EP Meeting: Challenges of Rare Cancers

Rare Cancers: Specific challenges to citizens and leading researchers

  • Date: 12 Jan 2011

On 12 January 2011, Marisa Matias (GUE/NGL, PT) organised in collaboration with the European Cancer Patient Coalition (ECPC) a FACE workshop on Rare Cancers: Specific challenges to citizens and leading researchers. The event gathered MEPs, patient organisations, representatives from the commission as well as from the industry. Please find below a summary of the discussions. Please note that presentations are available on demand.

Patricia Garcia from the ECPC opened the meeting on behalf of Marisa Matias who was delayed due to other commitments. She started by mentioning the support of 81 MEPs for FACE and also reminded the floor about the recent positive development on the cross-border healthcare proposal as well as the falsified medicines dossiers. She then briefly introduced the speakers of the first panel.

Rarer Cancers – An Urgent Need for Policy

Petru Luhan (EPP, RO) began by stating that today’s event was an occasion to continue to make all parties talk about what can be done in the field of cancer and specifically rare cancer. There is not much on the political agenda of member states nor the EU he explained, and there are today 3 million people who have cancer. This is a signal he said, that we have to do something. The question is also how many do not have access to a test and then how many people actually have cancer. The only efficient way to go is to combined technological evolution, financial support and the political framework. As regards technological evolution there is still a lack of research and development. Luhan then mentioned the new tool proposed by a Romanian researcher for cancer diagnosis and the improvement in the cancer research field. On the financial framework he regretted the lack of involvement of relevant applicants for funding under the FP7. Finally, on the political framework he stated that there are still huge discrepancies between member states regarding access. The objective would be to have equal access to treatment. But health is not a competence of the EU and this is to be addressed, unless there is a DG dedicated to these issues as well as money and people to deal with it at EU level. He then added that he will put pressure on the commission to take action on rare cancer and to dedicate money. Rare cancers are a specific challenge. There are often late and incorrectly diagnosed. There is a lack of research and therapies and there is a lack of interest in the development of new therapies.

Luhan then came on saying that 20% of cancers are considered to be rare. Cancers and rare diseases are on the EU agenda. But there are still many challenges to be addressed. The improvement of methodology and clinical trials is one of them. The development of new drugs, equal access to treatment and focus on prevention are others.

Rare cancer patients also face challenges that can only be addressed through the collaboration of all parties, politicians, the pharmaceutical industry, patients, health professionals and academics.

Luhan concluded by proposing a few changes.

  • More research and innovation. Prevention is important but not all types of cancer can be prevented. Rare cancers are not taken enough into consideration.
  • Better communication: MEPs are expecting patient organisations to educate them on these issues as they do not know everything.
  • Luhan also mentioned the issue of the use of off-label drugs. People are not aware of how to use these drugs.
  • Surveillance of rare cancer is essential.

He concluded by inviting all participants to an event he will organise in the European parliament between March and May to launch the new cancer testing tool he previously referred to.

Responding to a question from the floor, Luhan explained that there is no real control of which drugs patients are using. The use of off-label drugs is a huge problem but there is no solution yet. He then added that he will send a letter to the commission to ask about any action on this issue.

Another participant raised the possibility of enhancing the problem of rare cancer by pushing national plan for rare diseases. He mentioned the Europlan project that proposes templates for national plans for rare diseases. As regards off-label drugs use, he agreed that this is an important issue in the frame of rare disease but there is no clinical studies. It should be part of the reflection of the newly set up rare diseases working group he added.

Tom Hudson, ECPC president then spoke on behalf of Dr. Adamo Adamou former MEP who was unable to attend the meeting. Reading through the letter prepared by Adamou, he raised the problem of delayed and incorrect diagnosis, the lack of expertise, the lack of research, and the problem of access to clinical expertise. There are few clinical trials as they are few patients he added, and a lack of interest in these diseases. Rare cancers are not rare at all he stated, and they deserve attention. Treatments existing for cancer do not usually work on rare cancer. Hudson then remarked that if cancer is on the political agenda there remains a lot to be done notably on clinical trials, the development of drugs and on equal access. The visibility of rare diseases has to be improved. National plans on rare disease have to be supported and EU cooperation as well. Expertise has to be linked as well as knowledge sharing he explained. Finally all parties have to work together.

