The significant financial investment required for the research and development of a new medicine means that, in normal market conditions, there is generally insufficient commercial incentive for pharmaceutical companies to develop drugs intended for use by only small numbers of patients.
Rare diseases
It is estimated that between 5,000 and 8,000 distinct rare diseases exist, affecting between 6% and 8% of the total population. In the UK this amounts to approximately 3.5m people suffering from rare diseases.
To encourage the research and development of medicines for rare diseases, known as “orphan” drugs, the UK regulatory framework offers a number of incentives where a medicine meets certain criteria. If an organisation wishes to obtain such incentives, it must submit the Great Britain Orphan Drug Designation Application Form to the Medicines and Healthcare products Regulatory Agency (MHRA) with its marketing authorisation (MA) application.
Criteria for orphan designation
To qualify for orphan designation, a medicine must be intended for the diagnosis, prevention or treatment of a life-threatening or chronically debilitating condition. There must also be no existing satisfactory method of diagnosis, prevention or treatment for the relevant condition or, if there is, the new medicine must be of significant benefit over current methods.
In addition, the medicine must meet either the “prevalence” or the “insufficient return on investment” criterion. These are as follows:
- Prevalence - The applicant must show that the condition the medicine is intended for affects no more than five in 10,000 people in the UK when the application is made.
- Insufficient return on investment - The applicant must show that, without incentives, it is unlikely that the marketing of the medicine would generate sufficient return to justify the necessary investment.
Incentives for orphan designation
Ten-year market exclusivity
Once MA is granted for an orphan designated medicine, it will benefit from market exclusivity in Great Britain for up to 10 years in respect of the particular indication. During this period, no other marketing authorisation will be issued for the same therapeutic indication in respect of a "similar medicinal product" unless:
- The MA holder for the original orphan medicinal product gives consent to the second applicant, or
- The MA holder for the original orphan medicinal product is unable to supply sufficient quantities of the medicinal product, or
- The second applicant can establish that the second medicinal product is safer, more effective or otherwise clinically superior to the orphan medicinal product.
Fee reductions
Companies applying for regulatory approval in respect of a designated orphan medicine may also be entitled to pay reduced fees for MA applications.
GB and UK orphan medicines post-Brexit
If a medicinal product has been designated orphan in the EU before 1 January 2021, a GB orphan MA application can be made. A UK-wide orphan MA application can only be considered in the absence of an active EU orphan designation.
If a UK-wide orphan MA is granted and the medicinal product subsequently receives EU orphan designation, the MA holder would need to submit a variation to change this to a GB orphan MA.
Emily Heenan