Fund Discovery Research

Overview

Patient groups often choose to raise money to support research or seek other funding sources from the government or larger nonprofits. Some groups form their own grant program, where researchers submit a proposal and a scientific review committee recommends which projects to fund. Another option is to team up with other organizations that have overlapping interests to support mutually beneficial projects.

Funding Research

Your group can help fund academic research or start-up medical research companies directly to ensure that relevant research for your disease is pursued. Funding discovery research directly can occur in several ways.

  • Fundraise: Your group can raise money to fund research through crowdsourcing, special events, group fundraisers, and other endeavors.
  • Offer research grants: Research grants usually do not include any stake in intellectual property or future revenues for a therapy developed through the funded research.  
    • Adjust the grant size offered to the abilities of your group. Sizes of grants can vary:
      • Microgrants can be a few thousand dollars.
      • Small grants can range from $25,000 to $50,000.
      • Large grants can be multi-million dollars.
    • Stimulate research interest by awarding the grant money through a Request for Proposals (RFPs) or Request for Applications (RFAs). Your Scientific/Medical Advisory Board can help determine the winning RFPs/RFAs. Grants offered in this way can:
      • Support researchers who already have a research project in progress.
      • Encourage researchers to propose and then pursue new research ideas.
      • Entice new investigators in the field to become involved in your rare disease.
    • Offer a grant directly to a leading researcher in the field to pursue a project that fills a research gap identified by your group and scientific advisors.
    • Consider channeling the grant through larger umbrella organizations if you are uncertain whether your group has the time, experience, or energy to manage the process. For example:
    • List your grant programs with rare disease centers and organizations. For example:
  • Invest through venture philanthropy: Although usually requiring a large sum of money, venture philanthropy provides your group with an opportunity to fund the early, often risky stages of therapy development with for-profit companies. 
    • These investments usually involve millions of dollars.
    • You can fund start-up medical research companies or programs within larger pharmaceutical companies willing to focus on developing a treatment or cure for your disease(s).
    • Your group will gain an equity stake (a percentage) in the company or royalty rights of the developed therapy. 
    • Since profit is not the goal, if a therapy is successfully developed, equity stakes or royalty rights can be sold to raise money for future ventures. 
    • Venture philanthropy may also allow your group high levels of engagement with the company and/or program, thus enabling your group to provide patient perspective during the discovery stage.  
    • Due to the financial risk involved, it is important to work with your Scientific/Medical Advisory Board to determine whether the investment is a wise choice for your group’s efforts. You may also wish to consult with a business lawyer.
    • The Cystic Fibrosis Foundation pioneered the venture philanthropy model and shares their journey on the following webpage:  CF Foundation Venture Philanthropy Model.
  • Other resources: 

Federal Funding Opportunities

Your group can help researchers find funding opportunities for discovery research at the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA).

