The Cancer Drugs Fund achieved many of its aims. It transformed access to breakthrough oncology drugs in the UK, and brought the NHS closer to the levels of access seen elsewhere in Europe. If someone had pointed to the elephant in the room, it might still be here in it's former shape. 

Did I say elephant? I mean evidence.

Real-world evidence, or analysed Real-world data which has produced meaningful insights, is now foundational for pricing and reimbursement. As therapeutic value is linked to patients and outcomes across health systems, the burden of risk is being re-balanced with manufacturers now being required to shoulder more of the burden. On this basis, the proposed changes to the Cancer Drugs Fund by NHS England to incorporate it into the NICE HTA assessment should be welcome news. It’s welcome because NICE is increasingly placing Evidence in a formal assessment context, and with that adding yet more credence to the development of non-trials data. The CDF undertook assessments based on cost alone, whereas NICE bases its recommendations on real-world QALY. In theory, this should be good news for patients needing access to drugs for rare cancers.

The new managed access fund under NICE will have a £340m budget and will retain part of all payments to manufacturers pending end of year spend review. If the new fund over-spends, the retained funds will be used to balance the budget. So the core principle is one borrowed from the PPRS initiative – capped spend. Unlike the PPRS however, there doesn’t seem to be a commitment to free pricing below the spend cap though. Instead expect pressure from NHS England and NICE on manufacturers to discount, perhaps heavily, to access the fund as a means of access in what is effectively an alternative reimbursement pathway.


There is a carrot of course, which is an expedited assessment by NICE using Evidence generated through the fund patients. This Evidence will be used by NICE in determining whether to recommend for routine commissioning. Nearly half the drugs in the CDF were coming from only three companies – Novartis, Roche and Janssen. Companies with deep pockets and able to make the concessions to access the fund but other breakthrough therapeutics might not have the resources, or the nerve, to take a big hit on the promise of a later routine commissioned payday. Some early efforts in value-based pricing schemes under the CMC in the US showed a moderate success. Such schemes overcame the inherent reluctance of health systems to shoulder a burden of risk by capping costs, but such schemes also proved hard to scale there. Not in itself an issue in the UK.

Or in the world of patients, access to cancer drugs could be reduced, or even lost, to some 12,000 patients a year. This is the estimate of the Rarer Cancer Foundation when the proposals were first announced.

The CDF over-spend and the lack of assessment on many of the drugs in the original fund could have been mitigated with the collection and assessment in turn of clinical outcomes data from patients. The mechanisms were already in place elsewhere: commissioning through evaluation, which is where the CDF, under NICE, will arrive, would have continued access to speciality drugs to allow evidence to be collated. Adaptive Licencing. Early Access schemes. All these evidence generating schemes are in operation in the NHS already.

Initially, it looked as though an important alternative pathway for reimbursement in rare cancers, and most importantly patient access to innovative therapeutics, would be curtailed. A commitment to utilising the access fund to gather timely Real World Evidence while drugs undergo HTA assessment is a reassuring measure.

Evidence the elephant is still in the room, but looking a lot less like Dumbo.


Written by Kevin Acourt - Head of Healthcare and Life Sciences Practice at Consulting Point