In one of our previous blogs we discussed the US Food and Drug Administration’s (FDA’s) introduction of the ‘breakthrough’ designation concept as a way of improving earlier access to medicines for patients with high unmet medical need. In this blog we’re focussing on the Early Access to Medicines Scheme (EAMS), a similar scheme launched by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA) in April 2014. EAMS is a voluntary scheme that provides an accelerated route to market for drugs in the UK and aims to “give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have marketing authorisation”.[i]
Drugs under EAMS will be in phase III or, in exceptional circumstances (if data is compelling and there is a significant unmet need), late stage phase II trials. To gain marketing authorisation (MA) they will still need to go through the full clinical trials and drug licencing process, the MHRA highlight EAMS is not a replacement to the MA process and will only impact 5-10 drugs a year.
Three conditions must be met in sequential order for a drug to be accepted onto EAMS:
- a drug must first be awarded the status of ‘promising innovative medicine’ (PIM) by the MHRA, a step that mirrors the FDA’s ‘breakthrough’ status. PIM is reserved for drugs that are “likely to demonstrate significant benefit for patients in life-threatening or seriously debilitating conditions.”[ii]
- the drug manufacturer must then apply for ‘EAMS scientific opinion’ – the final MHRA evaluation process. The primary aim is to ensure an acceptable risk/ reward trade-off using available clinical trial data. The secondary aim is to develop guidelines and information to allow prescribers and patients to make informed decisions on drug use
- if accepted the manufacturer must make the drug available to the NHS free of charge for as long as it’s included in EAMS. Inclusion is guaranteed for a maximum of a year on the condition that subsequent clinical trial data is shared periodically and does not compromise the risk/ reward trade-off. Once an EAMS drug receives approval through traditional approval channels it is automatically removed from EAMS
There are a number of potential benefits of this scheme which impact a wide range of stakeholders:
- patients benefit from earlier access to treatments that could significantly improve survival and quality of life. It’s thought the scheme will allow patients and doctors access to new medicines two years earlier than is currently the case
- drug companies benefit from earlier regulatory guidance and collection of real world evidence of impact of the drug (evidence derived from patients outside of the tightly controlled and monitored clinical trial setting) to support licencing and health technology assessment (HTA) opinions. Although the granting of a PIM designation will not be made publically available, the UK Government believes it will be in a company’s interest to make this information available because it will signal to investors that a therapy is on the ‘right track’
- the UK economy should benefit as life science companies increase their investment in the UK as it offers the opportunity to work with doctors and patients earlier in the drug’s lifecycle, and allows them faster access to patients who will benefit from their new medicine.
For EAMS to realise its potential, the engagement of drug companies is essential. Before deciding to engage, drug companies will need to consider a number of potential risks of EAMS inclusion, such as:
- Reputational risk -The MHRA has provided reassurance that negative EAMS opinions, which might cast doubt on a drug’s effectiveness or safety profile, will not be made public. However, there needs to be more clarity of how the MHRA, as a public body, will respond to freedom of information requests. The impact of a withdrawn EAMS opinion also needs to be considered as do risks associated with releasing early clinical data as part of the approval process.
- Financial and other risk -The total direct cost of applying for EAMS approval, excluding the cost of preparing the submission, is £33,027 (£4,027 for a PIM application and £29,000 for a ‘scientific opinion’ of a new molecular entity). A positive EAMS opinion will incur additional costs in relation to providing the drug and any necessary non-standard diagnostic technology, given these are to be provided free of charge to the treatable patient population. There are also costs associated with keeping an EAMS opinion up to date. Financial risks are only likely to deter smaller biotech companies but, as the source of much of today’s innovation, these represent an important stakeholder group.
EAMS has so far been received positively by industry and patient groups but with caveats around funding, data clarity and how commissioning of the therapy by the NHS will work. A planned review of EAMS one or two years down the line may help to ensure these and any other concerns are addressed. Until then helping drug companies to better understand EAMS benefit on licencing and HTA opinions could increase interest in the scheme; engaging the National Institute for Health and Care Excellence (NICE) in the EAMS process could be one solution to provide reassurance of favourable HTA opinions. However, companies not considering the whole EAMS process may still apply for PIM status, costing just £4,027, a relatively small price to pay if it attracts potential acquirers or strengthens negotiating power when entering into in-licensing/ co-development agreements.
It is still early days for EAMS and as yet no formal applications have been made. Success will likely depend on the experience of the first few pioneering companies that ‘feel’ their way through the system and refine its evolution into something that can deliver on the goal of helping patients most in need.