Table 1 How to establish a rare disease SOAR collaborative

1. Find your fellow patients, families, and patient advocates who want to accelerate therapy-oriented research and development for the disease in question. Important characteristics: ability to give/raise research funds; good communication skills; willingness to devote time; openness to new ideas; ability to maintain confidentiality when needed within the SOAR.

2. Interview and select a small group of scientists/physician scientists appropriate to the SOAR’s objectives. Selection should be based on their research backgrounds, their genuine willingness to work collaboratively and openly share unpublished data and their familiarity and willingness to establish relationships with patients and families.

3. Clearly identify and state main objectives of the SOAR. Be as specific as practical with regard to time frame, research strategy and/or development of treatment strategies.

4. Establish a coordinated system of collaboration and communication between the scientists, ideally with one scientist acting as the Chair of the scientific working group.

5. Identify a SOAR Project Manager to monitor and coordinate the scientific collaborative and to link the activities of the scientists with the patient advocates.

6. Schedule regular briefings for SOAR patient advocates and twice-annual in-person joint meetings of patient advocates/families with SOAR scientists.

7. Identify a small group of senior scientists to serve as an outside advisory group to be called upon as needed for advice.

8. Develop payment plans, grant agreements and milestones for researchers.