Over the next two years, pharmaceutical companies will have to engage much more fully with payers and patients over issues of health care policy, ahead of the arrival onto the market of a whole slew of innovative and expensive new treatments which will require difficult decisions to be made on funding and affordability, a London conference heard this week.

55 new cancer medicines are due to come to market within the next five years, the biggest number ever, and many will be very expensive, Dr Brian Lovatt, director of Vision Healthcare, told the Pharmaceutical Pricing and Market Access Outlook meeting organised by Health Network Communications. The growing number of cancer patients will demand these treatments, and those with organised “political might” such as patient networks could very well succeed at the expense of other, less vocal patient populations, he warned.

If the National Institute for Health and Clinical Excellence (NICE) fails to recommend that the next two or three of these new treatments should be made available for use on the National Health Service (NHS) “that could be the tipping point,” said Dr Lovatt. He emphasised however that the UK is by no means the only nation where unequal access to innovative medicines is an issue, pointing for example to Japan’s “cancer refugees” who live outside areas where they are able to receive treatment, and the wide variety of levels of treatment which are provided across Spain’s 17 autonomous regions.

So it is essential that the industry commences this dialogue with patients and payers in order to define what really constitutes “valuable” innovation and help manufacturers to focus their research on what patients want and payers are willing to fund. The questions which need to be considered will include, for example: is purely medical need sufficient ? The concept of value varies from country to country but economic parameters are critical to obtaining the right pricing, reimbursement and market access decisions and, with the choice of medicines now coming down to issues of affordability, clinical proof of effect is absolutely critical, he said.

However, the industry is not yet addressing clinical value, according to Dr Lovatt; for example, while 90% of protocols in cancer research currently are powered for statistical significance, this is not the “real world” – it does not translate into clinical significance, which is what matters to patients and physicians.

So the dialogue must open now, he said. Industry and payers, providers and patients need to agree on what constitutes a “valuable” innovation. For researchers, there needs to be a shift in emphasis from statistical to clinical significance, and from research which is directed by science to a more indication-led approach. For payers and regulators, funding issues need to be addressed and health technology assessment (HTA) decisions need to reflect these agreed value judgements, Dr Lovatt told the meeting.