Clinigen Group plc – Written evidence (LSI0013)

NHS proprocurement and collaboration

11. How can the recommendations of the Accelerated Access Review be taken forward alongside the strategy? Will the recent changes to the NHS England approval process for drugs have a positive or negative effect on the availability of new and innovative treatments in the NHS? How can quick access to new treatments and the need to provide value for money be reconciled?

The objective of the Accelerated Access Review (AAR) as described by Sir Hugh Taylor in the AAR report introductory letter[1] is to “speed up access to innovative drugs, devices, diagnostics and digital products to NHS patients”.

At Clinigen, our mission is to deliver the right medicine to the right patient at the right time. We are specialists in supporting healthcare professionals and pharmaceutical companies gain access to medicines at any stage of a product’s lifecycle: in clinical trials; as unlicensed or commercial medicines. This provides us with a unique perspective and we believe that many patients could benefit even earlier from innovative products if a number of changes are implemented to the AAR process.

In the UK, we benefit from the Early Access to Medicines Scheme (EAMS), which aims to give patients with life threatening or seriously debilitating conditions access to medicines that do not yet have a marketing authorisation when there is a clear unmet medical need. The initial step in EAMS is the Promising Innovative Medicine (PIM) designation, which aims to highlight and increase patient access to such medicines.

Access to unlicensed medicines has proven to be successful through the PIM/EAMS route, and resulted in a number of products becoming available on the NHS earlier. For example, providing access to pembroluzimab, an innovative immuno-oncology medicine in late-stage clinical trials, to approximately 500 patients with advanced melanoma in the UK.

Despite previous successes of PIM/EAMS, we see from the MHRA’s website[2] statistics from April 2014 up to July 2017 that only about 50% of products that received PIM designation went on to an EAMS. During this period, 37 PIM designations were granted, which is approximately one per month or 12 per year.

As the PIM/EAMS process takes place directly between the manufacturer and the MHRA, we do not have specific data on all of the submissions. However, through our experience working with the vast majority of pharmaceutical companies, we are aware that many companies have not pursued PIM/EAMS for a variety of different reasons. These include: the lengthy two-step process (at least 5 months to be completed); the ongoing responsibilities for the company (such as periodic updates and renewals); and the inability to charge for the product via EAMS at any stage of development process.

Many pharmaceutical companies, therefore, prefer to supply unlicensed products following the Named Patient route, which can be implemented very quickly, is simple to manage and chargeable. These companies want to make the product available to patients as soon as possible, but the EAMS process does not help them to achieve this (it is often not deemed timely enough).

In addition, the process fees and the inability to charge for the product can be a limiting factor for small biotech companies, but may not be an issue for big pharma companies. Since biotech companies often have the most innovative products in their pipeline,[3] we recommend a reduction in fees using a suitable measure of affordability.

From an operational perspective, there are no significant benefits to patients needing immediate supply under EAMS versus using the Named Patient approach. On the contrary, as EAMS is valid for one year or until EMA approval and there could be a time gap until reimbursement and availability on the NHS, patients who receive treatment under this scheme may see their supply being interrupted. This means that when an EAMS ends due to EMA approval, Named Patient supply has to be implemented to ensure no treatment interruption. We recommend amending this for EAMS to ensure it only stops when it is available on the NHS and reimbursement is in place. However, as the supply cannot be charged for, there needs to be an approved timetable for reimbursement, otherwise pharmaceutical companies will not have an incentive to apply for the programme.

Whilst we feel that the PIM/EAMS process should be taken forward alongside the strategy, unless the recommendations above are implemented, we believe that the full benefit of the PIM/EAMS approach will not be realised.

We have one additional point to make. Providing quick access to innovative treatments whilst balancing the need to have value for money is not easy. Being able to collect and use real world data (RWD) from medicines access through EAMS could provide valuable information to help define the correct pricing for a product. This could include not only safety and clinical/medical data, but also healthcare resource utilisation such as hospitalisation days, visits to A&E services, use of additional medication, etc. Patients who access a product via a Named Patient programme or EAMS are different from the clinical trial patient cohort, and, as long as the protocol is not too prescriptive, their treatment will resemble more that of patients in the post-approval setting rather than in a controlled clinical trial. These data would therefore provide evidence more akin to what would be collected once the product is in use in the clinical setting but it would be available upstream in the development process allowing earlier assessment of its value for money.

In summary, the PIM/EAMS route to access unlicensed medicines has proven to be successful with several innovative medicines being made available to patients with life threatening or seriously debilitating conditions. However, there are still significant challenges including; the lengthy two-step process, the ongoing responsibilities for the company, and the inability to charge for the product via EAMS at any stage of development process. Process fees and the inability to charge for the product can be a limiting factor for small biotech companies so introducing a suitable measure of affordability should be considered. We recommend amending the EAMS process to ensure it only stops when the medicine is available on the NHS and reimbursement is in place. Finally, we would like to see less prescriptive protocols with regards to the treatment schedule and to collect additional RWD that will inform the true value of the product. This will enable a meaningful negotiation of pricing and reimbursement that will allow the NHS to demonstrate value for money and benefit patients.

13 September 2017