As a few potential changes, he called for a new vision and priorities. Prevention and screening are good but not for all kind of cancer. In times of economic crisis, pressure is put on the rare cancer area. Hudson also called for a new approach for drug development. Despite the orphan drug regulation, only a few companies can do investments. It also needs better communication and visibility including greater awareness of clinical trials to cancer patient organisations. Better access to treatment should receive focus. The right drug should go to the right patient at the right dose and at the right time. Patient mobility is a reality today for rare disease patients. Ideally, the expertise should travel rather than the patient. Moreover, there is 50% of use of off-label drugs. Clinical studies are difficult to make, and there is also a reimbursement problem and legal difficulties. Hudson concluded by explaining that it will need the commitment of all stakeholders, and that all achievements will be to the benefit of the whole community. The EPAAC should also go ahead. Surveillance is essential he said, as well as data collection. As such, national registers should be secured.

Kristina Andrekute head of the ECPC Rare Cancers Action Group stated that rare cancer is not just a question of prevalence. It involves a number of people. The strategy should be different from common cancer. Rare cancer is not adequately dealt with. Policy is needed. Rare cancer implies time for diagnosis. Cancer strategy and initiatives do not work on rare cancer. There is also a lack of clinical data she agreed. She then raised the problem of diagnosis and lack of drugs. High quality care is hardly achievable today. She concluded by saying that for the action group on rare cancer of the ECPC policy is definitely needed.

Kathy Oliver from the international Brain Tumour Alliance (IBTA) made a presentation on the light of her experience as a mother of a son who was diagnosed a brain tumour. She underlined the questions following the announcement of the diagnosis, about therapies, possible care, other patients, and support. She then highlighted some of the challenges for rare cancer noting that therapies are very expensive and not reimbursed, the lack of clinical trials, the denial of licensing approval, and that prevention and screening are not particularly relevant for rare cancer. She agreed that rare cancers are not rare. Kathy Oliver then evoked the difficulty of advocacy and for patients having rare cancer and agreed with the different medical challenges underlined by the previous speakers. Finally, she mentioned the European Action Against Rare Cancers and the 39 political recommendations developed. She then wished for a UN summit on Non-communicable diseases to take place in September 2011 to reach the same echo than the UN summit on HIV. There is some tension around the issue of putting the focus on prevention or research, but she expressed her wish that rare cancer will be highlighted.

Clinical Trials – Challenge for Rare Cancer Patients

The panel was opened by a video of Prof. Jean-Yves Blay, European Organisation of Research and Treatment of Cancer (EORTC) who was unable to attend the meeting. He started his intervention by explaining that rare cancer patients are facing more problems. Rare cancers are in-between cancer and rare diseases. There is a difference for the management of treatment he added. Prof. Jean-Yves Blay then made a few points on important aspects for him

  • Diagnosis: he agreed that standards of treatment are not well known or do not exist. Clinical research is also lacking.
  • Clinical trials are very important, and are windows of opportunities. The participation of patients is complex.
  • Participation of patients in clinical trials: it is important for standards and improvement of care. Patients might feel like guinea pigs but this is not true. Patients need to be aware of their existence but information is lacking. There are reference centres in certain countries but not all. Access to novel agents is not that difficult for rare cancers he explained. The pharma industry is involved and it is not too difficult for certain tumour patients to access clinical trials and novel agents. The question of reference centres and reimbursement is essential.
  • Role of patient advocacy groups: patient groups are more developed in the field of rare tumours than for other frequent tumours. Their role is about information and communication between physicians, patients and families.
  • Building research on people with rare disease, collecting data and education on clinical trials is also necessary. Patients groups should be involved in clinical trials process.

As a conclusion he re-said that patients with rare cancer are facing more problems than patients with more common cancer. Clinical trials are the way to improve care treatment and knowledge.

