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# EP Workshop on Rare Cancers: The Added Value of Closer Cooperation

On July 12, the Policy department A and the ENVI committee held a workshop on Rare cancers: the added value of closer cooperation.
12 Jul 2011

Alojz Peterle (EPP, SI) argued there are many rare cancers and the number is growing. More awareness and action is needed. They are council conclusions and many stakeholders wish to participate in a coherent manner.

#### Part 1: The importance of special recognition for rare cancers in the revision of the EU Clinical Trials Directive

Glenis Willmott (S&D, UK) said there are often not enough patients with the same type of cancer in order to carry out a trial. Transnational clinical trials are an important topic for the EU. She is relieved to see that the commission has seen the problems that exist with the clinical trials.

Andrezj Rys, Director of Health Systems and Products, DG SANCO, argued that SANCO is now responsible for the pharmaceutical dossier. It is important to have a platform for the stakeholders. The EU has an important policy in rare diseases and is a global leader in this field. There is a research fund in this field as well. In addition, the EU is responsible for the pharmaceutical regulations.

At the moment the public consultation on clinical trials is being finalised. The results will be published soon. The response was from a high level. The commission is aiming to adopt it by the second quarter of 2012 and hopes the work will be finished in the first reading agreement. A slim, fast and efficient approval process is needed; the amount of clinical trials is dropping. He would like to ensure that the current system will be changed in line with the needs of the patients, researchers and the industry. The subsidiarity principle is important in this domain; ethical issues have to be left to the member states.

It is necessary to simplify and risk adapting the rules in order to make the studies available to several levels of committees and so on. It is crucial to think about the legal instruments. There should be no discrimination between commercial and non-commercial sponsors. The directive is not about the kind of research that is conducted but how the research is to be conducted. The EU is building the system and has to make sure it is ready and good. With the database of clinical trials, it is necessary to develop good technical tools. He urges the researchers to let SANCO know if it works properly.

Paolo Casali, Organisation Committee of the European Action Against Rare Cancers, Board of Directors of the European Society for Medical Oncology (ESMO), stated that the action against rare cancers is a multi-stakeholder process. It is based on a meeting that was held in 2008. Its output was a set of 39 recommendations. At the moment they are trying to implement those recommendations. A higher degree of uncertainty should be accepted for clinical decision-making and regulators, if not, cancer patients are discriminated against. The regulators on orphan drugs are very important; they have been a major step forward. The problem is that all the benefits that pharmaceutical companies might apply only if the drug is approved and reimbursed. The decision-making of the industry has to do with the level of uncertainty that exists whether the drug will be approved. Now there is a widening gap between approval and reimbursement, between the risk benefit and cost benefit.

They are trying to improve the methodology of clinical trials. In February 2012 there will be a multi-stakeholder event on that. The preclinical rationale is important. There is a lot of evidence that must be put together in order to see if something is going to work on a patient. That is their task. As for the EU, he believes they should make it possible to have small clinical studies for rare cancers. Methodologies for small studies have to be developed from an organisational point of view. This should be supported by the pharmaceutical companies. In addition, networks have to be created to do trials as that might help finding money. Infrastructures are needed and the facilities that already exist have to be shared. Moreover, rules are needed. Limited centres of excellence are able to do those trials.

The directive is interpreted differently according to the member state. Moreover, good clinical practice depends on the quality of data. The quality of treatment is important in rare cancers. A limited amount of centres dealing with rare cancers ensures the clinical quality of trials is being done well. The data protection directive is also important because of the tissues. Patients would be willing to give up at least some of their privacy in order to get life in return. Patients are willing to give their tissues. Sometimes they are not possible to do so because of the different interpretations of EU directives.

Ruth Ladenstein, President of the European organisation promoting optimal standards of care for children and young people with cancer, coordinator of FP7 funded project ENCCA, said that children are often not protected when dealing with trials where they are considered small adults. There are 50000 cases in the EU each year. Due to the large academic engagement, the survival rate of 80 percent has been achieved. However, 3000 children still die each year. About 500 000 could be alive if they had access to the support that exists nowadays. Children are still pharmaceutical orphans. The drugs used in cancer are licensed but due to a lack of economic interest, they are a group left behind.

There is a need to stop daily experiments. Children need to come into the focus, they need support. The major challenge is that we are struggling to continue to run investigated driven academic trials, she said. The drug definitions currently used create the major barrier. Many drugs are used off-label. They appreciate the idea to have risk-based adjustment. The high level of bureaucracy and high insurance needs is a big problem. She pointed out there is still poor access to new drugs and there exists unequal access to standard treatment across Europe. She warned that the lack of sustainable funding will be a problem for future research.

Innovative research has to be fostered, training and education is important and well-approved drugs are needed. Bureaucracy and insurance are major constraints. Multinational clinical trials are vital in order to have quality assured treatment and foster research. Poland for instance has not brought up a new trial since 10 years. New regulations are needed because of the current regulatory burden. Member states should cover insurance risks, particularly in rare orphan disease cases. She wants to bring up the level of standards for children to the level of adults.

