Rare disease market access - strategic and tactical challenges facing orphan medicines entering Europe

Pharma Market Access Insights - from Mtech Access

We explore the challenges facing orphan medicines and other innovative rare disease treatments looking to achieve market access in Europe.
Orphan medicines and rare disease treatments are faced with a complex, ever-changing market landscape. These treatments also face unique tactical challenges on the route to securing market access.

Here we bring together specialists from our global market access (Rob Taaffe), systematic review (Regina Leadley), and health economic (Calum Jones) teams to explore this environment and some of the key hurdles that Pharmaceutical and Medtech innovators need to overcome in order to achieve patient access.

Rob, Regina and Calum discuss:

• Current and future market events and trends for rare disease products across Europe
• Clinical evidence development, including tactical challenges around evidence generation and how to address these
• Building the health economic case, including key principles and tactical nuances for health economic modelling in rare and orphan disease

This podcast was originally broadcast as a live webinar on 17th November 2021. To learn more and request a copy of the slides used please visit our website.

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1. Rare disease market access - strategic and tactical challenges facing orphan medicines entering Europe (00:00:00)

2. Today's speakers (00:01:15)

3. Today's agenda (00:02:10)

4. Who are Mtech Access and what do we know about market access for rare diseases? (00:03:40)

5. Current and future trends: what should we track and watch out for? (00:06:59)

6. Disease knowledge and awareness and clinical evidence: How important is the patient voice? (00:18:03)

7. Economic evidence: Are there health economic modelling principles for rare diseases? (00:24:50)

8. Summary & Q&A (00:51:24)