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Stephanie Sutton was an assistant editor at Pharmaceutical Technology Europe.
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) recently launched a 12-week public consultation for a national scheme that will look at the possibility of providing seriously ill patients with access to unlicensed medicines still in Phase III, or possibly even Phase II, clinical trials
The UK’s Medicines and Healthcare products Regulatory Agency (MHRA) recently launched a 12-week public consultation for a national scheme that will look at the possibility of providing seriously ill patients with access to unlicensed medicines still in Phase III, or possibly even Phase II, clinical trials. Similar initiatives, mainly through extensions of clinical trials or managed-access programmes, are already in place in certain parts of the UK, as well as in the US and other countries, but these are often ad-hoc in nature and locally based. The proposed scheme would provide a national approach to providing early access to certain medicines in the UK.
The need for new medicines to treat life-threatening illnesses, such as cancer, Alzheimer’s and heart failure, is well understood and both FDA and EMA offer fast-track approval processes for medicines that treat serious diseases and fill an unmet medical need. In the US, the fast-track process provides, among other things, more frequent FDA meetings concerning the drug’s development plan and eligibility for Accelerated Approval, which provides approval based on the effect on a surrogate or substitute endpoint that is likely to predict clinical benefit. These processes significantly shorten the time it takes for a medicine to be approved for the marketplace, although further tests are still required.
However, the scheme the MHRA is proposing would not provide accelerated approval, but would instead give clinicians the option to offer patients treatments that are not yet approved and which are still progressing through clinical trials. Patients, however, would have to accept a certain amount of risks in taking such unlicensed medicines. According to the MHRA, there is broad-based support for such a scheme, particularly for patients with life-threatening illnesses.
The Early Access to Medicines Scheme would only be open to patients with life-threatening, chronic or debilitating conditions, such as late-stage cancer. Under the scheme, the MHRA would give a scientific opinion on the benefits and risks of certain medicines before they are licensed. The opinion would then be available on the MHRA’s website to help clinicians and patients in making decisions about their treatments. The opinion would be based on scientific data submitted by the pharmaceutical company.
Unlike medicines using accelerated approval processes, pharmaceuticals using the MHRA’s scheme would remain unlicensed until fully assessed and authorised. In addition, it would be up to the UK’s National Health Service (NHS) as to whether the treatment should be funded.
The MHRA has conducted a lot of research on the subject of early access to medicines. I’ll look at some of their findings in my blog next week.