Orphan Unknowns: What the MHRA Guidance Will Mean After Brexit

Developers of orphan drug products face some important changes with the approval process in the UK after Brexit. To help companies prepare, the MHRA published new guidance: “How the MHRA will manage orphan medicinal products from 1 January 2021 in Great Britain (GB)”. While most of the criteria to qualify for orphan drug designation align with the EU, there are small but significant differences.

First, companies can only receive an orphan drug designation (ODD) when they apply for their marketing authorisation application, which means there will be no free protocol assistance or scientific advice. This sets MHRA apart from EMA, which offers discounted or free scientific advice as well as other avenues of support for orphan drug developers. The UK will continue to “offer incentives in the form of market exclusivity and full or partial refunds for marketing authorisation fees to encourage the development of medicines in rare diseases”, although the details on how these compare to EU ODD incentives are yet to be released. Those wanting scientific advice from the UK agency can still receive it, however, unless they are designated in the UK as a small to mid-size enterprise, they will have to pay for that expertise.It is an interesting move by the MHRA but while there are certainly negative aspects to it, I believe there are also advantages. The following are some of the pros and cons that my colleagues and I have observed …

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