Doslovno izvješće

Četvrtak, 13. prosinca 2018. - Strasbourg

14. Rijetke bolesti (rasprava)

  President. – The next item is the debate on the oral question to the Commission on rare diseases by Mairead McGuinness, Rory Palmer, Jadwiga Wiśniewska, Gesine Meissner, Tilly Metz, Estefanía Torres Martínez and Piernicola Pedicini, on behalf of the Committee on the Environment, Public Health and Food Safety (O-000118/2018 — B8—0419/2018).  

  Gesine Meissner, Verfasserin. – Herr Präsident, Herr Kommissar, liebe Kolleginnen und Kollegen! Wir sind ja nur wenige. Das ist klar: Es ist kurz vor Weihnachten, und die meisten sind schon weg. Vor Weihnachten möchte man sich auch mit schönen Dingen befassen und nicht mit Krankheiten, das ist klar. Wir alle wünschen uns eine stabile Gesundheit. Man könnte auch meinen, so, wie wir in Europa mit der medizinischen Forschung aufgestellt sind, ist eigentlich schon fast alles gut. Der Punkt ist der, dass es halt Krankheiten gibt, die so selten auftreten, dass dann eben genau nicht alles gut ist. Und das wollen wir verbessern.

Von seltenen Krankheiten spricht man in der Regel, wenn nur weniger als 2 000 Menschen davon betroffen sind. Das ist bezogen auf die Europäische Union eigentlich noch immer eine relativ große Zahl von Menschen, die seltene Krankheiten haben können. Insgesamt, sagt man, gibt es in der Europäischen Union 6 000 bis 8 000 seltene Krankheiten, aber insgesamt sind 30 Millionen von 500 Millionen Menschen davon betroffen – also eine erhebliche Zahl.

Und man muss überlegen: Was bedeutet das für diese Menschen? Das bedeutet für diese Menschen, dass es , wenn sie die Diagnose bekommen – die hoffentlich eine richtige ist, weil man eben bei seltenen Krankheiten auch mit der Diagnose unsicher sein kann –, große Fragezeichen gibt, große Unsicherheit, nicht nur bei den Medizinern, sondern auch natürlich bei der Familie. Normalerweise denkt man, wenn ein Mensch krank wird, dann hat man ein Medikament. Man kann das nehmen, und dann wird alles gut. Wie ich vorhin sagte, ist es in diesem Fall genau nicht so.

Ich werde jetzt sehr persönlich. Ich kann das aus eigener Betroffenheit sagen. Mein Vater ist an einer seltenen Krankheit früh gestorben, mein Bruder ist als Kind an einer seltenen Krankheit auch sehr früh gestorben. In beiden Fällen kam die Forschung zu spät, und es gab keine Möglichkeiten, ihnen zu helfen. Bei meinem Vater, der ein bekannter Politiker war – ich sage das auch sehr persönlich –, war es so, dass wir selber als Familie auch sagten: Wir wollen keine Forschung ermöglichen, wir wollen ihn nicht zur Schau stellen. Das war vielleicht verkehrt, das war vielleicht egoistisch, aber wir hatten uns so entschieden.

Mein eigener Sohn ist vor sechs Jahren an einer noch gar nicht erkannten Krankheit erkrankt. Er war der erste Fall weltweit, an ihm wird noch heute geforscht. Und auch da weiß ich, dass natürlich die Forschung ihm selber hilft, weil er sagt, er weiß, dass die Forschung an seiner Krankheit, die so einmalig war, schon vielen Menschen geholfen hat. Das ist natürlich gut. Aber es ist so, dass es ihm selber nicht helfen konnte. Es gibt dann halt nicht nur keine passende Diagnose, es gibt auch keine bekannte Behandlung. Es ist Versuch und Irrtum, das man von Herzen machen muss. Ich habe es eben selber gerade bei meinem Sohn mehrfach erlebt, wie Versuch und Irrtum zum Erfolg führen und das Leben retten können, aber natürlich nicht dauerhaft helfen können. Und das sind Dinge, die wir verbessern wollen.

Darum ist es ganz wichtig, dass wir die Forschung ermöglichen und dass wir auch sehen: Wie können wir Finanzmittel sicherstellen, wie können wir die Netzwerke, die es schon gibt, verbessern, um für die Menschen, die es betrifft, in Zukunft Besserung zu schaffen.