  • NIH Exploratory/Developmental Research Grant Award (R21) is available for research that may be too high-risk for other types of federal grants. 
    • Intended to fund exploratory, studies that break new ground or extend previous discoveries toward new directions or applications, as well as high-risk high-reward studies that may lead to a breakthrough in a particular area.
    • Non-renewable grant funding up to $275,000 over a 2 year period with no more than $200,000 in 1 year. 
    • Applications can be submitted 3 times per year. Check the Application Due Dates
    • Interested investigators are encouraged to contact participating NIH Institutes and Centers (ICs) prior to applying to make certain the project and scope are a good fit for the R21: Contacts and Special Interests
    • Non-participating ICs may offer R21 grant opportunities through specific funding announcements which would be listed on the individual IC websites and on the NIH Grants and Fundings Find Funding Search Tool (check R21 in Activity Code to narrow your search results).  
  • Americas Seed Funds offered by NIH ICs allow U.S.-owned and operated small businesses to engage in federal research and development that has a strong potential for commercialization. 
    • The programs are broken into Three Phase Programs:
      • Phase I: Feasibility and Proof of Concept
      • Phase II: Research/Research and Development
      • Phase III: Commercialization
    • The NIH offers special technical assistance programs to help small businesses in all 3 phases of the programs.
    • Small Business Innovation Research (SBIR) program funds early stage small businesses that are seeking to commercialize innovative biomedical technologies.
    • Small Business Technology Transfer (STTR) program is similar to the NIH SBIR program, but requires that small business to formally collaborate with a research institution in Phase I and Phase II.
    • The SBIR|STTR America Seed Fund website provides information about the differences between the grants and how to apply.
    • It is important to contact the NIH SBIR/STTR Program Officer for the specific IC prior to submitting an application: HHS SBIR/STTR Agency Contact Information
  • Parent Announcements (For Unsolicited or Investigator-Initiated Applications) lists other broad funding opportunities.  
    • Research grants include the R Series, K99/00, and U01: Types of Research Grants
    • Not all NIH ICs participate in all parent announcements, so it is important to check the ICs that may be associated with your disease about other possible  research grant opportunities.
  • Find Funding Search Tool within NIH Grants and Fundings lists all available grant opportunities. The filters on the left of the page narrow the search results. 
  • The National Center for Advancing Translational Sciences (NCATS) provides support for research projects, core facilities, scientific resources and tools, scientific conferences, and collaboration opportunities for small businesses, and other partners as well as grants programs and in-kind services: Funding and Notices
  • NCATS has additional programs listed in Discovery: Understand Discovery Research Tools.  
  • Other NIH resources:
    • NIH Tips for Applicants (2010) is a short video containing tips for grant applicants from reviewers and staff at the National Institutes of Health.
    • NIH Grants YouTube Page (updated periodically) is a page containing videos published by the NIH about a variety of topics related to NIH grants. There is a short Welcome Video on the homepage that introduces the organization of the content. 
    • NIH Grants Process Overview (updated periodically) is a webpage providing the steps required for the planning and submission of an application through to award and closeout.
  • The Office of Orphan Products Development (OOPD), a program of the FDA, offers 2 grants:
    • OOPD Pediatric Device Consortia Grant funds consortia capable of providing expert advising and support services to innovators of children’s devices through all stages of development. 
      • Specific areas of expertise provided by the consortia include:
        • Intellectual property advising
        • Prototyping
        • Engineering
        • Laboratory and animal testing
        • Grant-writing
        • Clinical trial design.
      • List of current Pediatric Device Consortia Websites
    • Orphan Products Natural History Grants support targeted studies that advance rare disease therapy development through characterization of:
      • The natural history of rare diseases/conditions.
      • Identification of genotypic and phenotypic subpopulations.
      • Development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics.

Industry Partnerships

Your group can form partnerships with a pharmaceutical company (industry) and/or facilitate formation of a larger partnership between key stakeholders, including your group. 

  • Strategic partnerships are traditionally collaborations between businesses with a common mission. However, many large pharmaceutical companies are beginning to build strategic partnerships for the development of therapies for rare diseases with academia, small businesses, and even groups like your own.  
    • The overall goal of strategic partnerships is to share resources in a way that promotes growth for all partners.
    • Choose a partnership with an  expertise different from your own. For example, if your researchers have the need to develop an animal model to test a promising therapy, find a strategic partner that has the resources to develop that model.
    • Since a strategic partnership involves contracts, if your group is going to establish a strategic partnership make certain your Board of Directors, Scientific/Medical Advisory Board, and possibly a business lawyer are involved in the process. 
    • Alternatively, you can help academic researchers or start-up medical research companies establish strategic partnerships by searching for possible industry partners. 
  • Venture funds are another way large pharmaceutical companies are investing in the early stages of rare disease therapy development. 
    • Venture funds however tend to support start-up medical research companies, rather than academic researchers or patient groups. 
    • Usually involve a high level of engagement with representatives from the large pharmaceutical company leading or co-leading the investment and playing an active role on company boards.

