The last six months have delivered a wave of clinical, commercial, and regulatory developments regarding the treatment of sickle cell disease (SCD). Indeed, the U.S. launch of two new therapies for SCD, a stream of early and interim clinical trial data on emerging SCD therapies, the FDA’s fast track and orphan drug designations for SCD therapies, and a controversial opinion from the Institute for Clinical and Economic Review (ICER) are just a prelude to what will be a dynamic market well into 2025. As we kick off several SCD market research studies, we find ourselves with an ever-growing list of questions about market dynamics, patients, physicians, value, and access, as well as new and emerging therapies. Below are several questions that are top-of-mind, especially as we think about the decision-making journey that lies ahead of each new SCD therapy.
- How and to what extent are Emmaus Medical’s Endari, Novartis’s Adakveo, and Global Blood Therapeutics’ Oxbryta being incorporated into the treatment of SCD patients?
- What are the key patient segments within the SCD population, and which ones will my product treat best?
- What is a clinically and commercially viable target product profile for my novel SCD treatment?
- How receptive are physicians and patients to gene therapies to treat SCD? How do physicians expect gene therapy to affect the treatment algorithm?
- What clinical outcomes have the most impact on prescribing decisions for SCD, and how does my product perform against these expectations and the competition?
- What are the key value and access considerations and requisite evidence generation to support the future uptake and reimbursement of new therapies for SCD?
- What is the potential impact of the draft findings from ICER regarding the cost-effectiveness of Novartis’s Adakveo and Global Blood Therapeutics’ Oxbryta?
The answers to these questions will require continuous refinement as the pharmaceutical market and the SCD therapy market, in particular, continue to evolve.