Es ist eben so: Die Medizin ist zum einen schon sehr weit fortgeschritten, zum anderen in mancherlei Hinsicht eben noch in den Kinderschuhen, weil es immer wieder auch neue Fälle geben kann, gerade auch durch tropische Krankheiten. Bei meinem Sohn kam ein tropischer Virus dazu, den man bis heute nicht entdeckt hat, obwohl viele versuchen herauszufinden, was es war.

In diesem Zusammenhang ist es für uns ganz wichtig zu sehen, was man machen kann, um auch überall in der EU den Menschen den Zugang zu Behandlung, guter Diagnose und auch entsprechenden Medikamenten zu ermöglichen.

Dazu haben wir verschiedene Fragen ausgearbeitet, die ich jetzt vorlesen werde.

Die erste Frage an die Kommission: Welche Maßnahmen hat die Kommission bisher ergriffen, um für eine genaue und frühzeitige Diagnose seltener Krankheiten sowie den Zugang zu erschwinglichen Arzneimitteln zu sorgen, die für deren Behandlung in der gesamten EU benötigt werden? Wie fördert die Kommission Forschungsmaßnahmen zu seltenen Krankheiten und die Entwicklung neuer oder besserer Früherkennungsmethoden, und wie gedenkt sie diese Anstrengungen zu fördern und messbar bessere Ergebnisse bei Patienten mit seltenen Krankheiten in allen EU-Mitgliedstaaten zu erzielen?

Zweitens: Wie gedenkt die Kommission den Zugang zu Informationen, Arzneimitteln und medizinischen Behandlungen für Patienten mit seltenen Krankheiten in der gesamten EU sicherzustellen? Wie begünstigt die Kommission angesichts dessen, dass der Zugang zu frühzeitigen und genauen Diagnosen erhebliche Auswirkungen auf die Prognose und die Lebensqualität der Patienten haben kann, die Verbesserung der Koordinierungs- und Lernprozesse in allen Mitgliedstaaten?

Drittens: Welche Pläne hat die Kommission, um die dauerhafte Finanzierung von Patientenorganisationen sicherzustellen, die die europäischen Referenznetzwerke für seltene Krankheiten unterstützen, beispielsweise mittels des gemeinsamen europäischen Programms für seltene Krankheiten?

  Vytenis Povilas Andriukaitis, Member of the Commission. – Mr President, first of all, as the Commission and other Commissioners and Members of this House have done along these past two days, please allow me to express my solidarity with the families of the victims of the terrible attack here in Strasbourg. Now we have three deaths and that is very painful for me as a medical doctor.

Secondly, I would like to tell Ms Meissner that she has just opened my heart and I give her my words of sympathy because of such an experience.

In the 20 years of work on rare diseases the Commission has fostered network structures for action and expertise. It has developed a policy framework with Member States and encouraged innovative ways of working.

Mr President, I have six questions, which – if I may say – will be impossible to answer in three minutes because I know those questions are very detailed.

Let me go question by question because I need to present a really comprehensive picture. Would you allow me to have more than three minutes? Thank you.

Going to the first block of questions, the first is about timely diagnosis of rare diseases, which is very important. We have taken concrete steps to ensure that the patients are diagnosed as rapidly as possible. First, with the adoption of the directive on the application of patients’ rights in cross—border healthcare, Member States set up national contact points. Then you have 28 national contact points. They are workable. These are key to inform doctors and patients, notably with rare diseases, on the way to access the appropriate expertise offered in the patient’s own home country or facilitate contact with a contact point in another Member State.

Second, eHealth digital service infrastructure now is workable, is in place; it is done. It helps to exchange views in cross—border areas, patient’s records and so on. The 24 European reference networks will launch in March and with more than 900 specialised healthcare units, from over 300 hospitals connected across the EU, the European reference network facilitates the access to timely diagnosis and treatment for patients with rare and low prevalence conditions. They contribute greatly to the sharing of information and linking of expertise between clinicians and researchers across the EU.

The clinical patient management system is now once again workable, from November last year, where healthcare professionals from European reference networks discuss real patient cases and conduct virtual consultations and treatment. These have led to very successful EU—wide cooperation between clinicians in identifying and addressing the needs of patients. From November until now more than 250 patients have received timely diagnosis and treatment. This shows effectiveness.

