Fund Discovery Research

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.
- Global Genes webinar Strategies for Effective Fundraising (2016) is a 1-hour video focused on different fundraising strategies. The tips are especially valuable to small rare disease groups.
- Global Genes also has online booklets to help you learn more about fundraising, including Rare Toolkit: Finding Your Fundraising Strategy (2016) and Rare Toolkit: Five Essential Tips and Tools for Effective Fundraising (2014).
- Fundraising by the National Organization for Rare Disorders (NORD) is an easy-to-read webpage outlining fundraising steps and includes links to examples of successful fundraising activities of other groups.
- Reach out to other groups to learn what has and has not worked for their group.
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:
  - NORD: Research Grant Program Funding .
- List your grant programs with rare disease centers and organizations. For example:
  - - NORD: Additional Funding.
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:
- Milken Institute Center for Strategic Philanthropy provides information about philanthropic investing through its newsletter and Giving Smarter Guides. For example:
  - Nonprofits: A Growing Force in Drug Development
  - Center for Strategic Philanthropy Newsletter
- FasterCures Toolkit Foundation-University Relationship provides:
  - Information about intellectual property, venture philanthropy, and foundation-university transformative partnerships.
  - Sample templates for agreement documents and reports.
- CureAccelerator FAQs Repurposing Research answers questions about repurposing approved therapies to treat a different condition, including ways for nonprofit groups to become involved. CureAccelerator is a non-profit organization that brings researchers and funding opportunities together in order to drive repurposing research.
- Section 4 : Funding Research of the Global Genes From Molecules to Medicine How Patients Can Share Their Voices Throughout the Drug Development Process (2018) reviews the ways a group can become involved in funding research and shares the success stories of two rare disease groups.

Resources

Funding Research

Strategies for Effective Fundraising Global Genes (link)

Rare Toolkit: Finding Your Fundraising Strategy Global Genes (link)

Rare Toolkit: Five Essential Tips and Tools for Effective Fundraising Global Genes (link)

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)

Milken Institute Center for Strategic Philanthropy Milken Institute (link)

Nonprofits: A Growing Force in Drug Development Milken Institute (link)

Center for Strategic Philanthropy Newsletter Milken Institute (link)

FasterCures Toolkit Foundation-University Relationship Milken Institute FasterCures (link)

CureAccelerator FAQs Repurposing Research CureAccelerator (link)

From Molecules to Medicine How Patients Can Share Their Voices Throughout the Drug Development Process Section 4 Global Genes (link)

Federal Funding Opportunities

NIH Exploratory/Developmental Research Grant Award (R21) National Institutes of Health (NIH) (link)

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)

Parent Announcements (For Unsolicited or Investigator-Initiated Applications) 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)

Information on the Orphan Products Natural History Grants Program U. S. Food and Drug Administration (FDA) (link)