Build Relationships With Key Partners

With Other Groups

An umbrella group is an association of groups or institutions who formally pool their resources. Many umbrella organizations, especially in the rare disease community, hold conferences and webinars and can assist with learning about therapy development and ways to combine multi-stakeholder voices to advocate for medical research more effectively. In addition, knowing the different groups involved in your disease space and engaging in the opportunities they offer is one of the best ways to empower your patient voice and become involved in driving research for your disease.

• Types of opportunities: Groups may offer a variety of resources in different formats. Learning about the groups, their missions, and the types of resources they offer can help you determine which groups may be most important to help accelerate your own mission. The most common types of resources include: 
  • Conferences: Although in-person attendance will be more expensive than watching a livestream, conferences, especially those by umbrella groups, offer ways to meet other groups and network. Scholarships may be available to attend a conference, so don’t be afraid to ask if you are unable to attend due to financial constraints. Advantages of attending include:
    • Learning from other patient groups who are further along in the process than you are.
    • Brainstorming with other patient groups who are facing the same challenges.
    • Mentoring patient groups who may not be as far along in the process as you are. 
    • Gaining insight into what your group can offer to key stakeholders via the conference speakers who may include:
      • Researchers.
      • Clinicians.
      • Representatives from both small and large pharmaceuticals.
      • NIH researchers.
      • FDA staff.
  • Webinars: As you likely know, webinars are seminars available over the internet. Webinars usually are focused on a single topic and last about an hour; they may be easier to incorporate in your busy schedule and may better fit your current needs. 
    • Some webinars are interactive and will take questions, most often through a chat box within the webinar platform.
    • Most webinars are recorded and archived on the group’s website.
  • Web-based materials: Umbrella groups often develop resources (sometimes labeled as toolkits) that cover several facets of a single topic. They may provide templates, checklists, and links to other helpful resources. The group may have special programs that are focused on therapy development or disease research. Taking the time to explore their websites to see all they have to offer can save you time in the long run. 
• Rare disease umbrella groups: Umbrella organizations focused on rare and genetic diseases and nonprofits focused on therapy development may be a good place to get started. Such groups include:
  • Global Genes: A non-profit organization with a mission to eliminate the challenges of rare diseases by building awareness, educating the global community, and providing critical connections and resources that equip advocates to become activists for their disease. Global Genes holds focused conferences and webinars and offers multiple resources, including RareToolkits, through their website. 
  • National Organization for Rare Disorders (NORD): A federation of voluntary health organizations helping people with rare diseases through programs of education, advocacy, research, and patient service. NORD offers a membership program for patient groups in rare disease. NORD also holds conferences and webinars, offers special programs to assist with building registries or funding a research project, and provides information on specific rare and genetic diseases. 
  • EveryLife Foundation for Rare Diseases: A nonprofit organization dedicated to accelerating biotech innovation for rare disease treatments through science-driven public policy. In addition to their many advocacy programs and activities, EveryLife holds an Annual Scientific Workshop that brings together thought leaders to address urgent issues impacting the development process for rare disease treatments. 
  • FasterCures: This “action” tank  center of the Milken Institute is driven by a singular goal: to save lives by speeding up and improving the medical research system. FasterCures focuses on cutting through the roadblocks that slow medical progress by expanding the science of patient input, fostering policies to support biomedical innovation, and spurring cross-sector collaboration in research to get better outputs. FasterCures offers a variety of programs, webinars, reports, and other opportunities to learn more about the therapy development process and practices that can help you speed up the time to your goal. 
  • Genetic Alliance: A nonprofit organization with a mission to engage individuals, families, and communities to transform health. One of their programs, Stakeholder Engagement, provides a framework, best practice tips, and tools to help you increase your effectiveness at forming and facilitating multidisciplinary teams. 
• Disease specific groups: Other patient groups focused on specific diseases can also present opportunities to build relationships that will be invaluable through the therapy development process. 
  • Patient groups for the same disease: In some cases, there is more than one patient group for the same disease. This may occur when the missions of the groups are different. For example, one group may be more focused on supporting patients and their families, while another may be more focused on one specific therapy research effort. 
    • Work together with other patient groups focused on your disease, so that patient data is shared and preferably joined in one place and you aren’t duplicating efforts.
  • Larger parent disease groups:  There may be a larger patient group that includes your disease and other related or similar diseases.The larger group may have more resources available, such as lawyers, an IRB, or industry, government, and research connections. 
    • Working with these larger groups when possible may allow you access to these resources, their experience and expertise, and save both time and money. In return, your efforts may allow them to focus on other diseases that do not have a specific patient group. 
  • Patient groups for related disease: You may find a group focused on a disease with similar symptoms, cause, or disease process. 
    • Forging a relationship can prevent needless competition for scarce researchers, funding, and industry interest.
  • Patient groups willing to mentor: Many who have been involved in the therapy development process are happy to share their journey with others. Although no two paths are exactly the same, you can take advantage of the time and effort that will be saved by listening to their experiences. 
    • Watch for news about a patient group who has a therapy headed to clinical trials or just approved, and then contact their lead staff members to set up a time to talk about their process.

