Pilar Pinilla-Dominguez is Senior Scientific Adviser at the National Insititute for Clinical Excellence (NICE) and will be speaking at the next Advanced Therapies Network (ATN) event on 17 September on the topic of reimbursement models. Here she gives an insight into NICE’s take on this complex subject.
The next Advanced Therapies Network event on reimbursement will be in partnership with Phacilitate Leaders Europe conference at the Royal Lancaster , London. Pilar will join speakers from NHS England, the Cell & Gene Therapy Catapult and Orchard Therapeutics.
Q. How is the UK approaching the reimbursement of advanced therapies? Is any particular reimbursement model preferred and why? What are the main considerations when deciding on how to approach this area?
A. NICE is not new to the evaluation of advanced therapies. It published its first recommendation on an advanced therapy on autologous cartilage transplantation for full thickness cartilage defects in knee joints in December 2000. However, it is acknowledged that the nature of advanced therapies has evolved since then and NICE is prepared for new advances associated with these technologies. We know this because we carried out an independent test in 2016, prompted by a recommendation from the Department of Health Regenerative Medicine Expert Group. The conclusions confirmed that NICE methods were applicable and highlighted that in situations where there is great uncertainty and potentially very substantial patient benefits, innovative payment models may be particularly suitable.
When more complex schemes are required, NICE encourages companies to engage early with both NICE and NHS England while recognising the need for these models to be feasible in the NHS without adding additional burden.
NICE’s remit is to evaluate the clinical and cost effectiveness of all new treatments and therefore it does not have a preference for a particular type of reimbursement model. However, the Commercial Liaison Team at NICE (which incorporates the Patient Access Scheme Liaison Unit (PASLU)) assesses schemes to ensure that these are feasible to be implemented and won’t add a substantial burden to the NHS. When more complex schemes are required, NICE encourages companies to engage early with both NICE and NHS England while recognising the need for these models to be feasible in the NHS without adding additional burden.
Q. The UK was the first major European country to agree market access deals with both Gilead and Novartis recently. You could say the NHS has previously been a slow adopter of novel therapies, what has changed?
A. The NHS in England has always been open to companies with promising new treatments that are willing to price these treatments responsibly.
The Cancer Drugs Fund has provided a tried and tested framework for all parties to understand how the significant uncertainties with these novel cancer treatments could be managed.
With the CAR-T therapies, both Novartis and Gilead were willing to engage early and openly with NHS England, NICE and other key stakeholders. The challenges associated with the introduction of these exciting new therapies were therefore understood early and solutions could be developed and implemented in a timely way. This includes the challenges of both the practical implementation and of the funding of these new technologies. The openness of the engagement was key to enabling discussions with all relevant parties present to identify and address these known challenges.
These technologies came with a high degree of uncertainty about their long-term efficacy and side effects which resulted in them being recommended within the Cancer Drugs Fund. This has provided a tried and tested framework for all parties to understand how the significant uncertainties with these novel cancer treatments could be managed.
Q. How is the application of ongoing evidence being used by NICE to inform reimbursement models?
A. Managed Access (e.g. via the Cancer Drugs Fund, or for Highly Specialised Technologies) provides NICE Technology Appraisal and Highly Specialised Technologies committees the option to make conditional recommendations for promising new technologies that are plausibly cost effective but for which the significant clinical uncertainty (e.g. immature data), would in other circumstances prevent a recommendation for routine commissioning.
A Managed Access Agreement includes a requirement for further data to be collected over a specified period of time from NHS patients (often alongside ongoing clinical trials) in order to address the clinical uncertainties identified by NICE’s committees. At the end of that period the additional data are then used in a re-appraisal of the technology resulting in either a positive recommendation for routine use in the NHS or not. This may require a new commercial arrangement to be put in place, which reflects the newly available evidence.
NICE committees have recommended 33 topics for use in the Cancer Drugs Fund and 5 non-cancer topics for managed access. To date, two topics have been re-appraised and exited the Cancer Drug Fund being recommended for routine commissioning in the NHS, and one topic was removed upon the withdrawal of the product’s marketing authorisation.
The 2019 voluntary scheme for branded medicines pricing and access contemplates the use of managed access agreements for other conditions in specific circumstances.
Q. Advanced therapies have struggled to prove themselves as commercially sustainable products and a big part of this are the high costs. In order to ultimately benefit patients, how do NICE and NHS work collectively with drug developers and other healthcare providers to tackle this challenge?
A. NICE encourages and promotes early and continuous engagement between all stakeholders. Technology developers can seek advice in their development plans and data collection through the different services that NICE Scientific Advice provides, including national advice from NICE or in parallel with other regulatory and health technology assessment bodies.
The NICE Office for Market Access also allows technology developers to engage with the system regarding how to navigate the NHS and the different routes available for market access. The Office for Market Access offers safe harbour meetings where technology developers can openly explore different issues with a range of system partners including NICE, the NHS, regulators, patient organisations or clinical experts, among others.
The newly formed NICE Commercial and Managed Access programme provides a vital liaison function between NICE and NHS England for all new commercial agreements and evidence generation activities.
Undoubtedly, efforts have been taken to facilitate discussions between different stakeholders at different levels in order to ensure accessibility to promising new technologies in a timely manner but ultimately it is the companies’ responsibility to price their products responsibly and work with NICE and NHS England to ensure timely access for patients to promising technologies associated with significant benefits.
Q. How are healthcare providers and payers across Europe taking a co-ordinated approach to pricing and reimbursement? Do you think that approaches will vary considerably across different countries?
A. European HTA bodies are already participating in the development of joint assessment of technologies coordinated through EUnetHTA. This joint assessment is based on the EUnetHTA HTA Core Model, which consists of the following nine domains: current use, technical, safety, clinical effectiveness, cost and economic, ethical analysis, organisational, patient and social, and legal. While some of these domains can be considered in a joint manner by member states, other domains such as the cost and economic effectiveness will need to be considered by individual member states through national appraisal.
While parallel early dialogues and joint assessments are accessible via EUnetHTA, pricing and reimbursement remains a national, regional or local responsibility.
Furthermore, European HTA bodies also participate in early dialogues with technology developers offering parallel scientific advice alongside regulators or multi-HTA advice. Through these early dialogues, technology developers have the opportunity to explore with HTA bodies specific issues regarding their development plans, and the HTA bodies will offer a consolidated position on each of the questions. Specific additional national advice could also be added to the joint consolidated positions to take into consideration any individual national position.
While parallel early dialogues and joint assessments are accessible via EUnetHTA, pricing and reimbursement remains a national, regional or local responsibility. There are some initiatives in Europe among groups of countries with similar healthcare systems to work together which may include elements of shared negotiation and decision-making for pricing and reimbursement.
Pilar Pinilla-Dominguez will be speaking at the next Advanced Therapies Network event on reimbursement on 17 September 2019. The event will be in partnership with Phacilitate Leaders Europe conference at the Royal Lancaster, Bayswater, London. Pilar will join speakers from NHS England, the Cell & Gene Therapy Catapult and Orchard Therapeutics. The event is free to ATN members and you can become a member of the network here.