevalence.” This subsection should include documentation to support the estimated prevalence of the rare disease or condition in the United States is fewer than 200,000. For studies proposing assessing multiple rare diseases, supportive prevalence data for each rare disease is required.

Additional information may be required upon request, for example, regarding population estimate and rationale. This additional information may be required, in part, to assure that natural history studies are eligible to receive funding under the Orphan Products Grants Program.

Do I need an Investigational New Drug Application (IND) or an Investigational Device Exemption (IDE) to qualify for an Orphan Products Natural History grant?
	No. An IND or IDE is not required for a natural history study to be qualified. However, if an IND or IDE is associated with the natural history study, we highly recommend that the IND or IDE number be provided in the application.

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). 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 theOffice 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 for multi-site research must establish a single IRB of record and assurance. The single IRB of record may be an IRB already being used by one of the performance sites, but it must specifically be registered as the single 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.

Do I need Institutional Review Board (IRB) approval at time of submission of grant application?
	No. IRB approval is not required at the time of the submission of grant application. However, documentation of IRB approval for the single 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?
	Yes. The protocol should be submitted in the application as an appendix. Informed consent and assent forms should be provided as an appendix as well.

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 funding opportunity as well as any program-specific instructions. Please see the current RFAfor receipt dates and information on how to apply.

The earliest submission date for the RFA is 60 days prior to the application submission deadline. A letter of intent is due (not required) a minimum of 30 days prior to the application due date. Applicants are encouraged to apply early to allow adequate time to make any corrections to errors found in the application during the submission process by the due date.

Applicants should be aware that on-time submission means that an application is submitted error free (of both Grants.gov and eRA Commons errors) by 11:59 PM Eastern Time on the application due date. Late applications will not be accepted for this funding opportunity.

**Application Review Process and Timelines**

How are applications reviewed?
	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 RFA. Applications found to be non-responsive will be returned to the applicant without further consideration. Responsive applications will be peer reviewed and evaluated for scientific and technical merit by a panel of experts in the subject field of the specific application. Consultation with the relevant 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. 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 grant program.

What criteria are considered for the application review?
	Reviewers will provide an overall impact score to reflect their assessment of the likelihood for the project to exert a sustained, powerful influence on the research field(s) involved, in consideration of the following review criteria and additional review criteria (as applicable for the project proposed).

Reviewers will consider each of the review criteria below in the determination of scientific merit as described in the RFA.

1. Rationale

2. Study Design/Data Quality and Interpretability

3. Inclusion of Patient Input

4. Investigator(s)

5. Infrastructure and Financial Resources

6. Ability to Advance the Current Field

In addition, reviewers will evaluate the following additional items while determining scientific and technical merit:

Budget and Period of Support
	Protections for Human Subject
	Inclusion of Women, Minorities, and Individuals Across the Lifespan
	Resource Sharing Plan
	Foreign Organizations
	Biohazards
	ResubmissionsSee the RFA for further information.

When will I be notified of the review results?
	Applicants will usually be notified of the review results via a summary statement approximately 6 months from the application deadline.

How and when are grant funds distributed?
	Once the grant applications are reviewed and scored by the ad hoc panel of experts, a cut-off score for funding will be established. Those applications within the anticipated fundable range will be ranked in order of their score with the earliest possible funding start date as specified in the RFA based on availability of funds.

**Post-Award Responsibilities**

What happens after the grant review is completed?
	All responsive applications will be issued scores and summary statements following the review of the applications. If your grant application received a favorable/competitive score you will be sent a cover memo and a Pre-Funding Certification Form (PCF) along with a copy of the Summary Statement. You will be required to complete and return the PCF and accompanying information (including your responses to the Summary Statement critiques, the current IRB approval letter, Federal Wide Assurance documentation and verification of product availability) by the date specified in the cover memo. This is not a guarantee that you will receive funding.

