A ten-year success story on tackling rare cancers

Written by Martin Banks & Brian Johnson on 11 October 2018 in Event Coverage
Event Coverage

EU action on rare cancers has soared over the last decade, thanks to a collaborative and unique multi-stakeholder approach.

Photo credit: François de Ribaucourt


EU action on rare cancers has soared over the last decade, thanks to a collaborative and unique multi-stakeholder approach.

This was one of the key messages to emerge from a recent European Parliament event addressing rare cancers, jointly organised by Rare Cancers Europe (RCE), the European Society for Medical Oncology (ESMO) and The Parliament Magazine.

The debate on 25 September centred on the challenges facing the 24 European Reference Networks (ERNs) for rare diseases, including rare cancers, the implementation of the Cross-Border healthcare directive, issues around generating and exploiting evidence on rare cancers and discussions on the probable impact of the EU’s draft regulation on Health Technology Assessment (HTA).


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The event also provided an opportunity for participants to acknowledge the decade-long positive impact that the Rare Cancers Europe initiative has had on driving EU actions on rare cancers as well as to reflect on other milestones such as the creation of the Joint Action on Rare Cancers (JARC).

RCE was established by ESMO in 2008 in a bid to improve care for rare cancer patients and is dedicated to putting rare cancers on the European policy agenda. Its activities have led to advances in improving rare cancer diagnosis and care, including the creation of JARC and supporting the formation of ERNs on rare cancers, and have contributed to bringing together scattered specialist knowledge.

Opening the event, Slovenian EPP MEP Alojz Peterle said multi stakeholder cooperation, channelled through platforms such as RCE and his own MEPs Against Cancer (MAC) group were committed to ensuring that political attention is given to rare cancers. Rare cancers, like other rare diseases, pose particular challenges due to their low frequency.

This can include late or incorrect diagnosis; lack of access to appropriate therapies and clinical expertise; a limited number of clinical trials due to the small number of patients and lack of interest in the development of new therapies due to limitations in the market and few available registries.

"Rare Cancers Europe is living proof that we need to pull our strengths together to ensure that better and more appropriate care is given to patients affected by rare cancers” Alojz Peterle MEP

Peterle, himself a cancer survivor, said that too little was still known about initiatives such as the ERNs. ERNs, he suggested, were a “perfect example” of what a multi stakeholder approach to rare cancers can achieve, adding, “Nevertheless, there is still a lot to do for patients and we see the establishment of the ERNs as the starting line, not the finishing line.”

His comments were echoed by Kathy Oliver, a founding member of RCE who, like Peterle had direct experience of cancer. RCE, she said, had worked for ten years to put the issue of rare cancers “firmly on the European policy agenda” while also “identifying and promoting appropriate solutions.”

Several “key challenges” remain though, including issues linked to the implementation of the cross-border healthcare directive, the EU legislation which frames the conditions for EU patients to seek treatment in an EU member state other than their own.

Paolo Casali, a medical oncologist and the chair of RCE said that the significant progress on addressing rare cancers couldn’t all be attributed to the work of the initiative. “But something happened over the last 10 years, that’s a fact: RCE was founded in 2008, so of course we are celebrating.” From the very beginning the initiative was participative and inclusive, he explained.

“We worked out 39 recommendations, which in a sense became the founding principles for RCE. We are still following those 39 recommendations”. Back in 2008 an official, agreed definition on rare cancers didn’t even exist and so the Commission bracketed rare cancers with every other rare disease. However, this failed to take account of the fact that 22 percent of all new cancer cases are rare cancers.

“The Joint Actions suggested that the European Commission recognised that rare diseases and rare cancers actually existed and that’s a very important message and a huge step forward. It’s safe to say that RCE’s work over the last decade has been instrumental in this” Chair of Rare Cancers Europe, Paolo Casali

“So clearly we were talking about a substantial proportion of cancer cases. That was important and had a number of implications.” Building on this, RCE made the case for more of a focus on rare cancers. “It wasn’t clear what rare cancers meant, compared to other rare diseases, so the conception in 2011 of the ERNs was a real step forward.”

