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Wednesday, 24 November 2021 - Strasbourg Revised edition

16. A European Action Plan Against Rare Diseases (debate)
Video of the speeches
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  President. – Good evening, colleagues. The sitting is resumed.

The next item is the debate on the Commission statement on a European Action Plan Against Rare Diseases (2021/2940(RSP)).

 
  
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  Stella Kyriakides, Member of the Commission. – Madam President, honourable Members of Parliament, I want to thank you for putting European action and cooperation on the agenda today on the issue to do with rare diseases, and I want to particularly convey my thanks to honourable Members Ries and Bilbao Barandica for this very important debate.

For ourselves and for those of us who know the area of rare diseases, we can understand that, given the limited number of patients and the scarcity of relevant knowledge and expertise, EU action is even more important in rare diseases, and this is where there is real added value. That is why we have been active in this area for over 20 years.

If I may just share with you, I have worked in the area of rare diseases with children, and I know the need to raise awareness on this, but I also know the loneliness faced by families when they have to deal with a child with a rare disease and not knowing where to turn. So it’s time we developed a wide range of activities to support the Member States and EU level cooperation.

The EU has really done a great deal over the last 20 years, and its response has focused on some key elements. First of all, promoting the development of national rare diseases plans and strategies, which need to be in place; establishing and supporting European Reference Networks (RFNs), which are possibly one of the best examples of EU cooperation, action and support; supporting the definition and inventory of rare diseases, which broadens the knowledge base; providing incentives for the development of medicines for rare diseases; and empowering patient organisations.

The EU Orphan Regulation has clearly stimulated research and development of medicines for rare diseases, but when we look at the numbers, we can see that more needs to be done, since 95% of rare diseases still do not have treatment, and orphan medicines are not accessible to patients equally in all Member States. That is why, as part of the EU Pharmaceutical Strategy, an important report will be voted upon in this House today. We will review the current rules to foster innovation, especially where it is needed most, and to make medicines more available across the EU.

The determination to ensure that all rare disease patients, children and adults, have access to the best knowledge, diagnosis and treatment is at the very core of our efforts. The European Reference Networks are key to achieving this goal. It’s a truly innovative system, a model of virtual healthcare and collaboration, bringing together the most specialised centres across the EU to speed up diagnosis, to improve standards of care and to generate knowledge for patients living with rare and complex diseases. Their guiding principle is quite a simple one: that it is the knowledge that travels, not the patient. The patient doesn’t have to travel, bringing those who are rare together, and this is really important.

Funding is key, and the EU4Health Programme includes the European Reference Networks as one of its specific objectives and will contribute to further evolution of the system. I also want to take this opportunity to emphasise the substantial EU investment into research on rare diseases, with more than EUR 1.8 million allocated to 320 multinational consortia. Horizon Europe, including the proposed European Partnership on rare diseases, will continue to support this effort.

Early next year, I will be here in this plenary to present to you a proposal to launch a European Health Data Space. A lack of data is a crucial challenge for rare diseases, and the European Health Data Space will make it easier to gather data from all corners of the EU and to make it available to researchers working on innovative treatments. Many rare disease patients also actively want to share their own data for research and development for this reason. The European Health Data Space will make it much easier for them to do so.

In 2022, we will conclude the evaluation of the Cross-border Healthcare Directive, including its provisions on the cooperation on rare diseases and European Reference Networks. It will provide us with important evidence and inform our assessment of the current approach to rare diseases.

There will be two additional processes that will inform that assessment. The evaluation of the Third Health Programme, which funded key actions in the field of rare diseases, and the outcomes of the ‘RARE 2030’ Pilot Project, which has been proposed by this House. We’re going to be building on all these collective lessons, and the conclusions will then be discussed in the Steering Group on Promotion and Prevention, and with all relevant stakeholders at the end of next year.

If appropriate, we will revise our policy framework for rare diseases in 2023, in addition to the upcoming reform of legislation for medicines for those with rare diseases and for children at the end of next year. I’m a believer that we need to evaluate and where we need to change, to bring about change.

Honourable Members, there are concrete plans that will improve our approach to rare diseases. I am really looking forward to hearing you today, to having the opportunity to discuss this with you, and to really bringing together all the expertise and knowledge. Because working together with all the stakeholders – including patients, families, doctors and others – we can really bring tangible benefits to citizens. Thank you very much, and I look forward to hearing your views.

 
  
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  Ewa Kopacz, w imieniu grupy PPE. – Pani Przewodnicząca! Pani Komisarz! Choć mowa dzisiaj o chorobach rzadkich, to cierpi na nie blisko 36 mln Europejczyków. 75 % z nich zaczyna chorować w dzieciństwie. 80 % tych chorób ma podłoże genetyczne. 50 % pacjentów z chorobą rzadką stanowią dzieci. Mówię to jako lekarz pediatra i jako były minister zdrowia w swoim kraju, który rozpoczął refundację leków na choroby rzadkie.

