Free of charge medicine schemes in the NHS. A local and regional Drug
and Therapeutic Committees (DTC) experience.
Introduction: Free-of-charge (FoC) medicine schemes are increasingly
available and allow access to investigational treatments outside
clinical trials or in advance of licensing or NHS commissioning.
Methods: We retrospectively reviewed FoC medicine schemes evaluated
between 2013 and 2019 by a single NHS trust and a regional drug and
therapeutics committee (DTC). The details of each locally reviewed FoC
scheme, and any nationally available MHRA Early Access to Medicines
Scheme (MHRA EAMS) in the same period, were recorded and categorised.
Results: Most FoC schemes (95%) allowed access to medicines intended to
address an unmet clinical need. Over 7 years, 90% were company-FoC
schemes and 10% were MHRA EAMS that were locally reviewed. Phase 3
clinical trial data were available for 44% of FoC schemes; 37% had
phase 2 data; and 19% were supported only by phase 1, retrospective
observational studies, or pre-clinical data. Utilisation of company-FoC
schemes increased on average by 50% per year, while MHRA EAMS showed
little growth. Conclusion: Company-FoC medicine schemes are increasingly
common. This may indicate a preference for pharmaceutical companies to
independently co-ordinate schemes. Motivations for company-FoC schemes
remain unclear and many provide access to treatments that are yet to be
evaluated in appropriately conducted clinical trials, and whose efficacy
and risk of harm remain uncertain. There is no standardisation of this
practice and there is no regulatory oversight. Moreover, no standardised
data collection framework is in place that could demonstrate the utility
of such programmes in addressing unmet clinical need or allow generation
of further evidence.