Jan Geissler, Founder of Chronic Myelogenous Leukemia Advocates Network, explained that he underwent trials himself. There are strong inequalities between groups of patients. Rare cancers are often neglected. The prevention and screening is mostly not relevant for them, they are diagnosed too late, experienced doctors are not available in their home town and there are access difficulties. He also mentioned the slowness of research. There is no commercial interest to drive improvement forward. Rare cancer patients face more stigma and discrimination. Patient groups help patients a lot with information, but they have problems with funding.

Rare cancers are often lost between big oncology and rare diseases. It is a challenge to build on the successes of rare diseases and oncology and find a common support for cancers. The directive was established in 2001. The main rationale was to ensure the safety of patients and guarantee rights. The implementation did not fully serve the interest of patients. He stated there is too much paperwork, presumed safety by documentation.

He argued that the number of patients has dropped in paediatrics and other trials. The protocol approval took longer after the establishment of the trials directive. All the elderly patients were often excluded from commercial trials. All the different patient groups have engaged when the commission showed the interest in the review of the directive. They have been working very closely with the European parliament and the commission. The patients want to be involved when consultations are being done. The clinical trial directive has increased the focus on industry research. He urged the EU to ask people what they need. Safety reporting must be adjusted to what people need. Patients are the only true representatives of patients.

Francoise Meunier, Director General of the European Organisation for the Research and Treatment of Cancer, explained that her organisation conducts independent clinical research. Multinational cooperation is needed in the field of rare diseases. It is impossible to deal with that on the national level. For many rare diseases, it is difficult to entice the industry. She argued that it is utopia to believe that each patient will be cured. Cancers cannot be cured so quickly and fast. Even the very frequent tumours become rare tumours. Multi targets are needed instead of single targets. Smart and robust methodology has to be developed and irrelevant national research has to be discouraged. Duplication has to be avoided, cooperation on a multinational level is important. The harmonisation of the legal framework is very important.

It is important to realise that 21 percent of the trials are multinationals. They involve 70 percent of the patients. 36 percent are investigator-driven clinical trials. The main goal of the revision of the directive should be to streamline, simplify and harmonise. Risk-based approach is also very important. Furthermore, she pointed out that one needs a multidisciplinary team for cancers.

What are we going to do when we lack nurses and young doctors in 10 years time, where are we going to find those resources, she wondered. It is necessary to promote clinical trials. Are we going to let other continents develop treatments for us, she asked. It is a real challenge for the EU to do the trails needed for its own population. The pharmaceutical industry is not the devil. New partnerships have to be developed. She pleads in favour of European independent driven clinical trial funds.

David Walker, Professor of Paediatric Oncology, University of Nottingham, and co-director of the Children’s Brain Tumour Research Centre, brought along a 14-year old young patient of him who talked about his disease. He had a cancerous brain tumour which imploded and rendered him unconscious. He had an operation to remove the tumour and had to stay in hospital for a long time.

Peter Wilkinson, patient (Sheffield) recovered from a rare cancer that was diagnosed in his teenage years. In 2007 he experienced personality changes, pain in his back and vision loss. He visited several GPs and went to the A&E several times. All the health professionals missed the symptoms and put it down to watching too much television or working too long on the computer. He did not understand how serious it was. An MRI scan revealed that he had a rare tumour and the cancer had spread down to his spine due to the late diagnosis. He stated that the awareness for tumours and cancers needs to be addressed. Since 2009 he has been cancer free. He experiences several consequences of the cancer and the treatment such as balding, fatigue problems, peripheral vision and memory loss. Nowadays he is a video editor at Jimmyteens.tv which is in a partnership with the Teenage Cancer Trust. They created a website where cancer patients can share their personal videos of their battle against cancer. He believes that a video tool is good therapy. Since 2009 he is a video editor there. Finally, he said that it is not because the cancer is rare that it should be forgotten about.

#### During the questions and answers

Michael Cashman (ALDE, UK) talked about the need to invest in specialisation around rare cancer and to review the clinical trials directive because it was preventing the kind of measures talked about earlier.

Andrzej Rys commented by agreeing that the regulation should also be reviewed as had also been proved by the consultation process and the debate. He then emphasized that public research money should subsidise research networks and should be discussed in the context of the next framework programme. He then spoke of centres of excellence and their definition and involvement in the clinical research and said the cross border directive was an example for the direction to take. Lastly, he said there had been good work put into making the paediatric legislation come into life but a regulation was not enough considering the way it was implemented. He then spoke of the data protection directive.

Paolo Casali said there was perhaps a wish to protect patients’ rights without listening to them. It was risky because having strong data protection was a good thing and not a bad one. He then said there were always trade offs especially at the time of a crisis and excellence was not always affordable.