Tips for Success

  • Check with other group leaders who have run successful fundraising programs, grant programs, or formed strategic partnerships early in the discovery phase of therapy development. 
  • Search for research funding that may be available through special programs in your state.
  • Think  outside the box, network, and stay alert for emerging innovative companies focused on rare disease research or branches of large philanthropic organizations newly supporting the development of therapies for rare diseases. 
  • Persistence is key. Successfully finding funding can rely on:
    • Timing 
    • Positioning
    • Luck

Resources

Funding Research
Fundraising National Organization for Rare Disorders (NORD) (link)
NORD Research Grant Program Funding National Organization for Rare Disorders (NORD) (link)
GARD Guide for Researchers: Finding Funding Opportunities Genetics and Rare Diseases Information Center (GARD) (link)
NORD Additional Funding National Organization for Rare Disorders (NORD) (link)
CF Foundation Venture Philanthropy Model Cystic Fibrosis (CF) Foundation (link)
Federal Funding Opportunities
Grants and Funding Application Due Dates National Institutes of Health (NIH) (link)
R21 Contacts and Special Interests National Institutes of Health (NIH) (link)
Find Funding: NIH Guide for Grants and Contracts National Institutes of Health (NIH) (link)
SBIR|STTR America Seed Fund National Institutes of Health (NIH) (link)
HHS SBIR/STTR Agency Contact Information National Institutes of Health (NIH) (link)
NIH Types of Research Grants National Institutes of Health (NIH) (link)
NCATS Funding and Notices National Center for Advancing Translational Sciences (NCATS) (link)
NIH Tips for Applicants National Institutes of Health (NIH) (link)
NIH Grants YouTube Page National Institutes of Health (NIH) (link)
NIH Grants Process Overview National Institutes of Health (NIH) (link)
OOPD Pediatric Device Consortia Grants U.S. Food and Drug Administration (FDA) (link)

Patient groups often choose to raise money to support research or seek other funding sources from the government or larger nonprofits. Some groups form their own grant program, where researchers submit a proposal and a scientific review committee recommends which projects to fund. Another option is to team up with other organizations that have overlapping interests to support mutually beneficial projects.

Your group can help fund academic research or start-up medical research companies directly to ensure that relevant research for your disease is pursued. Funding discovery research directly can occur in several ways.

  • Fundraise: Your group can raise money to fund research through crowdsourcing, special events, group fundraisers, and other endeavors.
  • Offer research grants: Research grants usually do not include any stake in intellectual property or future revenues for a therapy developed through the funded research.  
    • Adjust the grant size offered to the abilities of your group. Sizes of grants can vary:
      • Microgrants can be a few thousand dollars.
      • Small grants can range from $25,000 to $50,000.
      • Large grants can be multi-million dollars.
    • Stimulate research interest by awarding the grant money through a Request for Proposals (RFPs) or Request for Applications (RFAs). Your Scientific/Medical Advisory Board can help determine the winning RFPs/RFAs. Grants offered in this way can:
      • Support researchers who already have a research project in progress.
      • Encourage researchers to propose and then pursue new research ideas.
      • Entice new investigators in the field to become involved in your rare disease.
    • Offer a grant directly to a leading researcher in the field to pursue a project that fills a research gap identified by your group and scientific advisors.
    • Consider channeling the grant through larger umbrella organizations if you are uncertain whether your group has the time, experience, or energy to manage the process. For example:
    • List your grant programs with rare disease centers and organizations. For example:
  • Invest through venture philanthropy: Although usually requiring a large sum of money, venture philanthropy provides your group with an opportunity to fund the early, often risky stages of therapy development with for-profit companies. 
    • These investments usually involve millions of dollars.
    • You can fund start-up medical research companies or programs within larger pharmaceutical companies willing to focus on developing a treatment or cure for your disease(s).
    • Your group will gain an equity stake (a percentage) in the company or royalty rights of the developed therapy. 
    • Since profit is not the goal, if a therapy is successfully developed, equity stakes or royalty rights can be sold to raise money for future ventures. 
    • Venture philanthropy may also allow your group high levels of engagement with the company and/or program, thus enabling your group to provide patient perspective during the discovery stage.  
    • Due to the financial risk involved, it is important to work with your Scientific/Medical Advisory Board to determine whether the investment is a wise choice for your group’s efforts. You may also wish to consult with a business lawyer.
    • The Cystic Fibrosis Foundation pioneered the venture philanthropy model and shares their journey on the following webpage:  CF Foundation Venture Philanthropy Model.
  • Other resources: 

