OBJECTIVES : Many cancer medicines are made available on the National Health Service for England (NHS) based on a price discount agreed between the NHS and the manufacturer. However, such negotiations can have a power imbalance, lack transparency, and be protracted thus delay patient access. More innovative ways for the NHS to pay for medicines, such as Outcome-Based Payment (OBP), which links a medicine’s price to NHS patients’ treatment outcomes, could provide a solution. This research explores the feasibility of introducing an OBP approach for new cancer drugs in England.

METHODS : A literature review explores the current funding landscape in England, the available evidence on existing OBP schemes internationally, and which outcomes cancer patients value most. Two focus groups and an online survey with patients and carers, as well as interviews with NHS and government stakeholders, healthcare professionals, and pharmaceutical industry representatives, provided additional evidence on the feasibility and suitability of OBP schemes.

RESULTS : Implementation of OBP in the NHS is desirable and particularly suitable for medicines with the following characteristics: potentially large benefit to patients receiving the medicine; small to moderately-sized patient populations; immature clinical trials data; and a disease profile where improvements in outcomes measurable in the short-term (including overall survival and non-progression/relapse) are particularly valuable. As part of any future OBP schemes negotiated between NHS purchasers of cancer medicines and manufacturers, specific metrics should be included to measure the drug’s effects on patients in the NHS, on the following four types of outcomes as standard: survival; disease progression, relapse or recurrence; long-term side effects; and return to normal activities.

CONCLUSIONS : Significant advances in understanding the principles on which an OBP approach might be based for new cancer medicines for the NHS in England have been achieved. Future research will establish the necessary steps for implementing a pilot OBP scheme.