With Academic Researchers and Clinicians

Most groups start their multidisciplinary team by seeking out academic researchers and clinicians who are focused on their disease or related diseases. For some groups, finding a few interested researchers and clinicians is not that difficult and the group is quickly able to form a scientific/medical advisory board. But for many, especially rare disease groups, it is not only a very challenging first step, but a continuous challenge. Furthermore, it is not necessarily best practice to rely solely on your scientific/medical advisory board for the research needed for therapy development. Although there is no single method, patient group leaders who have successfully found researchers and clinicians suggest utilizing: 

• PubMed: You can find published medical and research articles on your disease (or associated gene or protein, if known) through PubMed, a searchable database of published medical articles provided by the NIH National Library of Medicine (NLM). Although not all of the articles are available for free online, most articles listed in PubMed have a summary (abstract) available. 
  • Using “your disease” (or associated gene or protein) as your search term should help you locate articles. 
    • Try using the disease term with and without quotation marks.
    • Replace the disease term with any synonyms if you are only finding a limited number of articles. 
    • Add terms such as “etiology” (disease cause), “pathology” (disease process) or “treatment” to the main search term or use the advanced search feature to narrow your search results. 
      • You can join any added terms to the disease term or each other using “AND” between the terms. This will limit your search results to articles that contain all the listed terms.
    • If you can’t find any researchers for your rare disease, substitute a more common disease name that may have similar symptoms, cause, or disease process as the main search term. This method may locate researchers who have the experience, knowledge, and possibly interest to include your disease in their research efforts.
    • If there is no summary available or you would like to read the full article before reaching out to the authors, the NLM can help you find libraries in your area where you can get the full text of medical articles. You can search for libraries at National Network of Libraries of Medicine Members Directory. Or you can contact the NLM toll-free at 1-888-346-3656.
    • You can set up email alerts to be notified if new articles are published in a specific search area by simply clicking “Create alert” under the search bar above the listed search results. If you don’t have an account with the NLM, the system will walk you through the process. Setting up an account is easy and free.
      • The NIH Library provides a bit more information, including a short video, if you need some help to set up or manage your PubMed alerts: Creating Alerts: PubMed
      • You can choose the frequency you will receive alerts and modify the search in the future.
    • PubMed Help provides answers to frequently asked questions and links to other tips that can help improve your search strategies and locate full text articles if one is not freely available. This resource is frequently updated by NLM.
  • Contact information for at least one of the authors of articles found through PubMed is often available. Just click on the title in the search list to take you to the abstract or summary view of the article. The contact information can be found by clicking the “+ expand” next to “Affiliations” just below the list of authors.
    • On case reports or case series, the first author may be a medical resident or fellow, and therefore you may want to try to find the contact information for the last author who is usually the most senior author. 
      • However, it can be helpful to find a medical resident or medical fellow who is just starting their career and may be interested in focusing on your rare disease. A quick internet search usually can help you determine if they continued to specialize in a relevant field and are focused on clinical medicine, research, or both.
    • Similarly, the first authors listed in research studies may be graduate students or postdoctoral fellows, but determining this is more complex. Sometimes when several labs or doctors work together, a senior researcher’s name is listed first. In some cases, most or all of the authors are senior researchers. 
      • Again, finding a graduate student or postdoctoral fellow who may be interested in making your disease one of the first focuses of their career can be helpful, especially if none of the established researchers or clinicians are interested in doing so. 
    • If the researcher or clinician you wish to contact does not have contact information provided, you can often find contact information by searching the researcher or clinicians name on the internet. 
      • Include MD, PhD or other professional title with the author name.
      • Add the university research or medical center, if needed.
      • Including “email address” after the doctor’s name can also be helpful.  
  • Encouraging Research Through Personal Connections, Section 3 (pages 7 to 10) of the Global Genes Toolkit on “From Molecules to Medicines: How Patients Can Share Their Voices Throughout the Drug Development Process” includes tips for using PubMed to find researchers and suggestions for reaching out to those you find.
• Medical professional information resources: The authors of the information on certain medical professional information websites may be specialists. For example, you may consider reaching out to the authors of a report on your disease or related or similar disease published on:
  • GeneReviews usually provides email addresses for all authors.
