#EAPM - Istraživanje zdravstvene skrbi: nastavak rasprave ...

| Siječanj 11, 2019

Research in health care is an ongoing discussion and the European Alliance for Personalised Medicine (EAPM) has always been vocal in this important field,writes EAPM Executive Director Denis Horgan.

The broad topic and its many elements is the subject of continuous engagement by the Alliance, and will be highlighted during its upcoming 7th annual Presidency Conference in Brussels on 8-9 April.

This event, during the auspices of the Romanian Presidency of the EU, comes on the back of a hugely successful Congress held in Milan at the end of 2018, which covered many of the issues.

EAPM notes that Manfred Weber, the European People’s Party’s lead candidate for the upcoming European elections, this week urged the EU to work together on what he called “an ambitious approach on medicine research”.

This was a part of his unveiling of a masterplan to fight cancer, which included him saying that “Nobody thinks that one single country can win the fight” against the disease.

The EPP’s Weber added that experts and researchers have told him that “if we combine our money and resources, we actually can cure cancer”.

EAPM recently published an article on the lead candidate, or ‘Spitzenkandidaten’. Možete ga pronaći ovdje.

As Weber suggests, when it comes to research in Europe, there is a clear need for more collaboration. As a case in point, the Alliance played a key role in the creation of the MEGA initiative, which was a topic of discussion at a forum on research innovation this week (more of the workshop below).

The Million European Genomes Alliance (MEGA) has so-far been signed by 19 member states and its foundation was based on the need to bring together a coalition of EU countries in order to cooperate in bringing innovation into health care systems.

Up until now, there has been something of a piecemeal approach across Europe in various slices of legislation, such as those covering clinical trials, IVDs and more.

Europe needs to be more proactive when looking at how best to bring innovation into health-care systems, not least when it comes to interoperability, but there are many other areas.

In fact questions have been asked, also this week, about the effectiveness of the general data protection regulation (GDPR), with EMA chief Guido Rasi surprising many by saying that he is not sure that the digital revolution and the regulatory environment are compatible.

There is a need for clarity “immediately” on two points, he said, citing secondary data use for health research and asking who is responsible if someone manages to identify data anonymized in good faith.

These are valid points and EAPM is also concerned that member states may implement GDPR’s provisions in different ways, which above and beyond the goal of a cohort of a million EU genomes, means that the ‘MEGA-style’ of a truly cooperative approach is key going forward.

STOA Workshop on health-care research

As mentioned, Thursday 10 January saw a workshop run by the European Parliament’s Science and Technology Options Assessment (STOA) Panel on the topic of innovative solutions for research in healthcare.

The discussion was based around a manifesto supported by the European Alliance for Personalised Medicine.

The high-level forum on Thursday (10 January) discussed ideas around developing a novel approach to deliver better precision medicine in Europe and, among others, was attended by Belgian Health Minister Maggie De Block and head of EORTC, Denis Lacombe.

EORTC was a key driver behind the gathering and takes the lead role in EAPM’s Working Group on Regulatory Affairs.

At the meeting, Paul Rübig MEP, who is STOA Vice Chair, set the ball rolling by saying that data plays an important role in healthcare, especially in cross-border healthcare situations. The use of data offers an opportunity to save lives and to know which kind of medicines work together.

Rübig’s view is that the establishment of a new framework between industry, patients, governments and other stakeholders could mitigate the somewhat problematic current situation.

Belgium’s health minister, who has previously taken part in EAPM events, said that she wants to offer access to medicines to all patients as soon as possible – pointing to an agreement signed in 2014 with the pharmaceutical industry to make clinically relevant therapies available to patients in a sustainable way.

The workshop also heard that gene therapies are able to improve the lives of patients significantly.

On the topic of modern concepts in health care in the 21st century, participants heard that there are very effective innovative new drugs which have been approved on the basis of a very limited number of patients, but they are used in a large number of patients for long periods of time afterwards.

More real-world data and evidence is needed.

Some challenges ahead…

The workshop heard that, in terms of challenges of new technologies in the research and societal environment, for researchers these lie mainly in the area of bio informatics solutions, benchmarking technologies and data interpretation. The situation is complex for researchers as well as for those bringing medicines to market, with the latter being challenged by new treatment approvals and off-label use. These issues go on to create challenges for pricing, health technology assessment and new therapeutic guidelines.