Your group can help researchers find funding opportunities for discovery research at the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA).

  • NIH Exploratory/Developmental Research Grant Award (R21) is available for research that may be too high-risk for other types of federal grants. 
    • Intended to fund exploratory, studies that break new ground or extend previous discoveries toward new directions or applications, as well as high-risk high-reward studies that may lead to a breakthrough in a particular area.
    • Non-renewable grant funding up to $275,000 over a 2 year period with no more than $200,000 in 1 year. 
    • Applications can be submitted 3 times per year. Check the Application Due Dates
    • Interested investigators are encouraged to contact participating NIH Institutes and Centers (ICs) prior to applying to make certain the project and scope are a good fit for the R21: Contacts and Special Interests
    • Non-participating ICs may offer R21 grant opportunities through specific funding announcements which would be listed on the individual IC websites and on the NIH Grants and Fundings Find Funding Search Tool (check R21 in Activity Code to narrow your search results).  
  • Americas Seed Funds offered by NIH ICs allow U.S.-owned and operated small businesses to engage in federal research and development that has a strong potential for commercialization. 
    • The programs are broken into Three Phase Programs:
      • Phase I: Feasibility and Proof of Concept
      • Phase II: Research/Research and Development
      • Phase III: Commercialization
    • The NIH offers special technical assistance programs to help small businesses in all 3 phases of the programs.
    • Small Business Innovation Research (SBIR) program funds early stage small businesses that are seeking to commercialize innovative biomedical technologies.
    • Small Business Technology Transfer (STTR) program is similar to the NIH SBIR program, but requires that small business to formally collaborate with a research institution in Phase I and Phase II.
    • The SBIR|STTR America Seed Fund website provides information about the differences between the grants and how to apply.
    • It is important to contact the NIH SBIR/STTR Program Officer for the specific IC prior to submitting an application: HHS SBIR/STTR Agency Contact Information
  • Parent Announcements (For Unsolicited or Investigator-Initiated Applications) lists other broad funding opportunities.  
    • Research grants include the R Series, K99/00, and U01: Types of Research Grants
    • Not all NIH ICs participate in all parent announcements, so it is important to check the ICs that may be associated with your disease about other possible  research grant opportunities.
  • Find Funding Search Tool within NIH Grants and Fundings lists all available grant opportunities. The filters on the left of the page narrow the search results. 
  • The National Center for Advancing Translational Sciences (NCATS) provides support for research projects, core facilities, scientific resources and tools, scientific conferences, and collaboration opportunities for small businesses, and other partners as well as grants programs and in-kind services: Funding and Notices
  • NCATS has additional programs listed in Discovery: Understand Discovery Research Tools.  
  • Other NIH resources:
    • NIH Tips for Applicants (2010) is a short video containing tips for grant applicants from reviewers and staff at the National Institutes of Health.
    • NIH Grants YouTube Page (updated periodically) is a page containing videos published by the NIH about a variety of topics related to NIH grants. There is a short Welcome Video on the homepage that introduces the organization of the content. 
    • NIH Grants Process Overview (updated periodically) is a webpage providing the steps required for the planning and submission of an application through to award and closeout.
  • The Office of Orphan Products Development (OOPD), a program of the FDA, offers 2 grants:
    • OOPD Pediatric Device Consortia Grant funds consortia capable of providing expert advising and support services to innovators of children’s devices through all stages of development. 
      • Specific areas of expertise provided by the consortia include:
        • Intellectual property advising
        • Prototyping
        • Engineering
        • Laboratory and animal testing
        • Grant-writing
        • Clinical trial design.
      • List of current Pediatric Device Consortia Websites
    • Orphan Products Natural History Grants support targeted studies that advance rare disease therapy development through characterization of:
      • The natural history of rare diseases/conditions.
      • Identification of genotypic and phenotypic subpopulations.
      • Development and/or validation of clinical outcome measures, biomarkers and/or companion diagnostics.