  • NORD Rare Diseases Information only lists the reviewing author, but contact information can be found with an internet search. Older NORD reports may not list the reviewing author.
  • UpToDate does not provide contact information, but, again, contact information can usually be found easily with an internet search. 
• The Research Portfolio Online Reporting Tool (RePORTER) is a searchable database of NIH supported research. Although these projects may not conduct studies on humans, this resource can be especially helpful if you are looking for researchers focused on research understanding cause and pathways, discovery of possible targets or translational research tools, and the preclinical trial phase of the therapy development pathway. 
  • To search for studies, enter the disease name (or associated gene or protein) in the “Text Search” box and click “Submit Query”.
• Clinical trials: There are several online search tools to help you search for clinical trials. Contact information is usually included for the Primary Investigator (PI) of the trial. You can search for studies using: 
  • ClinicalTrials.gov which was developed by NIH through the National Library of Medicine to provide the public with current information on clinical research studies. Try using the disease or associated disease or protein name. You can narrow studies to the United States and even some specific states, as well as limiting your search to active or completed trials .
  • CenterWatch which is a Clinical Trial Listing Service which provides a list of Institutional Review Board (IRB)-approved clinical trials being conducted internationally. The site is designed to be a resource for patients interested in participating in clinical trials and for research professionals. 
• University medical centers: Search the relevant specialty departments at university or tertiary care medical centers (or associated pediatric center, if your disease affects children) for specialists who have the knowledge and skill to become specialists in your disease.
  • Physician profiles often don’t list a rare disease a doctor may have experience treating, so can be most helpful to search for similar, more common diseases or the symptoms associated with your disease that are complicated to treat.
  • Many rare and complex diseases affect more than one body system and therefore might fall under more than one specialty, so you may wish to try several different departments.
  • If your disease is genetic, you may try reaching out to a genetic counselor in the medical genetics department of a university medical center to see if they have any recommendations.
    • The National Society of Genetic Counselors has an online tool to help you Find a Genetic Counselor
    • The American College of Medical Genetics (ACMG) has an online tool to help you Find a Genetics Clinic
  • Major university and tertiary care medical centers often have nurses available to help callers find the right specialist within their center. Although many times the nurse will have difficulties with rare diseases, sometimes they are able to help you find a specialist. 
• Rare Disease Clinical Research Network (RDCRN): If your disease has similarities to any of the diseases covered by the currently funded Consortia in the RCRNs, you may wish to reach out to one or more of the PIs.
  • Consortia include researchers, clinicians, and patient groups. 
  • The goal is to work to better understand rare diseases and move potential treatments closer to the clinic.
  • Each Consortia includes several diseases that may be related by body system affected, genetic change, disease process, or similar symptoms (phenotype). 
  • The Consortia list participating university and tertiary care medical centers and the PIs at each center. 
  • Although it is not easy for a disease focus to be added to an existing RDCRN, the PIs at the participating centers may have experience with your disease or know of a colleague who does or would be interested in focusing on your disease.
  • The RDCRN is supported by the Office of Rare Disease Research (ORDR)/National Center for Advancing Translational Science (NCATS) and collaborating institutes and centers.
• Medical professional society conferences: Exhibiting at medical professional society conferences can not only raise awareness among the specialists at the conference, but can also attract young medical residents and fellows who are just starting out in this specialty and are looking for their niche. 
  • If members of your medical board are willing to train with residents, fellows, or interested specialists, you can facilitate the connection. 
• Offer grants: If you already have scientific or medical advisors that can help you evaluate grant applications, you may consider offering one or more grants with specific areas of focus as a way to help you find or develop researchers in that area. 
  • For example, if research is needed to better understand how the changes in the associated gene or protein causes your disease, you can set up grants to attract this type of researcher. 
  • Funding Research, Section 4 (pages 11 to 13) of the Global Genes Toolkit on”From Molecules to Medicines: How Patients Can Share Their Voices Throughout the Drug Development Process” includes tips for funding research.
  • NCATS Toolkit provides more information about this topic in Discovery: Fund Discovery Research.
• Hold scientific conferences: As you begin to identify researchers already working on your disease or closely related diseases, you may be able to find or attract others through holding scientific conferences. 
  • NCATS Toolkit provides more about  this topic, including information about available grants, in Organize In-Person Scientific Meetings within Discovery: Facilitate Scientific Collaborations section.

