Legal framework for Early Access to Medicine Scheme planned

Responses to the consultation should be submitted by 17 September 2021.

The EAMS is one way through which a patient with a life threatening or seriously debilitating condition can gain access to a medicine before it has gained approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA). The aim of the proposed legal framework is to clarify the legal basis for the EAMS, to enhance the flexibility of the scheme and to ensure it remains an attractive mechanism for companies to provide medicines to patients prior to licensing.

Life sciences expert Helen Cline of Pinsent Masons, the law firm behind Out-Law, said: "The Early Access to Medicines Scheme is an important existing regulatory flexibility to ensure that UK patients are some of the first in the world to access new treatments, effectively delivering a scientific opinion which supports clinicians and patients when prescribing an unlicensed medicine."

Sarah Taylor, also of Pinsent Masons, added: “One of the driving forces behind the proposed changes to the scheme is to ensure that it, and therefore the UK market, remains relevant and attractive to companies following Brexit.”

The EAMS currently operates on a non-statutory basis. The government is looking for feedback on proposals to introduce new provisions into the Human Medicines Regulations 2012 to provide a specific statutory basis for the scheme. "The proposal suggests introducing provisions that simplify supply, provide a framework for collection of real world data, provide clarity around liability for prescribing and set out pharmacovigilance obligations," Cline said.

Under the EAMS, drug companies have the chance to follow a fast-track process for bringing their products to market. They can first apply for a 'promising innovative medicine' (PIM) designation from the MHRA.

A PIM designation signifies the potential of a drug for treating patients with life threatening or seriously debilitating conditions for which there is either no cure or where existing treatments are unsatisfactory during a stage at which the drug is still being developed and has not been finalised.

On obtaining a PIM designation, drugs manufacturers can then apply to the MHRA for a scientific opinion on whether available data shows that doctors should be able to prescribe the drug to patients before the product has gone through the full licensing process. For that to happen, the potential patient benefits must outweigh the risks.

Drug companies cannot charge for the use of their treatments under the scheme until they obtain a license for the product, but they can recover costs.

The government said: "Since there is no legislative provision that covers or specifically mentions EAMS, there are some aspects that would benefit from clarification in law, providing a legal basis for the delivery of EAMS." It said the new legal framework is aimed to improving legal certainty and reducing regulatory burdens.

The proposal includes provisions that would allow the EAMS scientific opinion to be withdrawn from companies who do not comply with an EAMS approval. There are also provisions planned to place obligations on companies to disclose information that may affect the decision on risks and benefits of a treatment.

Other rules aim to simplify the supply of drugs. One of the proposed amendments to EAMS permits any site with a human medicines manufacturers licence to allow manufacture, assembly or importation at the site. "This is intended to simplify regulatory requirements and avoid the current disincentives to a supply chain utilising UK sites, removing the need to classify supply aspects under existing legislation for unlicensed medicines or off-label provision," Cline said.

The new law would also provide a framework for the collection of real world data. Companies which hold an EAMS scientific opinion would be permitted to collect data in the context of EAMS regardless of whether the product is considered an investigational medicinal product or not. However, this would only be possible if the MHRA has no concerns about collection of data and patients have given consent. Cline said: "Currently it is thought that the collection of real-world data may fall under the requirements of the clinical trial regulations and therefore a clinical trial authorisation application might be required."

Other provisions are aimed at providing clarity around liability and the use of EAMS medicines in line with the General Medical Council (GMC) recommendations on prescribing unlicensed or off-label medicines.  Further, a number of pharmacovigilance obligations, to support safe use and promote patient and healthcare practitioner confidence, are being considered for inclusion in the legislation.