To successfully navigate a rare disease pathway, it is necessary to understand the interdependency of clinical, regulatory and commercial branches, as well as their different and sometimes competing challenges.
A panel of experts recently came together to share insights into this complex and evolving arena and discuss some of the ways forward. The panel included the author, Christian Schneider; Dennis Earle, Senior Director – Development Consulting & Scientific Affairs; Brad Carlin, Senior Advisor, Data Science and Statistics; and Erika Wissinger, Senior Director within Market Access and Healthcare Consulting, all at Cencora.
Meeting an unmet need
One of the challenges in the rare disease development space is convincing payers that the therapy meets an unmet need. Education of some healthcare providers may be required to demonstrate the effect a therapy will have on the care pathway for patients to secure their advocacy. This means closely examining the question: what is the symptom burden and actual impact of burden of the disease?
“That leads to quantifying the unmet need, which is important for payers, particularly with a new treatment for which there is no real standard of care, other than maybe symptomatic treatment,” Erika said.
Actively engaging patients in the pathway can also help boost their access to treatment, as they are best placed to provide a deeper understanding of the burden of rare disease. “Patient advocacy can also contribute to the generation of real-world evidence, which can be factored into the total package of evidence that’s put in front of payers,” Erika added.
Read the article in full here.
