Beyond clinical trials

Main activities and considerations:

The overall aim of this phase is to make sure that the repurposed medicine reaches the new patient population for which it has been trialled. Project teams should be entering this phase with a robust package of data that shows efficacy and safety of their repurposed treatment in the new indication, whether that has been newly generated, gathered from existing data or both. At this point it should be clear that your repurposed medicine will be beneficial to the patients you are aiming to treat.

This is a very complex area with many possible routes to patient access. We have put together some general points of guidance below and signposted to other, relevant sources of information.

Possible paths to patient access

If your repurposed medicine is still on patent you may be partnered with a company (usually the patent holder) who can drive this phase. If you are repurposing a generic compound the originator company (if interested) and/or the BGMA can offer advice, and may be able to help partner you with a generic manufacturer. The Medicines Repurposing Programme is another possible source of support. Visit Resources - BGMA and Funding - Medicines Repurposing Programme for further information

If you are aiming to repurpose a patented medicine and are not supported by the patent holder it may be possible to proceed with your studies and generate proof of principle data, however you need to be aware of potential limitations to the work you are able to do while the drug is still on patent.  Any data you generated could then be used to open a conversation with the patent holder, if appropriate. Alternatively, there may be other, “off-label” routes to patient

''On-label'' or ''off-label'' prescribing

The most usual path to patient access for a newly developed therapeutic in the UK would be via MHRA granting a marketing authorisation licence, and the National Institute for Health and Care Excellence (NICE), or the Scottish Medicines Consortium (SMC) in Scotland, conducting a Health Technology Assessment (HTA) to recommend reimbursement by the NHS. This route may apply to a repurposed medicine that is under market exclusivity, but probably will not apply to repurposed generics and biosimilars.

If the HTA is successful, your medicine will be recommended for adoption by the NHS and this will result in it being available for prescription “on-label”.

While achieving marketing authorisation is the “gold standard” for a repurposed medicine, as it may provide more robust and equitable access for patients, this may not always be possible. It can be a lengthy (18 to 24 months), and costly, process if a completely new marketing authorisation is needed. Repurposing an existing generic medicine is quick, with a variation to the licence only taking about six months.

The alternative is to prescribe the repurposed medicine “off-label”, which means that it has not been granted a marketing authorisation for the new use.

For prioritised projects the Medicines Repurpurposing Programme can provide a quick and free process for applying for licence variations for new uses of generic medicines. Visit Funding - Medicines Repurposing Programme for further information

Additional information

The preferred and most robust way to ensure patient access and consistency of supply is for the medicine to be licensed for the new indication. In the UK the MHRA is responsible for granting a marketing authorisation licence.

  • The National Institute for Health and Care Excellence (NICE) undertakes a Health Technology Assessment process, to determine whether the treatment represents value for money, before recommending whether it be reimbursed by NHS England. NICE charges fees.
  • In Scotland the body responsible for undertaking a Health Technology Assessment is the Scottish Medicines Consortium (SMC).
  • Evidence from clinical trials can be submitted to the MHRA and NICE or SMC at the same time so that these processes can take place in parallel.
  • It is vital that patient groups are engaged at HTA stage (and preferably earlier). Their lived experience is crucial to the assessment process and researchers should engage with them as early as possible.
  • In some circumstances the only choice may be to have the new treatment prescribed “off-label” (i.e. without marketing authorisation), but this can lead to inequality across the country and issues with consistency of prescribing or supply.
  • More information on marketing authorisations can be found in the MHRA Marketing authorisations, variations and licensing guidance
  • MHRA's Innovative Licensing and Access Pathway (ILAP) aims to accelerate the time to market, facilitating patient access to medicines.
  • Advice can be sought from NICE Office for Market Access
  • Education of prescribers and patients is important to drive through to clinical use.
  • The document Opportunities to Repurpose Medicines in the NHS in England, contains a wealth of information on how repurposed medicines may reach patients. Pages 5 and 6 contain a useful glossary of terms.
    The excerpt below describes the difference between “on-label” and “off-label” prescribing. A major benefit of “on-label” is equality of access for all patients, wherever they live in UK.
      On-label vs Off-label:
      A medicine will usually be prescribed to an individual in line with the scope of the medicine's marketing authorisation (or product licence). This will include:
      • use of the medicine only for specified therapeutic indications in line with the evidence base submitted to the regulators at the time of licensing
      • appropriate administration within dosing recommendations (as above)
      • use of a properly labelled product
      • provision of standardised patient information
      • taking into account listed contraindications and precautions
      Off label use is where a medicine is administered outside the scope of the marketing authorisation, for example by using a different dose, administering the drug in a different way or using a medicine licenced only for adults for a child. Clinicians are asked to consider using medicines off label only where there is not a suitable available licenced product and where they are sufficiently satisfied that there is evidence of the medicine's safety. More details can be found on the website: Off-label or unlicensed use of medicines: prescribers' responsibilities.
      Currently there are no clear guidelines describing the pathways to off-label use for a medicine, though this is something that is being researched.
      “NICE should consider whether it is possible to pilot an adapted approach for off-label medicines so that high quality guidelines can be produced to better inform commissioning decisions”