Interesting flipper, I talked to someone from Northwest Bio about a year ago, and he said they were all very excited about the new approval pathways in the UK. I mentioned the 150-day assessment and the rolling review and he said “yes, those . . . aaaand others.”
It wouldn’t surprise me if Northwest Bio attempts one of these pathways:
Conditional Marketing Authorization Applications The MHRA has introduced a national conditional marketing authorization (CMA) scheme for new medicinal products in UK effective from 1 January 2021. e eligibility criteria for this scheme, as that of the EU scheme, is intended for medicinal products that fill an unmet medical need and for serious and life-threatening diseases where no satisfactory treatment methods are available, or where the product offers a major therapeutic advantage.
The MHRA determines eligibility for a CMA at the time of MAA assessment. The MHRA does not have a specific application route for a CMA. e sponsor needs to file the MAA dossier for a full marketing authorization. At the completion of the MAA dossier assessment, the MHRA will determine whether to approve the application and grant a conditional MA or whether the benefit-risk ratio is negative and reject the application. e CMA may be granted where comprehensive clinical data is not yet complete and available. e sponsor must provide justification for a CMA, including the ongoing clinical studies’ status and timing of the availability of comprehensive clinical data. CMAs are valid for one year and can be renewed annually
Exceptional Circumstances Marketing Authorizations From 1 January 2021, the MHRA’s existing scheme for applications under exceptional circumstances will continue to be available for medicines where a comprehensive data package cannot be provided because the condition to be treated is rare or because the collection of full information is not possible or is unethical. This scheme has the same eligibility criteria as the EU scheme (see Chapter 2). e designation of a product as being eligible for an exceptional circumstances scheme by EMA or another jurisdiction may be taken into account by the MHRA, but the final decision on eligibility of the product for the GB scheme will rest with MHRA.
Orphan Medicine Designation, Orphan Medicine Development/ Marketing Authorization Application In the UK, no premarketing authorization orphan designation process exists, and if a medicinal product has been designated an orphan in the EU under Regulation (EC) 141/2000, an MAA can be submitted for the orphan medicine designation under regulation 50G of the Human Medicines Regulation 2012 (as amended). An orphan medicine designation application and the associated MAA (submitted together) in the UK can only be considered in the absence of an active EU orphan medicine designation.
The orphan designation criteria mirror those in the EU. Medicines need to fulfill the following criteria to qualify for orphan designation in an orphan condition:
* It must be intended for the treatment, prevention, or diagnosis of a disease that is life-threatening or chronically debilitating. *the prevalence of the condition in the UK must not be more than five in 10,000, or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development. * No satisfactory method of diagnosis, prevention, or treatment of the condition concerned exists in the UK, or, if such a method exists, the medicine must be of significant bene t to those affected by the condition. * Satisfactory methods may include authorized medicinal products, medical devices, or other methods of diagnosis, prevention, or treatment used in the UK.
To obtain the designation, the sponsor needs to submit an orphan medicine designation application form along with the MAA. e MHRA makes the decision on orphan status at the time it decides whether to approve the marketing application. This approach differs from EMA, which includes a process for granting the orphan medicine designation in advance of the MAA submission and offers orphan fee incentives and other benefits for sponsors of orphan medicines during development. The MHRA’s advisory committee, the Commission on Human Medicines (CHM), will examine the application for orphan designation concurrently with the MAA under review. Medicines with an orphan marketing authorization will be listed on the UK Orphan Register.
In the UK, no orphan designation is issued separately from the MA. Therefore, if a change of ownership application is submitted, the orphan designation will automatically transfer to the new marketing authorization holder.
It is assumed that the MAA requirements for approval will be similar to EU requirements. ere might be a risk if the MHRA requirements differ, and additional work or evaluation of a new parameter during development is requested. Also, there may be difficulties navigating differences between MHRA and EMA opinions on protocol design or development plans.
Scope of Orphan Medicine Exclusive Approval Once a medicinal product receives MA with orphan designation in the UK, it benefits from 10 years of market exclusivity. e market exclusivity period begins on the date of the first approval of the product.
The UK also will recognize remaining market exclusivity for centrally authorized medicines (granted prior to 1 January 2021) in the EU that are converted to UK marketing authorizations. Unlike the EU, it is not necessary to submit orphan maintenance reports to the MHRA, but they can be submitted as additional information.
Market Access The following three Health Technology Assessment (HTA) agencies have adopted special assessment criteria for orphan medicinal products (OMPs) in the UK:
1. The National Institute for Health and Care Excellence (NICE) includes a program for ultra-orphan medicine (highly specialized technologies [HST]). 2. The Scottish Medicine Consortium (SMC) includes orphan and ultra-orphan modi er criteria. 3. The Welsh agency, All Wales Medicines Strategy Group (AWMSG), includes additional criteria to consider the severity and unmet need.
A nominative prescription of the OMP from a National Health Service (NHS) doctor automatically provides the right to reimbursement to the patient.