Who will be my main contact within the Office of Orphan Products Development (OOPD) when I receive grant funding?
	If you receive grant funding, you will receive a formal Notice of Grant Award. Your grant will be assigned an OOPD grant Project Officer (PO) who will be your main contact. You will be required to keep the PO informed throughout the grant of any issues and changes including request for approval of protocol changes by OOPD and the appropriate FDA Review Division under which the IND or IDE for the study is held, adverse events, changes in key study personnel, etc. If you have any questions or concerns about the grant or the study, you may contact the PO for assistance. Additionally, if you experience any difficulties in patient enrollment, OOPD may be able to assist or suggest options.

After I receive grant funding, what can I expect as a grantee?
	You will receive a congratulatory letter from the PO outlining your roles, responsibilities, and requirements as a grantee. You will be required to follow and be in compliance with Good Clinical Practices and Current Good Manufacturing Practices. You will be required to maintain regulatory requirements such as IRB approvals, up to date FWA, IND requirements including submission of IND annual reports and appropriate adverse event reporting, up to date clinicaltrials.gov information (for applicable or voluntarily registered trials), etc.

The program project officer will oversee grantees' activities periodically through telephone conversations, e-mails, or written correspondences. To assist in monitoring your grant, your PO will establish enrollment and progress goals for each funding year with you upon initial funding. You will also be required to submit Quarterly Reports and Annual Reports to OOPD to update OOPD on information such as study progress, enrollment, problems, changes in protocol, and study oversight activities. There will be at least one grant evaluation (teleconference or on-site) with officials of the grantee organization during the lifetime of your grant to ensure extramural funded studies, which involve human subjects, are consistent with grant agreement terms and human subjects’ protection requirements. To ensure that funded studies support the long-term goal of product approval, regulatory milestone meetings will be initiated as needed. OOPD should be contacted before any protocol changes are made (including Key Personnel and Performance Site changes). Publication of study results is encouraged.

In addition, OOPD may hold periodic interactive webinars as part of the Orphan Grantees Unite Initiative to connect grantees and provide a forum to share useful information.

What reports will be required once my grant is funded?
	**Quarterly Reports**will be required typically every three months from the date the grant was issued. Information including, but not limited to, information regarding study progress, enrollment, problems, adverse events, changes in protocol, study monitoring activities, new collaborations, publications, financial and data leveraging, and changes in clinical guidelines based on the project will be requested. OOPD may request information related to the impact of this study on future approvals and other outcomes such as publications or data leveraging. For “applicable clinical trials” and voluntarily registered trials, updates on the ClinicalTrials.gov entry for the study will be requested.

**Research Performance Progress Report (RPPR)s** are required yearly for grants more than 1 year in length. These reports will include items such as progress made, budget information, updates on any revisions made, accomplishments, future goals, publications/presentations resulting from the award, and personnel updates.

A**Final Report**will be due within 90 days after the end date of the project period. Items that are needed include a Final Financial Status Report, Final Invention Statement Certification, and Final Performance/Progress report.

**Contact Information**

Who can I contact for questions concerning the budget of my potential grant application or funded grant?
	Questions regarding financial aspects of a proposed application and a funded grant should be addressed to:

Shashi Malhotra, FDA/Office of Acquisitions & Grant Services

Telephone: 240-402-7592

E-mail: Shashi.Malhotra@fda.hhs.gov

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, FDA/Office of Orphan Products Development

Telephone: 301-796-8660

E-mail: Katherine Needleman

Who can I contact for questions regarding technical issues with submitting the grant application?
	Technical questions regarding submitting through grants.gov should be submitted to:

eRA Service Desk (Questions regarding ASSIST, eRA Commons registration, submitting and tracking an application, documenting system problems that threaten submission by the due date, post submission issues)

o   Finding Help Online: Need Help? | eRA (nih.gov)

o   Telephone: 301-402-7469 or 866-504-9552 (Toll Free)

Grants.gov Customer Support (Questions regarding Grants.gov registration and submission, downloading forms and application packages)

o   Contact Center Telephone: 800-518-4726

o   Email: support@grants.gov

                                                                                  Content current as of:
      02/24/2023FAQs for Orphan Products Grant Applicants
 
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