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Early Access to Orphan Medicines: Highlights from the World Orphan Drug Congress Barcelona, 2017

By Kieron Lewis, Director of Strategic Consulting

Our team at Clinigen continued to support the World Orphan Drug Congress (WODC) by attending and presenting at this year’s meeting in Barcelona from November 13th-15th.

The meeting attracted over 350 delegates from pharmaceutical and biotech companies as well as patient organisations and service providers. It served as an excellent forum for orphan drug professionals looking at the complete value chain of orphan drug development that spans R&D, clinical and commercial development and market access.

Managed Access Programs and Real World Evidence

Clinigen has successfully delivered over 70 Managed Access Programs (MAPs) for rare disease treatments, partnering with small biotech companies as well the top 10 pharma. Rob Fox, Clinigen’s Head of Business Development for Managed Access, delivered an excellent presentation where he shared his insights into the future of MAPs, and how this contrasts with approaches that have been used by pharma and biotech companies over the last 10 years.

One of the key areas we see developing is the collection of valuable Real World Evidence (RWE) through MAPs. Now that companies are becoming increasingly aware of what’s possible, our clients want more than just a supply chain solution for their MAPs, therefore building in the option to collect RWE is just one way that the benefits of a MAP to payers, drug developers and patients with rare diseases can be enhanced.

Reimbursement of orphan medicines

Other major talking points at this year’s WODC included how countries in the EU are increasingly working together to better assess the value of orphan medicines and determine appropriate levels of reimbursement.

Many health authorities are also implementing schemes that accelerate access to orphan drugs, such as encouraging early dialogue on the clinical development roadmap and allowing MAPs for selected patient groups to form part of broader launch plans. Clinigen supports this approach, advising our partners that any plans for Managed Access are considered early in the life cycle, usually in phase 2 but for rare diseases this can be as soon as phase 1.

In order to reach the thousands of patients suffering from rare diseases whose treatment options can be extremely limited or even non-existent, we need to prepare companies for the high demand for access to treatment prior to approval.

Patient Groups

Patient groups representing many rare diseases, including Duchene Muscular Dystrophy, were very active in contributing to discussions on access to treatment, and the challenges faced when waiting for central EMA approval, local reimbursement and eventual drug availability. This whole process can often take years, and for smaller companies the costs involved on top of the investment already made into the development of a medicine can often present challenges.

If any of the topics covered in this blog are of interest then please get in touch, I would love to hear from you. Contact me at or +44 7788 313990

Watch out for our next blog, which will go into more detail about “Early Access to Orphan Medicines; A Glimpse into the Future”.

To learn more about Managed Access Programs and Real World Evidence read our article here.

Kieron Lewis Director of Strategic Consulting

Early Access
20 Dec 2017Insight Blog
Early access to orphan medicines – a glimpse into the future
By Kieron Lewis Director of Strategic Consulting
  • Orphan Medicines
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  • Medicine Lifecycle
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  • Experimental Medicines