The UK Medicines and Healthcare Products Regulatory Agency (MHRA) will launch a scheme next month to allow doctors to prescribe medicines without a marketing authorisation to patients with life-threatening conditions.
The Early Access to Medicines Scheme (EAMS) will allow sufferers with an unmet medical need to take unlicensed or off-label medicines if the MHRA believes there is a positive risk-benefit balance.
The MHRA will evaluate medicines for inclusion in the scheme via two steps. If early clinical data indicates a drug may be suitable, an MHRA scientific meeting applies a Promising Innovative Medicines (PIM) designation.
The second step assesses data submitted by pharmaceutical applicants on a drug’s quality, safety and efficacy, as well as information from the patients who the medicine will help. The MHRA will form a Scientific Opinion on the benefits and risks of the medicine, which will be available on its website for doctors and patients to make a decision on whether to use the medicine before its licence is approved.
The MHRA told In-Pharmatechnologist.com the trigger for an Early Access to Medicines Scientific Opinion does not necessarily have to be the submission of a dossier for marketing authorisation application, but a compelling case based on the evidence collected by the MHRA.
“It is conditional on data from the development process of the product that indicates that the benefit-risk profile of the medicine is positive. The scheme will be limited to medicines representing a significant advance in treatment in an area of unmet need,” said Nick Spears, MHRA spokesman.
“The MHRA will only give an EAMS opinion in cases where data from the development process of the product indicate that the benefit-risk profile of the medicine is positive, in areas of unmet medical need,” he added.
“In these circumstances, unmet need means that there are no suitable available therapies and this includes patients who have failed available treatments e.g. patients who have advanced and unresponsive cancer.”
The US launched a comparable Breakthrough Designation Scheme which approved its first treatments – for cystic fibrosis – in January 2013.
“Both schemes have similar aims,” said MHRA’s spokesman, “but different approaches for facilitating earlier access to innovative medicines for patients with serious and life threatening conditions.
“The UK EAMS is a scheme to support access in the UK to unlicensed or off-label medicines in areas of unmet medical need.” Conversely, he said, “the US breakthrough designation relates to the process of drug licensing” and is intended merely to expedite the development and review of drugs for life-threatening conditions.
The fee for an EAMS designation meeting for pharmaceutical companies will be in the region of a MHRA scientific advice meeting, said Spears, and the cost of entry into the Early Access Scientific Opinion step will be around £30,000 ($49,000).