As regards the sub—question on access to affordable medicine, the study that was published early this year on the economic impact of pharmaceutical incentives and rewards in Europe including supplementary protection certificates, draws attention to timely access treatment for rare diseases. An additional study will gather information and assess, on the basis of the acquired experience, the functioning of the orphan regulations. Also, an evaluation of the orphan and paediatric medicine regulation will be finalised before the end of my mandate – maybe in spring next year. Also, there have been measures to promote research in the field of rare diseases and to improve methods of early diagnosis. Horizon 2020 and the Seventh Framework Programme on research have supported more than 200 projects on rare diseases in very different areas, and now we provide more than EUR 1 billion to rare diseases. I will just mention a few examples. The EuRenOmics and NeurOmics projects focus on improving diagnostic matters for large groups of rare diseases including rare kidney diseases, for example, and the Solve-RD and Immune—A projects are now looking to improve the capacity in the coming years. We have a lot of common clusters which can help us address the complexity of rare diseases.

Regarding the questions on achieving measurable improved outcomes for rare diseases, let me stress this year’s opinion of the expert panel on benchmarking access to healthcare in the EU, including the area of rare diseases. We have also just finalised a pilot project called ‘towards a fairer and more effective measurement of access to healthcare across the EU’. The project was launched at the request of the European Parliament, and now we are pushing forward.

The second block of questions. Access to information, first of all. I would firstly like to mention the joint action on rare diseases, which has supported over the past three years the development and sustainability of the Orphanet database on rare diseases, and has also helped us to achieve an appropriate codification of rare diseases in the health information system in order to help really to understand more about such a complex issue. This database is the biggest repository of information about rare diseases globally and is run by a large consortium of European partners. You should also know that the steering group on health promotion, disease prevention and management of non—communicable diseases selected the codification of rare diseases as a priority area to be implemented as best practice, and is now in line with achievements.

The Orphacodes project will be supported with EUR 750 000 under the 2018 annual work plan. The project aims to support Member States in better gathering information on rare diseases from all 28 Member States. Last week, the Commission developed a European platform on rare diseases registration, and is promoting the interoperability of existing registers and will help to create new ones. Can I just mention two databases – Eurocat and CSBA – which have been implemented now.

On the sub—question on access to medical treatment and medicine for rare diseases, the Cross—border Healthcare Directive helps citizens in obtaining medical treatment in other Member States and to be clearly informed as to the rules on reimbursement that apply in cross—border situations. This means that now all Member States should draw attention to the possibilities to reimburse patients with rare diseases. This specialist treatment is particularly relevant for rare diseases. The Commission also supports the Orphanet database as a unique resource for pooling knowledge of rare diseases and guaranteeing access to treatment because it is also about medicines, new legislation on orphan drugs, redirecting investment into previously neglected therapeutic areas through incentive schemes. The challenge is to find a balance between adequate incentives for research and development and equitable access to affordable medicines for all, especially in the area of rare diseases.

We are conducting a study on these very issues which will be used for the evaluation of orphan and paediatric regulation. You know, lastly, speaking about health technology assessment, now such a tool, if we succeed, will help us to move forward in this area.

On your third sub—question about promotion of better coordination and learning across Member States, rare diseases is clearly an area where cooperation at EU level provides real added value. This is a real example of subsidiarity: when you have no chance to address issues at local level you should go to the regional – if not European – level, in line with subsidiarity.

By working together we can share data and expertise, overcome fragmentation and geographical disadvantages and guarantee patients the right diagnosis.

As you know, the newly—established European Reference Network has strengthened cooperation including between clinicians, researchers and of course different developers of treatment and they are building diagnosis networks and a network of working groups to see how they can address concrete issues. I count on possibilities to develop a network of networks – European reference networks – in communication with national healthcare systems and going deeply into primary care, so that all can find pathways, easy pathways, to achieve timely diagnosis.

On your questions on sustainable funding of patient organisation support in European reference networks on rare diseases, European reference networks are economically and logistically supported by the Commission through the eHealth Programme – one resource – and, second, the Connecting Europe Facility Programme. There is no direct funding to the patients’ representatives, but each European reference network receives EUR 200 000 annually from the health programme. This grant supports the network and coordination capacity of the networks, including the organisation of meetings with patients’ representatives.

