Anyone who has followed the recent debate about access to medicines for Cystic Fibrosis cannot help but feel deeply sympathetic for patients and their families, who are left without treatment because the company - Vertex - and the NHS can’t agree a price.
The stalemate has been going on for too long and it is not surprising that there are more calls for an urgent solution, including from the Health and Social Care Select Committee which launched an inquiry earlier this year to explore what more could be done.
Vertex is not a member of the ABPI and it is important to be clear that we do not know any of the commercial details being discussed between the company and the NHS. That said, as we are the organisation that represents the research-based pharmaceutical industry in the UK, we feel it is important that we set out some general principles and observations, both about the medicine price-setting process and also about recent developments in the Cystic Fibrosis case.
Prices for new medicines need to offer value for money for the NHS. At the same time, they need to allow for ongoing research and development efforts that will produce more innovation for the future. These dual principles are enshrined in the Voluntary Scheme for Branded Medicines Pricing and Access (VPAS), which the industry and government jointly developed to govern how prices are set, and how NHS spend on branded medicines (which covers the vast majority of new medicines) is managed.
The current VPAS scheme caps the growth of branded medicines sales to the NHS. Any sales in excess of the cap are paid back to the government by the industry as a whole. This provides significant budget predictability - in fact 100% certainty - for the Government for the next five years on how much it will spend on branded medicines. However, even though we've capped the budget, it is still critically important to ensure that individual prices reflect the value individual medicines offer patients. In fact, it's arguably even more important for industry that all companies are scrutinised in the same way because, if one company is allowed a higher price than its competitors, the rest of the industry would end up paying more through higher payments under the VPAS scheme.
Value for money in England, is assessed by the National Institute of Health and Care Excellence (NICE). We don’t always agree with the approach taken by NICE to assess the value of medicines. We believe that its methods need to evolve over time as new technologies present new challenges. For example, we will be submitting ideas to NICE shortly, as part of their forthcoming methods review, to address some of the challenges for treatment for rarer conditions. Having said this, we fundamentally support NICE as the cornerstone of NHS efforts to: ensure the price being charged by a company represents the value being delivered to the patient; maintain a level playing field between companies and to secure value for money in the NHS. The ABPI would always encourage companies to fully engage with NICE at all stages of the process.
Fortunately, most new medicines are recommended by NICE, but sometimes there are challenges. We have been working a lot recently with both NICE and NHS England to improve the process for how companies engage with the system to find solutions that can allow NICE to make a positive recommendation.
One of the key priorities for industry in these discussions has been to call for NHS England to be more flexible in its approach to constructing commercial deals. For example, arrangements that allow for the collection of real world effectiveness data , with price going up or down depending on how well a medicine performs can be useful in situations where such evidence is incomplete at point the company and the NHS are trying to agree a price.
In the case of Orkambi, NHS England's recent letter to the Health and Social Care Select Committee, makes clear that it has offered a two year managed entry agreement with data collection that would secure patient access in the short term, while allowing the price to go up or down later depending on the evidence collected. Whilst we would reiterate that we don't know any of the commercial details, i.e. the prices, involved in this offer, it is clear that the structure of the offer represents exactly the sort of flexibility industry has been calling for some time - and indeed responds to some of the observations made by the HSC Committee. It feels like a helpful step in the right direction and we certainly welcome that.
Will it be enough? Only time will tell - but for the sake of cystic fibrosis patients in this country, we hope it will not take much more time. Both sides need to be flexible to get to a solution and we encourage both sides to continue to strive for that as soon as possible.
Dr Richard Torbett
Executive Director, Commercial Policy: UK and International