Access all areas: Taking STEPs towards patient-centric health care

Personalised medicine (PM) is all about giving the right treatment to the right patient at the right time and is a sure-fire way towards a healthier Europe. But it comes with many challenges across all sectors operating in the health care field.

Industry and science, for example, need more collaboration, better interoperability, greater access to quality data, a rethought reimbursement process, more cash for research...the list goes on.

Meanwhile, doctors and nurses in the front line need more education about, for example, rare diseases and the treatments available.

For the patients, however, it’s all about access. Access to treatment and medicines, access to clinical trials, access to more (and clearer) information, access to decision-makers and legislators for the chance to get their voices heard and their needs understood from the top-level down.

The European Alliance for Personalised Medicine (EAPM) puts patients at the centre of its recently launched STEPs campaign.

STEPs stands for Specialised Treatment for Europe’s Patients and, among other things, EAPM’s campaign aims to highlight, to current and potential MEPs plus the European Commission, the possibilities surrounding PM and the advantages for their constituents in the run-up to this year’s European elections.

And, let’s be clear, this affects every country in Europe. While patients in member states with lower incomes face even bigger obstacles to all of the above, these problems also exist in the richer states. They are essentially pan-European issues.

Mary Baker MBE is immediate past president of the European Brain Council (EBC) which exists to promote brain research and improve the quality of life for those suffering from brain diseases.

She emphasizes what we all know: that society has changed, the population has grown and people now live longer. This has come with inevitable problems.

"The regulatory system is not fit for purpose anymore,’ says Mary. ‘It’s nobody’s fault, just the way things have developed. The whole health profile of Europe is different and adapting to that is certainly a new challenge."

So how do patient groups see the challenge?

Dr Stanimir Hasardzhiev is one of the founders (and current chairperson) of the National Patients’ Organisation of Bulgaria – the biggest patients’ umbrella organisation in the country with 85 disease-specific member groups.

Alongside his other patient advocacy work, in 2013 Stanimir became a board member of the European Patients’ Forum which, among other activities,  represents specific chronic disease groups at EU level.

‘Access’ is the key word. As Stanimir says: "The issues facing us, especially in this economic crisis, are access to healthcare and medicines, access to information, to screening and to the right treatments at the right time. Patients also need equal access to clinical trials and, from there, innovation."

With a new European Commission and Parliament on the horizon, Stanimir is clear about what he wants: "Access for patients to treatment and medicines should be among the highest priorities.  We need all institutions and stakeholders to sit around one table to find solutions to improve life for those most in need across Europe."

So what can patients do? Well, it depends. Ewa Borek, of the WE PATIENTS Foundation in Poland, says there’s a problem with empowerment for patients in her country.

"Patients in Poland don’t realise how powerful they can be. In many other EU countries they grasp this but Poland has only had democracy for 25 years. Often they don’t believe they belong to the NGO sector which is relatively new but has exploded in the ten years since accession."

But Warsaw-based Ewa adds that it is not just that. ‘Poland is low on the European health index with long waiting lists and so on. But politicians here are consistent in avoiding change and reforms.’

Stanimir echoes the sentiment that certain governments are resistant to change. He said: "In Bulgaria no new medicine can be registered for reimbursement through the system until the medicine has started reimbursement in five other countries in Europe. They say ‘we are a small country and we have to wait’ but that is an excuse."

However, in some ways governments’ hands are tied: "Then we move to the pricing procedure," addedStanimir. "This is the reference pricing system that operates across Europe - and this is another issue that prevents access.

"Bulgaria has to pay exactly the same price for medicines as other parts of Europe. But because countries’ prices are referenced to others this means that, if Bulgaria or Hungary were to have a lower price for a specific medicine, then this price would almost immediately appear in, for example, Spain, and then from Spain, the price would drop in another country with a domino effect.

"So, basically, no pharmaceutical companies can afford to have this happen, even if they were willing to offer some special prices for countries with lower GDPs. Also, if there was a lower price in Bulgaria, every country would start buying the lower-priced drugs because of the free-trade system in the EU."

Viorica Cursaru, of Myeloma Euronet Romania, works for the rights of patients suffering rare blood cancer with a low life expectancy after diagnosis of between 30 months and five years.