Dominika Trzaska from DG Research then made a presentation on the Commission action in this field through the FP7. She presented the health objectives of the FP7 and stated that clinical research is expected to be a major tool. Presenting the main policy drivers, health of citizens, competitiveness of the health industry, global health and the innovation union, she summarized the support for collaborative cancer research through the successive FPs. She then explained about the focus on clinical trials in the 2011 WP. The first objective is to bring research discovery into clinical testing. Second is to advance on the development of a new approach and then to compare treatments. Dominika Trzaska also explained that the commission is committed to involve patients in clinical trials. She then presented a large number of projects in this field.

Prof. Paolo Casali from the Instituto Nazionale Tumori (Milano, Italy) explained that difficulties exist in all phases of clinical studies. The problem, he explained is that in cancer we sometimes have the drugs but the decision is difficult to go ahead for rare cancer. Rules on orphan drugs have been very useful but one of the problems is the registration risk: Risk of not getting the authorisation. If the drug is not approved there is not benefit. After the approval, the question is whether the drug will be reimbursed. If it is not, it is as if it has not been approved. The issue is that the decision on reimbursement is local while the decision on authorisation is central. Two different approaches are used: risk/benefit vs. cost/efficient.

Prof. Casali then commented on the concept of quality of evidence. The need in rare cancer must be different that in common cancer he stated. If we do not allow drugs in rare cancer to be approved with a different quality of evidence we would discriminate rare cancer patients he explained. If we were able to support more academia sponsored studies it would be different he added. Prof. Casali then explained about the imbalance between academia and the industry and called for a more interaction between researchers, the industry and regulators.

Prof. Casani then went on saying that one of the problems today is that clinical studies cost a lot. Also, the clinical quality of studies is not stressed enough. He also added that it would be good to have multi-sponsored clinical studies. Networks are needed. Prof. Casani then explained that there is a methodological problem today as we are going from chemotherapy to targeted therapies. The problem comes from the non-targeted use of targeted drugs. He also raised the issue of statistic precision and clinical precision. The problem is to link clinical data to biological data. Today, donation of tissues is difficult (due to regulation constraints and privacy rules). Researcher need tissue sample for clinical studies but also tissue banks. He concluded by presenting different statistical approaches.

Marisa Matias then gave a few words and stated that there is still a long way to go. Rare cancers concern a large number of patients. Collaboration between researchers and patients need to be strengthened.

Julie Hearn from Cancer Research UK, after briefly introducing Cancer Research UK, explained that they fund €400 million on cancer research. She then indicated that in 2001 the National Cancer Research Network Consumer Liaison group was established and explained the involvement of patients in clinical study group. They participate in the design of trials and are also represented in the steering committees. As a funder, Cancer Research UK also asks applicants how they are involving patients. The position of the charity is unique because of the link with and existence of the NHS. There are currently 200 trials in the UK she said and 5% of them are on brain cancer. However, she regretted the small number of applications they received in response to calls for projects on rare cancers. Julie Hearn then stated that there is a crucial need for international trials. Conducting trials in the EU is difficult today and they are currently trying to identify the difficulties delivering trials across EU, also by analysing the systems existing in other world regions. She finally concluded by explaining the work they are doing with AstraZeneca on potential cancer drugs that would not otherwise be taken any further.

Dr. Filippo De Braud, from the Instituto Europeo di Oncologia (IEO) started by explaining that the development of drugs and innovation would improve the well-being of the society. The dream today is to have personalised medicines. He then explained that in rare cancer they are more advanced in research and designed drugs. Dr. Filippo De Braud then explained the progress made on cancer drugs, identifying strengths and weaknesses. He added that the process for authorisation of drugs is very long, going from two to ten years. His presentation also showed the results and stated that the most reasonable action for patients with rare cancer is to participate to clinical trials. Coming back on what was said by other panellists he added that the problem of off-label use of drugs is also a problem of safety. He supported the idea of clinical trials at European level and acknowledged the issue of reimbursement.

Prof. Henk Van Daele, ECPC Board member made a presentation on the problems encountered by rare cancer patients, giving the example of male breast cancer (MBrCA). He underlined the lack of data on MBrCA and of information. There is no dedicated treatment he explained and they are usually compared and treated through treatment used for other cancers. He called for research on this topic as well as to communicate and set up patients groups and clinical trials. As a conclusion he called for awareness, to strengthen partnerships, to support all existing rare cancer patients groups to create and support new rare cancer patients groups, to force all national cancer registers to register all rare cancer cases. As an urgent request he asked for the organisation of centres of excellence as well as guidelines for every rare cancer. He also called for the strengthening of academic research about all rare cancers and to improve the education of doctors, nurses and oncologists. He finally requested open borders for clinical trials and individual treatment of all rare cancer.

