#EAPM: Balancing societal needs and legislation in modern-day health care

Health care across the EU has never been more expensive, nor has the need for it to be more efficient been greater, writes European Alliance for Personalised Medicine (EAPM) Executive Director Denis Horgan.

With money tight and an ageing population suffering from ever-increasing co-morbidities, only innovation geared towards new forms of medicines and treatments, coupled with a smarter use of limited resources and greater stakeholder engagement, can improve the health outlook of 500 million citizens across 28 member states.

Europe as a whole clearly has its work cut out, as do individual EU countries and even the different regions within these countries.   The Brussels-based EAPM is a broad stakeholder organization made up of patients, researchers, scientists, academics and health-care professionals, plus law- and policy-makers. It tasks its members with finding ways to deliver the right treatment to the right patient at the right time and cites access, among others, as a major issue.Analysis has shown that there are many and varied reasons to explain why patient access is being delayed, blocked and made inequitable. In short, the ’system’ of getting efficacious medicine and treatments affordably to those who need them across Member States is currently less than ideal.

From a societal point of view, modern treatment should be about putting patients at the centre of their own health-related decisions as well as allowing and facilitating innovation, through investment in research and workable reimbursement policies at the pan-European level.   Today’s patients are more-and-more often demanding to be included in decision making that will affect their own health and will have an impact on their families. Better-trained health-care professionals (HCPs) are certainly needed, as is a higher degree of health literacy across the board. And that includes legislators working in the health arena.

On top of this, a question that needs to be answered urgently is how does the EU develop representative policies that take account of democracy and social welfare models while also allowing innovation in the healthcare area?

Well, given that one of the basic tenets of the European Union is equality among all citizens and the right to healthcare when needed, it is clear that there is a gap between aspiration and actual delivery. Legislators need to look and listen more.

For example, the bottlenecks in collection, storage and dissemination of what has now come to be known as ‘Big Data’ is something that regulators need to address if all the incredible information available today is to be put to the best use for patients in particular and society as a whole.

It is clear that patients, researchers and industry all need information. And there’s no doubt that there are ever-more new ways of collecting it. But to put patients at the heart of the Big Data phenomenon, individuals, and especially those who share all that very private data about their health, should be in control of their own information, become empowered through it, and use it to help themselves and others when it comes to health.

Obvious ways spring to mind – the sharing of clinical-trial data across borders due to many more rare diseases now being identified with, by definition, smaller patient groups, for example, and the usage of data that they have chosen for the benefit of research and development. This vital research would not be stymied by over-cautious legislation.

The World Health Organization (WHO) has stated that: “There is poor availability and quality of data on actual demand; inadequate management practices in procurement and the supply chain, combined with large tender contracts that do not sufficiently define quality standards but whose sole emphasis is on obtaining the lowest prices; and too small profit margins for manufacturers – all these factors may lead to shortages.”

What is clear according to EAPM and its stakeholders is that the EU’s institutions, notably the Commission, need to act quickly and effectively to ensure that patients are not suffering unnecessarily due to a lack of access to a particular medicine and diagnostics.   Pharmaceutical companies are often blamed for medicines deemed unaffordable or lacking in sufficient value to be added to lists of available medicines in health-care systems across Europe, but there is an argument that, because of the decade-and-more that it takes to get a medicine from bench-to-bedside (trials, HTA approval etc) and the high costs of doing so, more incentives are required to stimulate research and development.

A further argument is that any concept of ‘value’ should be defined by the customer, in this case the patient. And that’s not just a societal nicety but can mean the difference between life and death.   As mentioned earlier, EU citizens have been told to expect equal access to healthcare. But it is not happening, and the cross-border healthcare scheme, for example, while well-intentioned, falls down in many countries often because of the reimbursement systems.

If a particular treatment cannot be found in, say, a smaller, less well-off country then a patient could go to a richer one for the right treatment. Wonderful in theory. But how then does the poorer country reimburse at the price levels charged by the richer one?

The Alliance has always argued that new medicines have a positive contribution within society, providing effective treatments, but proponents of personalised medicine are not simply calling for access to all new medicines. This has to be balanced against the efficacy of older, cheaper medcines that have been proven to do a good job.   Legislation and authorisation has worked to a degree, for example in rare diseases, although the European Commission has stated that while “the number of products authorised has grown over the years, (this) remains limited bearing in mind the existence of 5,000-8,000 distinct rare diseases.”

The Commission added that: “just 1% of these (orphan medicine) are currently covered by authorised medicinal products in the EU. The incentives of the orphan drug legislation are therefore essential to facilitate pharmaceutical development.”

EAPM has asked the EU executive to take a long, hard look at the regulatory framework dealing with orphan medicines in order to speed up access to such medicine for the many patients across the EU that desperately need them.

Meanwhile, generally speaking, closing the education gap through multi-player interaction will surely help, but every stakeholder - including the legislators - needs to get up-to-speed quickly to make the most of today’s fast-moving medical sciences.