In a constantly changing market access landscape, industry has a greater range of options. But do these complicate the route to market further?
Centre for Health Technology Evaluation (CHTE): Develops guidance on use of new
and existing treatments within the NHS, including medicines and medical technologies.
Commercial Access Agreements (CAAs): The standard way for pharma companies to make cancer drugs cost effective for the NHS.
Managed Access Arrangements (MAAs): Consist of two components – a data collection arrangement, and a commercial access agreement.
Multiple Technology Appraisals (MTAs): Usually covers more than one technology, or
one technology for more than one indication.
Patient Access Scheme (PAS): Pricing agreements proposed by pharma companies to enable patients to gain access to high cost drugs.
Patient Access Scheme Liaison Unit (PASLU): Set up by NICE to work with companies considering a patient access scheme for their drug or treatment, to assess if it’s one that would work in the NHS.
Single Technology Appraisals (STAs): This process assesses a single drug or treatment; usually new pharmaceutical products or license extensions for existing products.
The market access landscape has changed substantially since the introduction of NICE in 1999 and the Pharmaceutical Price Regulation Scheme (PPRS, 2009, updated 2014). Increasingly formalised pricing mechanisms were introduced in the PPRS in 2009 to allow a range of options to industry, including patient access schemes.
The core focus of NICE at the start of its existence, in particular the Centre for Health Technology Evaluation (CHTE), was on Single and Multiple Technology Appraisals (STAs and MTAs). These still form the backbone of the CHTE work-streams, albeit mostly STAs. The current position for the NHS is that no Patient Access Scheme (PAS) should be required and if so, then it should be a simple discount, except in exceptional circumstances.
Historically these schemes evolved from less structured and robust offerings, to the current situation with simple discount (confidential upon request) and complex schemes being available. However, the introduction of NHS England also brought Commercial Access Agreements (CAAs) and Managed Access Arrangements (MAAs) around 2016, and the range of options in the future look to be even more innovative, albeit with simplicity at their core, relative to complex PAS.
The patient access scheme liaison unit (PASLU) was introduced in 2009, to provide the Department of Health (DH) with an independent and robust PAS review mechanism. PASLU provided the DH with advice, upon which the DH was able to recommend, or not, a PAS for ministerial approval. This arrangement changed on 1st January 2018, with NHS England becoming the new customer for PASLU, and instead of ministerial sign-off, senior NHS England staff now undertake this task.
The number of appraisals with an operational PAS has increased over time and the PAS-type moving between Simple Discount, to Complex, and back again, with the majority being implemented in the oncology disease area (see chart).
There are many reasons for this, including industry concerns of confidentiality, commercial viability of offering a simple discount, as well as the stronger stance by DH, PASLU and NHS England, that simple discounts in themselves should only be offered in exceptional circumstances.
Anecdotally, there is a greater preference for simple discount schemes to be proposed than previously. However, as long as the complex scheme option is available through the PPRS (2014), due for review this year, industry still has the option to propose complex scheme submissions when these are more appropriate to address the NICE cost-effectiveness hurdle. This is important, because complex schemes are more appropriate to address uncertainty in the clinical data when modelled to a specific time horizon, than simple discounts (cost focused).
The change of customer from DH to NHS England raises a number of questions, in particular the change in focus that may be placed upon PASLU. To clarify, NHS England take an equally firm position as the DH, however they are known to negotiate and be more directive in approach.
The complicated route to market
Up to this point, the options to improve the cost effectiveness of a product being reviewed by the NICE TA process were relatively simple; PAS or no PAS. The introduction of Commercial Access Agreements (CAAs) and Market Access Agreements (MAAs,) around 2016, provided additional options for industry to propose more innovative mechanisms by which to gain positive NICE TA Guidance. These are agreements that are submitted, reviewed and approved through NHS England, which like PASs, can be taken into consideration by the NICE Technology Appraisal committees.
When industry proposes CAAs, like simple discount PASs, it is possible for them to request that the details of the CAA remain confidential. Although details of CAAs, MAAs and all PAS-types remain confidential during the review period, this is not the case when they are included in NICE TA guidance. Only CAAs and Simple Discount scheme details can remain confidential in the NICE guidance, when it is published on the NICE website.
The introduction of the Early Access to Medicines Scheme, the Accelerated Access Review, the Budget Impact Test, Highly Specialised Technologies programme at NICE, and numerous other options, create opportunities and challenges to the uptake of new medicines. These new options to industry have assisted with speedier uptake through the NICE review, however it has provided a greater range of options to industry and complicates the route to market further; which option does industry choose?
A key driver for more innovative approaches comes from ground-breaking regenerative therapies, such as CAR T-cell therapy, when treatment is administered once. If a patient remains in remission for more than five years, they are considered cured.
The co-authored report ‘Exploring the assessment and appraisal of regenerative medicines and cell therapy products’ (University of York and NICE, 2016) explored a hypothetical scenario in which the new regenerative treatment of CAR T-cell therapy could be reviewed satisfactorily through the current NICE TA processes and potential new innovative pricing mechanisms, outside of the current options. One option that was considered to have promise was a leasing model.
While a conventional leasing model would spread the cost of obtaining a product, it encounters a range of barriers, namely the standing operating protocol and standing financial flows within the NHS. This is because the flow of money through the system has to follow that of the products (the treatment). However, with CAR T-cell treatment, a leasing arrangement would mean that there would be a one-off treatment, but future flows of money through the system against which no ‘new’ product is being purchased and allocated against that money.
While CAR T-cell therapy is likely to be provided only by a small number of specialist centres, this would not be the case for less specialised treatments, and more innovative approaches may be required for routinely commissioned treatments that are expensive upfront and approved through the NICE Technology Appraisal process. This raises the key questions about how innovative industry can be allowed to be, compared with the potential complexity of that innovation and whether or not they can be implemented by the wider NHS.
In addition, the current ability for the NHS to collect and maintain a data recording system for such an approach would place substantial additional and disproportionate administrative burden on the NHS treatment centres.