What lies behind ‘unmet medical need’?

Presented by EFPIA

By GIEDRĖ PESECKYTĖ

with CLAUDIA CHIAPPA and HELEN COLLIS

PRESENTED BY

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— Defining unmet medical need is not only semantics, it will determine which companies reap Europe’s biggest rewards.

— How can countries afford innovative treatments? Data may hold the key.

— Where is the push for equal access from Europe’s Health Commissioner Olivér Várhelyi?

Welcome to Friday’s Morning Health Care! With the geopolitical tensions rising “it is easy to forget how important health is,” MEP András Kulja told the Gastein Health Forum in Austria. “We have to fight every day to highlight and underline how important this topic is.”

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DEFINING UNMET NEED: The wording of “unmet medical need” in the EU’s pharma rules revamp isn’t just semantics — it decides which treatments get incentivized, prioritized and funded. It is about which products will move forward, and which products will be “abandoned,” Rosa Castro, public affairs director at rare disease patient group EURORDIS, told the European Health Forum Gastein on the third day of the event.

For regulators, clarity is key. The definition has to be “operational” said Steffen Thirstrup, chief medical officer at the European Medicines Agency (EMA), explaining that it must be clear whether a treatment fulfils the unmet need or not. “Otherwise, if it’s too ambiguous, we will end up spending time in the European Court of Justice,” he warned.

Further guidance will most likely be drawn up by the EMA. “The legislation more or less will define what we have to put in our guidance, and then we have our hands, if not tied, but at least to some extent restricted,” Thirstrup added.

Industry is pushing for breadth. “With a narrow definition, you run the risk that you exclude elements of a disease that are very important for particular patients,” warned Thomas Bols, head of government affairs and patient advocacy EMEA, at PTC Therapeutics. Bols also warned that if there is a “legal judgement” regarding the definition, companies will be risk-averse, calling this an “undesirable knock-on effect” on innovation.

Proposed definition: The Commission’s draft offered a rather broad definition in Article 83 of the pharma directive. Parliament widened it further, bringing in mobility, quality-of-life and treatment burden. The Council, however, rowed back toward “more traditional clinical interpretations, highlighting efficacy and safety,” said Tresja Bolt, senior adviser at Harwood Levitt Consulting.

Meanwhile, inside the EMA: Castro at EURORDIS said she “cannot believe” that patients’ involvement in EMA’s committees is at risk in the coming political negotiations on the pharma package. “I thought it was given, that it was very well recognized by everyone,” she said, calling on the legislators to keep patients in the decision-making processes.

Don’t go! EMA’s Thirstrup said that the agency has “a long-standing good experience in involving patients in decision making.” “I would be sad to see if [the EU] chooses to the opposite,” he said.

Recap: As we reported Wednesday, the Parliament and the Council take different positions in their proposals. The Council reduced patients’ participation in EMA committees and stripped them of voting rights, in contrast to the Parliament.

SOLVING THE ACCESS ISSUES: It’s an age-old conundrum for health systems ­— how to afford innovative treatments and diagnostics with limited budgets while also ensuring the basic care needs are met. A panel during Gastein proffered some suggestions.

What’s needed: In determining as a society which innovations to pay for, look at unmet need (once it’s defined!), suggested Elisabetta Zanon, CEO of the European Cancer Organisation. “Take pancreatic cancer, where we have not [made] progress in the last few years.”

Payment for reward: Ensuring access to tests and drugs quickly could be achieved through outcomes-based pricing models, she said. That means much faster market access to allow for further real world data collection, but “the price will be indeed adjusted on the basis of the outcomes,” she said, citing the U.K.’s innovative medicines fund model as an example.

Need for speed: Several people raised the need for faster access to innovation, blaming slow clinical trials and medical devices assessment bottlenecks, among others.

Hold up: Günter Waxenecker, head of the Austrian Medicines and Medical Devices Agency, cautioned that this cannot come at the risk of safety. “We kill the joy of innovators … almost weekly,” he said, because the agency wants to see more data. If the regulator makes a wrong decision — citing deaths from the infamous Karolinska Institute trachea implant study — “we will of course be blamed for that.”

Bring on EHDS: Uncertain benefits for innovative therapies can be part-solved with data, he said. “Our public health systems need feedback from the market,” he said. “So far, we have not established these systems … this is the idea of the European Health Data Space.”

But, but, but: People need a “change in mindset” to share their data for research. “We like to share our data on social media … but when it comes to health data, we are very conservative,” he said.

**A message from EFPIA: Digital health solutions are crucial to improve outcomes in cardiovascular care and play a role in the prevention, screening and management of CVD. As the Commission prepares its Cardiovascular Health Plan, the EU has a unique opportunity to turn digital innovation into real-world impact for patients across the region.**

WHERE’S EQUAL ACCESS IN THE COMMISSION’S COMPETITIVENESS MANTRA? European Health Commissioner Olivér Várhelyi admitted at the Gastein Health Forum that the current regulatory framework “does not deliver on the citizens’ expectations.” He vowed to fix that with a flurry of initiatives: the Critical Medicines Act, pharma legislation, the medical devices regulation revision, a new Biotech Act and the European Health Data Space. The pitch? Boost research and innovation to sharpen Europe’s competitive edge.

