Periodic Reporting for period 1 - RESTORE (RESTORE Health by Advanced Therapies (Advanced Therapy Medicinal Products and Biologized Medical Devices) – ALL for Advanced Therapies – with Passion – For Patients)
Reporting period: 2019-03-01 to 2020-07-31
What is the problem/issue being addressed?
The RESTORE consortium was initiated with the vision to not only become a game changer in health care within the next decade, but also to exert a high impact on Europe’s economy. It was selected in the multi-stage process of the former FET-flagship initiative and the project had the goal of preparing a European large-scale research initiative on the topic of Advanced Therapies.
Why is it important for the European society to invest more in Advanced Therapies?
As we age we become more at risk of developing chronic diseases. Many of these chronic diseases are currently incurable and therefore often require life-long treatment. Thus, the rise in the average age of the population is accompanied by increases in health costs. Therefore, chronic disease represents an increased socio-economic burden. In the last decade alone the direct health costs increased by 50%, reaching €1,526 billion in 2017, a staggering 9.6 % of Europe´s GDP (EU Health at a Glance 2018). For the increasing number of people suffering from them, chronic diseases mean life-long treatment with unwanted side-effects and a limited quality of life.
Advanced Therapies is an umbrella term for cell and gene therapies, tissue-engineering products and biologised medical devices.Unlike current treatments for chronic diseases, which often only treat the symptoms of the disease, Advanced Therapies offer the promise of a cure, restoring patient health.
Ground-breaking results with Advanced Therapies have been demonstrated for several rare genetic diseases as well as for common immune diseases, cancer, and tissue injury. Some Advanced Therapies have even made it to the market, mostly for rare diseases and specific blood cancers. However, so far only a few thousand patients worldwide have benefitted from them. The reason for this is that Advanced Therapies are different from conventional treatment options, not only in their superior therapeutic effect but in almost every other aspect too – from the way they are discovered, to the way they are produced, tested and approved for clinical use. These differences result in new obstacles and challenges. To overcome them, completely new approaches must be developed by those who wish to bring Advanced Therapies from the lab to the patient. RESTORE aims to do exactly that and to generate an Advanced Therapy ecosystem in Europe for the benefit of all European citizens.
What are the overall objectives?
The unifying goal of RESTORE is to make the transforming promise of Advanced Therapies a reality through the development of new Advanced Therapies and their implementation into clinical practice. Put simply, RESTORE aims to make potentially life-changing Advanced Therapies accessible and affordable to all patients in need.
The objectives of the preparatory phase project were:
-To expand the RESTORE community by engaging additional strategic partners as supporters
-To raise awareness among scientists, policy-makers and the public for the need to develop Advanced Therapies in Europe at a new level
-To refine the roadmap of the RESTORE initiative
-To discuss with stakeholders from academia, drug-developing biotech/pharma industry, enabling technology-providing tech industry, regulatory bodies, and patient advocates the nourishing elements required for boosting the field of Advanced Therapies in Europe
-To determine essential key elements to bring Europe to the top in this field
-To define recommendations for the European Commission and national funding organisations
The RESTORE consortium was initiated with the vision to not only become a game changer in health care within the next decade, but also to exert a high impact on Europe’s economy. It was selected in the multi-stage process of the former FET-flagship initiative and the project had the goal of preparing a European large-scale research initiative on the topic of Advanced Therapies.
Why is it important for the European society to invest more in Advanced Therapies?
As we age we become more at risk of developing chronic diseases. Many of these chronic diseases are currently incurable and therefore often require life-long treatment. Thus, the rise in the average age of the population is accompanied by increases in health costs. Therefore, chronic disease represents an increased socio-economic burden. In the last decade alone the direct health costs increased by 50%, reaching €1,526 billion in 2017, a staggering 9.6 % of Europe´s GDP (EU Health at a Glance 2018). For the increasing number of people suffering from them, chronic diseases mean life-long treatment with unwanted side-effects and a limited quality of life.
Advanced Therapies is an umbrella term for cell and gene therapies, tissue-engineering products and biologised medical devices.Unlike current treatments for chronic diseases, which often only treat the symptoms of the disease, Advanced Therapies offer the promise of a cure, restoring patient health.
Ground-breaking results with Advanced Therapies have been demonstrated for several rare genetic diseases as well as for common immune diseases, cancer, and tissue injury. Some Advanced Therapies have even made it to the market, mostly for rare diseases and specific blood cancers. However, so far only a few thousand patients worldwide have benefitted from them. The reason for this is that Advanced Therapies are different from conventional treatment options, not only in their superior therapeutic effect but in almost every other aspect too – from the way they are discovered, to the way they are produced, tested and approved for clinical use. These differences result in new obstacles and challenges. To overcome them, completely new approaches must be developed by those who wish to bring Advanced Therapies from the lab to the patient. RESTORE aims to do exactly that and to generate an Advanced Therapy ecosystem in Europe for the benefit of all European citizens.
What are the overall objectives?
The unifying goal of RESTORE is to make the transforming promise of Advanced Therapies a reality through the development of new Advanced Therapies and their implementation into clinical practice. Put simply, RESTORE aims to make potentially life-changing Advanced Therapies accessible and affordable to all patients in need.
The objectives of the preparatory phase project were:
-To expand the RESTORE community by engaging additional strategic partners as supporters
-To raise awareness among scientists, policy-makers and the public for the need to develop Advanced Therapies in Europe at a new level
-To refine the roadmap of the RESTORE initiative
-To discuss with stakeholders from academia, drug-developing biotech/pharma industry, enabling technology-providing tech industry, regulatory bodies, and patient advocates the nourishing elements required for boosting the field of Advanced Therapies in Europe
-To determine essential key elements to bring Europe to the top in this field
-To define recommendations for the European Commission and national funding organisations
WHO is behind RESTORE?