Choroby te są poważne, przewlekłe, postępujące, bardzo często mające swój tragiczny finał. W Europie wciąż wiele osób rodzi się, żyje i umiera z powodu chorób rzadkich, bez postawionej diagnozy. Jedna czwarta chorych nie ma dostępu do diagnozy przed ukończeniem piątego roku życia. 95 % pacjentów nie ma dostępu do leczenia. Nie mają wystarczającego dostępu do opieki medycznej i społecznej, która zapewniłaby im lepszą jakość życia. Obserwujemy brak specjalistów i ośrodków eksperckich dla poszczególnych chorób rzadkich. A jednak te ośrodki mogłyby powstać. Ośrodki, które by działały zgodnie z ujednoliconymi standardami opracowanymi dla państw członkowskich przez Komitet Ekspertów Unii Europejskiej do spraw Chorób Rzadkich.

Najbardziej istotna po rozpoznaniu choroby rzadkiej jest możliwość dostępu do nowoczesnych opcji terapeutycznych, które niestety nadal są zbyt drogie. W moim kraju, w Polsce, farmakoterapia jest dostępna jedynie dla 3-5 % pacjentów. Pozostałe 95 % chorych korzysta z leczenia zachowawczego i rehabilitacji.

Pani Komisarz, świetnie spisaliśmy się podczas pandemii. Potrafiliśmy negocjować z firmami farmaceutycznymi dobre ceny na szczepionki. Dzisiaj bardzo proszę o wzięcie pod uwagę stworzenia platformy zakupowej, w której to my z pozycji siły moglibyśmy negocjować z firmami dobre ceny na już wynalezione leki na choroby rzadkie. Chcę, aby taki sam dostęp do tych leków miały dzieci i dorośli chorzy na choroby rzadkie ze wschodu, jak i zachodu Europy, z tych biedniejszych i bogatszych krajów. Oni na to zasługują.

 
  
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  Jytte Guteland, för S&D-gruppen. – I EU är vi nästan 450 miljoner invånare, men det skiljer väldigt mellan medlemsländerna. I vissa medlemsländer bor drygt 80 miljoner människor, i andra kan det handla om en halv miljon. Och en vård i världsklass kräver både resurser, erfarenhet och specialistkompetens. Att varje medlemsstat ska kunna bygga upp den kompetensen för de allra mest sällsynta sjukdomarna är inte lätt.

Det är därför oerhört viktigt att vi koordinerar oss på EU-nivån, för att kunna hjälpa de minsta länderna och dem med de allra mest sällsynta sjukdomarna. Med gemensam upphandling kan vi pressa ner priserna och bättre säkra tillgängligheten på sällsynta mediciner, och med gemensamma resurser kan vi bygga en erfarenhetsbank som kortar ner tiden till rätt diagnos och till rätt åtgärder.

Det ska inte behöva ta fem år att ställa rätt diagnos, och vi måste hitta behandlingar för fler än de i dag sex procent som får en ordentlig, bra behandling för sällsynta sjukdomar. Jag hoppas att EU-kommissionen tar till sig revisionsrättens slutsatser och följer parlamentets uppmaning att ta fram en konkret handlingsplan med tydliga mål. Det är vi skyldiga de 30 miljoner människor som redan i dag lever med en sällsynt sjukdom.

 
  
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  Frédérique Ries, au nom du groupe Renew. – Madame la Présidente, Madame la Commissaire, je vous parle ce soir avec à l’esprit cette maman, l’une de ces très nombreuses mamans que j’ai rencontrées hier matin au Parlement européen. Elle travaille au service de recherche du Parlement et son fils est atteint d’une maladie métabolique très rare. Alors dans son cas, le diagnostic a été posé et c’est une victoire. Le traitement existe et c’est très rare. Vous l’avez rappelé, Madame la Commissaire, 95 % des maladies rares sont encore sans traitement. C’est après que, pour cette maman, ça se complique. Cette famille belge a dû déménager en Allemagne pour trouver le seul spécialiste apte à leur prescrire et leur donner ce traitement.

La priorité, aujourd’hui, est donc de sortir de ce fonctionnement en silos pour réfléchir à l’échelle d’une vie, celle des patients et de leur famille. Poser le diagnostic, assurer le traitement et faciliter l’accès aux soins, c’est un tout indivisible et ces patients, vulnérables entre tous, y ont droit. Ce manque de cohérence entre les politiques de santé européennes a été dénoncé par Rare 2030, un projet pilote que j’ai lancé en 2017 avec plusieurs collègues pour imaginer, avec les chercheurs et les patients, les politiques du futur pour les maladies rares.

Cette expérience inédite de deux ans, ce sont vos services qui en ont assuré la gestion, Madame la Commissaire, et le diagnostic est sans appel: il faut un plan d’action européen pour unifier nos politiques et leur apporter une cohérence à moyen et à long terme. Les planètes sont alignées; le Parlement est avec vous et les États membres sont avec vous. Repousser la question en 2023, c’est en réalité la renvoyer à la prochaine Commission.

Et c’est une autre maman belge, Isabelle, qui va conclure pour moi mon intervention: «Il n’y a pas de plan B, me disait-elle, il n’y a pas de vie B pour un enfant qui se bat contre une maladie rare». En d’autres termes, c’est maintenant que nous devons agir.

