Orkambi: The dispute is settled for Vertex, but what next for industry?
by Abigail Bishop-Laggett
A year after Vertex, NICE and NHS England resumed talks after negotiations hit an impasse last summer, a deal has now been struck to provide Orkambi on the NHS, with up to 5,000 cystic fibrosis patients in England set to benefit.
The deal is momentous for these patients, whilst also paving the way for future access to Vertex’s pipeline. But now the decision has been made, what will the long-term impact of the negotiations be? Will NHS England use this experience to adopt a new hard-line approach with industry, or might the dispute spark reform through the NICE methods review?
Positive signals for medicines pricing?
There are positive signals that NHS England is receptive to some reform, reflecting the life sciences industry’s calls for more flexibility in its approach to constructing commercial deals. This was reflected in NHS England’s offer to Vertex, through a two year managed entry agreement with real world data collection that allowed the price to go up or down depending on how well the treatment performed.
This is a positive step in itself, and more broadly the Orkambi negotiations have shone a much-needed spotlight on issues that would not otherwise be discussed by the public, media and policymakers, such as the ability of the STA system to effectively review and approve medicines. As Orkambi did not meet the criteria for end of life or the highly specialised technology process, it was assessed using NICE’s standard STA. However, it is clear that this process was not fit for purpose because, like an increasing number of treatments, Orkambi is a precision medicine, reflecting the growing demand for personalised healthcare. By their nature, personalised medicines often impact smaller patient populations, making data gathering challenging. The difficult negotiations around Orkambi are a real life example of where the system has not kept up with the science, and lives were lost whilst the process was drawn out over four years.
It is clear that action is needed to address this and NHS England’s offer to Vertex is a step in the right direction. Recent reports have also suggested that NICE, the Department of Health and Social Care and NHS England have agreed to the re-evaluation of an aspect of the QALY. A valuation study has reportedly been agreed to resolve concerns about how NICE measures and values health-related quality of life, through the EQ-5D questionnaire. This is welcome news for industry and indicates there could be a softening of NICE’s stance on the controversial QALY, although as history has shown, sweeping reforms of the QALY are unlikely.
Negative coverage for industry
Although the Orkambi dispute has drawn attention to the need for pricing reform and has seen some flexibility from NHS England, the row has intensified the focus on the pharmaceutical industry and medicine prices. Throughout the negotiations, the negative coverage centred mostly on Vertex’s negotiating position, rather than the capability of the regulatory system to deliver access to Orkambi. This defined a public narrative which has damaged the wider industry’s reputation.
At the same time, Parliament started paying close attention and a Health and Social Care Committee evidence session was held on the topic – the first session on specific drug pricing ever held. One of the main issues discussed was whether the current IP framework protects the profiles of companies over the interests of patients. The Cystic Fibrosis Trust and Parliamentarians began calling for powers to address the problem, with Just Treatment, the campaign for access to treatments on the NHS, calling for the introduction of crown licensing.
Whilst such sweeping powers were not required to breach the impasse with Vertex, the wider effect of this campaigning can be felt across the policy landscape. Labour’s ‘Medicines for the Many’ proposes crown licensing to clear the way for generics firms to produce patented medicines. Such policy proposals are likely to prove popular; ‘Medicines for the Many’ received broad support from the public with more than two thirds of voters (68%) supporting the idea of a UK Government run medicines development and manufacturing company.
What does this mean for industry’s relationship with policymakers?
When the dust settles and initial controversy fades, what will this mean for future pricing negotiations? Emboldened by their hard-line stance with Orkambi, we could see NHS England trying new approaches to pricing negotiations. For example, the choice by NHS England to publish its offer to Vertex was an unprecedented move in commercial negotiations. It was a crucial decision which greatly impacted public opinion and spurred media coverage. NHS England may now feel justified in publishing details of their negotiations in order to achieve a better deal in future.
However, whilst industry is seeking to recover its reputation, the dispute does have positive implications for pricing, broadening the scope with an increased debate on whether the system is fit for purpose and the need for reform. NHS England’s flexibility in its offers to Vertex is encouraging and reflects calls from industry for better use of real world data. Despite this, the negative feelings built towards industry both from policymakers and the public will have a lasting impact.
Going forward, the sector must prioritise positive engagement with policymakers to rebuild trust and demonstrate value, by partnering to improve outcomes and control costs across the NHS. To mitigate the risk of a new hard-line approach from NHS England and Government, such as crown licensing, industry must effectively communicate the negative impact such reforms would have on innovation and patient access to the best treatments in the UK.