Introduction and aim of the initiative

During the second half of 2022, the European Parliament hosted a number of initiatives on the topic of advanced therapies, promoted by the actors who are probably most committed to the topic at European level. From the TRANSFORM MEP Interest Group event on 13 October 2022, during which the “Charter with Solutions to Enable Safe and Timely Patient Access to Advanced Therapies in Europe” was launched, to the event organised by the Alliance for Regenerative Medicine (ARM) entitled “The future of ATMPs” on 29 November 2022, and the initiative of the 'VITA - Value and Innovation for Advanced Therapies' Consortium, realised with the support of the MEPs Interest Group "European Patients' Rights and Cross-border Healthcare", held on 24 October and titled "Securing Equitable Patient Access to Advanced Therapies across Europe", an initiative in which Active Citizenship Network actively collaborated in particular by promoting the Call to Action "Advanced Therapies Medicinal Products revolution & the respect of the patients' right to access to care" signed, so far, by 43 Patients' Advocacy Groups (PAGs).

In each of the aforementioned initiatives, opportunities and barriers have been highlighted so that Europe can take a leading role in the area of ATMPs and thus respond to the actual health needs of the many European citizens who see in these new treatment options a unique opportunity to improve their living conditions. Unfortunately, there are still many obstacles, economic and otherwise, that make access to these therapies extremely complicated and limited, despite the fact that for years the European Commission services and the European Medicines Agency, in collaboration with the authorities of the Member States, have initiated a number of initiatives to improve the regulatory environment for ATMPs so as to facilitate the development and authorisation of these products in the EU for the benefit of patients.

The 17^th European Patients' Rights Day aims to be a collector of the best and most advanced experiences existing at a European level on this topic, catalyzing the best professionals and the highest institutional authorities that are touched by the issue in their own areas of responsibility and intervention. The conference will be held at the European Parliament in Brussels on April 26 from 15:00 to 17:00. More details will be provided soon.

From the research towards a growing number of ATMPs to their use once approved, the unifying themes seem to be essentially the following: sustainability, innovation, equity. To these, from our point of view, two further elements must be added, which should also be taken into account as drivers in choices having to do with ATMPs: respect for and reinforcement of patients' rights. It is no coincidence that the issue of Advanced Therapy Medicinal Products is related to the following patients' rights set out in the European Charter of Patients' Rights, which is at the heart of the EPRD celebrations:

• the patients’ right of access to care(“Every individual has the right of access to the health services that his or her health needs require. The health services must guarantee equal access to everyone, without discriminating on the basis of financial resources, place of residence, kind of illness or time of access to services”);
• the patients’ right to personalized treatment(“Each individual has the right to diagnostic or therapeutic programmes tailored as much as possible to his or her personal needs”);
• the patients’ right to Innovation (“Each individual has the right of access to innovative procedures, including diagnostic procedures, according to international standards and independently of economic or financial considerations”);
• the patients’ right to Information (“Every individual has the right to access to all kind of information regarding their state of health, the health services and how to use them, and all that scientific research and technological innovation makes available”).

During the European Patients' Rights Day 2023 we want to give this topic the importance it deserves, certainly a complex topic where information is still scarce among the whole population and also among the various Patients' Advocacy Groups (PAGs). And for this reason we intend to invite as many leaders of patient associations as possible.

Promoted by Active Citizenship Network, the European celebration of the XVII Edition of the European Patients’ Rights Day will be officially endorsed by MEPs supporters of the MEPs Interest Group “European Patients' Rights and Cross-border Healthcare”, andMEPs supporters of the European Alliance for Transformative Therapies (TRANSFORM) MEP Interest Group.

Objectives of the event

• Collect ideas to fuel EU initiatives, exchange and discuss different experiences, best practices and more effective solutions for the inclusion of ATMPs by the national health systems of the various member states.
• Provide the leaders of civic associations and patient advocacy groups the opportunity to participate in an EU debate about this important issue.
• Raise the awareness of national and European institutions regarding the importance of ATMPs for the health of European citizens.
• Bearing in mind that in Italy Cittadinanzattiva is the only civic association officially involved in the national ethics committee for clinical trials with advanced therapies, set up in 2022, reflect on the role that such ethics committees, where established, can play in helping to ensure that in the field of ATMPs innovation and access go hand in hand.
• Advance proposals in view of the next European elections in 2024 in order to ensure a high focus on the topic in the next legislative term.

Background on the topic of ATMPs

The new frontier of personalized medicine (a medical model that proposes the personalization of health, with medical decisions, practices, and products tailored to the patient) is represented by Advanced Therapy Medicinal Products (ATMPs), which offer opportunities for innovative treatment and are classified into four groups:

1. Gene therapy drugs: capable of transferring a working copy of a defective gene.
2. Somatic cell therapy medicines: based on the use of cells or tissues manipulated to the point of treating, preventing, or diagnosing diseases.
3. Tissue-engineered medicinal products: contain cells or tissues to repair, regenerate, or replace human tissues.
4. Combined advanced therapy medicinal products: a combination of cell or tissue-based medical devices.