 
  
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  Tilly Metz, on behalf of the Verts/ALE Group. – Madam President, I think that the European Health Union is ready for an action plan on rare diseases. And as you know, I’m a strong advocate for rare diseases patients, especially the youngest concerned by them. About 75% of rare diseases affect children, and today paediatric cancers are unfortunately still the leading cause of death in children aged above one year, in Europe.

Rare cancer patients often face numerous challenges, like the difficulty in accessing timely diagnosis and specialised health care services, adequate treatments or social services. In order to tackle this issue, we should have a more holistic approach to rare diseases and should take into account the social aspects of a patient’s life. Rare disease patients often feel alone, and their quality of life is greatly reduced. People living with rare diseases should have the right to live a normal life, and we need to create equal opportunities, as well as social protection and inclusion for patients and their families.

As a former teacher, I am particularly attached to the educational aspect of children. Schoolkids with rare diseases still face inaccessibility to facilities, non-adapted teaching methods and are often behind in their education programme due to the frequency of medical appointments and their special needs. So a new action plan would therefore address areas such as research, diagnosis, treatment, social care, education, inclusion, as well as data collection compliant with the GDPR. Data is crucial and we should leverage European reference networks in the area of rare diseases to optimise data for patients and social benefit.

As a shadow for the upcoming European health status-based file, I am calling for a data space that is seamlessly connected to the European reference networks so that we can create better health care passwords and clinical research, because unfortunately, data on rare disease patients are still scattered amongst different healthcare systems. Collecting this data could help us better understand the specific diseases and develop appropriate treatment. Ultimately, data can help us increase the quality of life of millions of people suffering from rare diseases.

 
  
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  Rosanna Conte, a nome del gruppo ID. – Signora Presidente, onorevoli colleghi, le malattie rare colpiscono 30 milioni di cittadini europei. Ad oggi le competenze e conoscenze mediche non sono all'altezza. Le offerte di assistenza sanitaria sono inadeguate e gli investimenti nella ricerca sono limitati. Pensiamo addirittura che l'ultima politica generale dell'Unione europea su tali malattie risale al 2009.

Basterebbero queste indicazioni per incoraggiare, nei tempi più rapidi possibili, un piano europeo contro le malattie rare, a tutela di tutti quei pazienti orfani dei sistemi sanitari nazionali, che troppo spesso non garantiscono loro diagnosi accurate e trattamenti adeguati.

È fondamentale che il nuovo piano sia strutturato sulla base di un coordinamento con la nuova strategia europea per l'assistenza sanitaria a lungo termine, prevista per la seconda metà del 2022. Le parole chiave sono inclusione e accessibilità, ma perdono il loro senso se manca una forte strategia condivisa basata su un reale coinvolgimento dei pazienti, che possono e devono contribuire al meglio in questo processo. Sono loro i primi a conoscere i veri bisogni e le carenze attuali.

E non dobbiamo dimenticare il ruolo indispensabile degli assistenti ai pazienti, i care givers, figure fondamentali che troppo spesso non sono tutelati o riconosciuti giuridicamente.

Insomma, il confronto deve essere con i pazienti e non sui pazienti, e tutti quei finanziamenti a sostegno della lotta alle malattie rare non dovrebbero essere indirizzati solo a istituti e strutture, ma devono sostenere processi di aiuto all'autonomia delle persone affette da malattie rare e disabilità.

L'attenzione è alta, ma è necessaria maggiore concretezza. Oggi l'obiettivo è ancora lontano.

 
  
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  Joanna Kopcińska, w imieniu grupy ECR. – Pani Przewodnicząca! Pani komisarz! Popieram działania dotyczące rewizji planu działania Unii Europejskiej w zakresie chorób rzadkich, którego nadrzędnym celem jest to, aby do 2030 roku żaden pacjent dotknięty chorobą rzadką nie został pozostawiony bez opieki. Wydaje się rozsądne, aby europejskie sieci referencyjne oraz istniejące narodowe plany i strategie krajowe zyskały nową synergię zważywszy na fakt, że na płaszczyźnie europejskiej nie dysponujemy kompleksowo zaktualizowanym podejściem do tego problemu. Dlatego zgodnie uważam, że należy doprowadzić do wtórnej klasyfikacji rejestru chorób rzadkich oraz przemyśleć metody nowego wdrażania diagnostyki chorób rzadkich poprzez ciągłe upowszechnianie badań przesiewowych i testów genetycznych, a zwłaszcza należy przemyśleć funkcjonowanie sieci systemów ośrodków referencyjnych.

Opieka zdrowotna dla pacjentów z chorobami rzadkimi musi dotyczyć zarówno zwiększenia dostępności wysoko specjalistycznej opieki medycznej, jak i dostępności terapii lekowych wraz z aktualizacją dostępnych technologii.

