NICE Invites Pharma to Start the Conversation Early, with a View Towards Shifting Away from ‘No’ as the Starting Point
A brief history of NICE
- The ultimate goal for both parties (manufacturers and NICE) is timely market access for patients as well as a return on investment. To achieve this there will need to be closer collaboration between both parties prior to receiving a recommendation about a new technology or treatment.
The pharmaceutical bill in the National Health Service (NHS) has been rising considerably faster than the overall increase in expenditure. Since 1999 when England’s cost effectiveness watchdog, the National Institute for Health and Care Excellence (NICE) started operating, there have been several changes to the way appraisals for new technologies and treatments are consulted.
Healthcare funding is constrained, particularly in the UK, which has resulted in an overall trend focussed around managing budget pressure over the last few decades. The preliminary recommendation from NICE is often impacted by the misalignment on value, evidence, and price. As a result of this, the recommendation is often ‘no’, leading to delays in market access, and negative perceptions of both manufacturers and NICE. There have been several lengthy patient access delays which have been as a direct result of NICE’s recommendation.
NICE have expressed a desire to shift away from the current default of starting with a ‘no’, with Chief Executive, Sir Andrew Dillon, saying that there should be a shareholder return on investment (ROI) for novel medicines. It has been acknowledged that there is a requirement for alignment on value, evidence and price from all parties in order to make a well-informed decision. This would include industry, healthcare payers, and health technology assessment (HTA) bodies.
Early discussions will be required to mitigate uncertainty around a product’s evidence base and all parties will need to take a common view of the value assessment, agree on the additional therapeutic benefit the treatment offers, and be flexible and open to accommodating evidence uncertainties and share risk.
NICE: Increasing flexibility, innovation, and speed of assessment
NICE mechanisms that can be used to facilitate timely market access
The introduction of NICE’s highly specialized technology appraisal process is one way in which NICE has shown flexibility and acknowledged the unique challenges manufacturers face in bringing treatments to market for rare diseases. NICE identifies that for manufacturers their biggest challenge is making a ROI given the small target population.
The highly specialized technology (HST) appraisal process was designed to account for a greater range of criteria to better assess the value of, and account for, the challenges in developing a robust evidence package for rare disease treatments. Furthermore, NICE’s willingness to pay for treatments that target rare diseases is higher than for non-specialized treatments (GBP 300,000 vs. GBP 30,000 per QALY).
The Cancer Drugs Fund helps to give patients earlier access to promising treatments
The Cancer Drugs Fund (CDF) was initially established in 2011 as a temporary solution to enable patient access to cancer drugs not routinely available on the NHS. The major challenge associated with the initial CDF was the lack of clear criteria for how and when drugs should exit the fund, which resulted in the CDF becoming unsustainable from a financial perspective.
A reformed CDF was introduced in 2016 and represents a source of early funding for cancer drugs in England. Specifically, it provides access to promising new treatments (via managed access agreement), whilst further evidence is collected to address clinical and cost-effectiveness uncertainty. Interim funding is provided for all newly recommended cancer drugs, giving patients access to these treatments months earlier compared to the standard NICE process. Since 2016 when the reformed CDF was introduced, 29,900 patients have been registered to receive treatment with 74 drugs treating 138 different cancer indications. This showcases the breadth of patient access and impact the CDF can achieve.
NICE’s scientific advice consultancy service supports the development of robust evidence packages
NICE offers a service to developers of pharmaceuticals and biopharmaceuticals to support the development of robust evidence packages. This is to demonstrate product value, provide detailed feedback on evidence generation plans, and help integrate cost-effectiveness considerations into evidence generations plans.
It is suggested that scientific advice is to be undertaken prior to finalization of clinical trial or study designs, although economic advice can be sought after trials have started. Prices for early scientific advice projects range from GBP 30,000 – GBP 75,000, with more affordable options available for small and medium sized businesses.
NICE fast track appraisal (FTA) process
The fast track appraisal process involves a shorter process time resulting in faster patient access for the most cost-effective new treatments. Certain criteria must be met for a technology to be appraised through the FTA process including that the company base-case ICER must be < GBP 10,000 per QALY (the most likely plausible ICER is < GBP 20,000 per QALY, and it is highly unlikely that it is > GBP 30,000 per QALY).
NICE decides which products are suitable for the FTA process, and it involves a less resource-intensive appraisal process, resulting in funding within 30 days of NICE guidance publication vs the normal 3-month requirement in the standard NICE process.
The future of NICE’s recommendation
NICE’S commitment to adapting their processes and procedures have been made clear. They are continuing to accommodate unique situations while being mindful to the evolving market environment around them.
Manufacturers should revisit and challenge previous product launch perceptions based on NICE’s recent evolution, to understand how NICE mechanisms can be leveraged to achieve timely market access for novel treatments.
Furthermore, closer collaboration between NICE and manufacturers on evidence generation needs and best practices will continue to help support the goal of achieving timely market access for novel treatments, in addition to ensuring future R&D sustainability by providing a sufficient return on investment for manufacturers.