The eHealth Programme also provides operating grants for non—governmental organisations and patient organisations. Eurorodis is very important in this field.

In addition, the Horizon 2020 work programme for 2018 looks to set up a rare disease European joint programme co—fund. This encourages the participation of European reference networks and patient organisations in the programme over a five—year period.

In order to ensure sustainability beyond 2021, we made a proposal in the multiannual financial framework which envisaged support through direct grants, and this is now in the hands of all of us. Of course countries can also use structural funds, selecting centres, and use social funds supporting the qualification of medical doctors.

President, honourable Members, thank you for giving me the opportunity to update you on the broad spectrum of activities on rare diseases. These actions make up an important part of the Commission’s health and research policies and they have started to deliver clear added value to patients and their families.

Let me assure you that connecting expertise across Europe is a priority of mine.

  Sirpa Pietikäinen, on behalf of the PPE Group. – Mr President, I would like to thank the Commission to start with, because it has been really active and very good and efficient in its work on rare diseases. In some way, in rare diseases, Europe has things in place because rare diseases are in fact not rare – there are about 8 000 to 10 000 rare diseases and almost 10% of Europeans have some sort of rare disease.

They are only rare in the cases, and only rare in the cities, and only rare in the Member States that do not have the capacity or possibilities to have all the knowledge, the cure facilities, or for that matter, the required medication.

That is why we need Europe—wide cooperation that should serve as an example for other diseases. I think that in the longer term we should have the same type of reference centre networks for other diseases as we have at the moment for rare diseases.

As you mentioned, timely diagnosis using the cross—border healthcare initiative and eHealth network are good examples where Europe can help.

I hope that we can channel a bit more Horizon funding in the future into rare disease networks and reference centres. For example into how ‘Dr Watsons’ can be further developed, so that we would have artificial intelligence helping doctors diagnose rare diseases. That could be used by general practitioners already so they can identify and pick up and issue guidance for the further treatment of people with such conditions.

Last but not least is the question concerning orphan medicines. These are extremely expensive and it is a negative lottery for local health centres, but then again at European level the cost is not so much, so I think that we should establish a European fund to buy orphan medicines for rare diseases.

  President. – Thank you, but I need to underline that, even though we do not have a heavy agenda this afternoon, I will have to ask you to respect the time that is made available. I will be strict on keeping to the time.

  Andrejs Mamikins, on behalf of the S&D Group. – Mr President, approximately 5 000 to 8 000 distinct rare diseases affect between 6 and 8% of the EU population – between 27 and 36 million people. Patient registries and databases, clinical research in the field of rare diseases are very fragmented. This problem can only be solved at the level of the European Union.

The EU’s strategic objective for rare diseases is to improve patient access to diagnosis, information and care. Specific measures have to include, first of all, ensuring that rare diseases are adequately coded and traceable in all health information systems. Secondly, supporting national plans for rare diseases in EU Member States. Thirdly, improving recognition and visibility of rare diseases. And then creating European reference networks linking centres of expertise and professionals in different countries to share knowledge and identify alternative treatment options.

The EU has to continue to support research into rare diseases through Horizon 2020, in the EU framework programmes for research and innovation and the European Platform on Rare Diseases Registration, which was mentioned by the honourable Commissioner.

Rare diseases are the field where you can ensure progress, creating synergy in research and innovation and let’s work for it.

  Ivan Jakovčić, u ime kluba ALDE-a. – Poštovani predsjedavajući, gospodine povjereniče, statistike govore da skoro 10 % europskog stanovništva boluje od raznih rijetkih bolesti, a kojih ima i preko pet tisuća.

Kaže se da je rijetka bolest onda kada od nje ne boluje više od jedne osobe na njih dvije tisuće. Međutim, to je samo statistika. Sudbine su ono što je naša uvažena kolegica Meissner rekla, sudbine su ona tragedija našeg stanovništva i sudbine su ono za što mi ovdje u ovom Parlamentu moramo brinuti i govoriti. Zato, prijedloge koje smo djelomično čuli, mislim da trebamo itekako uvažiti.

