At times in recent months the number of competing claims to achieving a long term solution to evaluating new specialty medicines has resembled the race to the South Pole as NICE, NHS England and DH have all had review processes underway.
What emerged yesterday with the publication of the interim findings of the Accelerated Access review is a consensus around conditionality. The core recommendation of conditional approval from NICE strongly echoes Bruce Keogh’s idea floated in the summer that NICE should transition to be an interim process while a medicine proves its worth. In addition NICE has recently reached agreement for a “Managed Access” deal for introducing Vimizim® and signalled at its recent national conference that it is open to piloting new reimbursement models within the straight process.
What this means for those creating personalised medicines for ever more tightly defined genomic expressions of disease is a more complex and uncertain reimbursement environment where the level of proof no longer ends at pivotal phase III trial stage. Validity and agreement with regulators on use of real-world data in maintaining approval becomes more important. In compensation for this it should herald the end of the NICE “wall” of a once and forever judgement on medicines access – a barrier that has been growing ever more insurmountable in recent years. The new world will be less yes-no and more a ‘maybe while we collect more data’.
Challenges for the system include reconciling suspicion from big pharma that ‘maybe’ is a mechanism for the NHS to keep prices low, with suspicion from the NHS that ‘maybe’ is a mechanism for industry to keep prices high. Added to this high stakes game of trust are very real questions for patients and their families about who will pay for the drug while data is being collected, and who will pay for those patients already on treatment if a decision is taken to discontinue it after real-world data has been collected and analysed.
There are lots of questions that still need to be answered, and the next piece of the jigsaw for personalised medicines is the long-awaited consultation on the Cancer Drugs Fund. If it is consistent with the recommendations in the Accelerated Access review, then the goal of a more joined-up system for approving and reimbursing medicines may finally be drawing closer.