#EAPM: The need for early advice (and more) on novel medicines and treatments

In the fast-moving world of personalised medicine there are many issues and barriers - not least when it comes to getting novel drugs and treatments swiftly to where they are really needed, writes European Alliance for Personalised Medicine (EAPM) Executive Director Denis Horgan.|

Slow bench-to-bedside rates do not help the 500 million potential patients across the EU’s current 28 member states, and the timings are affected by several elements during the development and licensing phases.

As we all know, personalised medicine starts with the patient. It holds huge potential for improving the health of many of them and ensuring better outcomes of health systems' efficiency and transparency.

If personalised medicine is to be in line with the EU and member state principle of universal and equal access to high quality healthcare, then clearly it must be made available to many more citizens than it is now - citizens who are becoming more knowledgable and therefore powerful when it comes to engaging in dialogue with health-care professionals and making decisions about their own treatment.

A major pillar in bringing novel medicines to patients is, of course, innovation. This, in the realm of health, means the translation of knowledge and insight into what we can call ‘value’. And that value covers the value to patients but also has to take into account value to healthcare systems, society, the taxpayers and, of course, the manufacturers.

The technology now exists to apply the personalised medicine, and all of this is built upon thorough research. Which then needs to be translated via various process on the way to market, not least of which are regulatory approval and pricing.

But there is also a necessity for up-to-date incentives and rewards in research, yet this is so far failing to happen.

Across the Atlantic, former US president Barack Obama famously brought in his Precision Medicine Initiative with committed initial funding of $215 million. The intention is to build a research cohort of at least a million Americans, incorporating information from genomic analysis as well as clinical information and integrate this into routine health care.

It was a bold, but necessary move, and Europe is now in danger of falling behind. In this post-Brexit vote world, with the EU seeking a new focus, the healthcare of its citizenry down the line would seem to be a good point for future emphasis.

OK, generally speaking, the EU has recognized that innovations in healthcare can contribute to the health and well-being of citizens and patients through access to innovative products, services and treatments that have added value.

It is also aware that in order to stimulate development, there is a need to facilitate the translation of scientific advances into innovative medicinal products that meet regulatory standards, accelerate patients’ access to innovative therapies with added value for patients and are affordable to the member states’ health systems.

And it is clear that early dialogue between technology developers, regulatory, health technology assessment and, where relevant, pricing bodies will promote innovation and quicker access to medicines at affordable prices, to the benefit of patients.

There is an obvious need for researchers and pharmaceutical companies, as early as possible in what can be a very long process, to receive suitable and accurate advice.

This should ideally be forthcoming from regulators, payers, HTA bodies and, of course, the patients.

On a purely cost issue, if a drug ‘fails’ this can be extremely expensive. Therefore, the stakeholders already mentioned can keep such a risk as low as possible by giving early input.

Not only that, but these stakeholders can help to bring about what are vital evidence-based assessments.

Unfortunately (and not a little surprisingly), there are too few such forums that allow this necessary inter-stakeholder dialogue with the result that the people who need the fastest results most, the patients, end up losing out.

With all the amazing new medical science now at Europe’s fingertips, such a situation should not be acceptable in the 21st century.

Fortunately, at least one suitable forum does exist and the European Alliance for Personalised Medicine is working towards the first ever pan-European, multidisciplinary Congress specific to this fast-moving field.

The 28-30 November 2017 event is entitled ‘Personalising Your Health: A Global Imperative!’. It will be held in the capital of Northern Ireland in partnership with Queen’s University Belfast and Visit Belfast.

The over-arching idea for the event is that it will act as a one-stop shop for everything connected with personalised medicine, and will also take on board conclusions emerging from this month’s EAPM conference to be held in Brussels.

It will allow for cross-fertilization between the different disease and policy areas, allow delegates to gain a greater depth of knowledge into barriers in the field of personalised medicine,  and offer up valuable evidence and stakeholder opinion on which policy makers can base their decision making on how better to integrate personalised medicine into the EU’s healthcare services.

The Belfast event will provide the biggest ‘space’ to date to allow for such a meeting of minds and expertise.

Personalised medicine clearly has the promise of seeing healthcare move away from ‘trial-and-error’ therapies to evidence-based individual ones.

It is an approach that removes the ‘one-size-fits-all’ philosophy and instead sees sub-groups of good and bad responders identified and stratified before treatment on a patient begins.

Politicians will tell you that, if you ask any citizen, health and health care will be high on the agenda – and as we live longer that will surely become more the case, rather than less.

Indeed, the Luxembourg Presidency published its Council Conclusions on personalised medicine in December 2015. During this process it became clear that new approaches are needed in the following areas:

• Research

The undeniable challenge is how best to translate new knowledge and expertise into medical advances that improve outcomes and enhance well-being for European patients.

• Regulation

The development of personalised medicine will require regulatory bodies to adopt new approaches to product approval.

• Incentives

Innovative models and methods are needed to calculate the benefit and risk factors of medicines. Drugs and biomarkers need to be approved through effectiveness and benefit to society.

Currently, there is a lack of incentives for investing in the developing of diagnostics which work hand-in-hand with medicines. Coordinated timing when it comes to reimbursement and approval of a companion diagnostic is essential. Without this, there will be potentially life-threatening delays when it comes to patient access

• Budgets and performance

New drugs are becoming unaffordable. Therefore, much-better economic evaluation is needed in order to provide decision-makers with a theoretical framework to inform resource allocation.

EAPM’s Co-Chairman Gordon McVie, the founder of eCancer, has recently worked with Pfizer to produce an exceptionally useful video on health economics, which you can view here.

• Making better use of data

There is so much information out there now, but the gathering, storage, and sharing of health-related information can be a legal and ethical (not to mention practical) minefield.

Clearly it is incumbent upon all stakeholders in the realm of healthcare – and especially the policymakers and legislators – to ensure that every citizen of Europe has the same rights and access to the same high quality care as his neighbour.

At the moment we have different standards of health care in different countries, different price structures in many of them, and problems in affordability when it comes to cross-border access for patients.

Despite health remaining a member state competence, the individual healthcare systems can play a vital role in offering affordable disease prevention, diagnosis and treatment.

Now is the time to make health care more personalised, more preventive, and more cost effective, and the technology, data and more are there already. What is needed most now is the will.