2022年2月2日

Patients with rare diseases and their caregivers face many unique challenges along the road to diagnosis, treatment and—ultimately—optimized quality of life. With all of the complex information and systems out there, the landscape can be difficult to navigate. Where can they go for guidance?

Ray Huml, Vice President, Medical & Scientific Strategy and Head of the Rare Disease Consortium at Syneos Health—and editor of the recently published book Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver Perspectives—returns to the podcast to discuss his dual experience as a scientist and caregiver, what drove him to create two informative books, and insights on cracking the code to reimbursement in rare disease.

For more on the topics covered in this episode, be sure to check out:

How Competition Affects Market Access for Rare Disease Therapeutics – Part I

How Competition Affects Market Access for Rare Disease Therapeutics — Part II

サイネオス・ヘルスのポッドキャスト:Real World Evidence and Rare Diseases

Innovation Perspectives in Rare Disease Drug Development

Find the books discussed in this episode:

Rare Disease Drug Development: Clinical, Scientific, Patient, and Caregiver Perspectives

Muscular Dystrophy: A Concise Guide

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