 
  
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  Kateřina Konečná, za skupinu The Left. – Paní předsedající, vážená paní komisařko, přiznejme si to, potřebujeme plán pro třicet milionů našich spoluobčanů žijících se vzácným onemocněním v Evropské unii na další desetiletí. Ten poslední je totiž dvanáct let starý, a to je ostuda. Potřeby pacientů se vzácným onemocněním vyžadují větší úroveň přeshraniční spolupráce v oblasti zdravotní péče. Navzdory tomu, že existuje směrnice o přeshraniční zdravotní péči, která má takovou spolupráci usnadnit, přetrvávají překážky, které nejen znesnadňují přístup ke zdravotní péči v jiné zemi Evropské unie, ale nechávají na straně pacienta administrativní, finanční a emoční zátěž. Je třeba také podpořit zlepšení v oblasti diagnostiky, dat, výzkumu, jejich integrované péče a léčby. Potřebujeme zavést masivní screeningy novorozenců, abychom jim dali šanci žít normální život. Je zcela jasné, že bez celounijního vedení se tento boj prostě neobejde. Jsem tak velmi ráda, že se nadcházející předsednické trio v Radě, v čele s Francií, Českou republikou a Švédském, k otázce nového plánu staví kladně.

Ráda bych se však ještě krátce zastavila u financování evropských referenčních sítí, o kterých jsme tady dnes tolik slyšeli. Přibližně před měsícem jsem na problém jejich financování upozorňovala na Výboru pro životní prostředí, veřejné zdraví a bezpečnost potravin. Evropské referenční sítě jsou nejúspěšnějším projektem evropské zdravotní politiky. I přesto, že jsme pro jejich financování dali jasná pravidla v rámci programu EU4Health, čelí dnes finančním potížím a hrozbě zániku. Je nutné, paní komisařko, co nejdříve reagovat a poskytnout jim chybějící peníze na rok 2022. Ono totiž nejde o to, co bude po roce 2023, kde ty peníze asi máme, ale je velký problém s tím, že my se potřebujeme udržet financování právě v roce 2022. Já vás prosím, udělejte všechno proto, aby tento zářný projekt úspěšné spolupráce nemusel zmizet jen proto, že bude chybět řádově pár milionů eur.

Děkuju všem, kteří pomáhají v péči o pacienty, především rodinám a lékařům, kteří se snaží najít nejlepší cestu pro jejich léčbu.

 
  
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  Λουκάς Φουρλάς (PPE). – Κυρία Πρόεδρε, κυρία Επίτροπε, οι σπάνιες ασθένειες δεν είναι τόσο σπάνιες. Τριάντα εκατομμύρια άνθρωποι πάσχουν από σπάνια νοσήματα στην Ευρώπη. Θα επικεντρωθώ στον ρόλο των εταιρειών. Είναι ανεπίτρεπτο να μην υπάρχουν φάρμακα για παιδιά με σπάνιες μορφές καρκίνου, γιατί δεν συμφέρει οικονομικά στις μεγάλες φαρμακευτικές εταιρείες να τα παράγουν. Πολλές από αυτές τις ασθένειες απαιτούν χρόνιες, πολυδάπανες θεραπείες. Αν θέλουμε να μιλάμε για ανθρώπινη Ευρώπη, πρέπει να εστιάσουμε σε μια ενιαία πολιτική υγείας. Οφείλουμε να στηρίξουμε τους ανθρώπους αυτούς. Η υγεία τριάντα εκατομμυρίων ανθρώπων που ζουν με μια σπάνια ασθένεια δεν μπορεί να αφεθεί στην τύχη ή στα συμφέροντα μεγάλων εταιρειών που βλέπουν τους ασθενείς απλώς σαν νούμερα. Πρέπει να διασφαλίσουμε την πρόσβαση σε θεραπείες για όλους τους Ευρωπαίους πολίτες. Κυρία Επίτροπε, ευχαριστούμε που ήσασταν δίπλα μας. Η ανθρώπινη ζωή δεν θυσιάζεται στο βωμό του κέρδους των φαρμακευτικών εταιρειών και οφείλουμε να θέσουμε τέρμα σε αυτές τις συμπεριφορές.

 
  
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  Sara Cerdas (S&D). – Senhora Presidente, Comissária, Colegas, de acordo com os novos desenvolvimentos a nível de investigação, ciência e tecnologia, as atuais políticas europeias em matéria de doenças raras encontram-se desatualizadas. Apesar da sua baixa prevalência, as doenças raras afetam cerca de 30 milhões de europeus. São 30 milhões de pessoas sem respostas e com necessidades em saúde não correspondidas.

Precisamos, assim, de um novo Plano Europeu de Ação para as Doenças Raras. Este novo plano de ação deve assentar em objetivos e metas bem delineadas e seguir as últimas recomendações. Deve unir as políticas e iniciativas a nível nacional, regional e local sobre doenças raras num só quadro. Deve também tirar maior proveito da era digital, dos dados em saúde para mais informação, melhor investigação, mais ferramentas de diagnóstico e melhores tratamentos. Um novo plano europeu de Ação para as Doenças Raras transversal que nos torne mais competitivos ao nível da inovação e da investigação, de novas terapias e tecnologias e que permita responder adequadamente às necessidades atuais, tais como os diagnósticos mais precoces e terapêuticas mais adequadas.

Em linha com a recente resolução das Nações Unidas, precisamos de uma estratégia europeia que vá para além da prestação de cuidados de saúde, que olhe para a saúde em todo o seu pleno, incluindo as componentes sociais e económicas, e que responda, assim, aos inúmeros desafios vividos por aqueles com doença rara na União Europeia. Tem de ser um novo plano de ação que não deixe nenhum cidadão para trás. São 30 milhões de razões multiplicadas pelos seus familiares e cuidadores.

