Rare Disease Day: Driving Progress through Collaboration

Rare Disease Day 2017

Rare Disease Day: Driving Progress through Collaboration

On February 28, rare-disease communities around the world come together to advocate for an increase in research focused on rare diseases. When it comes to clinical research for rare and orphan diseases, the relationship with the patient and advocacy groups is critical to success. In support of Rare Disease Day, we’ve asked Shazia Ahmad, Director, Patient and Physician Services (PPS) a specialist in rare diseases, how sponsors can make working with advocacy groups a win-win collaboration.

Q. What are the benefits of collaborating with an advocacy group?
A. Clinical trials in rare and orphan diseases have unique recruitment challenges because of the small patient populations and complex diseases. Patients and patient advocacy groups bring a perspective that is unique and critical to the success of clinical trials – especially in rare and orphan diseases. In-depth interviews and focus groups can help us gather patient opinions and preferences. Qualitative research helps us single out any study requirements that may be too burdensome for patients. You may learn that the visit schedule is too demanding or the patient reported outcomes tools are not suitable for the patient population at hand. By partnering with an advocacy group that represents patients from a specific therapeutic area or with a particular disease, sponsors are able to obtain critical disease and patient perspective that help optimize protocol design and allow for a successful clinical trial.

Q. How can sponsors and advocacy groups work collaboratively to support clinical research?
A.
Understanding as much as possible about the patient and caregiver is a critical component to ensure a clinical trial is designed to work well for participants. When I worked at the National Institutes of Health (NIH), advocacy groups and patient representatives would help identify questions for researchers that were important to them. This allowed us to think through critical questions around the disease and to better understand the patient journey. From long-distance travel to dietary requirements, participating in a clinical trial can be very difficult for rare-disease patients and their caregivers. Patient advocacy groups can help sponsors identify the burdens and challenges that patients and their caregivers face on a daily basis. Sponsors need to commit to incorporating appropriate solutions, like concierge travel services, to help patients and their caregivers overcome any barriers to study compliance.

Q. What are some dos and don’ts when working with advocacy groups?
A.
Sponsors should provide information to advocacy groups in ways that they can use it and share it with their members. This provides resources they don’t already have and can help develop a strong relationship built on trust, transparency and commitment. They should also interface with local chapters – this helps create a personal connection. Sponsors should ask advocacy groups about their concerns and challenges about a specific indication.  Sponsors should not stop communicating with advocacy groups after the initiative is complete or a goal has been met. Patients trust and rely on advocacy groups, so by neglecting or damaging the relationship with an advocacy group, sponsors risk the potential of tarnishing their company or brand image.

Q. What are the critical elements sponsors need in order to develop an advocacy group partnership for their next study?
A.
Building trust, transparency, and alignment with patient advocacy groups is critical. It’s important to engage advocacy groups early and often. If possible, sharing data or sharing how patient information will be used in a particular program is impactful for a successful partnership. Sponsors and patient advocacy groups share the same objective: improving patient outcomes. By aligning efforts around this same objective, both parties can experience success and generate actionable results.

Click here to learn more about our big solutions for small patient populations, and contact us to find out how UBC can help you do more for your rare-disease patients.