Graham March, Founder and Chairman of Special Products Limited,
discusses the "specials" sector
We in the "specials" sector focus on rare, life-threatening illnesses, which typically affect only 40 or 50 patients and sometimes significantly fewer. There are usually no approved drugs for these illnesses because the number of patients is so small and the patients are often children, both of which militate against a conventional approach to drug development.
In the hope of treating these very small numbers of patients, doctors have turned to existing drugs developed and licensed for other illnesses. With the help of the reformulation skills of the "specials" industry, such drugs become "specials" - the official term in the UK for a medicine being used to treat an illness that it isn’t approved to treat
Whilst "specials" are unlicensed, they are subject to rigorous quality standards imposed by the Medicines and Healthcare products Regulatory Agency (MHRA), which ensures they are made in proper manufacturing facilities and are stable, suitable for use and subject to an effective recall system
Doctors take full responsibility for prescribing "specials" but in my view it would be better if a doctor could base decisions on clinical evidence. The MHRA requires that any Serious Adverse Drug Reactions are reported but efficacy data is not currently being fed back to the MHRA. If this data were collected from the moment that a special was first prescribed, it would form an excellent starting point for a simplified regulatory approval route.
I think that any special medicine that has been in use for 10 years, in either paediatric or adult patient populations, should be subject to a simplified approval procedure. Many of those products have a long history of efficacy and safety.
It would be useful if the MHRA could issue guidance on how "specials" could be licensed.
I think that the approval procedure for special medicines could be based on the orphan drug approval process, in which several years’ market exclusivity is awarded to the company whose product is the first to be approved in a particular indication.
The orphan drug process was introduced as an incentive for the pharmaceutical industry to fund the development of medicines for small patient populations - the marketing exclusivity period creates the opportunity to recoup the investment in drug development. Special medicines are prescribed for even smaller patient populations so an appropriately structured arrangement would be required.
I think that the development of a regulatory approval structure would help doctors, patients and all other stakeholders including the "specials" industry. It would also underline the vital contribution that the "specials" industry makes to the nation’s healthcare system.