In 2015, the Commission launched the Joint Action on Rare Diseases, which was quickly followed in 2016 by the three-year Joint Action on Rare Cancers. “The Joint Actions suggested that the European Commission recognised that rare diseases and rare cancers actually existed and that’s a very important message and a huge step forward. It’s safe to say that RCE’s work over the last decade has been instrumental in this.”

Annika Nowak, a member of health and food safety commissioner Vytenis Andriukaitis’ cabinet, explained that EU member states are responsible for ensuring that the ERNs are integrated into national health systems. But integration of the systems is one of the most difficult points, with more needing to be done to improve patient pathways.

“This is about how patients’ cases are referred to the ERNs and also about what kind of changes are needed within national frameworks when it comes to prioritisation and reimbursement issues under future cross-border care.”

The long-term sustainability of the ERNs was also a major challenge, with Nowak warning the EU needed to make the best use of its financial resources to ensure that within the framework, the ERNs “benefit to the optimum”.

“We have to advance research into this field, but I believe initiatives like HTA can help maximise the EU’s added value” Lieve Wierinck MEP

Belgian ALDE group MEP Lieve Wierinck chaired the second panel on patient access to therapies. She told participants that she believes politicians like her have a “duty” to help improve the current plight facing rare cancer patients: “We have to advance research into this field, but I believe initiatives like HTA can help maximise the EU’s added value.”

Romanian EPP member Cristian- Silviu Busoi highlighted the potential impact of the EU draft regulation on HTA. This, he noted, aims to improve treatment options for rare cancers patients while helping safeguard the sustainability of healthcare systems: “HTA should be given a fair chance to become more structured, sustainable and efficient, better allowing for the optimal use of resources and sharing of expertise.”

Cancer patients, he said, face a “paradox,” adding, “for the first time in history there are many promising, effective life-saving treatments and yet patients don’t have access to them because of delays in the medicine’s reimbursement procedures or because they cannot afford them.” This, he said, leads to an “unacceptable” lack of essential life-saving medicines.

One of the moderators, Francesco de Lorenzo, of the European Cancer Patient Coalition, wished to see the work done by ERNs rolled out more evenly across Europe. He spoke enthusiastically about the HTA, believing it will empower cancer patient organisations and improve patient access to drugs and treatment.

From his side, Ortwin Schulte, of Germany’s Permanent Representation to the EU, explained why Berlin was taking a more cautious approach to HTA than many other member states, outlining concerns, also shared by Paris, he said, on the mandatory national uptake of EU joint scientific assessments and market authorisation.

“HTA should be given a fair chance to become more structured, sustainable and efficient, better allowing for the optimal use of resources and sharing of expertise” Cristian- Silviu Busoi MEP

“There are some real national competencies at stake”, said Schulte, adding that despite the European Parliament’s “constructive contribution to the decision-making process”, unease from the Council’s side would likely push the HTA regulation into the next European Parliamentary legislative term.”

In a final interjection, Paolo Casali argued that people with rare cancers were “more exposed and more vulnerable” to “discrepancies” such as variations in pharmaceutical funding and said it was important to also consider the “efficacy” of treatments and the drugs available to people with rare cancers.

He added that RCE had recently been focussing its eff orts in discussions with the European Medicines Agency (EMA) on how member states could be tempted to use the ERNs to develop innovative solutions to drug licensing and reimbursement.

“These kinds of networks are devoted to rare diseases and rare cancers and are made up of centres of excellence selected by governments. Therefore, governments should trust them.”

Closing, Peterle reminded the audience that “Cooperation with all stakeholders is key to deliver good policies and achieve tangible benefits for European citizens. Rare Cancers Europe is living proof that we need to pull our strengths together to ensure that better and more appropriate care is given to patients affected by rare cancers.”

About the author

Martin Banks is a Senior Reporter for the Parliament Magazine

Brian Johnson is managing editor of the Parliament Magazine

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