The National Institute for Health and Care Excellence (NICE) has published a new Manual, which sets out how the latest medicines and health technologies will be evaluated.
The ABPI’s Director of Value and Access Policy, Paul Catchpole, provides further commentary on the publication.
So, what does this mean for NHS patients and for pharmaceutical companies operating in the UK? And what do we think needs to happen next to ensure the UK is a go to place to research, develop and use new medicines? Getting this right is fundamental to delivering the Government’s Life Science Vision.
With the publication of the new NICE Manual for health technology evaluation today, ABPI is examining the detail of the final changes made - as many will be doing. Today represents a significant milestone on the journey to improving patient access to new medicines but the ongoing work on methods and processes must continue. Further action is also needed to consider what NICE should prioritise in the short term, including ensuring the changes make a tangible difference to patients’ lives through faster and broader access to treatments.
There are a number of areas where the new Manual should, if implemented by NICE and its Appraisal Committees with the right intent, provide much needed flexibility and pragmatism.
However, there are significant missed opportunities and changes that have been made which do not reflect the feedback from industry and other stakeholders made to the consultation.
The new modular update approach proposed by NICE can help. It needs to be implemented quickly - with input from stakeholders - and a sharp focus and ambition to make progress on access to medicines for all NHS patients.
How has NICE improved the system?
- Appraisal Committees accepting a higher degree of uncertainty when evidence generation is particularly difficult, for example in rare diseases and for innovative and complex medicines.
- Broadening NICE’s definition of ‘severity’ beyond just conditions which are imminently life threatening. Medicines treating patients living with debilitating conditions should now be able to benefit from the new severity modifier.
- Supporting the use of more comprehensive evidence base for decision making, including real-world evidence sources and the lived experiences of patients.
- Clarifying that NICE accepts health effects of carers as well as patients in the evaluation of a medicine is a welcome step forward.
- Better flexibilities for challenging or unusual scenarios. For example, NICE being unable to recommend a medicine even if it were priced at zero and when there are costs to the healthcare system that should not be fully borne by one individual medicine alone.
Areas of concern which require ongoing focus as a priority:
- NICE’s decision to not change the discount rate despite concluding there is an evidence-based case for change – with strong support from many stakeholders – is hugely disappointing. This is a significant missed opportunity to properly value the longer-term benefits medicines provide. This decision restricts the level of ambition the review has delivered and risks the UK not being seen as a priority market to launch new medicines.
- The implementation of the severity modifier in an “opportunity cost neutral” way means some end-stage cancer patients may miss out on last resort treatments, due to the retirement of the end-of-life modifier. Monitoring the application of the modifier in practice and progressing the additional work NICE has identified as necessary to inform further broader evolution of the modifier needs to be a high priority.
- Further work and research on a health inequalities modifier is needed, including considering how it can support rare disease medicines in relation to the inequity in access to treatments these patients continue to face.
- The revision of the Highly Specialised Technologies (HST) selection criteria risks preventing some medicines treating very rare diseases from being able to enter this evaluation programme. The impact of this change needs to be closely monitored to consider whether further adjustments are needed.
- There remains a particular challenge for rare disease medicines which will not meet the HST entry criteria or benefit from the new severity modifier.
Swift implementation of the Manual is now needed, with a fair approach taken to managing the transition from previous to new methods, for example so those patients currently benefiting from end-of life cancer medicines in the Cancer Drugs Fund do not lose out.
ABPI welcomes NICE’s commitment to closely monitor and review the impact of the changes to ensure they are working in practice as intended.
Finally, the Life Science Vision set out to make the UK a leading global hub for life sciences. The role of NICE and the methods it uses to evaluate new medicines are observed internationally, signalling to global investors the extent to which the UK values innovation. Currently the UK has one of the lowest levels of investment in medicines. This funding gap versus comparable countries needs to be addressed by government for NICE and the NHS to be seen as world leading.
We look forward to working together to make this happen so that patients in the UK can benefit from early and sustainable access to new medicines.
 IQVIA - Drug Expenditure Dynamics 1995–2020: Understanding medicine spending in context. Real net drug percentage of healthcare, 2018
- Health and Access to Medicines
- NICE Methods Review