Regulatory trials which aim to document new drugs are certainly needed, but as downsides their primary end-points are frequently purely drug centred, and are based on heavily selected populations.

The control arm may not, in fact, represent real practice, leading to the possibility of poor external validity, which doesn’t adequately serve day-to-day patients and doctors. Today’s clinical research looks at optimal patient populations, drug combinations and sequences, and duration of treatment, but a way needs to be found to re-engineer how to work together.

The workshop heard about an EMA study which showed that, out of 48 cancer medicines approved between 2009 and 2013, only just over one-third showed a prolongation of survival.

Attendees were told that the critical gap to be addressed, at the European level, is to understand how to move from drug-centred research to patient- and society-centred research, while also ensuring the interests of all stakeholders.

The European Federation of Pharmaceutical Industries and Associations (EFPIA) was represented at the workshop, and said that they were inspired by the manifesto, especially the ideas on how to create a collaborative research space to make a quality change.

To make a step change, attendees were told, there is a need to bring together stakeholders that are not used to working together. The health arena is unique, and there are conflicts of interest that may arise plus the need to maintain a balance between access and innovation.

A neutral broker is extremely important, and resulted in the creation of the Innovative Medicines Initiative.

The workshop heard about the need to break down silos in developmental phases and between research and care, with the latter two needing to be brought closer together.

Also, in the development and registration process there are uncertainties. It is necessary to have spaces in which patients and researchers can come together to understand how to deal with these.

IMI has a project called ‘Big Data for Better Outcomes’, which seeks to define outcomes that are patient-centred, but also relevant for HTA bodies, doctors and researchers. This relies on data from all areas.

Genomics, imaging and patient stratification

The workshop heard that genome sequencing used to cost €10 million, as much as the most expensive house in London at the time, but now it would cost less than an Arsenal season ticket. Many people can now have their genomes sequenced, should they wish to.

Big changes he also occurred in imaging. Applying these technologies can be done in research and in practicing medicine, although the two areas are quite different. But they are both relevant in terms of patient stratification.

In clinical practice, stratification can help with better diagnosis and prognosis, better use of medicines, such as in respect of personalised medicine, and with specific care pathways optimised for individual cases.

In medicine discovery, stratification can bring more clarity regarding therapeutic goals in early development and make clinical trials less costly and more likely to succeed in phases II and III.

The workshop heard that for the best stratification, four pillars are required. These are at-scale genomic assays, a clear legal basis to access appropriate data and approach patients, a very large virtual cohort, ideally with population scale ascertainment, and harmonised representation of key aspects of electronic health records (EHRs).

Attendees learned that Europe has the largest cohort of EHR records in the world, with some of the most advanced clinical and population genomics programmes globally. Europe’s formative one-million genomes project, known as MEGA, was highlighted and the workshop heard that the target will easily be surpassed.

Disruptive medicines and HTA

The workshop heard that highly innovative, potentially curative medicines require a re-definition of value. Emerging business models are also driving the need to improve cooperation between competent authorities at national and regional level on key elements of drug-pricing decisions.

There is a paradigm change on the way as healthcare moves from treatment to potential cure and prevention, from anatomical to molecular, from drug prescription to therapy delivery, from risk/benefit to clinical added value, and from approval to access.

This shift will all require changes on the part of researchers, developers, patients and doctors.

Meanwhile, on health technology assessment, the workshop heard that it is important for HTA bodies to assess what happens to patients after treatment and, as such, real world data is vital.

Traditionally there has been the initial HTA to assess market authorization, but there should also be a comparative or full HTA later, not only for reimbursement purposes but also to support appropriate use.

New HTA methods are needed to support internationalization, and for adapting to a new era of personalised medicine.

Pacijenti

Attendees heard that if health care works to address the real needs of patients in the development of medicines, it decreases the risk for all parties.

The development of and access to medicine is a sequential process, with the sequence meant to bring efficient products to the market. It is not optimal and could be better, but it works. However, there is a conundrum between access versus evidence and the fundamental question is “What is enough evidence?”

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Kategorija: Naslovna stranica, EU, Europski savez za Personalizirana medicina, Zdravlje, Personalizirana medicina

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