This article is the product of a POLITICO Working Group presented by Sanofi.
The debate over how to make pharmaceutical companies treat all European markets fairly is raging. But there’s a second front opening in the industry’s fightback against the European Commission’s pharmaceutical reform: the category of unmet medical need.
The idea seems innocent enough. It’s an incentive that rewards new drugs for patients with serious diseases who don’t have any other treatments and whose medical needs are, well, unmet. But it’s already provoking strong reactions in some corners, with objections spanning from accusations of more red tape to worries that it will actually stymie innovation.
“Having another step of evaluation [in the drug approval process] would just add complexity, and create unbelievable discussions between member states,” said Christian Deleuze, deputy managing director for innovation at Sanofi, who was speaking at a working group on rare disease drug development organized by POLITICO.
Deleuze and his colleagues in the industry are worried that drugs that don’t fall into the category — the majority — will be stigmatized, fetching a lower price when it’s time to negotiate with national health systems.
“Is the idea to incentivize those who are trying to reach diseases with high unmet need, or is it a way to look at how to reduce, artificially in a way, the perception of the value brought by part of the products?” asked the Sanofi manager during the panel.
Policymakers, on the other hand, see the label as a way of directing pharmaceutical research spending to what the public needs, and not just what the market wants.
How it works
The category is included in the draft pharmaceutical package published by the Commission earlier this year, which aims to increase European patients’ access to medicines.
It comes in two flavors. There’s the basic unmet need category, which could apply to all newly approved drugs that meet the criteria. Then there’s the high unmet medical need category, specifically for products for rare diseases, which affect fewer than 1 in 2,000 people.
They both work the same way: If a medicine approved by the European Medicines Agency is granted the label, that product gets more time on the market without competition from rivals (six months for unmet medical need, or a year of market exclusivity for high unmet need). In both cases though, this bonus comes after a deduction in the baseline of protection compared with the status quo.
A medicine will be granted the unmet need label if it treats a “life threatening or severely debilitating condition” for which there is no treatment — or where existing treatment is considered unsatisfactory — and produces a “meaningful reduction in disease morbidity or mortality.” Meanwhile, “high unmet medical need” is reserved for drugs for rare diseases for which no treatment exists, or for which the medicine would be considered an “exceptional therapeutic advancement.”
The logic is simple enough: Drug developers that invent a medicine to treat unserved patients should be rewarded. And the current system clearly isn’t working. While pharmaceutical companies have made important strides in the past decade to tackle some diseases, many more are languishing neglected. A 2020 review of the EU’s rare disease rules found that 95 percent of rare diseases still have no treatment options, for example.
Even when a new drug is approved, often its effect is negligible. A study of cancer drugs authored by the Belgian Health Care Knowledge Centre found that new drugs reimbursed in the country since 2004 didn’t improve survival rates in six of 12 cancers studied, and only slightly improved rates in the other six. Drug spending, meanwhile, ballooned over the period.
It’s such a problem that some countries want to go further than just tweaking data protection. They’d like the category to become a way to direct private investment into where research is most needed, by having national reimbursement authorities take the label into account when deciding how much to spend on a medicine.
“The time has come to think of new systems, allowing for a change in business models. Models that focus more on societal and patient needs rather than on supply,” reads a position paper authored by the so-called Beneluxa group of countries, which includes Austria, Belgium, Ireland, Luxembourg and the Netherlands. The hope is to cajole pharmaceutical companies to diversify their research, and not churn out copycat medicines for diseases that already are addressed.
They appear to have a friendly ear in the Commission. In June, DG SANTE’s head of the medicines unit, Olga Solomon, said that the aim was “to catalyze change beyond incentives” and that the Commission was seeking a “lifecycle” approach with medicines development. She explained the Commission hoped the category could help spur discussion “across stakeholders, including [health technology assessment] bodies, pricing and reimbursement bodies, industry, patient associations.”
This is exactly what the pharmaceutical industry fears.
Speaking at the same working group, Alexander Natz of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE) — the lobby group for pharma SMEs — said the risk was that once a drug wins the unmet needs label, it discourages other medicines launching in that same space. That’s a problem because different medicines for the same disease might be better suited to different patients. Competition can also help drive down prices.
There could be other problems too.
Under the Commission’s draft plans, the unmet need designation is only awarded when a drug is approved. That means that when investors and companies are making their business decisions, they can’t be sure if a drug candidate will be eligible — after all another medicine might come on the market first, said Natz. That uncertainty dampens the incentive effect.
The Commission’s definition of high and normal unmet medical need is just one of many — and it might not stick.
The European Parliament’s first draft of the pharmaceutical directive puts on the table a looser definition that also includes medicines that have a “meaningful positive impact on quality of life” or produce “meaningful delay of the onset of the disease or its complications.”
In some countries, it’s already a reality, even if it works differently from what the Commission is proposing. Belgium in 2014 introduced a law enabling medicines that meet an unmet need to be fast-tracked so that patients get earlier access to them.
“It’s a sort of scoring and grading system which takes into account the input from various stakeholders, so that we have a real picture of what the medical needs are,” said Diane Kleinermans, the president of the commission of drugs reimbursement for Belgium’s National Institute for Health and Disability Insurance (INAMI-RIZIV). The end result is a ranked list of unmet needs. Belgium is now working to refine and improve the methodology, in what could be a possible template for the European proposal as well.
With the country in the driving seat of the European Presidency at the start of next year when discussions on the pharma package will be happening in earnest, both supporters and detractors of the unmet need concept better pay attention.
“I can tell you that for Belgium, unmet medical need is really one of the priorities … that will be addressed during the next presidency,” said Kleinermans at the working group.
This article is the product of a POLITICO Working Group presented by Sanofi and was produced with full editorial independence by POLITICO reporters and editors. Learn more about editorial content presented by outside advertisers.