אַפפאָרדאַבלע געזונט זאָרג אין טעסטינג מאל: דער וועג פאָרויס

By EUROPEAN אַלליאַנסע פֿאַר פּערסאָנאַליסעד מעדיצין (עאַפּם) אויספֿיר דירעקטאָר דעניס האָרגאַן

With more and more researchers, front-line clinicians, pharmaceutical companies, patients’ groups and individual citizens becoming aware of the potential of personalised medicine, the question now is how to make the best use of its ability to offer the right treatment to the right patient at the right time in a way that is affordable.

Arguments continue to rage in a world of cost/benefit analyses but the fact remains that we live in a 500 million citizen-strong EU of 28 member states with an ageing population that will inevitably become ill at some stage.

Thus, the health-care burden is increasing all the time – and will continue to do so – and giving affordable access to the best possible treatments available in Europe is a huge issue and a massive challenge.

Modern patients want to have their illnesses and treatment options explained in a transparent, understandable manner (from a clinician with up-to-date knowledge) to allow them to become involved in co-decision, and, crucially, they want greater access to treatments that could improve their lives and, in some cases, save them.

The Brussels-based European Alliance for Personalised Medicine (EAPM) believes that treading the path to better and more-affordable access involves a greater understanding of value, more focus on new technologies, better use of resources, more collaboration in the medical community and markedly improved engagement with innovation.

Of course, EAPM stakeholders are not the only people talking about this. At a recent European Parliament plenary session, the representative of the Latvian Presidency of the EU, Zanda Kalniņa-Lukaševica, said that this issue “must be addressed both at national and EU level”.

אַדווערטייזמאַנט

She added: ”It involves several aspects, namely: availability – meaning that new medicines are developed or existing products are adapted; also accessibility – bringing the products to patients who need them. It is also about affordability – ensuring that patients, healthcare providers and governments can afford the products; and lastly, ensuring quality so that the medicinal products work as intended and are efficient and safe.”

Despite the existence of innovative new drugs, new technologies and developments in medical science, many citizens are not able to access them, often due to high costs, while further issues include overly bureaucratic reimbursement procedures in the EU and a lack of implementation of the Cross-Border Healthcare directive.

The reality is that to introduce new medicines requires considerable investment and time. There is clearly a need for more modern and realistic approaches to reimbursement and agreement over the use of so-called Big Data for vital research purposes, bearing in mind that it is incumbent on European lawmakers to protect citizens from unwanted and unnecessary exploitation in this regard.

There is also an obvious need for all medicines, as well as in-vitro diagnostic devices, to be proven to be safe and cost-effective if much-needed innovative and personalised medicines are to come onto the market.

The creation of a robust evidence base is vital for good decision making to ensure that resources are used for the maximum benefit of patients and this must take into account the views of different stakeholders as well as decision making in any given member state health system. Payers clearly need to trust that any evidence base is solid before decisions are made.

Pricing is clearly a major issue and is being handled in differing ways. And it is plain to see that, currently, there is a huge difference in how healthcare systems handle funding across the EU. It’s a massive issue and is being closely watched by advocates of, and stakeholders in, personalised medicine.

It is clear that there are large organizational, fiscal, clinical, and practical barriers to the full introduction of this radical and exciting way of treating patients into a single European healthcare system, and many more when it comes to implementing it on a pan-European scale.

Let us take an example regarding access to treatment and its cost: Cancer, a massive killer representing a set of diseases that are a major economic expenditure even in rich countries, is seeing not only an increase in numbers who are (and will) suffer from one form or another but also an increase in expenditure. This is due to, for instance, a lack of suitable clinical research, an out-of-date regulatory system, and a lack of evidence-based data.

Meanwhile, bearing in mind that aforementioned ageing population, plus fast-developing technology that needs to be implemented and ever-growing healthcare costs on a general level, it is no surprise that these are reflected in the rising costs of treating cancer. More generally, fifty years ago, average health-care spending represented 5% of GDP. In five years time, that will be around 20% and, of course, that across-the-board increase is reflected in the costs of treating cancer patients.

Worldwide, roughly 12 million new cancer patients are diagnosed each year with more than 7.5 million dying in the same period. Had they not developed a cancer many of these patients would otherwise have lived healthy – and productive – lives for years to come. Prevention and better treatment, therefore, surely have an inestimable value.

A proper perception of value is certainly among the variety of solutions to the affordable access problem, especially when it comes to personalised medicine. To understand ‘value’ one must first understand a product and consider what it can provide, weighed against cost and other considerations. For example, in vitro diagnostics, or IVD, tests give vital information to a medical professional regarding the likelihood of a patient responding to, or benefiting from, a specific treatment.

