Repurposing is a strategy for identifying new uses for approved or investigational drugs that are outside the scope of the original medical indication. It typically involves taking an existing medicine that has a marketing authorisation or licence for human use in one condition and using this medicine to treat another condition. Drugs may be prescribed off-label. Off-label use may occur where they are administered outside the scope of their marketing authorisation, for example by using a different dose, where they are administered in a different way, or where a medicine licensed only for adults is used for a child.
A 2017 report found that although it was likely that there were high levels of off-label prescribing already happening within the NHS, more consideration was required to address the potential to repurpose medicines more systematically. At the moment, off-label prescribing is part of normal clinical practice. When prescribing, clinicians follow the 'prescribing hierarchy': the first choice should be a licensed medicine used within its licensed indication; if that is not clinically suitable for the patient, clinicians can use a licenced medicine outside its licenced indication (off-label prescribing); and then finally if that is not clinically suitable for their patient then they can use an unlicensed medicine. Off label prescribing is therefore at the discretion and professional liability of an individual clinician, leading to significant variation in practices. Concerns can include lack of an agreed protocol, fear of litigation and not having resource to review research findings or undertake the additional administration and monitoring required when prescribing outside of a licence.
However, off-label prescribing can be beneficial to patients and the NHS. Many new uses of medicines have been discovered through off-label prescribing and are now commonplace. A good example is aspirin, which is used primarily for cardiac problems rather than as pain relief.
Enabling access to unlicensed or off-label drugs could also provide faster access to an effective treatment either ahead of planned licensing or where the cost of developing and licensing a new medicine may not be financially viable such as with rare diseases.
Sometimes, a pharma company may actually have sufficient evidence that a drug is clinically effective in a new indication or population, but there is no commercial incentive to license it if the drug is low cost or generic. On other occasions, it may decide not to go through collecting evidence to license the drug where the patient population for the new indication is very small (for example, for gene therapies) or is challenging for ethical or regulatory reasons (for example, for children).
Licensing an off-label medicine can have other benefits - by reducing the individual burden on individual clinicians and reassuring them and their patients that the use of a medicine in a given indication is evidence-based, and providing assurance that the drug is subject to ongoing pharmacovigilance.
The Repurposing Medicines Programme has been established to identify and pursue opportunities to strengthen the evidence base, licensing, supply, cost-effectiveness and equitable adoption of currently unlicensed or off-label medicines, where there is benefit to the NHS and patients. The latest published report identifies opportunities and possible ways of working to achieve it. That includes a reference to working with the Office for Life Sciences on a 'catalyst' scheme to financially incentivise generics companies to support the licensing process for repurposed medicines in prioritised areas where current market and or regulatory requirements are acting as a bar to applications.
Although medicines repurposing is not unique to the UK, there is a unique opportunity for the NHS to take the lead, as was shown in the pandemic response where there were trials of effective drugs, repurposed to treat coronavirus (COVID-19), such as dexamethasone and tocilizumab. The report states that it is in a unique position to build on opportunities to work with key partners in the statutory, third sector and pharma industry to identify potential candidates for future licensing.
The repurposing programme will therefore have future implications for how the NHS and partners can improve clinical outcomes. In addition, the NHS England and NHS Improvement report provides further evidence of their commitment to maximising opportunities for working with the UK's pharma and life sciences sector.
The report can be accessed here.
In the COVID-19 pandemic, the UK's NHS has had great success in using repurposed drugs, and there are real opportunities to continue to take a lead in this area, for the good of the NHS, patients and the pharma and life sciences industry. As has happened in the COVID-19 response, this can show global leadership. It's great to see the UK wanting to build on these successes over the last year and achieve much more going forward.