Ruth Ladenstein said that considering the history of academic trials, such patents were known to have their chances to survive 20 % lower. She said drug development was one major issue but data protection poses threat to patients because clinical trials cannot be run anymore.

David Walker said there should be thought over how to influence hospital trust boards to think that research was important to target on. When new trials are introduced the hospital demands proof for the need of a new drug because it brings about costs.

#### Part 2: Improving Radiation Therapies and Drug Development for Rare Cancers

Alojz Peterle (EPP, SI) introduced the second part of discussions and said they would be speaking about improving radiation therapies and the research on rare cancers.

Maria-José Vidal-Ragout, Head of Medical Research Unit, DG Research and Innovation, European Commission, said she would speak of actions under the 7th framework programme, rare cancers and the future. She then clarified that the commission’s means to do cancer research was through collaborative research with national actors and also by using public-private partnerships. The second invested area was the policy initiatives. Between 2007-2010, 350 million euros had been devoted to cancer research via different programmes, of which one third was devoted to the research of rare cancers. The aim of the programmes was to establish cooperation and evidence based strategies via capacity building and the fostering of education and training. The reason for the interest in rare cancers was that there were practical needs for basic understanding which called for enhanced collaboration, best practices and facilitating the constitution of critical mass of data resources through comparative studies, clinical trials, etc. The latter was one area in particular which had been supported under the 7th framework programme.

The 7th framework programme had also focused on investigation into clinical trials in particular in the area of rare cancers. Moreover, another area investigated during last years was establishing proof of concept for new methods and approaches. There were several projects addressing trials to obtain the “authorization for a paediatric use marketed authorization”. Thirdly, she mentioned networks, for example on the improving and understanding of connective tissue cancers, the European leukaemia net (successful in issuing guidelines for CML) and horizontal networks coordinating research centres and institutes by joint programmes on data collection, clinical trials and diagnoses. Furthermore, the programmes focused on capacity building for cancer research, clinical trial infrastructures and also included a network on cancer registries from all member states which was important for underpinning research on cancer.

As called for by the communication on action against cancer, a forum in Madrid in June had coordinated member states actions for cancer. From the commission’s point of view, the focus would be on key societal challenges, one of the most important one being the health and ageing of society. The first European innovation partnership pilot project would be on the active and healthy ageing. The idea would be to balance research closer to market application to help the validation in the clinical context. The commission had put forward a common strategic framework following the competed public consultation and in September there will be a first draft proposal on it. The proposal would bring both academia and industry into more application of the basic knowledge in the clinical context.

Gilles Vassal, Head of Translational Research at Institute Gustave Roussy; SIOPE President-Elect, emphasized the patients’ rights and needs for an equal access to expertise, treatment and innovative therapies in due time.

He then gave four pillars for improving drug development in rare cancers:

• networking in expertise and research
• investment in research
• incentives/obligations towards pharmaceutical industries that develop drugs to treat rare cancers

He continued by explaining the third point and spoke about the valid European legislation on the matter which covered two regulations on the development of drugs on rare cancer voted by the European parliament. Many rare cancers were still not treated and were orphaned under the applying regulations and many patients still did not have access to medicines they required and which were not authorized. More importantly, major needs of children were not always recognized by European legislation. The problem was that companies had no obligation to do research on children’s cancer and it was not their priority. Moreover, children were denied access to drugs even if the standard treatment had not worked whereas in some countries drugs for adults were subscribed to children in quite a relaxed manner. Concluding his speech, he said there was need for investment in sustainability and to resolve the question of how to make paediatric medicine regulation applicable to children. Thirdly, there was need for academia research which did not rely solely on the input of pharmaceutical companies. The public-private partnerships should develop drugs for children with rare cancer as well.

Pamela Cohen, Associate Vice-President, Oncology Clinical Research, Sanofi, spoke about recent drug approvals in rare cancers and brought up the idea that common tumour types could also be looked at as rare cancers. She then spoke of her research and about a drug they had come up with. She then said that authorities should give a higher degree of uncertainty for the development of the drugs. There should be thought over the random nature of the patients chosen for the trials.

Stephanie Combs, Radio-oncologist, University Hospital of Heidelberg, Partner of FP7 funded project ULICE (Union of Light Ion Centres in Europe), explained that in radio-oncology the challenge was the achievement of local tumour control. The current status of cancer treatment was that there were many patients whose cancer could not be treated locally which was naturally an area which could be improved. She then mentioned a new technique to improve radiation called particular therapy which was already used in the USA. It was currently used in Germany and some other countries. She then said the ULICE-project was trying to do research on the way individual biologies could handle the therapy.

Alojz Peterle (EPP, SI) and Glenis Willmott (S&D, UK) summed up the event by stating EU regulation would have to be improved and knowledge would have to be shared.

The complete workshop brochure, including Executive Summary, Introduction, Policy Background, Proceedings, Programme, Biographies and Presentations, can be downloaded here.

Last update: 12 Jul 2011

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