Commercial arrangements in access to medicine
The introduction of new medicines in England requires the cooperation of industry and the NHS. Industry has long been familiar with the need to prove that new treatments are both clinically-effective and cost-effective; and for the majority of new treatments, the standard NICE procedure is viewed as a fair procedure for assessing their value.
However, fairly assessing the value of a new treatment becomes more difficult where there are gaps in the data or where there are questions over how well the clinical trial data will translate to real world practice. In such instances, a more complex commercial arrangement may be required.
There are currently three main options open to manufacturers: Complex Patient Access Schemes (PAS), Commercial Access Agreements (CAAs) and Managed Access Arrangements (MAAs)
Patient Access Schemes (PAS)
When companies wish to improve the cost-effectiveness of a product under appraisal by NICE without lowering the list price, the default option is to offer a simple Patient Access Scheme (PAS), which consists of a fixed price or a percentage discount on the NHS list price of the product.
These schemes have a relatively low administrative burden and consequently are widely used and accepted by the NHS. Whilst these schemes fill a commercial need to maintain a higher list price whilst still offering the NHS value for money, they do not answer more complicated questions such as when it is unclear what proportion of patients will respond to a therapy in practice, or uncertainties over required treatment duration.
In these situations, a complex PAS might be more relevant and involve for example, the manufacturer committing to paying for the treatment of non-responders or providing treatment a set price, regardless of number of cycles required. These schemes limit the risk to the NHS whilst at the same time being able to offer the manufacturer a price for their therapy that reflects its benefits. By their nature, complex PAS schemes involve a complex proposal and the administrative burden on the NHS is high.
Companies therefore require a strong justification for their use and they will only be considered if a simple discount has been demonstrated to be unsuitable. To date, just 21 complex PAS schemes have been accepted in contrast to approaching 200 simple PAS schemes1.
Commercial Access Agreements (CAAs)
More recently, NHS England has outlined plans for CAAs2, which recognise that there are circumstances where companies may face significant commercial challenges in launching clinically effective products that could benefit patients and the NHS. Unlike complex PASs, CAAs are at the discretion of NHS England and are currently only considered where the therapy has a value proposition at or below the lower end of the standard NICE cost-effectiveness threshold and the company wants to propose an enhanced value offer, or where there are unusual or unique circumstances that mean launching a product is considered particularly challenging or commercially unviable.
Examples of potential CAAs are provided in the table below:
|Budget cap||Maximum budget impact for a product beyond which a central rebate is payable|
|Price/volume agreement||Price agreed for set volume of patients and then reductions staged based on additional patient numbers, or company pays back the full amount (similar to budget cap).|
|Cost sharing||The company funds initial cost of therapies such as offering the first month free.|
|Stop/start criteria||Rules on eligibility criteria for when patients should start/stop therapy.|
|Outcomes-base agreement/payment by results||Discount or rebate applied if a product does not perform as expected or for non-responders|
*Table taken from NHS commercial framework for new medicines*
The exact terms under which a CAA will be entered into are not clearly defined and as such the process remains at the discretion of NHS England, who will consider all applications on a case-by-case basis.
Managed Access Agreements (MAA)
MAAs are another method through which to address uncertainties in the data at the time of NICE review. MAAs can be suggested either by the manufacturer at the point of submission or NICE may wish to recommend the technology in the context of a MAA; that is, that the technology is recommended as an option, but that formal data collection is conducted alongside routine use.
The purpose of such data collection being that it addresses the uncertainties, thereby enabling NICE to issue a revised recommendation in light of the new evidence. To date, MAAs have mostly been used to support Highly Specialised Technology (HST) submissions, where data availability at launch is often limited by the very rare nature of the conditions in question.
Launching a challenging product
Until recently, complex PASs were the only option available to companies wishing to offer the NHS a discount other than a simple percentage reduction on the list price. However, the fact that NHS England have included CAAs and MAAs in their Commercial Framework suggests that their use may become more frequent in future and companies presented with a challenging commercial situation should consider all of the options available to them, including PASs, CAAs and MAAs to ensure patients have access to new effective medicines at a price that is sustainable for both the NHS and industry.
- Technologies with approved patient access schemes and other commercial arrangements (Excel). Accessed at https://www.nice.org.uk/about/what-we-do/patient-access-schemes-liaison-unit August 2021
- NHS commercial framework for new medicines published 23 February 2021