The Steering Committee provides leadership, strategic direction and guidance in identifying the main questions that will be addressed as well as recommend individual experts from academia, industry, government, and community to ensure adequate representation of expertise. The Forum rotates the steering committee membership to ensure leadership opportunities to more individuals, especially those from industry. The industry co-chair position will be rotated on a two-year basis. (* Denotes Steering Committee co-chairs.)
| Categories | Members | Affiliations |
| Academic | Steven Gray | University of Texas Soutwestern Medical Center |
| Scott J. Steele | University of Rochester, Medical Center | |
| Marshall L. Summar* | Children's National Health System | |
| Patient Representatives & Advocacy | Tara J. Britt | Patient Advocate |
| Sandra Lehrman | Patient Advocate | |
| Caroline Loewy | Patient Advocate | |
| Susan Nichols | Patient Advocacy | |
| Peter L. Saltonstall | National Organization for Rare Disorders (NORD) | |
| Frank J. Sasinowski | EveryLife Foundation for Rare Diseases | |
| Forum | Veronica Miller | Forum for Collaborative Research, University of California, Berkeley, School of Public Health |
| Government | Anne Pariser | National Institute of Health (NIH), National Center for Advancing Translational Sciences (NCATS) |
| Industry | John F. Crowley* | Amicus Therapeutics |
| Timothy J. Miller | Gene Therapy Company | |
| Jeffrey Sherman | Horizon Pharma, Inc. | |
| Regulatory | Violeta Stoyanova-Beninska | EMA/ Committee for Orphan Medicinal Products/ Policy, Regulatory & International Affairs |
