Doctor with patient

Rare Disease Day 2015 is February 28, and as the day approaches I find myself thinking about how we as a biopharmaceutical industry can help the millions of patients living with a rare disease. There are more than 6,000 rare diseases affecting hundreds of millions of people around the globe. Yet because the population of sufferers from each disease is small and often widely dispersed, it can be difficult for researchers to connect with physicians and recruit enough patients to perform clinical trials. It is also much more difficult for them to get a sense of common treatment patterns, how those treatments are working and where treatment gaps exist – all of which is vital to the research process.

These barriers become much more manageable through the use of patient registries.

Q proof point - rare disease registriesRare disease registries, where physicians, patients and caregivers record information and track details of the patients’ diagnosis, condition and treatment, can address many of the scientific and communication challenges faced by rare disease researchers. Patient and caregiver reported outcomes provide a valuable glimpse into how various treatment strategies impact their quality of life. These insights, which can’t be gleaned from labs or physicians assessments, often shape the direction of their investigations and help them validate the choice of clinical outcomes. Registries also give researchers access to the stakeholder community, where they can connect with physicians, patients and even advocacy groups, to share information about their studies, build rapport with the broader patient community, and more efficiently and cost-effectively recruit participants. 

But these benefits can only be achieved if researchers make the most of this valuable clinical tool.  To ensure the success of your registry, consider doing the following:

  1. Start early. Building a patient registry and accruing meaningful data takes time, and the sooner you start the sooner you will have a robust population of patients and a collection of real world  outcomes to inform your research. Ideally, the registry should be part of the clinical development plan, and launched early (i.e., during preclinical or early phase of the research).
  2. Engage stakeholders. Patient registries can become a hub for the patient and treating physician communities, which gives researchers an amazing opportunity to make connections and get the word out about their work. If you use the registry to engage with stakeholders and participate in conversations about the disease, you can build trust, gain important insights and create a network that may facilitate recruitment and retention in your clinical studies. 
  3. Think big picture. To make the most of your registry, set goals from the outset about what you want to accomplish with your research, whether it be exploring clinical outcomes, validating patient-reported data, better understanding healthcare resource utilization or determining what the appropriate population for your clinical studies might be.
  4. Make and maintain connections. In rare disease research, every patient is important, so you cannot afford to lose them to follow-up.  To keep them engaged, stay actively involved in the registry by sharing updates about study progress and outcomes. This ensures that the patient community will be aware of your work and it will encourage them to talk about your study. The more buzz you can generate, the more successful your recruiting and retention efforts will be.
  5. Provide and collect feedback. A registry can be more than a passive database. It can be a place where people connect and communicate with each other about their disease. Take advantage of that networking opportunity by asking for feedback about what would make a clinical trial more appealing to them, and by providing information that they will find valuable. Participating in this two-way dialog will engender trust and demonstrate to participants that you are invested in helping them treat their disease and improve their quality of life.

Registries are an invaluable tool to any clinical research program, but for rare disease research, they can act as a lifeline between patients and the researchers trying to help them. By following these guidelines you ensure everyone derives the greatest return on their investment in the registry community. For more insights into patient registries, read about the Registry of Patient Registries.

Topics in this blog post: Biopharma, Registries, Rare Diseases, Collaboration