 
  
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  Véronique Trillet-Lenoir (Renew). – Madame la Présidente, Madame la Commissaire, chère Stella, chère Frédérique, merci d’avoir organisé ce débat essentiel. Être atteint d’une maladie rare, c’est être confronté au risque de diagnostic tardif, à des difficultés d’accès aux traitements, aux essais cliniques, dans un sentiment de perte de confiance, d’incertitude et d’isolement. Un plan d’action efficace contre les maladies rares se doit d’être européen parce que nous avons besoin de cette masse critique de compétences.

L’Union européenne a créé le précieux modèle des centres européens de référence permettant l’échange de pratiques entre spécialistes, la réponse aux questions posées par des médecins souvent démunis, l’accès à des consultations de qualité, à des bases de données, à des patients experts. Conservons cet acquis et renforçons-le, ainsi nous ne laisserons personne de côté. Ces centres ont besoin d’un budget à la hauteur des attentes des 30 millions de patients atteints d’une maladie rare dans l’Union européenne. Madame la Commissaire, financez-les.

 
  
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  Alessandra Basso (ID). – Signora Presidente, onorevoli colleghi, una malattia rara ha solo, dal punto di vista della visibilità, uno svantaggio, vale a dire quello di colpire solo 5 persone su 10 000 nell'Unione europea. Questo non la rende meno dolorosa, ma semplicemente meno statisticamente rilevante.

Sin dai tempi di Ippocrate curare i malati è stata una priorità e la storia del nostro continente ha visto centinaia di uomini e donne che hanno dedicato la loro vita allo studio per sviluppare la medicina. In Europa siamo sempre stati in prima linea dal punto di vista medico e farmaceutico, ma non abbiamo fatto abbastanza.

Abbiamo delle eccellenze riconosciute in tutto il mondo e da sempre supportiamo la ricerca, come ha dimostrato lo sviluppo dei vaccini per fronteggiare la COVID-19, ma non è ancora abbastanza. Ciò che manca sono i fondi, e non possiamo aspettare una pandemia per ricordarcene.

Ci sono famiglie le cui vite dipendono dalle nostre decisioni ed è per questo che dobbiamo fare tutto ciò che è in nostro potere per aiutarle. Questo significa creare network informativi che migliorino la diagnostica e affrontare il problema dei medicinali orfani.

Dobbiamo investire ancora più risorse e dare un aiuto concreto alla sperimentazione di nuove cure e nuovi farmaci, per tutelare al massimo il diritto alla vita dei nostri cittadini e garantire la loro integrazione nella società, perché una vita non è solo un numero e il dolore non è qualcosa che si pesa.

 
  
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  Anna Fotyga (ECR). – Madam President, to successfully combat rare diseases, we have to overcome many and enormous difficulties. Yet, I acknowledge progress achieved until now.

The existing European Joint Programme on Rare Diseases is an example of a very good European cooperation, bringing together all stakeholders engaged in this area. I think that a revised action plan is necessary.

In particular, Commissioner, I appeal to you and to your other colleagues to ensure broadening the scope of EU finances, in particular to those who are in action – parents, caretakers, as well as doctors directly engaged in providing treatment to patients of rare diseases.

 
  
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  Sandra Pereira (The Left). – Senhora Presidente, muitos milhares de pessoas na União Europeia são afetadas por doenças raras, exigindo uma resposta abrangente. Tenho acompanhado as dificuldades que passam as cerca de 500 famílias que em Portugal são afetadas pela paramiloidose amiloidótica familiar, também conhecida como a doença dos pezinhos, uma doença rara hereditária e degenerativa para a qual escasseiam respostas.

Este é, aliás, um exemplo paradigmático. Existindo um medicamento com elevado grau de sucesso para o tratamento da doença, a lógica mercantil por trás da sua distribuição faz com que este seja completamente inacessível a muitas destas famílias. Só o reforço das capacidades de intervenção e investimento público que garanta um serviço nacional de saúde universal e acessível, capacidades de investigação científica que desenvolvam tratamentos, capacidades de produção e distribuição de medicamentos que não sujeitem os doentes à voragem das multinacionais farmacêuticas, pode responder às necessidades e aos anseios destes doentes.

Para além do necessário apoio aos pais cujos filhos são portadores de doenças raras, é preciso promover iniciativas e programas que visem eliminar o estigma que algumas dessas doenças, geralmente por desconhecimento, causam nos outros. Deste plano de ação da União Europeia, sempre tão apologista da liberalização e do desinvestimento, dificilmente encontraremos a resposta necessária.

 
  
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  Maria da Graça Carvalho (PPE). – Senhora Presidente, Senhora Comissária, as doenças raras afetam mais de 30 milhões de pessoas na União Europeia e mais de 600 mil em Portugal. Existem cerca de 6 mil doenças raras, cada uma atinge poucas pessoas, mas no seu conjunto representam muitos doentes e não estamos a dar-lhes a resposta digna. O diagnóstico pode demorar até cinco anos. Os tratamentos são escassos, abrangendo apenas 6% das doenças.