With Government

The ability to connect with FDA in meaningful ways continues to increase thanks to those patient group leaders who blazed the trail before you. The different opportunities to connect to the FDA are covered in more detail in subsequent sections, but a few resources that provide overviews are highlighted here. In addition, you will want to identify the NIH Institutes and Centers (ICs) that may be focused on research related to your disease. Building relationships with relevant NIH ICs increases your awareness of grants, special workshops, and other opportunities that may help you accelerate developing a therapy for your disease. 

• FDA: The FDA recognizes that patient perspectives are primary, and that these perspectives might differ significantly from what would be expected by clinicians and researchers who do not experience the challenges of living with the disease. This is particularly true in the context of rare diseases. Therefore, the FDA recommends early and continued contact as your group begins the process of therapy development.
  • Resources with information about ways to connect to the FDA include:
    • For Patients is a website developed by the FDA Patient Affairs staff to help patients and patient group leaders learn about therapy approvals, public meetings, special opportunities to connect with the FDA, and more. 
    • Learn About FDA Patient Engagement is a summary of organizations and opportunities at the FDA that incorporate patient involvement. Opportunities include the FDA Patient Network, a Patient Engagement Initiative, and the Patient Representative Program. 
    • Developing Products for Rare Diseases & Conditions explains the mission of the FDA Office of Orphan Products Development (OOPD) as well as the different programs that may be available to help with the development of therapies for rare diseases. The contact information for the OOPD is also provided.
    • FDA Patient Affairs Staff coordinates and supports patient engagement activities across the FDA to facilitate awareness and collaboration with patients, patient groups, and the FDA. Staff can be contacted directly at (301) 796-8460 or PatientAffairs@fda.hhs.gov.
  • To identify what is most important to patients, the FDA has multiple patient focused initiatives. Each of these programs is described in more detail in sections of Discovery: Collect Patient Experience Data. For example, the FDA:
    • Hosts internal Patient-Focused Drug Development (PFDD) meetings.
    • Assists with organization of external PFDD meetings.
    • Supports the development of benefit-risk analysis in the form of clinical outcome assessments (COAs).
    • Offers grants for natural history studies and clinical trials focused on rare diseases. 
  • Other resources to learn more about engaging with the FDA include:
    • Your Voice Matters: How to Engage with the FDA (2020) is a NORD RareEDU webinar in which three key FDA staff members share information about FDA programs designed to incorporate patient and caregiver voices into therapy development, including patient-focused outcomes. The video is one hour long. 
    • Regulatory Advocacy: Patient Organization Case Studies on Working with the FDA (2016)  is a Global Genes video presentation with suggestions for ways to work with the FDA and better understand regulatory processes and advance along the research and development continuum. The video is approximately an hour long. 
    • Working with Regulators: A Focus on the FDA (2015) provides patient group leaders with information about working with the FDA. Some of the topics in the 32-page document include the role of the FDA, the relationship between the FDA and patient groups, and new initiatives that allow patients to have better access to therapies. The document was prepared by the Cancer Policy Institute at the Cancer Support Community.
  • It is important to remember that although the FDA is open to and encourages the involvement of patient groups and patients, it is a federal regulatory agency focused on public health safety and has rules and regulations it must follow when considering approval of a therapy. 
• NIH: The different ICs at NIH may provide opportunities to connect your researchers and clinicians with specialists at the NIH. Many of the ICs offer grants or engagement opportunities for patient groups. Finding the ICs that may be involved in research for your disease early in the process will allow you to use their resources to their fullest.
  • List of NIH Institutes, Centers, and Offices can help you learn more about the 27 ICs at NIH. Each has its own specific research agenda, often focusing on particular diseases or body systems.
    • Many rare and complex diseases lacking treatments affect multiple systems and therefore may fall under more than one IC. 
    • If you are uncertain whether you found the right IC, use the contact information to call and find out. 
    • Find NIH Clinical Center Trials is a registry of publicly supported clinical studies conducted mostly at the NIH Clinical Center. Searching for studies for your disease or similar, more common diseases may help you identify NIH researchers and Institutes with knowledge about your disease.
  • The National Center for Advancing Translational Sciences (NCATS) was established to transform and accelerate the translational research process. NCATS collaborates with researchers, the public, and other stakeholder groups to design new approaches and technologies that ultimately will deliver more treatments to more people more quickly. NCATS:
    • Complements other NIH ICs, the private sector, and the nonprofit community.
    • Focuses on research that can broadly be applied to other diseases. 
    • Supports multiple programs focused on rare diseases through ORDR including NCATS Toolkit. 

With Industry 

Many groups begin to connect to the pharmaceutical industry by reaching out to potential partners that include similar diseases or specific symptoms in their scope of work. Establishing relationships with industry can drive a stronger industry focus on therapy development for your disease and its associated symptoms. 