Advanced therapies are becoming not only a hope but a reality of cure for patients suffering from serious diseases who have not yet found a concrete therapeutic answer.

Advanced therapies are the emblem of personalized medicine since they are treatments that start from the patient's cells and therefore act on the genetic cause at the origin of the pathologies, also in some cases even with a single administration the ability to offer benefits to the patient forever.

The costs of this technology are high so that access by patients is often hindered by public health budget limits; the issue is to balnce this budget contraints with the high expectation of patients, having these therapies the ability to guarantee results over the years even with a single administration, are often one-shot.

The importance of access to advanced therapies by the highest possible number of patients is represented by the fact that by 2030 there could be as many as 60 advanced therapies that could change the life of 350 thousand patients, but to do this, many changes are required.

Origin & value of the European Patients’ Rights Day

In 2002, Active Citizenship Network (ACN), together with a group of European citizens organizations, drafted the European Charter of Patients’ Rights, which has been translated in 16 languages and includes the following 14 rights: the right to preventive measures; access care; information; consent; free choice; privacy and confidentiality; respect of patients’ time; observance of quality standards; safety; innovation; avoidance of unnecessary suffering and pain; personalized treatment; to complain; to receive compensation. All these rights, based on the Charter of Fundamental Rights of the European Union, are crucial for European citizens and healthcare services. The majority of these rights were also embodied within the Council conclusions on Common values and principles in EU Health Systems adopted in June 2006.

Vytenis Andriukaitis, former EU Commissioner for Health and Food Safety

attending the 10^th European Patients’ Rights Day in 2016

The reinforcement of these rights would become effective only with the cooperation and commitment of all healthcare stakeholders in every European country. It is thus essential to increase awareness regarding the importance of patients’ rights and everyone’s responsibilities in guaranteeing their respect. The celebration of the European Patients’ Rights Day every year in all the EU Member States would greatly contribute to this goal, as a common occasion to inform, discuss and take commitments to improve patients’ rights in Europe and in each Member State. For this reason, ACN - together with citizens and patient organizations throughout Europe - have taken the initiative to organize for the last fifteen years a European conference to celebrate the European Patients’ Rights Day.

Now, the European Patients’ Rights Dayhas become a regular annual event on the European and national political agendas to inform, discuss and take commitments to improve patients’ rights in Europe and in each Member State. Every year, also thanks to the more than one hundred patient and civic associations of our network, the EPRD is celebrated in hundreds of cities and ways all around Europe, allowing civil society to fully enter policy making processes and achieve important objectives. For further information, please visit the dedicated session of ACN website.

Recent articles published by Cittadinanzattiva-Active Citizenship Network on ATMPs

• HEALTH EUROPA QUARTERLY (31st Jan. 2023, issue 24, pags. 124-127): The value of investing in Advanced Therapy Medicinal Products
• HEALTH EUROPA (5th January 2023): The value of investing in Advanced Therapy Medicinal Products
• IL SOLE 24 ORE (25 October 2022): Terapie avanzate trattate come un investimento (IT)
• SANITÀ24 (24 October 2022): Riclassificare la spesa sanitaria per le terapie avanzate come investimento: la proposta al Parlamento Ue di esperti italiani e 43 associazioni di pazienti (IT)
• ABOUTPHARMA (24 October 2022): Spesa per terapie avanzate riclassificata come investimento: la proposta all’Europa di esperti e associazioni di pazienti (IT)
• REDATTORE SOCIALE (24 October 2022): Salute, proposta al Parlamento Ue: riclassificare la spesa per le terapie avanzate come spesa di investimento (IT)
• HELP CONSUMATORI (24 October 2022): Terapie avanzate, la proposta: riclassificare la spesa sanitaria come spesa di investimento (IT)
• IL FARMACISTA ONLINE (24 October 2022): "Riclassificare la spesa sanitaria per le terapie avanzate come spesa di investimento". La proposta presentata al Parlamento Ue da un gruppo di esperti e da 43 Associazioni di pazienti (IT)
• HEALTH EUROPA (21 October 2022): Equitable patient access to advanced therapies
• ST, EUROPE (7 October 2022): 24 October – Securing Equitable Patient Access to Advanced Therapies across Europe
• LA REPUBBLICA (28 April 2021): Entro il 2030 in arrivo 60 nuove terapie geniche (IT)
• PHARMASTAR (22 April 2021): Sedicimila terapie avanzate in sperimentazione, 60 in arrivo. La sfida alle malattie (oggi) incurabili (IT)
• PANORAMA DELLA SANITÀ (22 April 2021): Terapie avanzate, serve cambiare le politiche di bilancio europee (IT)
• FORTUNE ITALIA (23 Nov. 2020): Le opportunità e le sfide delle nuove terapie avanzate (IT)
• ISTITUTO DANTE ALIGHIERI (23 Nov. 2020): Le opportunità e le sfide delle nuove terapie avanzate (IT)