Patient Group Assessment by Industry

Knowing the criteria Industry uses to assess your group can help you maximize and articulate your expertise and assets.  

• The vision, mission, and goals:
  • Ensure that your group’s  vision, mission, and goals are clearly stated in all materials and  demonstrate that your vision, mission, and goals are at the center of your activities and are reasonable in respect to your budget. 
  • Your goals and objectives are summarized in your vision and mission statements. 
  • A vision statement concentrates on the future; what is your ultimate goal?  The vision statement should be clearly written and engaging, but at the same time it should also express realistic desires and give a clear timeline. A vision statement guides later decision-making. 
  • A mission statement is about the present; it outlines your current goals and the processes you have in place to meet those objectives. What is the purpose of your organization, who does your organization serve, and what is its primary focus? This statement should avoid the use of generic endpoints (e.g., our goal is to lower therapy prices) and should instead be as specific as possible (e.g., our goal is to provide support for the Orphan Drug Act by broadening public knowledge of Wilson’s Disease). 
• Operations:
  • Group operations are evaluated in several places to ensure that the group is sophisticated enough to demonstrate a measurable impact. 
  • Specifically, it is important to have scientific and medical community members involved either directly or as advisers.
  • Your outreach methods to partner with other groups, researchers, and Centers of Excellence will also be evaluated. 
  • Check your ratings with the Better Business Bureau and Charity Navigator and take the necessary steps to correct any inaccuracies. 
• Budget:
  • The amount of your income is less important than the diversity of sources and fundraising capacity. 
  • The ways in which you use your resources is also relevant; most of your budget should be going towards operations and activities instead of overhead. 
• Communications:
  • Groups are also evaluated on the effectiveness of their communication system, including their social media presence, publications, and outreach activities. 
  • The impact of informal publications (e.g., blogs, press releases) and formal publications (e.g., white papers) is also considered.

Clinical Trial Sites

An established Network of Clinical Centers of Excellence can provide the clinical sites needed for a clinical trial. When a new therapy is being tested to determine safety and efficacy, it is important that the Sponsor is confident that the data collected during the trial is consistent across clinical research sites. Using established Centers for clinical trials sites website can:

• Increase participation in a clinical trial, since the trial will be available at Centers many patients and their families are already going to for their clinical care.
• Ensure participant eligibility is correctly determined using the established diagnostic criteria for the disease as well as other clinical trial specific eligibility requirements.
• Decrease time needed to train clinicians and staff in the clinical trial procedures and data collection for the clinical trial.
• Make sure patients enrolled in the clinical trial are also receiving established clinical care.

Overview

Developing clinical centers that have expertise in diagnosing and caring for patients with your disease increases the ability to conduct clinical trials and optimizes clinical care. When clinicians and staff have specialized experience and established relationships with patients, they are better prepared for conducting clinical trials. Additionally, when clinical centers have been uniformly trained in evaluation procedures, variability among the sites in the trial Network is minimized.

Clinical Care Networks

Establishing certain hospitals as Clinical Care Centers of Excellence for your disease can increase the quality of care every patient receives. In many cases, the diagnostic criteria, clinical care guidelines, and measures to monitor disease progression have not yet been established. Clinicians and staff at the Centers can develop these important elements, forge relationships with patients and their families, and increase the readiness for performing clinical trials when a new therapy is developed.

Advantages to having clinical centers include:

• Diagnostic criteria can be developed and published to clearly define the patient population and decrease time to diagnosis.
• Clinical care guidelines (standards of care) can make certain that patients with your disease are receiving the best medical care no matter where they live.
• New clinicians and staff can be trained at established Centers, as well as at future Centers.
• Clinical measures can be developed to track changing clinical outcomes overtime.
• Strong relationships between patients and their families and the clinicians and staff at Centers can increase the medical and quality of life knowledge about your disease.
• Clinical Centers of Excellence can work together as a network to collect medical data, perform natural history studies,  establish and validate clinical outcome measures, and help design clinical trials.

Tips for Success

Even though it is clear that establishing a Center of Excellence Network is important for clinical care and research efforts, it may seem like an overwhelming task. If your disease is newly discovered or very rare, there may not be many identified patients and they may be scattered across the globe. Learning how other rare disease patient groups have established their own Network can provide you with the inspiration you need to move forward. 

• Ask members of your Medical/Scientific Advisory Board whether they are willing to establish a Center of Excellence at their associated university medical center. 
• Find  a university or tertiary care medical center or centers that have been caring for at least a few patients with your disease.
  • Contact 1 or more of the clinicians who have seen the patients to determine whether they would be willing to be involved in a Center of Excellence for your disease.
  •  If specialists for your disease tend to be in one main department, approach the department chair.
  • Reach out to the university medical center administration to determine whether their center has a framework for establishing a Center of Excellence. 
• Find a university or tertiary care medical center that already has established multidisciplinary clinics for other diseases. 
  • Determine whether there are any open time slots in the space used for the multidisciplinary clinic that can be used to establish a multidisciplinary clinic for your disease.
  • Contact relevant specialists at the center to determine whether they would be willing to participate in a multidisciplinary clinic for your disease.
  • Start small, maybe offering 1 morning or afternoon a month, and grow as needed.
  • Grow the Network in other areas of the country using the same strategy.
• Provide families with resources that can help with travel costs or consider partnering with other organizations or a donor to more directly provide assistance.
• Consider establishing a Data Coordination Center to which all data are sent, maintained, and analyzed as the number of clinical centers in your Network grows.
• Offer research grants to help support the Principal Investigator and Clinical Trial coordinator when a clinical trial begins. Partnering with other, larger organizations or philanthropic organizations may make this more feasible.
• Read Centers of excellence in healthcare institutions: what they are and how to assemble them (2017), a published medical article detailing 1 university medical center’s framework for establishing multidisciplinary clinics.

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 non-profit organization that aims to improve health through the authentic engagement of communities and individuals. The organization is committed to transforming health through genetics and promoting an environment of openness centered on the health of individuals, families, and communities. Genetic Alliance’s network includes disease-specific advocacy organizations, universities, private companies, government agencies, and public policy organizations.
• 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.

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).

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. 

Overview

When you establish and nurture relationships with all the stakeholders in the therapy development process, including academia, government, industry, and other patient groups, you can form multi-stakeholder partnerships that maximize your ability to help accelerate therapy research and development (R&D) for your disease(s).