There are more than 10,000 identified rare diseases impacting an estimated 400 million people globally1
Rare populations come in all shapes and sizes and depending on which rare indication your organization is targeting your strategies will vary. More than 15 years’ experience in rare disease showed us the following to be true:
Every rare disease and its associated clinical trials are unique. Cookie-cutter approaches to site identification, patient identification and recruitment do not work.
Rare clinical trials often force sponsors and CROs to scour the globe to find the right patients.
Rare diseases push their stricken individuals and caregivers to join communities and seek online support to find appropriate help and guidance.
When sponsors & CROs target a new rare indication, collaborating with the corresponding communities is critical to navigating the community networks and finding the target populations around the world.
Rare communities and rare patient advocacy groups are diverse regarding their development and support capabilities.
Proven strategies for rare advocacy collaboration
In this article, we share our tips and best practices for finding rare disease communities and advocacy groups and offer insight on how best to partner with them to maximize value and impact on your planned clinical trial. Rare disease researchers need every possible tool to help drive patient identification and recruitment. Working directly with patient advocacy groups and communities can be a highly effective tool for finding the targeted rare patients and making them aware of your clinical trial.
When masterfully executed, patient advocacy groups can be a true catalyst for your trial’s enrollment success.
Finding rare advocacy groups and communities
Identifying these groups and communities can be challenging, requiring a multifaceted approach. This is particularly true when searching across several regions, compensating for language barriers, and navigating locally preferred social media networks. Here are some strategies to help you to get started.
Leverage established doctors and investigators
Rare disease doctors typically have a national or global network they access. While this may be sufficient to meet your needs, it is more likely that you will need to further augment their network and rare population connections. While collaboration with doctors and hospitals can be helpful, you will face communication challenges and need to work through getting the right people involved (on both the Sponsor and CRO sides).
Use Advanced Search Engine Techniques
Search engines are vital resources for finding online communities, regardless of the country. For more advanced Google search tips, please refer to this helpful article. These commands will help you to segment your search regionally.
Engage Facebook groups
While traffic on the platform is declining overall, Facebook groups continue to attract active and engaged users. Reaching out to these groups can be a useful exercise, considering the maturation of an indication and the awareness surrounding it. Here is a helpful article for exploring Facebook groups.
Accounting for incidence and geographic approach
One of the key strategies for working with patient advocacy groups is to map both the geographic implications of the disease and its incidence rate. When selecting groups, understanding their individual goals will help you identify those whose goals align with those of the clinical trial. To establish a successful communication channel, finding the right contact person – whether from the client, CRO team, or principal/local investigator – can be a tremendous difference-maker.
Please note, it is important to be open to all advocacy groups, and we suggest keeping the door open for new groups until enrollment is complete.
Integrating advocacy groups into patient engagement plans
Advocacy groups don’t only bring patients to the study. They may also bring the Sponsor or CRO closer to patients, allowing for a better understanding of the nature of the disease, its impact on all aspects of the condition, and any associated difficulties. As you search globally, look out for opportunities to connect with local, national, or regional communities alongside global ones. Integrating diverse groups into your patient outreach and engagement strategies will be critical to on-time enrollment success.
Not all Sponsors or CROs take enough time to thoroughly investigate the communities associated with the targeted disease, nor do they always fully leverage the potential of an advocacy group and community collaboration. Consider the following tips for maximizing your outreach potential:
Partner to understand the patient population
This is particularly relevant if this is your first study of the disease. Connect with several few groups to ensure you understand the unique demands and challenges of the population. This is especially helpful when launching a first-in-human clinical trial.
Collaborate on study awareness and enrollment
Pending the capabilities of the advocacy groups and communities you are engaging; they may be able to provide support using exclusive communication channels with their populations (where available):
Driving study awareness through their website
Tapping into their community newsletters
Communicating via social channels
Support your rare community
These communities are safe and trusted spaces for people. It will be important to support any virtual or face-to-face gatherings normally conducted by the group (if possible). This can help to drive understanding, awareness, engagement, and trust.
Precision’s experience in rare disease partnerships
Precision has worked with advocacy groups on several rare disease studies and has the experience to help you start the process of finding and working with the right groups. With patient advocacy group collaboration, clinical trials can be more effective and efficient in finding the right patients and advancing the development of new treatments and therapies. At Precision, we believe rare disease studies call for ‘Rarefied Thinking’. A difference-making factor for our Sponsor partners. Learn how this approach can make a difference in your next study:
Reka Zsambok is a Director of Project Management, Senior Director Clinical Trial Management and Medical Director at Precision who brings 15 years of experience in managing Phase II-III clinical trials. Reka is experienced in providing directional oversight on rare disease, cardiology and oncology trials. Her experience covers a wide variety of therapeutic areas, with strong experience in Paroxysmal Nocturnal Hemoglobinuria, heart failure, AF, infections disease, stroke, Parkinson-disease, breast cancer and multiple myeloma. She provides strategic support to the project team.
Sarabeth Velazquez is an Associate Director of Project Management at Precision for Medicine, having 20 years of experience in managing Phase I-III clinical trials. Sarabeth’s expertise covers a wide variety of therapeutic areas in rare disease, oncology, dermatology, ophthalmology and cosmetic clinical trials. Her experience notably includes: abdominal bulging, submental subcutaneous fat, molluscum contagiosum, onychomycosis, neurofibromas, herpes labialis, hyperhidrosis, breast implants, colorectal cancer, solid tumors, breast cancer, ovarian cancer, lymphoma, cataracts, pulmonary arterial hypertension, classic galactosemia, and sorbitol dehydrogenase deficiency.
Precision for Medicine is part of the Precision Medicine Group, an integrated team of experts that extends Precision for Medicine’s therapeutic development capabilities beyond approval and into launch strategies, marketing communication, and payer insights. As one company, the Precision Medicine Group helps pharmaceutical and life-sciences clients conquer product development and commercialization challenges in a rapidly evolving environment.