Cutting the combination therapy access issue down to size

While the access to combination treatments problem is nothing new, its sheer magnitude means it has, to date, gone unsolved. But now, an initiative spearheaded by Takeda is starting to shift the dial.

This time last year, the pharma giant published two whitepapers on the topic, setting out proposed frameworks for valuing combination treatments and for compliant inter-organisation arbitration.

Since then, the team have been taking part in ecosystem-wide discussions and workshops, and a solution could soon be in the pipeline, says Emma Roffe, Oncology Country Head (UK & Ireland) at Takeda Oncology.

A long-standing challenge

Securing access to combination treatments is a long-standing challenge that only promises to become more pertinent in the coming years, Roffe told pharmaphorum.

“As our understanding has advanced, we now know that we often need more than one medicine, with more than one mode of action, to target the different underlying mechanics of disease,” she says. “The challenge we face in the UK is the way NICE and the Scottish Medicines Consortium use cost effectiveness to assess combination therapies.”

The crux of the issue is that combination treatments are evaluated as single technologies, composed of component treatments, each with an individual price. In some scenarios, the initial market entrant, or the ‘backbone treatment’, can account for the whole willingness-to-pay threshold, meaning any add-on components have a much-reduced opportunity to demonstrate value.

“Essentially, the whole burden falls on the company bringing the new medicine to market, but they get no benefit from the extended use of the ‘backbone treatment’ in terms of revenues,” says Roffe, adding that when the component treatments are manufactured by different companies anti-competition law prohibits them from discussing and re-aligning prices to meet cost effective thresholds.

“Sometimes, even if the add-on medicine is priced at zero, the treatment is judged not to be cost effective.”

The consequences of this – for individual patients, healthcare systems, innovation, and the wider R&D community – are as obvious as they are far reaching, she goes on.

“Science is always innovating and moving, but we need to be able to keep up in terms of access. If people in the UK cannot access these new medicines, our standards of care will start to fall behind.”

“That will impact future research. Companies will not want to place research in the UK if we are not working to global standards,” says Roffe.

Too hard to fix?

This access conundrum was acknowledged by NICE as far back as 2014, and has been sitting in pharma’s “inbox” for some time. Roffe believes it has largely been ignored as organisations see it as “too hard to fix”.

Yet as medicine and medical knowledge continue to evolve, we must act or risk being left behind, she says, adding that last year’s whitepapers were part of Takeda’s bid to “grab the problem by the horns”.

An Attribution of Value Framework for Combination Therapies tackles the technical challenge of how to attribute value to each of the different components in a combination treatment, while the Voluntary Arbitration Framework for Combination Therapies sets out how pharma companies could have conversations about value within the confines of existing competition law.

Both papers were developed by an advisory group that included representatives from the patient, clinical, academic, and health economic communities, and with input from NICE and NHS England.

Growing momentum

The response to Takeda’s proposed solution has been overwhelmingly positive, and highlighted the appetite in the industry to get this job done, says Roffe, adding that the team held a virtual roundtable event, bringing various experts with different perspectives together to discuss and challenge the proposed solutions, in May.

She says: “We had some really great suggestions from legal people and from the patient group representatives on how we could adjust things slightly to make them more applicable. It’s clear that we have started something that a lot of people want to get behind and find a solution to.

“The response has really highlighted that this is something people are thinking about at a senior management level. This is no longer just an oncology challenge; many diseases are moving towards combination therapies, making this a whole-industry challenge.”

This is reflected in the fact that the Association of the British Pharmaceutical Industry (ABPI) and the European Federation of Pharmaceutical Industries and Associations (EFPIA) have also recently picked up the mantle. The ABPI, for example, is working with the Competition Markets Authority to develop guidance on compliant pricing conversations.  At the same time, interest from NICE and NHS England is growing.

“I think there is an urgency now, and that is coming to the fore. The level of interest is clear to see,” Roffe explains, adding that progress could only be achieved with concurrent policy change.

“Certain things have to be put in place before any solution becomes transactable – and a key element of that is flexible or non-uniform pricing.  At the moment in the UK, medicines have one price that applies to all indications or combinations.”

“But every different combination treatment has a different value, and we need policy that reflects that.”

The road ahead

There is still a lot of work to do, but Roffe believes Takeda has started to make robust inroads: the more people talking about access to combination treatments, the closer we are to finding a solution.

Said Roffe: “It does not matter if it is our solution, or someone else’s solution – we just need a solution that we can implement so that, ultimately, patients get access to world-class, life-changing medicines.”

  • For more on Takeda’s proposed solution for the combination treatment issue, including roundtable discussion reports, videos, and infographics, go to the website.