Posted 01 October 2020 | By
The guidances are the latest in a suite of post-transition guidance from the agency as it prepares for the Brexit transition period to expire at the end of the year. (RELATED: MHRA posts suite of post-transition guidances, Regulatory Focus 2 September 2020; MHRA: Post-transition guidance on licensing medicines, Regulatory Focus 3 September 2020; Pharmacovigilance and QPPV: Post-transition guidance from MHRA, Regulatory Focus 4 September 2020).
In its guidance on orphan medicinal products, MHRA explains that it will provide incentives to encourage the development of products to treat rare diseases, including market exclusivity, refunds for marketing authorization fees and by waiving scientific advice fees for UK-based small and medium enterprises (SMEs).
MHRA says it will review applications for orphan designation at the time of a marketing authorization application (MAA), and explains that, “There is no pre-marketing authorisation orphan designation.”
The requirements for orphan designation mirror those in the EU. For a medicine to qualify for orphan designation it must be intended to treat, prevent or diagnose a disease that is life-threatening or chronically debilitating and occurs in no more than 5 in 10,000 patients in Great Britain. Alternatively, a medicine may qualify for orphan designation if it is unlikely a company will recoup its investment to develop the product. Additionally, a medicine may only qualify if “no satisfactory method of diagnosis, prevention or treatment of the condition concerned exists in [Great Britain], or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.”
To apply for orphan designation, applicants must submit a Great Britain orphan drug designation application form along with their marketing authorization application. MHRA says it will make a decision on orphan status at the time it decides whether to approve the marketing application.
As in the EU, the UK will offer marketing authorization holders 10 years of marketing exclusivity that will begin from the date of first approval in Great Britain or in the EU/European Economic Area. The UK will also recognize market exclusivity for centrally authorized medicines that are converted to UK marketing authorizations. Similarly, the UK is offering the same two years of additional exclusivity for medicines authorized with the results of studies from pediatric investigation plans (PIPs) included in the product information.
Supplying medicines to Northern Ireland
According to the EU Withdrawal Agreement, “Goods placed on the market in the European Union or the United Kingdom before the end of the transition period may continue to circulate between these two markets from 1 January 2021.”
MHRA explains that medicines are considered “placed on the market” if they are available for sale or supply. To be considered on the market before the end of the transition period, medicines must “have been certified by a qualified person and released for sale or supply before 11 p.m. on 31 December 2020.” Such products may remain on the market between the three jurisdictions, Great Britain, Northern Ireland and the EU, “Without additional regulatory checks.”
However, MHRA explains that wholesalers or manufacturers in Great Britain are responsible for confirming that the person in Northern Ireland is authorized to receive any medicines before agreeing to supply the products.
To demonstrate that a batch of medicines was placed on the market in time, MHRA says a written statement from the manufacturer or wholesaler or reference to company internal systems showing the batch certification.
Orphan medicinal products, Supplying medicine to Northern Ireland