As a final comment and during the Q&A session, Prof. Casali explained that in a way rare cancer patients are privileged as they are on both sides, cancer and rare diseases. But some of the problems have to do with the peculiarity of rare cancer. Male Breast cancers have a counterpart in women. It is a privilege but still needs specific studies. Casani also explained that regulators might be ready to accept a new approach. He then regretted that physicians have often understood very little about statistics. The community should be more involved in the methodological issue. Dr. De Braud added that one of the problems is the validation of results.

National Perspective – Towards a European Rare Cancer Plan

Belgium: Prof. Simon Van Belle, University Hospital Ghent started by introducing the proposal based on discussions in National College Oncology in Belgium. He was not sure whether the proposal would fit in the European law. He then said that National Cancer Plan was established in 2008. There are 32 initiatives. One of the initiatives is taking care of patients with rare cancers. In 2009 working text was prepared to centralise treatment in Belgium. However, that proved impossible to achieve. There is an aversion to centralised care system in Belgium he explained. They found the agreement indicating that incidence under 1% is a rare cancer. Basing on this they made a list of possible cancers.

He then said that they want to have quick and adequate registration and adequate follow up of patients. He said that they defined all elements needed in registration. The quality control of pathology is a problem he added. 40% of patients have a different histology comparing to the one done at the beginning. A lot of rare cancer cases are not discovered because histology is not well defined. He noted that they want to do elaboration on standards of care guidelines by College of Oncology. It would be good to monitor the standards of care guidelines he noted. He said that they tried to have audit of the system, but it proved to be problematic. Evaluation and adjustment of the system would be needed. Federal services would analyse database. He said that the problem is that privacy system is not easy. He then said that they would like to have a possible link between the College and the funding. They also would like to participate in the international studies. Finally, he said that we should try to have national plan that should be adopted at national level and then at European level.

Frank Boeye, Study Group Brain Tumour, said that health policy is of national concerns. However, European initiative is needed for rare cancers. The obstacle is the complexity of procedures and relatively low number of patients. Brain tumour happens 2 or 3 times per year in the country. Expertise needs to be established at the European level. He added that there is a high standard of expertise and unlimited possibility for data management. As for the clinical trial directive to register new therapies, he said that there are three phases and it takes long time. He also said that the access to medicine should be better and that the major concern is safety of patients. He then noted that the procedure for registration is too expensive for patients.

Regarding the brain tumour he said that the life expectancy is very low. Five years of survival applies only to two percent of people. There is 98% of mortality. This proves that methods that have been used so far do not work. He then asked what safety means for brain tumour patients. With regards to new paradigm, he said that the absolute safety philosophy is leading to deaths. He stressed that another approach is needed, adding that they must not be limited in the choice of a new type of medicine. He then said that they proposed changes in legislation and facilities in registration proposing better terms and conditions for registration of therapies only designed to orphan diseases. They also propose that conditional registration would be limited in time giving access to refunding prior to full registration. Other statistical methods should be added as well. They also proposed that cancer registry could be used for clinical and scientific research. He added that most of data is already accessible through the Belgian National Registration. He said that since this would start working they should get in touch with registries in other European countries. He noted that the system is a transparent way of working. There is no distortion to preliminary conditions. He added that linkage to tissue bank is possible. He concluded by saying that use should be made of National Cancer Registries and that European Expertise Centre for rare diseases should be created.

France: Jeanne Marie Brechot, National Cancer Institute (INCa) presented a number of challenges such as the lack of medical expertise, poor knowledge of rare cancers, poor information and lack of communication. Regarding the French National Cancer Plan, she said that there are developing reference centres – a dedicated hospital. National network will be organised in each administrative region. She noted that this year, seven clinical referral expert networks for rare cancers were identified. She added that two clinical guidelines at national level are in progress. She said that the last call for application will be next year.