Vi ste spominjali e-zdravlje ili e-medicinu. Mi bismo trebali imati, osim ove mreže o kojoj govorite, mi bismo trebali ne samo na nacionalnom nivou nego i na regionalnim nivoima imati centre, imati mogućnost zajedničke centralne europske baze podataka, gdje bi netko iz moje Pule ili, ne znam, iz Rige ili Tallina, mogao zaista negdje u nekom dijelu Europe biti prisutan, imati informaciju i znati o čemu se radi. Dovoljno rano, naravno, jer to je ono što je najbitnije.

Dakle, mislim da su ti prijedlozi o kojima razgovaramo ovdje danas nešto što sigurno može doprinijeti da se borimo protiv rijetkih bolesti. Ono što bih vam htio predložiti gospodine povjereniče je nešto što nije ovdje izrečeno. Mislim da je Europska unija zrela, Europska komisija s Vama na čelu, budite Vi taj koji će biti predvodnik svjetske koalicije za borbu protiv rijetkih bolesti.

Uključite Svjetsku zdravstvenu organizaciju, uključite druge bogate države! Danas smo ovdje glasali za Sporazum o slobodnoj trgovini s Japanom, uključimo Japan u borbu za svladavanje rijetkih bolesti, uključimo SAD, uključimo bogate zemlje, uključimo sve one zemlje koje to žele! Što nas više bude, to ćemo biti jači u suzbijanju rijetkih bolesti jer ako 10 % stanovništva ili skoro 10 % stanovništva Europske unije pati od rijetkih bolesti, na svjetskom nivou to vjerojatno može biti samo još i više, pa u tom kontekstu mislim da bi bilo jako dobro da Vi preuzmete tu ulogu i da Europska unije bude predvodnik borbe protiv rijetkih bolesti na svjetskom nivou.

  Paloma López Bermejo, en nombre del Grupo GUE/NGL. – Señor presidente, existen más de 6 000 enfermedades raras que afectan a 30 millones de ciudadanos europeos. Muchas veces son crónicas y ponen en riesgo la vida de quienes las padecen. Como dice Eurordis, las enfermedades raras son poco frecuentes, pero los pacientes de enfermedades raras son muchos. También son muchos los problemas con los que se encuentran: entre otros, falta de acceso a diagnósticos e información correcta; consecuencias sociales, educativas, laborales, de ocio, etc.; desigualdad en la disponibilidad de tratamientos y alto coste de los medicamentos.

La lista de problemas es muy larga, pero las soluciones se reducen a dotar de recursos suficientes para realizar políticas públicas de salud. El refuerzo de la sanidad pública es imprescindible porque es la única garantía de que todos los pacientes puedan tener acceso a diagnósticos y tratamientos en igualdad de condiciones y no dependiendo de los recursos económicos de las personas o familiares. Porque esto, precisamente, genera una desigualdad adicional.

Es imprescindible, asimismo, dotar de recursos a la investigación, y —vuelvo a insistir— tiene que ser pública: no podemos dejar en manos de la investigación privada, precisamente por los costes que genera, la solución de estos problemas. Y recursos también para la atención no médica: para dependencias, apoyo familiar, apoyo social, para rehabilitación y un largo etcétera.

Tenemos en nuestras manos la posibilidad de promover estas políticas de carácter público, necesarias para que los pacientes no tengan que sufrir, además del aislamiento y la vulnerabilidad, la desigualdad y la indiferencia.

  Ana Miranda, em nome do Grupo Verts/ALE. – Senhor Presidente, Senhor Comissário, as doenças raras enquanto assunto de saúde pública preocupam-me como política. Uma das maiores confusões é o conceito de raridade. As doenças raras são raras, mas as pessoas com doenças raras são muitas na Europa.

Conheço muitas famílias que pagam custos elevados, que têm de fazer mesmo campanhas nos meios de comunicação para falar, para pedir dinheiro para pagar esses tratamentos. É duro para essas famílias; sentem-se sós.

Uma doença pode afetar apenas um punhado de doentes, mas outra pode chegar a atingir

300 000 pessoas em conjunto ou 10% da população da Europa.

A Comissão reconhecia que os afetados se encontram particularmente isolados e vulneráveis. O que é importante, como disse o Sr. Comissário, é codificar, inventariar essas doenças raras, investigar e ampliar os centros especializados em redes europeias de referência para doenças raras.

E também, para nós, precisamos de mais fundos, possibilidades para cooperar nas fronteiras, para cooperar de um país para outro e ter esses tratamentos adequados. Também para os medicamentos órfãos e, a nível europeu, mais fundos públicos para que estas pessoas sejam tratadas com igualdade e não fiquem isoladas nem com esta vulnerabilidade.

