A novel UK approach to cancer access: yes, no, or maybe?

What are the implications of the new, reinvigorated Cancer Drugs Fund, for pharma and patients? Read the views of an expert panel, gathered to discuss the key issues.

A recent webinar discussion on changes to England’s Cancer Drugs Fund (CDF), hosted by IMS Health and pharmaphorum, began with an exclusive, as panellist Nina Pinwill revealed that she would play a key role in running the scheme.

Pinwill, currently associate director at NICE’s Office for Market Access, will become operational lead for the CDF in NHS England, beginning this autumn. It’s a job with a daunting remit – ensuring the CDF does not overspend its £340 million budget, while also striving to allow patients access to critical medicines.

The new CDF is a very different beast from the one it replaces. The old scheme was £126 million overspent, despite several attempts by NHS England to de-list drugs not perceived as delivering value. Something clearly had to give, but whether the new arrangements succeed in bringing expensive cancer drugs to patients, while still balancing the books, remains to be seen.

While the proposals for the fund, which were first floated last year by NHS chief executive Simon Stevens, seemed like a practical solution to a seemingly intractable problem, not everyone is convinced that the new-look scheme will bring cancer drugs to the patients that need them. Some pharma companies, including Roche, have said the new arrangements, for a ‘managed access’ scheme that funds oncology medicines where NICE is unsure of their cost-effectiveness, still rely on a flawed assessment regime. Within the new approach, manufacturers can expect a draft ‘yes – no – maybe’ decision from NICE prior to marketing authorisation, followed by a final ‘yes – no – maybe’ recommendation after regulatory approval and, in the cases of a ‘maybe’, NICE will turn to real-world evidence collection (funded by the manufacturer) for a period of up to two years to make the case for a drug’s cost-effectiveness.

In a discussion following the webinar, Pinwill said that it was unlikely that there would be any other ring-fenced schemes along the lines of the CDF. But the option to “try it and see” for drugs with “plausible promise” would probably be extended to other therapy areas, she explained. “Having a binary decision does not get us the right answer for personalised medicines,” said Pinwill. “When there is uncertainty there is another route than just saying no – it is so important. That’s what I want to see in the coming years.”

The ‘managed access’ approach outlined in the CDF standard operating procedure is likely to become more common, the panel suggested. The agreements are signed not just by the company, and the NHS, but also the patient. They will play a crucial role as they must agree for the data they generate to be used in the ‘real world’ studies that could provide proof of cost effectiveness.

Malcolm Qualie, pharmacy lead for specialised services for NHS England, noted that the approach had already been used in a scheme that was agreed with Biomarin for its medicine Vimizim (elosulfase alfa) for the rare genetic disease Morquio A Syndrome, outlining how these agreements will be vital to ensuring that patients keep their end of the bargain and adhere to any treatments prescribed. Qualie described how compelling patients to turn up for treatment is the best way to ensure they can attend hospitals to get their medication, and provide test data for cost-effectiveness trials, stating, “If the patient doesn’t sign up, they don’t get treatment.” In contrast, the panel was concerned that, in many cases, mobile phone apps were actually poor at helping provide data remotely, as patients often lost interest after a short period.

Questions from the webinar audience, submitted online, showed confusion about who would be in charge of real-world study design. Panellists said they believed that, under the new standard operating procedure, Public Health England would be likely to conduct most studies required for cost-effectiveness data. Companies would have input into the initial design and they would only get to see the final analysis at the end. Timelines would also be a factor as, under the CDF’s standard operating procedure, there would be a maximum of two years of reimbursement.

But I believe the scheme should not be seen as ‘paid-for research’ as it will probably be very different from the clinical trials used to support regulatory filings. Deadlines could be tighter as the emphasis will be on ensuring the scheme remains in balance.

“There is some flexibility but we don’t want drugs hanging around forever,” said Pinwill. Companies will still be able to draw on clinical trial data to support their case for cost effectiveness.

One webinar viewer raised the thorny issue of pricing according to indication, and asked if this could be used to ensure drugs are cost-effective. Katie Pascoe, value and access manager at the Association of the British Pharmaceutical Industry, said: “I think it is something we need to find a solution to at European level – it is not just a UK problem.” But Pinwill countered that there was “no way” that such a system was possible at the moment, as it would be too expensive and bureaucratic to implement. Qualie said a “blended price”, based on an average calculation across all indications was a feasible alternative.

One stumbling block was that the pricing team at the Department of Health, which is a separate body from NHS England, is understaffed. The small team does not have the capacity to strike elaborate pricing deals with drug companies. This has resulted in a lack of flexibility, as anything other than a simple discount could set a precedent, causing an impact on workload.

NICE will also be under enormous pressure. The body is already assessing the drugs that were paid for by the old fund and deciding which should receive routine funding on the NHS. This is a huge task as the cost-effectiveness body will have to make decisions on data from dozens of drugs and indications. It has already caused controversy, as some drug appraisals have been kicked into the long grass if they have been considered of low budget impact.

Ultimately, NHS England will be under significant pressure to make the scheme work. Overspends will not directly hit NHS budgets as industry will have to reimburse the NHS should the scheme exceed its £340 million. While it is understandable why NHS England has introduced this financial safety net, it will not be popular with those in the pharmaceutical sector.

Indeed, pharma already reimburses the NHS as part of the voluntary Pharmaceutical Price Regulation Scheme (PPRS), should drug spending exceed pre-agreed limits and, as yet, there is very little evidence that the cash has been used to stimulate use of new medicines, as was originally planned.

So ensuring the scheme stays on track financially, while ensuring eligible patients get their medicines in the turbulent environment of an under-pressure and changing NHS will be an enormous challenge. When she makes the move from NICE to the NHS in the autumn, Pinwill will take on one of the toughest jobs in the health service.

The expert panel included: Malcolm Qualie, Pharmacy Lead, Specialised Services, NHS England; Nina Pinwill, Associate Director, NICE; Katie Pascoe, Value and Access Manager, ABPI; Angela McFarlane, Market Development Director, IMS Health; Toby Gosden, PhD, Senior Principal in Health Economics, IMS Health, with Paul Tunnah, CEO, pharmaphorum as moderator.

In order to better understand the new CDF, listen to the full, on-demand debate here.

About the author:

Richard Staines is Senior Reporter at pharmaphorum. Contact him via: Richard.Staines@pharmaphorum.com