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

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.

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.
  • 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.

Organize Virtual Meetings

Virtual meetings or conference calls can be a good starting point to bring researchers and other key stakeholders together. Virtual meetings require a smaller time commitment from the participants since there is no travel required. Although your group may be familiar with holding virtual meetings among staff, volunteers, medical advisors, or your board of directors, you may want to make a few changes when involving outside medical and scientific researchers.

Consider your goal for the meeting. You may want to choose only 1 or 2 goals, especially for the first meeting. Some possible goals include:

• Introduce researchers to one another and to key stakeholders.
• Have researchers present a brief overview of current projects and goals.
• Review relevant experiences of patients with researchers.
• Brainstorm tentative short- and long-term research goals.
• Highlight areas for collaboration.
• Establish open sharing of data and analyses. 

Successful meetings rarely just happen. Knowing how to focus planning efforts can save time and energy, while still providing the elements needed to achieve the meetings goals.  There are a number of steps you can take before the meeting to ensure it is productive.

• Restrict meetings to key participants:
  • Virtual meetings with limited participants can be easier to moderate and allow everyone a chance to have their voice heard. 
  • Researchers may speak more freely in a smaller group.
  • Minutes from the meeting can be sent to a larger group of interested parties after the meeting.
  • Edited recordings of the meeting can be made available to staff, volunteers, or even posted on your group’s website (with proper consent from participants).
• Use meeting goals to guide invitations:
  • Smaller meetings are better for enabling discussions, making connections, and making decisions.
  • Larger meetings are better for information dissemination and presentations.
  • Patients, families, and caregivers may not need to attend a meeting designed to make connections between academic researchers.
  • Your group’s Medical/Scientific Advisory Board may offer helpful insight or help translate technical research terms.
•  Establish the meeting platform:
  • A voice-only conference line may be the easiest for all to access and is the least technologically demanding, but it will limit the type of sharing of information available. Materials to be shared will need to be sent prior to the meeting.
  • Online meeting or web conference platforms will allow screen sharing but are not always accessible to everyone. Skype, Google Hangouts, GoToMeeting, and Zoom are the most popular programs/applications for virtual meetings.
•  Tips for successful meetings:
  • Familiarize yourself with the technology being used in the meeting. If you are not tech savvy, you may wish to appoint a staff member with the right skill set to the task. Generally, you will need to know how to:
    • Start and end the meeting.
    • Share your screen or webcam, as well as help others share their screen.
    • Mute and unmute microphones.
    • Share files.
    • Send and read text chat messages.
    • Record the meeting (if applicable).
  • Draft an agenda for the call and include when inviting participants and then share a finalized version at least a week prior to the meeting.
    • Align the agenda with the meeting goals. Is the meeting to generate ideas, facilitate collaboration, or to make decisions?
    • The agenda should also include a list of topics, attendees, facilitators, and the time and meeting platform. 
  •  Assign meeting responsibilities. If you are working together with multiple people to plan the meeting, you can divide up the tasks each person will be responsible for before, during, and after the meeting.
    • Consider having someone other than the facilitator being responsible for watching the clock, taking notes, and sending meeting notes after the meeting. 
    • Depending on the technology being used, you may want to have someone assigned to help the facilitator in case any technological issues happen before or during the meeting.
  •  Be mindful of time.
    • Start the meeting on time even if all the attendees are not present and finish at the time listed on the agenda, in case participants have other commitments.
    • Keep the first meeting under an hour. Longer meetings can be scheduled later as specific topics are identified that need a more in-depth discussion.
    • Ask the timekeeper to chime in when you have reached the end of allotted time for a specific agenda item and at 3 breakpoints: halfway, 15 minutes left, and 5 minutes left.
    • Moderate to keep meeting on topic, but be flexible. Be willing to stop a discussion or move non-urgent items to the next meeting.
  •  Agree on time/date/format of next meeting.
    • Finding a meeting date and time may be difficult to accomplish between meetings when more than 3 people must coordinate.
    • During your meeting wrap up, try to get a general idea of when the next meeting should be held (a week, a month).
    • If you cannot get this done, use an organizing tool like Doodle, where participants can enter their own schedules instead of asking for availability via email.
  •  Send meeting notes highlighting action items from the meeting.
    • Notes should provide a summary of discussions, record important decisions, list action items, and function as a document to remind everyone of what was agreed upon and why.
    • Compile notes sooner rather than later. The meeting notes should ideally be sent out within 24-48 hours after the meeting, so that participants can make appropriate corrections as needed.

NIH Programs Supporting Collaboration

Learn about special programs offered by the NIH at early stages of therapy development.