But does competitiveness mean access? What Natasha Azzopardi-Muscat of WHO missed in Commissioner’s speech was “the focus on ensuring that what we incentivize industry to produce is very much directed towards meeting the public health needs.”

“It’s not just about producers producing the products but we need to make sure they are truly available to all those who can benefit,” she said.

Equity, anyone? “We’re hearing less and less about equity,” warned Ilona Kickbusch, founding director and chair of the Global Health Centre in Geneva. What’s “essential,” she added, is finding the balance: “[We need] to make sure that the equity dimension remains part of this new strategic approach.”

Youth voice: “As the Commissioner says, we want a smart, just and resilient Union,” said Sanja Šišović, president of the International Youth Health Organization. “But I would add that the pre-condition for all of that is that each individual in Europe is actually healthy,” she said, with her words followed by applause.

On the cardiovascular plan: Várhelyi in his opening speech did mention the cardiovascular plan, however, Azzopardi-Muscat, while welcoming the effort, cautioned: “We really [need to] think carefully how such a plan … is truly going to be used to make progress on all fronts and tackle some of the very difficult issues.”

Commissioner’s response? None. Várhelyi left straight after his keynote, skipping the feedback. Don’t worry, Commissioner — we covered it for you.

AS US STEPS DOWN, OTHERS NEED TO STEP UP: The U.S. retreat from global health with withdrawal of funds will have “drastic impacts on health,” Katharina Hauck, a professor in health economics and deputy director of the Jameel Institute at Imperial College London said at Gastein. She pointed to a looming shortfall in prevention programs: “What will fall back on the wayside will be preventive interventions.”

WHO is feeling it: The WHO is already feeling it, forced to let go if “30 percent of its staff … by closing down programs,” WHO’s Azzopardi-Muscat told an earlier panel.

Can Europe fill the gap, especially with the EU’s focus on its own competitiveness? Muscat believes that “Europe may not realize that it has to have that ambition, but it will come,” she told Giedrė. Asked whether she sees such ambition in Várhelyi’s vision, she said “yes,” adding that “he’s well positioned to do so.”

Let’s use this momentum: While the U.S. political climate regarding health is “frightening,” MEP Kulja believes that Europe should also see this as an opportunity to attract talent and investment. “If we can show to our scientists, the researchers, and even companies, which invest in research, that Europe is a safe place … maybe we can reverse the brain drain and also bring those innovation investments,” Kulja told a panel on disruptive technology.

Talking about bringing in talent, the Commission has launched a new initiative as part of its half-a-billion-euro Choose Europe for Science plan to attract foreign researchers. The latest call, backed by a €22.5 million budget, will support organizations looking to host and fund postdoctoral researchers.

PARLIAMENT’S HEALTH WORKFORCE REPORT DELAYED: The joint report from the Parliament’s SANT and EMPL committees on Europe’s health workforce will land later than initially planned. The draft publication has been pushed to Oct. 16, an official close to the matter told Giedrė at Gastein. Amendments will be due by Nov. 3.

DOCTORS RECOMMEND SEMAGLUTIDE AS FIRST LINE DRUGS: Weight-loss drugs like Ozempic and Wegovy should be the first-line treatment for people with obesity, according to new guidelines from the European Association for the Study of Obesity (EASO).

Diving in: The team of doctors, public health and obesity experts evaluated medication for obesity based on their effectiveness in promoting total weight loss, their impact on complications, and their safety profile. In a new framework for the pharmacological treatment of obesity and its complications, set to be published in Nature Medicine, they concluded that tirzepatide and semaglutide should be considered the medications of choice when a substantial total body weight loss is required.

“Even though there are several options on the market, the reality is that semaglutide and tirzepatide are so effective that they should be the first choice in almost all cases,” said co-first author Andreea Ciudin.

From POLITICO’s Gastein coverage: Várhelyi open to new EU funding model for health and EU health commissioner vows to hike vape taxes to match cigarettes.

Doctors who have been banned from practice due to “major wrongdoing” in one country can easily relocate and pick up their careers abroad, the Organized Crime and Corruption Reporting Project found.

STAT News looks at how shutting pregnant women out of gold-standard clinical trials in order to protect them and their babies has had the opposite effect.

**A message from EFPIA: Digital health solutions are crucial to improving heart health. They play a role in prevention, early detection, disease management, and helping patients stick to their treatment. However, barriers remain, including the digital divide, lack of internet access, privacy concerns, and uneven digital skills. Health systems generate great amounts of data, though most of it is not used correctly or not used at all, leaving huge potential untapped. To harness this potential, the forthcoming EU Cardiovascular Health Plan must tackle these barriers and create the right conditions for using data and digital innovation. Scaling up existing pilots and initiatives under IMI/IHI can strengthen prevention and disease management, while a dedicated pilot on cardiometabolic diseases within the European Health Data Space would unlock the power of data to transform care. Imagine what could be achieved if Europe fully unlocked the power of health data and digital tools to transform cardiovascular care.**

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