#EAPM – Congress build consensus for progress on innovative personalised medicine

#EAPM – Congress build consensus for progress on innovative personalised medicine


The second and final day of the Third Annual Congress organized by the European Alliance for Personalised Medicine (EAPM) saw multi-stakeholder consensus reached on many facets of facilitating innovation in modern-day European health-care systems, writes European Alliance for Personalised Medicine (EAPM) Executive Director Denis Horgan.

The event was held under the auspices of the Finnish Presidency of the EU, was titled ‘Forward Together with Innovation: The Importance of Policymaking in the Era of Personalised Medicine’, and took place at the Fondation Universitaire in Brussels (3-4 December).

The meeting of multidisciplinary personalised medicine experts followed two successful annual congresses in Belfast and Madrid, plus seven annual Presidency Conferences.

What happened on Day Two

As ever, Congress showcased different objectives which both the public and private sector can support, with a view to allowing the EU to present a common objective. It took place in a focused format to allow concrete issues to be tackled and to have a dialogue with policymakers.

Day Two featured full and productive with sessions on the current hot topic of the Orphan Regulation, evidence frameworks, plus value-based outcomes and biomarkers.

Biomarkers were discussed recently at an EAPM satellite event which took place at the ESMO Congress in Madrid and a pre-event roundtable on the topic of biomarkers and molecular diagnosis took place ahead of the main Congress in Brussels.

Among those speaking at the event were Ortwin Schulte, head of Health Policy Unit, German Permanent Representation

Germany has its EU presidency coming up in the second half of 2020, and Ortwin told attendees that his country is working on a trio presidency” with the two that follow it in 2021, that’s Portugal and Slovenia.

He told attendees of his concern that the proposed health programme’s budget may not cover “all the new tasks we have, including formal HTA co-operation” as well as follow-up work on European Reference Networks.

Meanwhile, Europe needs consistency across disease areas, and that potential efficiencies for agencies need to be identified, he said.

“The ultimate aim,” he said, “is faster patient and citizen access to diagnostics with proven value and subsequent treatment where required.”

Later, Stephen Robbins, scientific director at the CIHR Institute of Genetics, in Montreal, Canada, explained that personalised medicine is commonly understood as providing the right treatment to the right patient at the right time. He said: “I would also add to this definition at the right location, by the right provider and at the right cost. This includes the equality dimension to the definition of personalised medicine, which makes quite some sense given the geography of Canada, but also of Europe.

Stephen told attendees that the CIHR is he largest research body in Canada dedicated to improving the health of Canadians. It is in partnership with more than 250 organizations – including charities, hospitals, universities, local governments, and academics.

The CIHR launched its personalised medicine initiative in 2012 to enhance health outcomes through patient stratification approaches by integrating evidence-based medicine and precision diagnostics into clinical trials. 

“This programme progressively aligned to another programme on eHealth – the eHealth Innovation service – to become the personalised medicine initiative in 2016. As we went forward, the team became more and more complex and multi-sectoral,” he added.

In the session on the Orphan Regulation, Member of the European Parliament Tilly Metz, meanwhile, spoke about the need for more EU action on rare diseases. 

It is estimated that about 30 million people living in the European Union suffer from a rare disease. Since the adoption of the orphan drug directive, between 6000-7000 rare diseases were identified in Europe, and this is a good indicator of the incentive to innovate in this field. 

“However, we dont hear about ultra rare diseases that affect one-in-1/50,000 Europeans. they must be part of the debate despite the small numbers of patients affected.”

Tilly added that: “The debate on personalised medicine and the debate on rare diseases should not be mixed. Otherwise, we risk hampering the development of therapy for rare diseases. They should be treated in two different frameworks. Removing the special incentives on rare disease from this specific framework is risking endangering patients with rare diseases.

“There is a urgent need to raise awareness on rare diseases, especially for children, so that patients can access to diagnostic in specialised centres more quickly.,” the MEP said.

Simone Boselli, public affairs director at EURORDIS, told attendees that Europe “needs to encourage more research into rare diseases, evaluate current screening population practices, and supporting rare diseases registries”.

EURORDIS, she said, has described a rare disease patient as the orphan of health systems, often without diagnosis, without treatment, without research: therefore, without reason to hope”.

