Are We Getting The Medicines We Need? Speakers Ask At European Health Forum Gastein
Executive Summary
Speakers at the recent European Health Forum Gastein conference in Austria claimed that many new drugs don’t really bring a meaningful clinical benefit, and that new ways are needed to assess the value of innovation.
Are we getting the medicines we need? Are we getting the medicines we can afford? These were two of the questions discussed at the recent European Health Forum Gastein, a conference that is held each year in the Austrian alpine resort of Bad Hofgastein, where policy-makers, industry, health activists, academics and other stakeholders try to find ways of addressing a range of topical health policy issues.
This year, which sees the 20th anniversary of the founding of the EHFG, a session entitled “Medicines: new game, new rules – pathways to better and affordable medicines” focused on whether new drugs really offer value for money, the need for more evidence on which governments and payers can base pricing and reimbursement decisions, and the ability of healthcare infrastructures to properly assess and reward innovation in the future.
The tone was set by Courtney Davis of the department of GlobalHealth and Social Medicine, Kings College London, who told the audience that while there have been genuine breakthroughs where new medicines have changed standards of treatment, most are not in that category. “They either offer incremental benefits or none, or we simply don’t know. Too many drugs are coming on the market that don’t have a strong enough evidence base to inform patients and doctors and funding decisions in a way that will allow the efficient and appropriate use of medicines,” she declared.
“Of 23 indications associated with a survival benefit, less than half were judged to show a clinically meaningful benefit” – Courtney Davis, Kings College London
Davis illustrated her case by citing a new (open-access) case study just published online by the British Medical Journal entitled “Availability of evidence of benefits on overall survival and quality of life of cancer drugs approved by European Medicines Agency: retrospective cohort study of drug approvals 2009-13.”
This showed, she said, that in that period 48 cancer drugs had been approved for a total of 68 indications, and most were “approved without evidence that they extend survival and improve quality of life.” After five years of follow-up, only 35 (51%) of the 68 indications had shown significant survival or quality of life gains, and for 33 (49%) the evidence remained uncertain. Of 23 indications associated with a survival benefit, “less than half were judged to show a clinically meaningful benefit according to the ESMO [European Society for Medical Oncology] threshold,” she said.
In order to incentivize “real innovation”, regulators should think about raising regulatory standards regarding the evidence base required, and “when drugs come onto the market that have shown survival and quality of life gains, they must be clinically meaningful to patients,” Davis said.
Incidentally, the BMJ study, published on Oct. 4, has been criticized by the European pharma industry body EFPIA, which said it “predominantly focused on clinical trials, rather than on real world data on actual patient outcomes,” and noted that gathering overall survival data on new cancer medicines generated in clinical settings “continues to represent a significant challenge both in terms of complexity and time.”
The issue of meaningful innovation was taken up by Ajay Aggarwal, consultant clinical oncologist at the London School of Hygiene and Tropical Medicine, who said: “I need to know what the value is compared with what we have already.” What was needed, he said, was “smart thinking – innovative is not always better.”
Aggarwal asked whether we are “investing in those things that will bring benefit for the money we are spending? Currently we are not. We need good regulation that says we want something that will improve quality of life and extend life and save lives, and then we can have the price debate. I am being honest about what is going to improve outcomes for my patients,” he said to applause from the audience.
Steering The Elephant
The session then turned to whether, in areas like cancer drugs, regulators and payers have the right evidence to hand when deciding which products should be approved or how they should be priced or funded.
Sueri Moon, director of research at the Global Health Centre, illustrated the problem using the Indian parable of six blind men examining different parts of an elephant and coming to very different conclusions about the nature of the object.
“The point is that when you can’t see the whole thing you can’t know what it is, or steer it in the direction you want it to go,” Moon declared. “Society wants to steer the industry to deliver medicines that are safe and effective and improve quality of life and are affordable, but there is concern that we are less and less able to steer the elephant.”
“We are doing this blind,” she said. “We don’t necessarily know the effectiveness at launch or even after five years, and we don’t know the cost effectiveness. And how do we judge fairness of the price?”
Noting that she had tweaked the parable a little and that the men in her slide of the elephant were blindfolded rather than blind, she said it was necessary to “take off the blindfold and see more clearly what you are dealing with… so you can help steer the elephant.”
This meant, for example, ensuring that the prices paid for medicines were made public, requiring the disclosure of the real costs of R&D – as well as manufacturing costs – associated with new drugs, and ensuring all the clinical trial data are published.
Moreover, she said, when policy-makers put in place incentives, they might not deliver the results they hoped for – she asked, for example, whether the award of orphan drug status, along with fee waivers and periods of exclusivity, was always appropriate.
An alternative approach to incentivize R&D could be to offer more money for publicly funded trials “to generate the kind of data we need,” or to learn from the experience of not-for-profit initiatives, such as those on neglected diseases, which have “very targeted aims” and share the risks, benefits and costs, with public disclosure of all research generated.
Price Pressure And Transparency
Also under debate were the high prices of some new drugs and how to approach pricing strategies. Referring to Novartis AG’s recent $475,000 price tag for its anticancer drug Kymriah ((tisagenlecleucel), Moon said, “I find it shocking” but “we are almost at a point where $50,000, $80,000, $100,000 is no longer shocking.” (Also see "Novartis Beats CAR-T Competitors To The Pricing Punch With Kymriah Approval" - Scrip, 31 Aug, 2017.)
Some delegates suggested international price transparency as a tool for keeping prices down, but others asked whether that would be a good thing given disparities in the resources available to healthcare systems in different countries.
A representative of the European Cancer League wondered whether it was realistic to have one country say “this is what we are paying, and then another country says we want that as well. Is it right for different healthcare systems to pay the same price?”
Moon said it was possible, and that there were some areas with almost full price transparency at the international level, as with HIV medicines, although a broader debate was necessary on whether what is fair for one country is fair for another. For example, how much should a poorer EU country pay compared with a higher income one?
She also noted the issue of parallel trade arising in Europe where there were price disparities, and suggested that “in the medium or longer term you could have one price, but transfers [of money] across governments… this is more open and transparent, and is based on what is fair for each country.”
“We are focusing only on spending, and not on other parts of healthcare like savings on hospitalization” that medicines can bring – Elizabeth Kuiper, EFPIA
Elizabeth Kuiper, executive director of European affairs at EFPIA, questioned the session’s focus on prices. “If prices are rising as you say, and are unaffordable, how do you react to the OECD report in 2016 that says since 2013 medicines spending has been falling, accounting for only a fifth of healthcare expenditure,” she queried. “We are focusing only on spending, and not on other parts of healthcare like savings on hospitalization” that medicines can bring.
A representative from Merck Sharp & Dohme was also “surprised at the focus on drug spending. The rationale for going after drug spending is that healthcare spending is unsustainable. But if healthcare is an important part of the social contract, shouldn’t we see it as an investment?” Healthcare would in future take a bigger share of resources in ageing societies and “we must not see it only from an accounting standpoint,” he said.
The second part of the session looked at what governments can do to address the inherent tension between encouraging much needed innovation and countries’ ability to afford them as populations age. This will be covered in a subsequent article in the Pink Sheet.
From the editors of Scrip Regulatory Affairs.