Your group can form partnerships with a pharmaceutical company (industry) and/or facilitate formation of a larger partnership between key stakeholders, including your group. 

  • Strategic partnerships are traditionally collaborations between businesses with a common mission. However, many large pharmaceutical companies are beginning to build strategic partnerships for the development of therapies for rare diseases with academia, small businesses, and even groups like your own.  
    • The overall goal of strategic partnerships is to share resources in a way that promotes growth for all partners.
    • Choose a partnership with an  expertise different from your own. For example, if your researchers have the need to develop an animal model to test a promising therapy, find a strategic partner that has the resources to develop that model.
    • Since a strategic partnership involves contracts, if your group is going to establish a strategic partnership make certain your Board of Directors, Scientific/Medical Advisory Board, and possibly a business lawyer are involved in the process. 
    • Alternatively, you can help academic researchers or start-up medical research companies establish strategic partnerships by searching for possible industry partners. 
  • Venture funds are another way large pharmaceutical companies are investing in the early stages of rare disease therapy development. 
    • Venture funds however tend to support start-up medical research companies, rather than academic researchers or patient groups. 
    • Usually involve a high level of engagement with representatives from the large pharmaceutical company leading or co-leading the investment and playing an active role on company boards.
  • Check with other group leaders who have run successful fundraising programs, grant programs, or formed strategic partnerships early in the discovery phase of therapy development. 
  • Search for research funding that may be available through special programs in your state.
  • Think  outside the box, network, and stay alert for emerging innovative companies focused on rare disease research or branches of large philanthropic organizations newly supporting the development of therapies for rare diseases. 
  • Persistence is key. Successfully finding funding can rely on:
    • Timing 
    • Positioning
    • Luck

Resources

Funding Research
Fundraising National Organization for Rare Disorders (NORD) (link)
NORD Research Grant Program Funding National Organization for Rare Disorders (NORD) (link)
GARD Guide for Researchers: Finding Funding Opportunities Genetics and Rare Diseases Information Center (GARD) (link)
NORD Additional Funding National Organization for Rare Disorders (NORD) (link)
CF Foundation Venture Philanthropy Model Cystic Fibrosis (CF) Foundation (link)
Federal Funding Opportunities
Grants and Funding Application Due Dates National Institutes of Health (NIH) (link)
R21 Contacts and Special Interests National Institutes of Health (NIH) (link)
Find Funding: NIH Guide for Grants and Contracts National Institutes of Health (NIH) (link)
SBIR|STTR America Seed Fund National Institutes of Health (NIH) (link)
HHS SBIR/STTR Agency Contact Information National Institutes of Health (NIH) (link)
NIH Types of Research Grants National Institutes of Health (NIH) (link)
NCATS Funding and Notices National Center for Advancing Translational Sciences (NCATS) (link)
NIH Tips for Applicants National Institutes of Health (NIH) (link)
NIH Grants YouTube Page National Institutes of Health (NIH) (link)
NIH Grants Process Overview National Institutes of Health (NIH) (link)
OOPD Pediatric Device Consortia Grants U.S. Food and Drug Administration (FDA) (link)