UK Early Access to Medicine Scheme to have statutory footing soon

The Medicines and Healthcare products Regulatory Agency (MHRA) said the relevant new regulations will be laid before the UK parliament “in early 2022”. The decision follows a public consultation on the plans last year.

The MHRA said: “These changes will provide the UK with an opportunity to maximise the scheme’s impact by accelerating availability of medicines for patients, reducing the burden on manufacturers supplying EAMS medicines and facilitating the collection of real-world data which may potentially be used as evidence to support regulatory decision making for future authorisations. This will help support more patients benefiting from important EAMS medicinal products and ensure that the UK remains internationally competitive in the pre-market access landscape.”

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 MHRA. The scheme currently operates on a non-statutory basis. 

Under the EAMS, pharmaceutical companies can follow a voluntary 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 – which lasts for a year and can be renewed – 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.

Pharmaceutical 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 from the NHS bodies that prescribe it.

According to the MHRA, since the introduction of the EAMS in 2014, 100 medicines have been granted PIM status and more than 40 scientific opinions have been awarded.

The plans to provide a specific statutory basis for the EAMS will involve making amendments to the existing Human Medicines Regulations 2012. The legislative changes are to be made under Part 2 of the Medicines and Medical Devices Act 2021, which provides powers to make regulations about human medicines.

The MHRA confirmed that the government will not consult on the detail of the regulations but that they “will reflect the policy as proposed in the consultation” and that industry and other stakeholders will be given the chance to have their say on supporting “detailed guidance”, which is to be developed to underpin the statutory regime, “to ensure proper interpretation and implementation”.

According to the government’s response to its consultation, the new regulations will, among other things, describe the scheme and its goals, and seek to simplify and harmonise expectations for the manufacture, assembly and importation of EAMS medicines.

Life sciences expert Helen Cline of Pinsent Masons said: “It is proposed to allow manufacture, assembly or importation at sites with any human medicines manufacturers licence. 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.”

The new regulations are also expected to provide a framework for holders of an EAMS scientific opinion to collect real-world data from users of EAMS medicines in certain circumstances.

MHRA said these plans were “largely welcomed” by respondents to its consultation, while Cline said they reflect one of the core features for a future EAMS process that was identified in a government-sponsored independent review of the EAMS by PWC in 2016.

Cline said: “The proposals would allow companies who hold an EAMS scientific opinion to collect data in the context of EAMS regardless of whether the product is considered an investigational medicinal product or not, providing MHRA has no concerns about collection of data and patients have given the appropriate consent. 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.”

According to the MHRA, the new regulations will also allow for the withdrawal of an EAMS scientific opinion and place obligations on companies to disclose information that may change the risk profile of EAMS medicines. In addition, matters of liability and the use of EAMS medicines are to be legislated for to reflect recommendations from the General Medical Council on prescribing unlicensed or off label medicines. A number of pharmacovigilance obligations that are currently set out in guidance are also to be enshrined in law to support safe use and promote patient and healthcare practitioner confidence.