  André Elissen, namens de ENF-Fractie. – Voorzitter, laat ik beginnen te zeggen dat ik de betrokkenheid en de inzet van collega mevrouw Meissner begrijp en waardeer. Waarschijnlijk zijn er weinig onderwerpen in dit Parlement waarover zoveel overeenstemming is. Natuurlijk verdienen de tientallen miljoenen mensen in de Europese Unie die dagelijks kampen met de gevolgen van een zeldzame ziekte de allerbeste zorg. Natuurlijk moet iedereen een correcte en tijdige diagnose kunnen krijgen. En natuurlijk moet iedereen toegang hebben tot betaalbare medicijnen. Niemand zal dat tegenspreken, ook ik niet.

Maar, Voorzitter, zo werkt het helaas niet. Er zullen keuzes gemaakt moeten worden en die keuzes horen thuis in de lidstaten. Het is aan de Nederlanders om te bepalen hoe de gezondheidszorg in Nederland wordt vormgegeven. Of een bepaald medicijn wordt vergoed door een zorgverzekeraar, is een kwestie die thuishoort in het nationale parlement. De hoogte van de zorgpremie, de invulling van de zorg voor ouderen, kortom het hele zorgstelsel is een nationale bevoegdheid en dat moet zo blijven.

Er zijn 28 lidstaten met 28 verschillende zorgstelsels. Het is een slecht idee om te proberen deze systemen te integreren in iets wat uiteindelijk moet leiden tot één groot Europees zorgstelsel. Want, Voorzitter, één groot Europees zorgstelsel is het uiteindelijke doel van de vele eurofielen hier in het Parlement. En dat moet ten koste van alles worden voorkomen. Europa bestaat uit verschillende naties, verschillende volkeren. Ieder land, ieder volk heeft zijn eigen tradities en kenmerken. Het is onzinnig om te denken dat er één model kan bestaan dat op alle lidstaten van toepassing is. Zorg is maatwerk. Laat het daarom over aan de lidstaten.

Voorzitter, het is natuurlijk wel een goed idee om internationaal samen te werken. Niet vanuit de ivoren torens in Brussel en Straatsburg, maar vanuit de lidstaten. Vanzelfsprekend moedigen wij samenwerking aan, bijvoorbeeld tussen academische ziekenhuizen, wetenschappers en de Wereldgezondheidsorganisatie.

  Vytenis Povilas Andriukaitis, Member of the Commission. –Mr President, let me react to the last speaker.

I can tell you that if you are in the Netherlands, you have ten people with a rare disease. You have no clinicians, no professors, no expertise or possibilities to treat those ten people. I spent more than 20 years in the field of treatment. This means that you can immediately ask in the EU: ‘where is the centre of knowledge, expertise, diagnoses and the possibility to provide assistance and treatment?’

It is also about a second possibility. Let me address the issues raised by Mr Jakovčić. This is a very good idea and we are moving in such a direction. The European Reference Network is like the backbone that can help us to join together different universities, institutes, researchers, different developers of treatment, especially in advanced therapies, or so called precision therapies.

Of course, we are now trying to encourage Member States to sign the One Million Genomes Agreement because real diseases are genetically—driven diseases.

This means that we can build a ‘collider’ of systems at EU level, joining different actors and being a leading entity worldwide, developing new treatments, new devices, new diagnostic tests, and helping people worldwide to stay alive.

Cooperation here with different actors is welcomed and we are doing as such. Big data needs big actors and it is very important to understand this. The different national registers are too weak to address the issues we are now facing and I am very keen to promote such possibilities to build networks – a ‘big collider’ – in bringing different actors together and establishing mission—driven initiatives on different rare diseases cases. This is brilliant.

On the Joint Procurement Agreement, you know that we have in our hands a Joint Procurement Agreement which allows us to encourage Member States to cooperate, especially knowing that treatment and new medicines are very costly and only economies of scale can help us to guarantee access to treatment. We are also in favour of not discriminating against patients – they could be in the Netherlands, Lithuania or in Cyprus – access to innovative treatment is a universal human right and we need to do more to achieve this right. It doesn’t matter whether a patient is in Malta or in Finland. We should speak about and think about those issues.

