Time to 'STAMP' out lack of access to new treatments

By European Alliance for Personalised Medicine (EAPM) Executive Director Denis Horgan

More and more patients’ groups and individual citizens are becoming aware of the potential of personalised medicine, with its ability to give them the right treatment at the right time.

They want empowerment, they want to have their illnesses and the 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.

In a 500 million citizen-strong EU of 28 member states with an aging population that will inevitably become ill at some stage, giving patients access to the best possible treatment available in Europe is a massive issue.  Indeed, the importance of access to medicines and innovative treatments is undergoing particular scrutiny at the moment and is currently a big issue in the European institutions.  Representing the Latvian Presidency of the EU at a recent Parliamentary plenary session, Zanda Kalniņa-Lukaševica, President-in-Office of the Council, stated that “access for patients to medicines that efficiently treat illness is an important issue that must be addressed both at national and EU level”.

”It involves several aspects”, she added, “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, health-care providers and governments can afford the products; and lastly, ensuring quality so that the medicinal products work as intended and are efficient and safe.”

At the same plenary, MEP Cristian-Silviu Bușoi explained that 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.

Other issues include overly bureaucratic reimbursement procedures and a lack of implementation of the Cross-Border Health Care directive.  Bușoi believes that EU policymakers should ensure that regulatory decisions on the value of innovative therapies are based on what matters most to patients, and ensure that they get access to innovative treatments following a centralized cost-benefit analysis by the European Medicines Agency.

The minister, Kalniņa-Lukaševica, highlighted the creation of the Expert Group on Safe and Timely Access to Medicines for Patients, otherwise known as STAMP, which began its work in January with the next meeting scheduled to take place at the beginning of May.

According to the Commission, the STAMP expert group has been set up to provide advice and expertise to Commission services in relation to the implementation of European Union pharmaceutical legislation, as well as programmes and policies in the field.  STAMP is intended to “exchange views and information about the experience of member states, examine national initiatives and identify ways to use more effectively the existing EU regulatory tools with the aim to further improve safe and timely access and availability of medicines for patients”.

A further goal is to explore ways to increase information-sharing and cooperation among member states.  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.  There are many issues surrounding early, and timely, access and, during the first meeting of STAMP, several member states gave presentations, including Belgium, France and Spain, offering their perspectives on how to improve it, especially in certain cases.  The Belgian presentation highlighted that there is a need to find a balance between addressing unmet medical need while providing complete information on benefits, risks and relative effectiveness.

There was also a focus put on new national legislation regarding early temporary authorization (known as ETA) with a possible link towards early temporary reimbursement.  In Belgium, there is leeway for the provision of medicinal products that are not yet authorized, to patients with a chronically or seriously debilitating disease or whose disease is considered to be life threatening, and who cannot be treated satisfactorily by an authorized medicinal product. This is enabled by ‘Compassionate Use Programmes’.

Meanwhile, products authorized in Belgium (but not yet licensed for critical indication), or licensed but not commercially available yet, can be given to patients via Medical Need programmes.

Spain’s presentation also covered Compassionate Use and its possible implementation in cases where patients would benefit from accessing the medicine and when any delay would mean a lost opportunity. Also, where no alternative is available, or where alternatives have been previously tried but failed.  Spain highlighted the use of Compassionate Use programmes in severe clinical situations, in a hospital setting only while not hindering medical research - as far as possible, a clinical trial should be considered.

France, meanwhile, highlighted the TAU scheme - Temporary Authorization for Use – in the case of a drug fitting an unmet medical need, where there is no option for patient enrollment into clinical trials and where the drug is intended for treatment, prevention or diagnosis of a rare or serious disease with no satisfactory alternative available.  So far, 240 drugs have been used for individual TAUs, with 20,000 patients currently under TAU programmes.

Of course, in all of the above circumstances the pricing is an issue and is being handled in differing ways. And it is plain to see that, currently, there is a huge difference in how health-care systems handle funding across the EU. It’s a massive issue and is being closely watched by advocates of, and stakeholders in, personalised medicine.

The Brussels-based European Alliance for Personalised (EAPM) is a key player, having a multi-stakeholder membership that includes patients, clinicians, academics, industry representatives and health-care professionals.

The Alliance regularly meets with Members of the European Parliament and the Commission to discuss many of the goals of, and barriers to, personalised medicine.

As well as its Working Group on Early Access and Better Decision Making, it also hosts an MEPs’ Interest Group. The Alliance has been in continuous dialogue with Members since its formation, on access and many other topics.

EAPM notes that among the basic tenets of the EU are equality and access to the best health care for all, regardless of who or where they are.  Yet it is clear that there are large organizational, fiscal, clinical, and practical barriers to the full introduction of personalized medicine into a single European health-care system, and many more when it comes to implementing it on a pan-European scale.

The Alliance believes that what the European Commission needs to do, in tandem with the Parliament, is to create a regulatory environment which allows early patient access to novel medicines and treatments – and to do this as soon as possible.  A close scrutiny of, and changes to, the current system for incentives and reimbursement, right across Europe, may represent a very solid start.

Meanwhile, in an ideal world, the Commission’s new initiative will leave its 'STAMP' for the benefit of all Europe’s patients.

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