Simone added: “We believe that major efforts are needed to foster and stimulate research in order to increase existing knowledge, which is nowhere near enough to meet the challenges.

Despite the undoubted hard work undertaken by the EU over almost 20 years, there is still a long way to go,” she said.

During the session on HTA, MEP Tiemo Wôlker told attendees that: “The European added value of the (Commission’s) HTA proposal is obvious. It would avoid duplication and enable a better allocation of resources. And it will minimize the risk of fragmented access to therapy across member states.”

He added: “We also need to recall that reimbursement and pricing are not covered by the proposal and that will remain a competence of Member States. EU cooperation is needed to ensure constant exchange of information between HTA authorities in the EU.”

Time pointed out that: “Germany and France are blocking the proposal for subsidiarity concerns, but we should act now as the EUnetHTA is coming to an end.”

Floria Giorigio, of DG SANTE at the European Commission, said that the objective of HTA proposal is to reduce duplicity, promote convergence, strengthen quality of HTA across Europe, ensure the uptake of joint outputs In member states, and ensure long term sustainability. 

She said: “The proposal sets up a collaboration framework of HTA. This is also about ensuring appropriate evidence for HTA across the EU. We know this is doable, we know that it is useful, but we need structure to ensure this is efficient.”

Floria went on to say that Parliament has added more flexibility to the proposal and supported the overall approach of the Commission. Meanwhile, the Finnish and the upcoming Croatian EU presidencies are willing to make progress.

Significantly, Floria said: “I notice that many stakeholders don’t grasp the added value and the knowledge that patients can bring their expertise of the disease is a crucial element.

“Their involvement in the process is also key to their empowerment. But involving the patient is not a given for everybody.” 

On biomarkers, Laetitia Gambotti of INCa, in France, said: “Many predictive biomarkers are available for target therapy for different cancer types. Ten years ago, our goal was to provide the best quality, nationwide molecular diagnostic tests for all patients. 

“A network involving 28 regional centres was set up, partnerships were signed with laboratories, hospital research centres…and, in 2013, we launched another pilot programme on the implementation of targeted next-generation sequencing that involves 11 molecular genetics centres.”

She added: “We also have opened five screening centres for BRCA with 1100 patients screened in two years.

“Patients should have the same access to the best treatment available. These programmes and organizational framework enable equality of treatment no matter the location of patients,” Laetitia said, adding that there is a need to “raise awareness about biomarkers and diagnostics” as well as organizing a prevention and screening campaign. 

“We need to inform citizens before they are patients”, she said.

On the topic of reimbursement, meanwhile, Christine Chomienne, professor at the Université de Paris, told the audience that doctors have always practiced personalised medicine. “We integrate both the disease’s characteristics as well as the patient’s characteristics,” she said.

Christine explained that a GP takes into account the age, gender, life-style, and history of a patient when treating him or her.

“But I have never asked about the wealth of my patient to make a treatment decision so far, as we always took reimbursement for granted,” she said. 

“But this is not true, as reimbursement depends on several parameters: the healthcare system, private health insurance, and so on…” 

Christine went on to explain that reimbursement for treatment and/or a diagnostic is a decision taken at member-state level. After Approval by the EMA and the national authority, usually a national body gives recommendations regarding pricing and reimbursement, and the health ministry takes the final decision.

This decision is based on several criteria assessed based on data, literature, clinical trials, meta-analysis and, as personalised medicine develops: “this evaluation and reimbursement decision process becomes more-and-more complex”. 

“We need…to avoid refusal due to lack of evidence. We need to make patients and practitioners aware and involve them in the process of making good decisions on reimbursement and pricing to ensure access to personalised medicine,” Christine said. 

In the final session of Congress, Petra De Sutter MEP said that: “Regarding personalised medicine, the IMCO committee (which she chairs in Parliament) is very much interested in the regulation of data sharing, and the collection and transfer of data, while protecting the rights of the provider of the data, meaning the patient, consumer or citizen. 

“This will be a very important issue that concerns the health sector as the new Commission has put the digital single market as a priority for the coming mandate.”

“Trust is a key issue for data sharing,” the MEP said, “and that concerns the future of personalised medicine in Europe.” 

“I’m confident that the Parliament will support the work of the European Commission on this. We really need to act now, as in five years, it will be too late. If Europe doesn’t act now, other players will fill the room,” Petra said.

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