Under this new framework, regulators may be able to approve the treatment even when large patient populations don’t exist, if researchers can clearly show:
- How a disease works at the genetic, cellular, or molecular level;
- How a treatment addresses that mechanism, and
- Improvements that would not have been seen in patients without treatment.
This shift towards evaluating potential therapies based on how well they address specific mechanisms could speed up the development of rare disease treatments. It also means that it’s more important than ever to make sure patients and healthcare providers have a clear understanding of the science behind the disease and treatment so that they can make confident, informed decisions about their care.
Our other ideas worth exploring
Prioritizing Patient Messaging: A framework centred in patient experience
We use a patient-centric framework to help prioritize the focus of patient messaging across three phases: activate, evaluate, and initiate.
AI search is transforming patient marketing
AI search is transforming digital patient marketing. We outline how biotech marketers can create content that is more likely to show up in AI overviews.
Bedside Manners: How an OBGYN approaches patient education
An interview with Dr. Hava Starkman, a clinical fellow in midlife women’s health, about how she approaches patient education.