The changing regulatory landscape brings new challenges to Health Technology Assessment (HTA). Marketing authorizations are being granted as the evidence base evolves to facilitate timely patient access to promising health technologies. Consequently, some products come to HTA bodies sooner in their development cycles with less evidence, which ultimately leads to greater uncertainty in decision making. A key challenge for payer and HTA bodies is providing access to promising medicines while the evidence is still emerging, in a financially sustainable way.

Changes to the Cancer Drugs Fund (CDF) have resulted in a managed access fund for cancer medicines in England. The National Institute for Health and Care Excellence (NICE) can now recommend a treatment for use within the CDF if there is plausible potential to satisfy the criteria for routine use in the National Health Service (NHS) at its current price, but the evidence is not robust enough and associated with significant uncertainty. Further evidence is then generated in clinical trials, through observational data collection, or a combination of the two, while the drug's price reflects the decision uncertainty. At the end of the managed access period, NICE reviews the guidance to determine if the treatment can be recommended for routine commissioning.

The first treatment recommended for use within the new CDF was osimertinib for non-small cell lung cancer (1). At the time of NICE appraisal, there was considerable uncertainty in osimertinib's clinical and cost effectiveness because only short-term phase II trial results were available. NICE's independent appraisal committee considered there was plausible potential for osimertinib to be cost effective and identified that an ongoing phase III trial would provide longer-term data addressing the key uncertainties.

An integrated approach between payer and HTA decision-maker has significantly changed how cancer treatments in England are appraised. This collaborative way of working heralds a more sustainable approach to introducing promising cancer treatments.