Not only that, but value-wise there are questions such as whether an intervention that is proven to, say, shrink a tumour but brings no overall improvement in chances of survival actually represents ‘value’. Who decides?

Clearly, earlier diagnostics and earlier treatment have many benefits, among them fiscal, because while, yes, there are key questions about the cost-effectiveness of new and even existing treatments, better diagnostics will help to ease the burden on health-care systems.

Also, cost-wise, given that despite access problems for many, it is a fact that more patients are being treated for multiple diseases as they age and, with the advent of personalised medicine, are being treated in different ways. This clearly drives up spending to a large degree.

Much of this extra cost involves screening and diagnostics, plus actual treatment. Follow up measures also eat up funds and there is a need to acknowledge that extra diagnostic studies are often required in the support phase of treatment.

And while innovation and new technologies clearly have the potential to be highly beneficial, there is a huge cost aspect in developing, testing, certifying, marketing and implementing new treatments and drugs - not only in cancer, of course, but in respect of all innovative measures for all disease areas.

It is also a fact that, in general, healthcare systems are spending more because they are in a position, through innovation, to help more patients in better ways. As a simple example, four decades ago in the United Kingdom there were 35 oncology drugs approved. This has almost trebled to around 100 today.

And, of course, the better we get at diagnosing the various forms of cancer, then the higher the number of cases rise. And we are getting much better, not just at diagnosis but at prognosis and individualized therapies. At lot of this has to do with imaging technology which, again, is driving up costs substantially.

Meanwhile, over-utilization is another large factor when it comes to the costs of cancer care in the 21^st century. There are still too many instances of healthcare systems using a one-size-fits-all model and, thus, treating patients who in many cases do not need that particular treatment, will fail to respond to it and/or will suffer side-effects that could have been avoided if another treatment were available.

And, if another, better treatment actually is available, the doctor may not know of it and by him or her prescribing a ‘population-based’ treatment (which certainly saves time in the short term, at least), we will have an over-utilization scenario once again. All of which makes an argument for the up-to-date and continuous training of healthcare professionals – not cheap, either, but certainly necessary in such fast-moving times.

Interaction with the patient is of vital importance, too, when it comes to cost as a large amount of spending on cancer-care is packed into the last weeks and months of a patient’s life. It is difficult to assess value but, in many cases, the spending achieves no tangible results and often ignores what would be the wishes of a well-informed patient.

Aside from the moral issues involved and improvements in care, EAPM strongly believes that the empowerment of patients in these, and all other, cases would also lead to a cut in overall costs. Patients cannot be empowered, however, unless their physician is likewise empowered to discuss all the options and cut out unnecessarily expensive – and counter-productive. Again, we are back to the education of health-care professionals.

So how do we start to make access affordable? There are many ways and among them is further investment in research and new technologies. The personalised medicine approach relies, in large part, on genetic information (although the lifestyle of a patient also helps to determine a final treatment choice). The cost of gene sequencing is falling all the time but, with member state health-care systems - and disciplines within these very systems - sticking to a silo mentality and failing to collaborate, a lot of vital research information is being duplicated and remains unshared.

Quite aside from the issues surrounding the collection, storage, dissemination and ethics of Big Data, this lack of coordination is in-and-of-itself obviously highly inefficient, and extremely costly.

Big Data is not going to go away, nor will science cease progressing. The key to improving affordable access lies partly in building more effective ways in which member states and their healthcare systems can engage and exploit new technologies and fully embrace innovation.

This cannot be done effectively in one country alone. There is a need for a pan-European approach, notwithstanding the fact that each of the 28 member states has competency for its own health issues. While the EU has introduced many laws and regulations in the product and standards arena, Member State healthcare per se is not an EU competency.

However, lest we forget, one of the basic tenets of the European Union is equality – and, thus, the concept of equal access for all to the best medicines and treatments is an issue that the EU has embraced on a moral level. Currently, this is far from the case and – in the 21st century and beyond - unfair access to affordable and effective care cannot be justified, not can it be tolerated.

Yet the issues of value and cost-effectiveness remain and the arguments will rumble on. Unfortunately, that means patients are suffering needlessly and, in many cases, dying long before their time while valuable cash is being wasted on many one-size-fits-all methods that quite plainly do not work for everyone and, indeed, often let down a sizeable number.

It is time for a new approach and EAPM and its stakeholders firmly believe that personalised medicine represents the way forward, through an emphasis on reassessment of ‘value’, the embracing of – and engagement with - new technologies, a proper EU-wide foundation for the training of health-care professionals, a new clinical trial model that acknowledges the relatively smaller disease groups, and increased collaboration at all levels. That would represent an excellent start.

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