As industry leaders, we should be breaking down these barriers and driving patients to the answers they need, and ultimately, to life-changing treatments.
The need for education among rare disease patients is three-fold. First and foremost, there have to be patient populations that receive a diagnosis and are ready and willing to participate in a trial. Secondly, as gene therapy approvals increase, there will be a need for a significant shift in the mindset of clinicians as they evaluate these therapies and how they communicate that value to patients. And finally, tough conversations will have to happen to determine when and if such a gene therapy is “worth it” for a family.