• Your group can help educate your industry partners on:
  • The natural history of your disease.
  • Unmet medical needs. 
  • Symptoms or features most affecting patients’ quality of life.
  • Feasibility of protocol design.
  • Possible patient reported outcome measures.
  • Patient/disease compatibility with drug administration.
• Patient groups with registries that are also actively involved in the therapies development process can provide reassurance to industry that there is an increased possibility that enough patients will be able to be recruited for clinical trials. 
• Global Genes provides several resources focused on working with industry from the patient group perspective.
  • Building the Bridge Between Foundations and Industry (2018) is a webinar providing advice on how and why to establish relationships with industry partners and some important aspects of managing those relationships. The webinar is an hour long. 
  • Searching for Answers: Contacting Biopharmaceutical Companies Effectively (2014) provides helpful tips about reaching out to biotech and pharmaceutical companies. Other topics include what to do before reaching out to the companies, how to request financial assistance, and resources to connect with patients and medical researchers. 
  • Research Development with Industry: Understanding Their Motivations (2015) is a video of a presentation and question and answer session focused on the role of industry in the development of drugs used to treat rare diseases. The presenters provide the industry perspective on collaboration with patient advocacy groups in drug development. The video is about one hour long.
  • Partnering with Pharmaceutical Companies in a Changing Landscape (2016) is a video of a presentation exploring the relationship between patient advocacy groups and pharmaceutical companies, including historical perspectives of this relationship and how it is evolving. A case study is presented about how to effectively work with industry, and thoughts about future changes in this relationship are discussed. The video is about 45 minutes long.
  • Patient’s Role in Designing Clinical Trials (2016) is a video of a presentation covering patient engagement in the research and development process. The presenters speak about the development of a set of specific recommendations for patient engagement: Effective Engagement with Patient Groups Around Clinical Trials. The presenters also provide case studies for turning these recommendations into real practice. The video is 40 minutes long.
• Foundation-Company Partnerships Toolkits provides information on how to find the right partners and structure agreements with biotech and pharmaceutical companies in order to incentivize research. Other topics include venture philanthropy, nondisclosure agreements, negotiating strategies, and intellectual property. The information is compiled by The Research Acceleration and Innovation Network (TRAIN), the online platform for venture philanthropy in medical research supported by Faster Cures.
• Foundation-Industry Relationships – A New Business Model: Joint-Venture Philanthropy in Therapy Development (2014) is a published article describing the collaborations and financial relationships between foundations and industry partners. This paper provides examples of how foundations for cystic fibrosis, spinal muscular atrophy, and Friedreich ataxia have worked to build a new joint venture philanthropy business model.