• NIH ICs: Determine which NIH ICs are involved in researching the rare disease(s) supported by your group.
  • Contact the specific IC to learn about any special programs they have in place for working with patient groups. For example:
    • The National Cancer Institute has an Office of Advocacy Relations , dedicated to working with patient groups. You can contact them directly by phone: (240) 781-3360 or email: nciadvocacy@nih.gov 
• National Center for Advancing Translational Science (NCATS): NCATS has a number of different programs and funding opportunities focused on the discovery stage of therapy development.
  • General information is available on Patient/Community Engagement & Health Information. 
  • Programs that may be of interest to researchers working with your groups are outlined on Work with NCATS.
• Rare Disease Clinical Research Network (RDCRN) : The RDCRN is a special program supported by the Office of Rare Disease Research (ORDR), NCATS, and collaborating ICs. The program funds research teams, called Consortia, that work to better understand rare diseases and move potential treatments closer to the clinic.
  • Consortia are a network of scientists, clinicians, patients, families, caregivers, and patient groups.
  • There are 20 funded programs per cycle, and a cycle lasts 5 years.
  • Consortia can reapply for another 5 years of funding, but new networks will also compete. 
  • Most Consortia include a group of related conditions. 
  • Each Consortia includes multiple clinical and research sites.
  • The most recent Consortia funding was announced Fall of 2019. 
  • The opportunity to apply to become an RDCRN Consortia is usually announced late spring or early summer the year prior to when the funding cycle will start. Thus, currently a new announcement will occur late spring, early summer 2024: RDCRN Funding Information.
  • In addition, RDCRN Conference on Clinical Research for Rare Diseases (CCRRD) occurs about every 2 to 4 years and focuses on rare disease research methodology.
    • Topics and slide decks from past conferences are available.

FDA Critical Path Innovation Meeting

A Critical Path Innovation Meeting (CPIM) is a meeting to discuss the science, medicine, and/or regulatory aspects related to innovation in therapy development. The meeting is between FDA staff and individuals from industry, academia, and/or groups like your own. 

• CPIM provides an opportunity to have an open scientific discussion and to hear the FDA’s perspective on the method, approach, or technology being developed.
• The goal is to improve the efficiency and success in drug development. 
• If a CPIM is accepted, the FDA may invite subject matter experts from other Centers in the FDA, including Center for Biologics Evaluation and Research (CBER) and Center for Devices and Radiological Health (CDRH).
• Materials to be presented at the CPIM are requested 2 weeks prior to the CPIM.
• The FDA staff participating in the CPIM meet 1 week prior to the actual CPIM to review the information. Other experts may be identified.
• The meeting lasts about 90 minutes.
• A brief, high level summary will be written by the Office of Translational Sciences  and shared with the person who requested the meeting.
• Topic area is added to the CPIM website. 
• Outcomes include:
  • CPIM staff may help your group connect with other investigators sharing similar medical development challenges.
  • FDA may facilitate discussions with review divisions or other FDA scientific staff.
  • Recommendations may include convening a public workshop, collaborating with other groups.
  • May lead to technology transfer activities such as a research collaborative agreement or a Cooperative Research and Development Agreement (CRADAs ) between investigators and FDA.
• CPIM topics include:
  • Rare disease progression studies
  • Biomarkers as clinical trial endpoints
  • Therapy development tools
  • Innovative trial designs
  • Clinical trial networks
  • Natural History Studies
  • Clinical outcome assessment development
  • Databases
  • Registries
• FDA Critical Path Innovation Meetings provides more detail about CPIMs.
• If you have any questions about the CPIM Program, please contact CPIMInquiries@fda.hhs.gov . 

Other FDA Meeting Opportunities

The FDA offers additional opportunities to meet with patient groups early in therapy development. If you are uncertain which meeting type and the timing of your request to meet, contact the FDA Patient Affairs Staff at (301) 796-8460 or PatientAffairs@fda.hhs.gov. Other FDA meeting opportunities include:

• Patients Ask FDA: FDA Patient Affairs Office and the FDA medical product centers developed the Patients Ask FDA portal to allow patients and the groups like yours that advocate for them to submit questions and requests for meetings.
  • Requested meetings may focus on sharing patient disease experience or better understand the FDA’s marketing review process.
  • The staff of the FDA Patient Affairs Office will route the questions or meeting requests to the appropriate medical product center or office.
• Patient Listening Sessions: Patient Listening Sessions are hosted by the FDA Patient Affairs Office and the National Organization for Rare Disorders (NORD) , a nonprofit group uniting rare disease patient groups. 
  • FDA, patients, caregivers, and advocates participate in the session.
  • Small, informal, non-regulatory, non-public discussions.
  • About disease experiences, not a specific therapy.
  • FDA staff will either ask questions or simply listen to better understand your experience with a disease or condition.

Overview

Therapy development, including translational research, is a team sport. Determining areas where your patient group can support and drive research efforts will help you become an effective part of the team. Organizing virtual and in-person meetings between academic researchers and  your disease community can foster collaboration, maintain productive communication, and fuel interest in research. Meetings can promote an understanding of which therapy/treatments are currently within reach and which may be longer term goals. Special programs and meetings with the specific Institutes and Centers (ICs) of the National Institutes of Health (NIH) and the U.S. Food and Drug Administration (FDA) may increase collaboration.