Jaye Bea Smalley from Celgene and Linda Brennan from Cystic Fibrosis Foundation ran a workshop at the DIA (International Meeting for the Drug Information Association) on this topic using the audience responses to a case study/questionnaire as either sponsors or patient advocates.  The results were interesting as the audience evaluated both the benefit and level of investment required to achieve the benefit of a surprisingly long list of patient engagement opportunities. The case study setting was for an orphan drug in development by a medium-sized biotechnology sponsor.

We were asked to identify three opportunities that were perceived by both sponsors and patient advocates as having the ideal: high benefit with low investment.  Although investment was typically defined by the group acting as sponsors as being financial, for the patient group investment wasn’t just money, but also included their time and other difficulties (for example childcare) that they would have to overcome in order to bring about the benefit. Engagement opportunities included the patient group:

• giving insights to the sponsor regarding how the orphan drug might meet the needs of the patient community
• facilitating the development and validation of translational tools
• providing the sponsor with funding for their research
• conducting preference studies to support benefit-risk assessment
• giving feedback on the informed consent process
• helping with the setting of eligibility criteria
• helping determine which endpoints are the most meaningful and practical for patients
• accompanying the sponsor to FDA meetings to give insights and support
• helping the sponsor determine details of the natural history of the disease
• supporting recruitment and retention, and increasing awareness of the clinical research process
• helping disseminate study results in patient-friendly language
• giving feedback on the overall trial experience.

Although the workshop audience assessed most of these opportunities to be of high benefit, it was interesting to see how the perception of the investment needed differed between sponsors and patients. In fact, few of these assessments matched; but as a participant noted, what is significant investment to a patient may only be a fraction of what a sponsor would consider a high investment. By taking only those opportunities where both parties agreed on High Benefit, Low Investment, the most important opportunities might be excluded. He suggested that the best way to judge which opportunities should be followed up, would be by engaging the patients in the discussion.  Which, after all, is the whole idea in the first place.