• An Essential Partnership: A Guide for Charities Working with Industry (2014) is a guide for medical research charities looking to collaborate with industry. The guide illustrates the relationship between charities and industry through case studies, what charities should consider before entering partnerships, and different types of agreements and contracts. The resource was developed by the Association of Medical Research Charities (AMRC) which is based in the United Kingdom.
• Guiding Principles for Interaction with Patient Advocacy Organizations briefly describes a set of principles that can be used by industry when interacting with patient organizations and thus offers what industry may be looking for from your group. This resource was developed by Biotechnology Innovation Organization (BIO).
• Medical Device Innovation Consortium is a non-profit public-private partnership with the goal of improving the medical device environment. MDIC forms partnerships between the FDA, industry, and non-profit organizations in order to improve patient access to medical devices. 

Tips for Success

• Review Best Practices for Effective Patient Group Engagement (2015 to present) which provides resources that include recommendations on how and why patient groups should engage early and often with all stakeholders in the therapy development process. It also provides methods for assessing the financial value of patient engagement and evaluating the capabilities of patient groups to engage with other partners. The recommendations are presented in several formats, including an executive summary, full report, webinar, and tools. The resources are developed  by Clinical Trials Transformation Initiative (CTTI).
• Keep first email contact attempts brief and with a clear description of why you are contacting the person. 
  • If there is no answer in a few weeks, try reaching out again. Sometimes responding to an email just falls off a busy researcher’s list.
• Develop a 5 to 10 minute elevator pitch that explains how far along your group is in the therapy development process, current goals, and a few of the obstacles. 
  • Remember to update the pitch as you move along in the process or goals or obstacles change.
  • The pitch can be helpful when you are at conferences and have a quick moment to talk to researchers, industry, and FDA and NIH staff—any of whom may become interested in your disease research, provide suggestions, or be aware of developing advances that may help you overcome current obstacles. 
  • The pitch can also be helpful in those chance meetings, so having one prepared means you won’t miss an unexpected opportunity.
• Learn what motivates the person with whom you are hoping to build a relationship and adapt your approach. For example, in addition to the rules and culture of their employer (academic, industry, or government), individuals may be motivated by a combination of:
  • A specific patient’s story.
  • Statistics and data.
  • Grant opportunities.
  • Publishing opportunities.
  • Wanting to make a difference.
  • Wanting to make a name.
•  Understand what an individual can and not compromise when forming a relationship.
  • Negotiables and “must haves” are usually defined by their employer’s rules and culture, as well as regulatory rules and state and federal laws.
• Be clear about areas you will and will not compromise to form a relationship. 
• Prior to signing any agreement or contract, you may consider having a lawyer with experience in the therapy development process review the document(s).

Resources