The core RESTORE consortium is comprised of 10 partners from academic translational centres, biotech industry and non-for-profit organisations. RESTORE is also supported by an increasing number of various stakeholders in the healthcare and high-tech sector (currently >300) (Fig.1).
The engagement of a diverse and interdisciplinary RESTORE community is key to future development of Advanced Therapies. To solve the obstacles and challenges in the development of Advanced Therapies, it is necessary to bring together actors from academia, industry, and patient organisations and have intensive exchanges with regulatory bodies and political actors. Thus, within the last several months, the RESTORE community has held or participated in events to engage different audiences (Fig 2). The consortium has actively sought the opinion of the European public by conducting an online survey, in which the thousands of participants from many European countries rated healthcare the number one most important topic among big political issues, with the vast majority expressing support for using EU- and state-funding to support medical innovations in general and Advanced Therapies in particular. Finally, the RESTORE consortium has outlined its recommendations for how to proceed in making Europe a key player in the emerging field of Advanced Therapies, for the benefit first and foremost of patients, but also more broadly for society and the European economy.
The core RESTORE consortium is comprised of 10 partners from academic translational centres, biotech industry and non-for-profit organisations. RESTORE is also supported by an increasing number of various stakeholders in the healthcare and high-tech sector (currently >300) (Fig.1).
The engagement of a diverse and interdisciplinary RESTORE community is key to future development of Advanced Therapies. To solve the obstacles and challenges in the development of Advanced Therapies, it is necessary to bring together actors from academia, industry, and patient organisations and have intensive exchanges with regulatory bodies and political actors. Thus, within the last several months, the RESTORE community has held or participated in events to engage different audiences (Fig 2). The consortium has actively sought the opinion of the European public by conducting an online survey, in which the thousands of participants from many European countries rated healthcare the number one most important topic among big political issues, with the vast majority expressing support for using EU- and state-funding to support medical innovations in general and Advanced Therapies in particular. Finally, the RESTORE consortium has outlined its recommendations for how to proceed in making Europe a key player in the emerging field of Advanced Therapies, for the benefit first and foremost of patients, but also more broadly for society and the European economy.
The consortium has worked intensively and successfully together and has addressed all the objectives formulated above. A large number of analyses and conclusions have been developed, which are available in open access on the RESTORE website (https://www.restore-horizon.eu). The following recommendations to the EC are derived from this:
What does RESTORE recommend?
- Create a consortium of all relevant stakeholders - from academia, industry, and patient advocates that will have strong exchanges with regulatory bodies and policy makers. It should have a transparent and dynamic governance that provides support, guidance, education, advice, outreach, networking, and expertise for the European large-scale research initiative on Advanced Therapies.
- THINK SMALL! take advantage of many small, independent research teams scattered over the European landscape with their high potency for creating disrupting new ideas and technologies.
THINK BIG! bring innovation to the market and patient by forming 15-20 Academic Hubs in Europe as virtual and real docking point for the European-wide scattered smaller teams, technology and drug developing Biotech/Pharma industry, special interest venture funds, patient representatives, and experts in ethics, data management, and artificial intelligence (AI).
- Accelerate and de-risk the development of new, highly efficient Advanced Therapies, by replacing futile competition (e.g. reinvention of the wheel) with collaboration. Do that by implementing innovation platforms that deliver new core technologies for a broad range of applications to the centres.
- Implement an ecosystem that is supporting Public-Private-Partnership.
Impact of the RESTORE initiative
RESTORE foresee a strong impact of pushing Advanced Therapies in Europe on not only Science and Technology but even more so in Societal Benefits for society and the economy. RESTORE believe that by pursuing our initiative, we will be able to improve the quality of life of chronically ill patients, decrease the financial burden of chronic diseases on society, support industry and build up a highly specialised workforce in Europe in the coming decades. Make Europe a Player and not just a Payer in this emerging field.
RESTORE Health by Advanced Therapies (Advanced Therapy Medicinal Products and Biologised Medical Devices) – ALL for Advanced Therapies – with Passion – For Patients
What does RESTORE recommend?
- Create a consortium of all relevant stakeholders - from academia, industry, and patient advocates that will have strong exchanges with regulatory bodies and policy makers. It should have a transparent and dynamic governance that provides support, guidance, education, advice, outreach, networking, and expertise for the European large-scale research initiative on Advanced Therapies.
- THINK SMALL! take advantage of many small, independent research teams scattered over the European landscape with their high potency for creating disrupting new ideas and technologies.
THINK BIG! bring innovation to the market and patient by forming 15-20 Academic Hubs in Europe as virtual and real docking point for the European-wide scattered smaller teams, technology and drug developing Biotech/Pharma industry, special interest venture funds, patient representatives, and experts in ethics, data management, and artificial intelligence (AI).
- Accelerate and de-risk the development of new, highly efficient Advanced Therapies, by replacing futile competition (e.g. reinvention of the wheel) with collaboration. Do that by implementing innovation platforms that deliver new core technologies for a broad range of applications to the centres.
- Implement an ecosystem that is supporting Public-Private-Partnership.
Impact of the RESTORE initiative
RESTORE foresee a strong impact of pushing Advanced Therapies in Europe on not only Science and Technology but even more so in Societal Benefits for society and the economy. RESTORE believe that by pursuing our initiative, we will be able to improve the quality of life of chronically ill patients, decrease the financial burden of chronic diseases on society, support industry and build up a highly specialised workforce in Europe in the coming decades. Make Europe a Player and not just a Payer in this emerging field.
RESTORE Health by Advanced Therapies (Advanced Therapy Medicinal Products and Biologised Medical Devices) – ALL for Advanced Therapies – with Passion – For Patients