On January 27, 2022, The EveryLife Foundation for Rare Diseases, in partnership with the Biotechnology Innovation Organization (BIO), National Health Council, and Pharmaceutical Research and Manufacturers of America (PhRMA), hosted a webinar which introduced a vital new resource for rare disease nonprofit organizations searching for treatments: the Guide to Patient Involvement in Rare Disease Therapy Development.

Patient Focused Drug Development

This resource has been developed through a collaborative process. Patient focused drug development has become something of a buzzword in the rare disease space in recent years. This approach is intended to prioritize the needs of the patient and the treatment outcomes desired by them. This means that patients are involved in the development process from the beginning.

Speakers for this webinar program included:

1. Annie Kennedy, EveryLife Foundation for Rare Diseases
2. Ryan Fischer, Parent Project Muscular Dystrophy
3. Cara O’Neill, Cure Sanfilippo Foundation
4. Kristin Van Goor, Vertex Pharmaceuticals
5. Kim McCleary, The Kith Collective
6. Eric Gascho, National Health Council

This webinar is part of a series of workshops that have the purpose of educating participants about the patient focused drug development process and how it can be applied to rare diseases in the context of US Food and Drug Administration (FDA) guidelines. Work on the guide began almost a year ago.

Ryan touched upon some of the goals that the guide was created to meet such as:

• How should organizations prioritize resources?
• How can organizations better engage with drug developers?
• How should nonprofits steer their strategy for facilitating drug development?

The FDA has released some of its own guidelines, but for patient advocates whom often have little in the way of formal training or education in the medical field, these can be confusing. Ultimately, the guide is meant to help save time first and foremost.

Kristin shared some of the industry perspective and noted that initially patient focused development was little more than companies organizing communications and meetings with patients. However, it has become much more in-depth over the years. The guide is a vital tool that can help stakeholders in the field set their own priorities more effectively.

Cara noted that there is actually a lot of information out there for nonprofits on the subject of patient oriented drug development, but it’s hard to know what is really worthwhile and legitimate, and it’s easy to get overwhelmed. The guide will serve as a trustworthy and convenient resource that also has some greater validity due to some input from the FDA itself.

Kim says that the guide has been organized into three sections, including a context and orientation portion that will help bring in new organizations. It also provides more detail about the involvement of the FDA. The guide includes eight different areas of focus and is delivered in an action oriented format that is explicitly directed to different stakeholders, such as sponsors, companies, and advocacy leadership. This will also create opportunities for different stakeholders to work together.

Eric notes that workshop summaries are included as an appendix, and the guide is also heavily sourced. A total of 112 sources are included for reference, all of which are freely accessible to the public.

You can check out the complete guide for yourself here.