Patients could receive medicines before formal approval in exchange for monitoring data
Critically ill patients in the UK could receive new medicines before they are formally approved under a new scheme beginning in April. For diseases where no approved treatment exists, or patients who have exhausted other treatment options, doctors will be permitted to prescribe experimental therapies, which will be supplied by companies to the NHS free of charge.
In order to qualify for the Early Access to Medicines Scheme (EAMS), the product must first be designated as a ‘Promising Innovative Medicine’ by the UK Medicines and Healthcare products Regulatory Agency (MHRA). Then, once the MHRA is satisfied that the drug is safe and the potential benefits outweigh the risks, it will issue an EAMS ‘scientific opinion’. The company will then supply the medicine for free until it is officially approved.
One benefit of the scheme to industry is closer liaison with the regulators over requirements of clinical trial design and manufacturing. Firms will also get much earlier access to data about how their drugs perform in real world settings. While this is not a substitute for comprehensive Phase III clinical studies, it provides a route for small companies, patient groups and charities without the necessary funds to attract investment and bring drugs to the market more easily.
The scheme will not replace the existing pathway to European approval for drugs, and once approved, the medicines will still be subject to value judgements from the UK’s National Institute for Health and Care Excellence (Nice).