Like Bulgaria, Romania is a relatively low-income state and Viorica said: "There should be no first- and second-class citizens when it comes to healthcare. We need a minimum standard of care across the whole EU, a standard everywhere below which we cannot fall.

"When it comes to cross-border treatments for patients, it is prevented to a certain extent by the reimbursement policy. If the treatment is lower-priced in Romania but you need to go elsewhere for quicker or better treatment, which costs more, you will only get reimbursed at the Romanian rate. So that could be 1,000 euro, but you have to pay the difference on a treatment that could cost 5,000 euro if you went to, say, Germany."

Adds Stanimir: "One-size-fits-all pricing doesn’t work in smaller countries. Basically, we can’t afford the drugs. Change has to happen at EU level."

This is clearly a complicated issue, which Stanimir confirms: "The solution is not simple. It requires the European institutions and member states to find a way to create a fairer system. There has been a debate for several years and, under the Belgian Presidency in 2010, it was already being flagged that the current system may prevent access to patients in low-income countries."

"This," he insists, "will happen only with commitment and real dialogue in the spirit of the solidarity of the European Union."

So what about access to information and clinical trials? Šarūnas Narbutas, president of Lithuanian Cancer Patient Coalition, says: "When it comes to oncology, clinical information for the patient has been almost non-existent in Lithuania. There is a huge communications gap.

"To combat this we have been working with other groups to produce literature which is reviewed here by oncologists and the patients."

Ingrid Kossler, of the Swedish Breast Cancer Association, echoed this, saying: "In Sweden there is perhaps a lack of convincing information. Take breast cancer – women have a choice between mastectomy and breast-conserving treatment. Many needlessly choose mastectomy because of a fear of cancer. They think that if they have been diagnosed with breast cancer and don’t have a mastectomy, they will die next Monday."

In a rich country such as Sweden, at least patient access to clinical trials should be pretty-well covered? Well, no. Ingrid adds that because of the sparse population in certain parts of her country, access to clinical trials can be a big problem.

"Because of Sweden’s regional structure, patients may not be informed about them. The information should come from doctors and be available on the internet but it doesn’t happen as there is a lack of time and specialised knowledge. Add to this the fact that most trials are held in Stockholm so are difficult for many to get to."

Back in another richer EU country, Jayne Bressington is Patient Director for the PAWS-GIST national alliance and a trustee of GIST Support UK.

GIST stands for gastrointestinal stromal tumour, which is a type of sarcoma found in the digestive system, most often in the wall of the stomach. While generally rare it’s even less common in the under-25s and the group’s stated aim is to find the cure for these rare GIST cancers in young people, which react differently to drugs already developed and used successfully for adult sporadic GISTs.

Jayne said: "Smaller national patient groups should join with others on an international scale and the best existing treatment should be made available to all patients regardless of country.

"And given that we’re talking about relatively small numbers when it comes to rare cancers, we also need clinical trials on an international scale to maximise the amount of patients involved.

"On top of that, we need to educate front-line doctors so that they can recognise rare cancers earlier, grow cross-border cooperation and an infrastructure that supports translational research, plus allow tumour banks to benefit from easy transfer to aid that research."

That sounds fair enough, but what about all that data required for cutting-edge research? Are patients comfortable with sharing this kind of personal information? Back to Mary Baker, of EBC, who said: "Patients often don’t want to hand over sensitive personal information which could be vital for research."

She cited a historical example for this: "Remember that ethical committees grew out of (the) Nuremberg (trials), after the evidence of DNA prejudice, and some patients have a fear of stigma. Others fear that, say, insurance companies will misuse their information."

"These are ethical and humanity issues," Mary added, "and if you leave ethics and humanity out of medicine then you have a problem. We need to be able to communicate with patients to explain the benefits. There needs to be a debate in society and that is currently lacking."

"Nevertheless," she concluded, "there is tremendous hope hanging on personalised medicine and we need to communicate this to society. There’s some great science out there, but we need to build a motorway between the various disciplines – and include patients at all levels."

No patient organization, in whatever member state, would argue with that.

The author, Tony Mallett, is a Brussels-based freelance journalist.
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