By Danielle Whitney & Kate Varnum
As ever, healthcare in the UK is a fiercely debated topic. Opinions have flared again on the ‘hot topic’ of access to new medicines with the unveiling today of the Early Access to Medicines Scheme (EAMS) by the UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) as a part of the government’s overall Strategy for UK Life Sciences.
Here are the key components of the scheme, which is said to be designed to work alongside and not replace the current system of clinical trials:
- When the MHRA has enough data to reach an opinion that a new drug works and is sufficiently safe, it can issue a promising innovative medicines (PIM) designation; the designation will provide an early indication that a product may be a possible candidate for the EAMS. This could occur years before a medicine gets licensed while large scale Phase III clinical trials are still continuing or while drugs are waiting for authorisation.
- Before it could be prescribed there would need to be a scientific opinion from the MHRA describing the risks and benefits of the medicine, which would enable doctors and patients to make an informed decision about whether to use the drug.
- The programme will be entirely funded by pharmaceutical companies; no support from the National Healthcare System (NHS)
- The new scheme will be launched in April
The scheme is being compared by UK media to the FDA’s Break Through Therapy designation. The aim of both programmes is to expedite access to new treatments. However, the FDA scheme focuses on procedural improvements that will allow for more rapid approval within the existing review system. The UK scheme will not expedite approval of treatments through the centralised European Medical Agency review system, but will potentially give patients access in advance of this approval on a physician supervised, individual patient basis.
If this sounds similar to the Named Patient Programmes (NPP) that are run outside the US, and Expanded Access Program (EAP) run in the US by many pharmaceutical companies in advance of approval, that’s because they are in essence the same, at least based on the information currently available. See the below diagram outlining where the scheme would fit in relation to the current drug development pathway (parallel to the NPP and EAP). According to the MHRA, “…NPPs and EAPs are usually locally based and ad-hoc in nature. One potential benefit from the Early Access Scheme is that the provision of an opinion from the regulator and a nation-wide approach from the company to funding and exit strategies may reduce these local variations in uptake, although given NHS funding mechanisms this cannot be fully managed away.”
This is one of the reasons the scheme is controversial for stakeholders, including the UK Faculty of Public Health Response (FPH), who have questions about the value such a scheme will provide to patients given its current scope. The key criticisms that have been shared by stakeholders include:
- The program undermines the current centralised (EMA) system to review clinical trial data to ensure they meet the standard for efficacy and safety prior to approving drugs for public use.
- New medicines can have serious side effects that could have an adverse effect on a patient’s quality of life and could shorten rather than prolong the patient’s life.
- The program will not improve public health given its small scale (potentially 2 drugs a year are estimated to be eligible for the scheme by the MHRA) and the fact that it provides access to individual patients while the majority will have to await the usual EMA timelines for approval.
The Association of the British Pharmaceutical Industry welcomed the initiative. However, they are concerned by the lack of central funding and requested the scheme be reviewed after the first year.
The story has already generated significant media buzz. Impacting multiple significant players in the healthcare arena — including access, patient and industry communities — this announcement is likely to generate continued interest over coming weeks and months. We will also monitor for additional information on the Government’s Strategy for UK Life Sciences.
Government leaders have confirmed the Strategy will also include:
- A £180 million catalyst fund to help speed new medical treatments through the so-called “valley of death” between development in the laboratory and use on patients.
- A consultation on changes to the use of NHS patient data, which could see more information shared with private healthcare companies and data automatically included in clinical research unless individuals opt out.
- A £50 million new cell therapy technology and innovation centre in London to help turn scientific discoveries into commercial products to help patients with illnesses like Parkinson’s.
- A new app and web portal to help members of the public participate in clinical trials.
- Investment of £10 million in collaboration between the Medical Research Council and AstraZeneca to fund academic research into a broad range of diseases.
If you have additional questions about the EAMS, please reach out to one of your London colleagues, or click here for more information.