Changing the way we develop orphan drugs – The need of early dialogue

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Martin Andrews, SVP of Rare Diseases, GSK talking about adaptive licensing

I attended the Fast Tracking Orphan Drugs Terrapinn Briefing in London last week and some interesting common themes were discussed: the nuisance in regulatory unpredictability, importance of early regulatory and patient engagement as well as the challenges and advantages of adaptive licensing. With the orphan drug market set to reach a value of 127 Billion USD in 2018 (according to Carlos Camozzi, Chief Medical Officer of Orphazyme), how should the industry be navigating existing fast track orphan schemes for breakthrough therapy designation?

Of particular interest, Martin Andrew Senior Vice President of Rare Diseases at GSK talked about how there is nothing new in the concept behind adaptive licensing. He discusses how mechanisms already exist to allow access to new medicines. What adaptive licensing changes is the mind-set and approach to this, the opportunity for safe-harbour dialogue between regulators and patients.

Therefore adaptive licensing could allow valuable regulatory advice without commitment, discharging some of the risk in orphan drug development and approval. To allow adaptive licensing, some obvious criteria must be met, these include easy identification of the sub population of patients affected, having robust surrogate end points to measure, that the potential orphan drug justifies early access (treating a rare unmet need) and that clinical trials can be expanded. Of course there are also clear disadvantages to adaptive licensing, the advice from regulators are not binding and there is disconnection with HTA and pricing issues, but what it does allow is early dialogue and alignment.

Martin Andrews will be presenting the progression of his case study on adaptive licensing as well as downstream CMC challenges at the 5th World Orphan Drug Congress 12- 14th November in Brussels. To hear more about this please visit our website here.

 

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