Em 2009, a União Europeia adotou a estratégia para as Doenças Raras que se manteve até hoje. Eu própria, na altura como relatora do programa específico Horizonte 2020, incluí as doenças raras nas prioridades, o que possibilitou o Joint Programme que hoje existe. Registámos progressos, mas estamos aquém das expectativas. Precisamos de rever a estratégia o mais tardar até 2023.

Na minha opinião, há dois pontos que temos que salvaguardar: o reforço da investigação científica, o reforço da cooperação nessa investigação, não apenas na Europa mas a nível global. Precisamos de criar um ecossistema de inovação forte na área das doenças raras e uma nova estratégia para a partilha de dados na área da saúde. A troca de informações e de conhecimento é particularmente importante no combate destas doenças.

 
  
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  Alessandra Moretti (S&D). – Signora Presidente, onorevoli colleghi, signora Commissaria, per affrontare la questione delle malattie rare e della strategia europea da realizzare in questo senso si devono tener presenti due aspetti: il problema della disponibilità dei farmaci e delle terapie adeguate e, allo stesso tempo, il problema dell'accessibilità e il diritto di cura di tutti i pazienti, e in tutti i paesi europei, quando queste terapie siano già disponibili.

In questo contesto l'Italia, pochi giorni fa, ha approvato una legge organica e approfondita sulle malattie rare, alle quali dedica fondi e programmi sia per la ricerca che a supporto dei pazienti. Tuttavia, non può essere un tema lasciato ai singoli Stati, perché uno dei problemi fondamentali delle malattie rare è la scarsità di dati che ostacolano l'individuazione di terapie e percorsi clinici.

Ma soprattutto è necessario che l'Unione si faccia carico di un ruolo fondamentale di coordinamento, promozione di buone pratiche e, in modo ancora più deciso, di garanzia di pari diritti per tutti i pazienti affetti da malattie rare. Penso soprattutto ai bambini colpiti da diagnosi così terribili e alle loro famiglie.

Queste malattie possono essere affrontate efficacemente solo a livello europeo. Serve quindi fare del piano europeo per le malattie rare una priorità da inserire nel contesto dell'Unione europea della salute, la cui realizzazione diviene sempre più urgente, giorno dopo giorno.

Il gruppo dei socialisti è al fianco di tutti i malati e delle loro famiglie e chiediamo alla Commissione di agire tempestivamente per affrontare con misure concrete questo tema così urgente.

 
  
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  Billy Kelleher (Renew). – Madam President, we need to have a uniform new-born screening programme. We need to ensure there’s early diagnosis. We have too many mis-diagnoses, non-diagnosis, delayed diagnosis of children when they are born in the area of rare diseases. And we also have to accept that we have to have collaboration between Member States in terms of research, innovation, specialist services, medical personnel in the areas of the very rare diseases, because we simply cannot assume that every country will have the capacity, or the population base, or the expertise, or the wherewithal, or the resources, to make available the therapeutic medicines, the orphan medicines and the care that is required in every area of rare diseases.

So, this European Action Plan Against Rare Diseases is very welcome, and we have to ensure now that it is bought into by the Member States in areas of, as I said, support for people with rare diseases.

My specific area of interest is in the context of PKU (Phenylketonuria) – I am the co-chair of a cross party alliance here in the European Parliament, and we do want to bring the issue of rare diseases right to the very top and to ensure that when medicines are approved by the European Medicines Agency that every citizen in the European Union can get access to them, not based on individual Member States and their ability to pay or not. So I commend the review and I hope that we will engage positively with all Member States.

 
  
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  Tanja Fajon (S&D). – Gospa predsednica!

Danes nas je okoli 30 milijonov Evropejcev, ki imamo diagnosticirano eno od redkih vrst bolezni. Imamo 30 milijonov razlogov, zakaj potrebujemo usklajen in celosten pristop za reševanje težav, s katerimi se dnevno soočamo.

Nocojšnja razprava me kot ambasadorko za redke bolezni navdaja z optimizmom. Zakaj? Prejšnji teden je odbor Generalne skupščine Združenih narodov sprejel resolucijo o obravnavi izzivov oseb, ki živijo z redko boleznijo, in njihovih družin. To je velik korak za skupnost bolnikov, ki so prevečkrat stigmatizirani in potisnjeni na rob družbe.

Tudi študija Rare 2030, ki jo financira Unija, ugotavlja, da sedanja strategija potrebuje posodobitev. Spodbuditi moramo tudi inovacije.

Slovenci beležimo izjemen dosežek na področju presejalnega programa za novorojenčke, ki odkriva kar 40 redkih bolezni pri otroku in zagotavlja pravočasno ukrepanje.

Zagotoviti moramo, da bo Evropski akcijski načrt za boj proti redkim boleznim ena izmed prioritet tudi naslednjega predsedujočega tria Svetu Unije.

 
  
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  Ondřej Knotek (Renew). – Madam President, I am very proud that within the TRANSFORM MEP Interest Group, we help to promote the enormous potential of transformative therapies for rare disease patients. One of the key challenges today is lack of data. Methods based on the collection and use of real world data represents a possible solution.

Therefore, the European Health Data Space shall enable usage of real world data in the lifetime management of transformative therapies. Furthermore, cross-border patient data exchange and adequate funding for a European reference network are crucially important.

