On 17 July 2012, the UK Medicines and Healthcare products Regulatory Agency (“MHRA”) published proposals on developing a UK scheme to provide access to certain new medicines before they are formally licensed. The proposals outline the scheme in general, detail the types of medicines to which it will apply and at what stage companies will be able to apply to have their medicines made available under it. The consultation process will be open until 5 October 2012 after which MHRA will finalise the details of the scheme.
Unlicensed medicines are currently available in the UK through clinical trials or on a named patient basis. This system has led to media and public anger at perceived unfairness when one health authority makes a promising unlicensed medicine available to the public, while another does not. Campaigns by patients affected by rare diseases aim to speed up access to promising medicines to alleviate patient suffering.
A stated aim of the UK government’s Strategy for Life Sciences is the creation of a more progressive regulatory environment that supports and promotes innovation with a view to providing faster access to new medicines for patients. In this context, MHRA has been tasked with introducing a national scheme to regulate early access to unlicensed medicines in the UK.
Key features of the proposed early access scheme
MHRA has said that the scheme will operate within current regulatory structures and will be voluntary and non-statutory. Although currently lacking in detail, the key features of MHRA’s proposals can be broken down as follows:
- Promising medicines: The scheme will cover particularly promising medicines which have a positive benefit to risk balance. These will typically be medicines that are between the end of Phase III trials and being granted a marketing authorisation.
- Manufacturer request: The company developing the new medicine can decide whether to request an opinion from MHRA as to the medicine’s suitability to be made available under the scheme.
- MHRA review: MHRA will review the information submitted to it by the medicine’s manufacturer and will produce a scientific opinion describing the benefits and risks of the medicine’s early release. This will be available on MHRA’s website to assist clinicians and patients in making treatment decisions.
- Local funder decision: No additional budget will be made available to fund the purchase of the unlicensed medicines. So local NHS funding bodies will decide whether to fund unlicensed medicines from existing budgets.
Price: NHS funding bodies have said that their willingness to fund unlicensed medicines depends on the purchase price asked by manufacturers. MHRA proposes that costs of unlicensed medicines be controlled as follows:
- Providing the medicine for free. This is unlikely to be popular with industry, except in limited circumstances.
- Carry out a NICE (National Institute for Health and Clinical Excellence) cost effectiveness review. This will take a long time.
- Introduce a price cap. This will depend on the quality of the data submitted to MHRA.
- Product information: Information supplied to clinicians and patients must include not only what is known about the medicine, but also highlight what is not yet known.
- MHRA fees: The initial fee for MHRA’s review will be £29,068 per annum.
MHRA’s proposals are fairly general at this stage and further work will be required before the scheme will be up and running. It is important that feedback is given by industry.
MHRA is very clear that the scheme will only apply to particularly promising medicines being developed. It does not anticipate that more than one or two medicines will be made available each year under the scheme. In addition, the manufacturer must still complete the marketing authorisation process. In an era of growing budget constraints, we will have to wait and see if NHS funding bodies will divert cash to fund these new products.