The NICE Process and What Happens in the Rest of the UK
Last updated 5th December 2018
Possible topics for what is called a ‘Technology Appraisal’ are filtered through a ‘Topic Selection process’ to ensure it’s a correct topic for NICE to look at. Topics usually reach what is called the scoping stage around the time the company plan to apply for a licence for its drug in the UK or Europe.
Once NICE selects a ‘correct topic’ they then prepare a draft ‘scope’ – a document that describes how NICE would look at the new drug, and what questions it would ask and how, if the drug were to be referred to NICE for a full appraisal. (See draft scope for AveXis gene therapy treatment for infants with SMA Type 1 ‘Onasemnogene abeparvovec’ released in September 2018).
At this stage, NICE collects information from stakeholder groups about the condition and the treatment so that it can make a preliminary report to NHS England and the Department of Health about whether an appraisal should go ahead and what questions need answering if there is to be a full appraisal. This includes consideration of whether the treatment should be assessed via a Single Technology Appraisal (STA) for ‘common diseases’ or via a Highly Specialised Technology (HST) for rare conditions which also meet a number of other criteria (See NICE website and scroll down on this page.)
Following the submission of this draft scoping report to NHS England and the Department of Health, it is then up to Government Ministers to decide whether or not to make a referral to NICE to fully appraise the product for the specific indication expected in the licence. This can only happen if a treatment is licensed by the European Medicine Agency (EMA). If NICE does receive this referral from the Minister, they will write to the pharmaceutical company and all the consultees, including all the patient organisations, to advise them of the decision and, if there is to be an assessment, what appraisal route it will follow and what the timeline will be. They are asked to publish guidance within six months of the product receiving its licence from the EMA. However, as we know only too well from nusinersen, EMA published its licensing report on 1st June 2017 and there was still no NICE guidance as late as December 2018.
Full scoping / appraisal
Once NICE receives a referral for a technology appraisal, it undertakes a full and detailed assessment of the drug. This considers all aspects of the drug and condition, including how well the drug works, who it is for, any side effects, how it can be used, how the condition affects people and other areas including the cost of the treatment. Evidence is also taken from expert healthcare professionals, and patients and families on the impact of the condition.
If NICE finally recommends the drug for use through the NHS, then NHS England is normally expected to fund the treatment within 90 days.
The full NICE approval process (including possible appeals) takes an absolute minimum of 43 weeks, and this process can be much longer.
The HST route officially takes 39 weeks. However, there is often further delay around, for example, requests for further data or further consultations. If at the end of the appraisal process NICE does recommend that the drug is approved for reimbursement, then the NHS is mandated to provide funding within 90 days following the decision.
The decision to reimburse the treatment Translarna for muscular dystrophy took 2 years from the time of regulatory approval by the EMA to a final decision by NICE to have the drug funded by the NHS. As for nusinersen, we are still waiting.
What about the rest of the UK?
In Scotland, the process for assessing new medicines is usually conducted by the Scottish Medicines Consortium, following a licence by the EMA. This has happened for nusinersen, with recent changes positive to the system. Read more here: SMC and nusinersen.
Wales, Northern Ireland and the Isle of Man will often follow the guidance issued by NICE.