Madam Commissioner, let me invite you to propose an EU Rare Disease Action Plan based also on the recommendations of the Rare 2030 foresight study. This is very important, because this concerns some 30 million EU patients who are waiting for us to take action.

 
  
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  Stella Kyriakides, Member of the Commission. – Madam President, I thank you very much for all the input to this debate. In fact, you are in a sense preaching to the converted, if I may say, because I agree with the importance of this issue and with the need for us to all work together and for the action needed to be taken. Rare diseases will remain an important focus for the Commission and patients’ voices and families’ voices will continue to be central to our approach, also, in the context of the revision of the legislation on medicines for rare diseases in children, planned for the end of next year.

Mrs Kopacz, you said that all patients, no matter where they live, should have equal access to treatment, and I would agree totally that this is not negotiable. And Mrs Guteland said it’s not easy to build up expertise and competence in all Member States and that shows exactly the importance of European reference networks.

Several of you, I think it was Mrs Kopcińska and others, mentioned the importance that we support a European reference networks, and I totally agree. I’m working on this too. They deserve the support. I am working to find the solution. We will be supporting their further activities and looking how to do this within our EU4Health programme.

Mrs Ries, you mentioned Red 2030 and I think I need to just mention here that we are already looking at the outcomes of this study, and I am pleased to note that many of these recommendations of the study are already being taken on board.

Mrs Metz also mentioned the European reference networks, and several of you, and I made some notes here, spoke about rare diseases in the forms of paediatric cancers. I know that Madame Véronique Trillet-Lenoir is an expert in the area, and this is why we dedicated a specific area of Europe’s Beating Cancer Plan to paediatric cancers. And this is why we are, in conjunction with the plan, looking at the revision of the orphan and paediatric regulations, which aim to address the problems that we now face in terms of treatments and innovation. I want to assure you that all medicinal products, which could be efficacious in treating paediatric cancers, are effectively studied in children. And we would look at the experience acquired in the US with their recent change of legislation for medicines for children to address this issue.

And Mr Fourlas, we will be giving incentives to industry to boost innovation in areas where we have medical needs. But we will also impose obligations to ensure better access to medicines, to ensure that inequalities disappear within the EU.

Several of you also mentioned the issue to do with stigma and awareness and the need to really change it, and I can’t agree more with this. And Mrs Moretti also mentioned the issue to do with the (Red) 2030 project and the need to have the evaluation of this, but we need to widely consult and I just wanted to say that before deciding the way forward.

So I just wanted to say a few words in terms of where we are with the European Reference Networks. We will also grow the ERNs and these are already providing answers and diagnosis and treatment plans for more than 1.7 million patients who before only had, as many of you said, uncertainty and doubt. And in January 2022, the new ERNs will be invited to join the existing 24 networks, almost doubling their size to comprise around 1500 specialised healthcare units across the EU.

And so we are consistently moving forward with this. We’re supporting Member States in the development of their national rare disease plans. We’re working within the Pharmaceutical Strategy for Innovation and increase support for our medical needs. And as many of you mentioned, we’re preparing the proposal for the European Health Data Space and through the European Platform on Rare Diseases Registration. We’re working towards a framework to counter data fragmentation.

So, all I can say in ending is that we as a Commission and myself, as Commissioner of Health and Food Safety, we are committed. We’re committed to continuing to make concrete and tangible improvements and changes to the lives of rare disease patients, of their families, of those who work in the areas of rare disease, with commitment and dedication, as we have already been moving for the last 20 years and I look forward to working very closely with this Parliament and having the opportunity to stand before you again in the not so distant future and share with you how we have moved on with this.

 
  
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  President. – The debate is closed.

Written statements (Rule 171)

 
  
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  Ioan-Rareş Bogdan (PPE), în scris. – În prezent, sunt identificate 7 000 de boli rare, care afectează 30 de milioane de persoane numai în Europa și peste 300 de milioane de oameni din lumea întreagă. În Europa, există încă multe persoane care se nasc, trăiesc și mor cu boli rare, fără a fi diagnosticate. 95 % dintre pacienți nu au acces la tratament, nu au acces suficient la asistență medicală și socială pentru a le asigura o calitate mai bună a vieții. Pacienții cu boli rare sunt, de asemenea, afectați în mod disproporționat de probleme psihologice, financiare și sociale.

De aceea, consider că este esențial un plan de acțiune concret și cu obiective clare, care să vină în ajutorul tuturor celor care trăiesc deja cu o boală rară în prezent. Sănătatea a zeci de milioane de persoane care trăiesc în Europa cu o boală rară trebuie să fie în centrul preocupărilor noastre și este obligatoriu să facem tot ce ne stă în putere pentru a asigura accesul la tratamente pentru cetățenii europeni.

 
  
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  Josianne Cutajar (S&D), in writing. – 30 million people across Europe suffer from a rare condition: a number which accounts for 60 times the size of my own country, Malta. The findings of the Rare 2030 Foresight Study are clear: we need a new EU policy framework for rare diseases for them to remain a public health priority. We can achieve it through European actions that enhance cooperation of long-term national plans and policies while monitoring their achievement with measurable goals. Our fight is to eliminate the inequalities persons living with a rare disease and their families experience every day! From combating isolation to the possibility of obtaining special leave to take care of a member of the family, a European Action Plan should fully support the potential and wellbeing of all people living with a rare disease. We can make the difference in many ways, such as enhancing the awareness of people living with a rare disease in society as well as supporting their better access to medicine, employment and education. I urge the European Commission to act swiftly: leaving no patient and no family behind is not just a matter of fairness; it is a moral imperative.

