NHS made big strides in market access this year with the Accelerated Access Review and changes to the CDF – but will they work?
The new face of the CDF
England's Cancer Drugs Fund (CDF) reopened in late July with a new operating model, but faced immediate criticism from the pharma industry and charities, who said it would do little to solve fundamental issues with drug reimbursement and access.
The original CDF was launched in 2011 as a 'silo fund' for cancer drugs deemed by NICE to be too expensive, but was shut down in March this year after costs spiralled. Under the new system, NICE can use the CDF to fund use of a drug for two years while further evidence is collected, when findings will be fed into a second appraisal.
Critics argued that the new CDF arrangements fail to address the issues that prompted the creation of the fund in the first place. "The CDF was set up because NICE's methodology was not working for cancer drugs and this new process offers little change," said Baroness Delyth Morgan, chief executive of the charity Breast Cancer Now. "With the fund's drug assessment now being handed back to NICE, we worry that patients in England will miss out on effective drugs that are available in other countries."
This sentiment was echoed by a number of pharma leaders, including Richard Erwin, general manager of Roche UK. He said: "Now that the assessment of new cancer medicines for reimbursement has been returned to NICE, we must, as a matter of urgency, address the challenge they have in assessing the real clinical value of cancer treatments – which necessitated the creation of the original CDF in 2010," he said. "We are calling on government to review NICE's assessment methodology to stop patients facing ongoing anxiety around the availability of existing and new cancer medicines".
The firm had been waiting for more than two years for a final decision on Perjeta (pertuzumab) for neoadjuvant treatment of HER2 positive early breast cancer, he said, noting: "NICE's methodology is unable to find combination treatments cost-effective, even when they significantly extend overall survival for late stage disease."
While the ABPI welcomed the way the new Fund "joins up the dots" between NICE and NHS England, it also stressed that further evolution of NICE's appraisal process is required. "Importantly, we also believe that given the fact that the old Fund consistently overspent significantly on its allocated budget, and that industry already underwrites the majority of expenditure on branded medicines over and above agreed levels, a fairer and more equitable system of financial risk must be prioritised," the trade body said.
A previous analysis by the Rarer Cancer Foundation estimated that over 12,000 patients a year could lose out on treatment under the proposals.
However, Cancer Research UK has voiced its support. "We welcome the decision to move forward with reforms to how the NHS makes certain cancer drugs available to patients," said its chief executive Harpal Kumar. "We see this as a positive step in creating a more sustainable and flexible system. Importantly, we believe the proposals will bring more certainty to patients and doctors about the drugs that can be prescribed, and swifter access to promising drugs."
In October, the government published long-awaited findings of an independent review on how to ensure patients can benefit from healthcare innovations much more quickly.
The Accelerated Access Review was commissioned with the view to making the UK "the fastest place in the world for the design, development and widespread adoption of medical innovations" as well as to "stimulate new investment, jobs and economic growth to support the NHS". The Review, developed in partnership with the Wellcome Trust, made 18 recommendations that could propel a step change in access and uptake of new medicines and technologies across the country.
For one, it proposed the creation of a new Accelerated Access Partnership, to help quicken and simplify the process for getting the most promising new treatments and diagnostics safely from preclinical development to patients.
Through the new Partnership, which would span NHS England, NHS Improvement, NICE and the MHRA, innovators would have access to "joined-up help with clinical development, regulation, and assessment of cost-effectiveness".
This could potentially bring forward patient access to drugs by up to four years if a scientific opinion from the Early Access to Medicines Scheme is used (saving 12-18 months) and there is no delay at the NICE appraisal stage (which takes around two years) or during the process for NHS commissioning and adoption (which takes two years or more).
The Review also suggested that a new Strategic Commercial Unit be established within NHS England to facilitate flexible arrangements through commercial dialogue with product innovators, the idea being to secure faster access to the NHS market and better value for the taxpayer at the same time.
Other recommendations included improved horizon scanning and prioritisation for innovative new products, better data on the impact of technologies on patient outcomes and uptake; local support for the spread of innovation, through Academic Health Science Networks; and stronger incentives for local NHS organisations to use and spread the benefits of innovation.
Wider reaction to the plans has been largely positive. The ABPI called the review "a significant first step in addressing poor patient access to new medicines in the UK".
"The Accelerated Access Review… opens the door to greater collaboration between innovators, patients and the NHS to make the UK a world leader in researching, developing and using new treatments and technologies," said the Association's chief executive Mike Thompson. "Turning this into reality now relies on a positive government response to the Review, and a clear implementation plan."
Professor Paul Workman, chief executive at The Institute of Cancer Research in London, added: "I'm especially pleased that this excellent report recognises that improving access to truly innovative medicines and other products requires that multiple partners in the ecosystem need to work closely together to find solutions to the major medical problems we face." But, he also expressed concern "that only a handful of treatments per year will benefit from the proposed Accelerated Access Pathway", and called for "changes across the board that provide incentives for a much greater number of innovative cancer drugs to be approved and made available… very quickly once their potential becomes apparent."
The government has said it will consider the proposals "and respond more fully in due course, mindful of the need to ensure affordability".