It’s clear that no single country has the knowledge or capacity to tackle these diseases alone. If we work as individuals, patients will suffer from a lack of diagnosis and delayed or ineffective treatment. But together we can share best practices and expertise. We can coordinate resources. We can pool data and act more efficiently – more effectively and more decisively. Patients across Europe will benefit.

So rare diseases are a prime example of the power of cooperation. This is true for research support, registers, codification, and in particular the European Reference Network.

But we also need to make sure that patient groups and NGOs maximise the potential of the EU’s health policy platform. Access to medicine is one of the challenges facing patients suffering from rare diseases. We are aware of this. Indeed, access to healthcare is one of my priorities and I ask Member States to cooperate. Now we have different Member States who have decided to cooperate. There is the Valletta Group cooperation – I am speaking about access to treatment. Benelux, the Netherlands, Belgium, Luxembourg and Austria, they are cooperating. We are now moving in this direction. Soon I should have the results also of an analysis of EU orphan medicines legislation. This analysis will help us ensure that patients have access to innovative medicine and medicinal products when they need them.

Let me finish by assuring you that the Commission will continue its support for patients suffering from rare diseases. That commitment is reflected in the Commission’s proposals – in particular the health strand of the European Social Fund+ and the Horizon Europe framework programme.

I would now ask Parliament to support the Commission’s proposals in the next multiannual financial framework.

  President. – Commissioner, thank you very much indeed for your response.

The debate is closed.

Written statements (Rule 162)

  Dominique Bilde (ENF), par écrit. – Les maladies rares, qui ne sont d’ailleurs pas si rares puisqu’elles concernent trente millions d’Européens, restent le parent pauvre de la recherche. Les progrès sont certes incontestables: il faut saluer l’action concertée des Européens, notamment au travers de la base de données Orphanet. De même, la recherche privée se mobilise davantage que par le passé, sans compter les promesses de la thérapie génétique. Les défis sont toutefois de taille: ainsi, Le Figaro relevait-il que deux tiers des 30 000 enfants qui consultent à l’Institut des maladies génétiques n’ont toujours pas reçu de diagnostic. Nombre d’entre eux (35 %) mourra avant l’âge d’un an, alors même que la durée moyenne d’obtention d’un diagnostic est de quatre ans. C’est donc un véritable chemin de croix qui s’impose aussi bien aux patients qu’aux familles, qui doivent souvent faire face seuls à l’errance diagnostique ainsi que, d’une manière générale, à une charge émotionnelle et matérielle considérable. Il est urgent de poursuivre nos efforts en faveur d’une recherche européenne, notamment au travers de programmes tels que E-Rare, tant une action commune semble dans ce cas particulièrement indiquée.

  Urszula Krupa (ECR), na piśmie. – Dane dotyczące chorób rzadkich są niepełne i choć statystycznie na te choroby może cierpieć od 5 do 6 % społeczeństwa, jednak zastanawiające są szacunki dotyczące takich krajów jak Francja czy Niemcy o populacjach dwukrotnie większych niż Polska, gdzie występuje podobna liczba około 3 mln szacowanych chorób. W poszczególnych krajach występują znaczne różnice zarówno w testach wykonywanych u noworodków, jak i w możliwościach diagnostycznych i leczniczych, co wydaje się związane z możliwościami finansowymi państw, w których zapewnia się znacznie lepszą opiekę socjalną i psychologiczną oraz refundacje, co dotyczy zwłaszcza Francji i Niemiec czy Wielkiej Brytanii, gdzie jednak w Anglii refundowane jest 60 leków sierocych, natomiast w Irlandii refundacja dotyczy tylko 12. Według nie tylko polskich lekarzy, leczenie chorób rzadkich i refundacja jego kosztów jest problemem ogólnoświatowym, praktycznie nigdzie jeszcze nierozwiązanym chociaż przełomem było potraktowanie wszystkich chorób jako jednej całości i konieczna jest centralna koordynacja zarówno informacji, badań i finansowania, nie tylko wskazania w art. 12 i 13 dyrektywy z 2011 roku. Wydaje się, że wiele zależy od zaangażowania Komisji Europejskiej i propozycji regulacji, jakie powinny dotyczyć nie tylko badań naukowych, ale i procedur terapeutycznych, a szczególnie finansowania bardzo kosztownych leków sierocych.