 
  
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  Jarosław Duda (PPE), na piśmie. – Działania na rzecz osób dotkniętych chorobami rzadkimi to obszar, w którym oczywista jest wartość integracji europejskiej. Istnieje ok. 6000 chorób rzadkich, a wiedza o nich jest znikoma. Koszty badań naukowych są wysokie, a terapie nierentowne. Dlatego bez wspólnych, strategicznych działań na poziomie europejskim wielu pacjentów nie będzie miało nigdy szansy na właściwą diagnozę, leczenie i rehabilitację.

Choroby rzadkie dotykają 30 mln ludzi w Europie. Często uwarunkowane genetycznie, rozpoczynają się w dzieciństwie i z powodu trudności w diagnostyce i braku leków prowadzą do postępującej utraty zdrowia, niepełnosprawności, izolacji, stygmatyzacji i wykluczenia.

Ostatnie 10 lat pokazało, że na poziomie europejskim można zdziałać wiele: powstały m.in. sieci referencyjne skupiające wiedzę i talenty najwybitniejszych specjalistów. Mamy pierwsze doświadczenia, podsumowania oraz świetny materiał – studium perspektywiczne RARE2030 będące wynikiem intensywnej współpracy ekspertów z całej Europy. Studium zawiera konkretne rekomendacje i scenariusze.

Przyjęte podejście, warte upowszechnienia, oparte na przewidywaniu przyszłych trendów, pozwala przygotować się do wyzwań i szans, jakie przyniosą kolejne lata. Potrzebujemy strategii, która umożliwi wcześniejszą, szybszą diagnostykę, dostępność leków, holistyczne podejście do opieki i leczenia. Dlatego też z niecierpliwością oczekuję dalszych działań Komisji, w tym przeglądu strategii na rzecz chorób rzadkich i osiągnięć sieci, a także podjęcia dynamicznych prac nad wdrożeniem rekomendacji RARE2030.

 
  
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  Ádám Kósa (NI), írásban. – A ritka betegségek gyakran krónikus, progresszív lefolyásúak, és rendkívül romboló hatással vannak az érintettek jólétére, életminőségére és a mindennapi tevékenységek végzéséhez szükséges képességekre. A Petíciós Bizottságban is több olyan petícióval találkoztam, melyeket ilyen betegséggel élők adtak be. Sokszor innovatív és drága kezelésre van szükségük, ami anyagilag nem megtérülő, de mégis feltétlenül támogatni kell EU- és tagállami szinten is gyógyulásukat. Ebben a témában az Európai Bizottsághoz is kérdéssel fordultam már 2020 tavaszán. Csökkenteni kell a betegek diagnosztikához, információhoz és gondozáshoz való hozzáférése terén fennálló egyenlőtlenségeket. A tagállamoknak segítségre van szükségük e betegségek ismertségének és láthatóságának fokozásához.

Támogatni kell a kutatásokat és az innovációt az ilyen betegségek alaposabb megismerése céljából. Javítani kell a diagnosztikát és a terápiás megközelítéseket. A ritka betegségben szenvedők többsége mobilitási problémákkal küzd, és súlyos egyenlőtlenségekkel kénytelen megküzdeni a foglalkoztathatóság és a társadalmi befogadás terén, ezért fontos, hogy az ENSZ CRPD egyezmény alapján ugyanolyan szintű védelemben részesüljenek, mint azok, akiknek a fogyatékossága nem függ össze valamely ritka betegséggel. A foglalkoztatás terén történő megkülönböztetéssel szemben a fogyatékossággal élő személyek számára védelmet biztosító uniós jogszabályok, valamint a szociális jogok európai pillérének 17. alapelvében foglaltak szintén alkalmazandók a ritka betegségben szenvedőkre is.

 
  
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  Manuel Pizarro (S&D), por escrito. – O debate sobre o plano de ação europeu contra as doenças raras é um claro indicador de que a Europa tem vindo a evoluir positivamente na tentativa de mitigar as assimetrias entre Estados-Membros. A atual situação no que diz respeito ao diagnóstico neonatal de doenças raras, quando vista Estado-Membro a Estado-Membro, prova que o único caminho a prosseguir na construção de uma europa mais justa e solidária passa por identificar boas práticas e uniformizar os bons procedimentos. Nesta matéria, é com imenso orgulho que posso referir Portugal como um exemplo muito positivo, fazendo diagnóstico neonatal de 27 doenças raras. Importa, por isso, continuar a construir uma União Europeia também na área da saúde. Provado está que esta é uma das grandes preocupações dos cidadãos europeus e que vai muito para além das situações de crise, como a atual pandemia, ou da prevenção e tratamento do cancro (claramente entendido como uma prioridade pela Comissão Europeia), estendendo-se a todas as áreas da saúde.

 
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