Czech Republic: Jana Pelouchova, Diagnoza CML started by illustrating examples of some of the goals of Czech National Programme such as public education on cancer prevention, early cancer prevention by doctors, cancer research, professional education, cancer registry and cancer diagnosis. The programme was approved in 2004. There are 13 specialised centres for ontological treatment. There is also a plan to build a Cancer Research Centre. A screening programme exists. But she added that only 16% of population is using it. According to the Society of Oncology the treatment of cancer in the Czech Republic is at the same level as in the founding member states. She added that there are economic challenges.

She then said that rare cancer patients have lack of support with experts being few. She said that access to information in Czech language plays a key role. Referral to experts for a second opinion should take place. Open communication across the society should take place as well. She said that since 2010 there is a new tendency from insurance companies to reduce the number of patients to whom treatments could be given. An exodus of Czech doctors is taking place. She stressed that this should be solved at political level.

Policy Actions

Andras Fehervary, Novartis Oncology Europe, focused on the strengthening of access to orphan drugs. He cited a recent study proving that the net impact of the Orphan Drug regulation on European society and economy was positive in terms of number of orphan drugs registered, as well as clinical trial activity. Small and medium sized enterprises in particular have benefited. This has been achieved at a reasonable cost to European society - less than 3% of pharmaceutical spending, which in turn is a fraction of total health care spend. However, there remains a problem with access to medicines for rare disorders, including rare cancers, which is most acute in Central and Eastern Europe as well as some Western European countries such as Portugal. As a result, there are many patients who do not have access to such life-saving medicines. One cause are the differences in the amount spent on health care across member states, with the poorest countries often also spending the lowest % of GDP on health care. Changes in reimbursement policies would help stimulate more innovative pricing and market access solutions, which can often expand access and end waiting lists at an affordable cost. Fehervary noted that as we are facing difficult economic times, it is even more important than ever today to implement such innovative policy imperatives.

Member states acceptance of orphan drugs, efforts to expand access through expanding programme and reimbursement are important in improving access to orphan drugs. Regarding the evaluation of access to orphan drugs in the European Union, he said that there is an ambition to start addressing the factors that limit access to drugs. With regards to member states’ acceptance of orphan drugs value, he said that for orphan drugs, reimbursement problems can be created in member states due to the economic and clinical uncertainty which orphan drugs face due to small clinical trials. To address this issue, he cited the potential to expand risk-sharing schemes, following the example of Italy which is the leader in this area. Further, acceptance of the CAVOD proposal endorsed by EuropaBio/EBE regarding clinical value of time of marketing authorisation would be a step forward. Another other way to improve early access is through expansion of the French ATU system - a means of enabling patients to have access to promising new drugs not yet covered by a marketing authorisation in France, for which a major unmet medical need exists. He added there is another pricing issue which endanger access to orphan drugs: International Reference Pricing limits the flexibility for companies to offer innovative solutions, and hence orphan drugs should be exempted from the application of international reference pricing. Instead, more flexible pricing solutions should be encouraged.

Based on progress made during the past three years including the ESMO 39 Recommendations, Fehervary concluded there is agreement that only a holistic and collaborative approach involving all partners will achieve better standards for care for patients suffering from rare cancers and other rare disorders.

Sandra Craine, ECPC Board Member, said that being diagnosed with rare cancer that is classified as rare disease detaches us from the place we should be as patients of cancer. She noted that it cannot be said that the market is flooded with new products. Patients should be informed and involved in the decision-making process. More innovative approach to the treatment is needed. She, as a patient, was supported by doctors who took un-calculated risks. She then said that Novartis took innovative decisions and followed ‘the God instinct’. The courage and the rational decision to take risks resulted in saving a lot of lives. Novartis took a successful decision she said. She was fully informed about all possible possibilities of therapies. She stressed that the decision taken by Novartis resulted in a step change in our knowledge in novel therapies. She concluded by saying that innovation saved her life.

Marisa Matias (GUE/NGL, PT) said that she is frustrated about the fact that people keep dying from cancer. She then said that it does not matter whether it is rare or regular cancer, choices are crucial for our lives. Individual needs should to be faced. She concluded by saying that there are small victories that should not be wasted.