The RESTORE position paper represents the cumulative efforts of the RESTORE community, and the steering committee since the start of the RESTORE initiative in 2019. It outlines, in depth, the RESTORE view on why Europe should invest in Advanced Therapies, the roadblocks in Advanced Therapy development and implementation into patient care, and makes recommendations about how Europe can tackle these hurdles to establish a sustainable pan-European ecosystem integrating research, clinics, industry and patients. 

The RESTORE network enthusiastically and actively engaged with the creation of the RESTORE roadmap for Horizon Europe. This resulted in participation of 256 supporters, 26-118 contributors in each of the 17 working groups, actively contributing to the groups via an online file sharing system and during numerous Face-to-Face (F2F) meetings.

The results of this work are published as open access content on the RESTORE website, and further documents will follow as the team is continuing to work together even after finishing the official H2020-funded preparatory phase of the LSRI (https://restore-horizon.eu).

WHAT does RESTORE address?
Objectives:
• To deliver a pipeline of revolutionary Advanced Therapy Medicinal Products “developed and made
in Europe” and transform the disruptive promise of Advanced Therapies into reality to cure chronic diseases
• To make them accessible and affordable as “standard-of-care” for every patient in need
• To make Europe a spearhead of Advanced Therapies in Science, Clinics and Biomedical Industry
• To make use of the enormous socio-economic potential of novel breakthrough-therapies in Europe

Key elements to achieve these aims and to overcome roadblocks are:

1. Coordination and support action for all efforts.
2. Combine the benefits of thinking SMALL and BIG to unify disrupting inventions and innovation.
3. Broadly accessible Technology Research and Innovation (TRI) platforms and Cross-theme platforms –
   Collaboration replaces competition.
4. A Network of Academic translation incubators (Hubs)
5. New forms of Private-Public-Partnerships (PPP) and support of SMEs.

RECOMMENDATIONS

Tackle the rising problem of chronic diseases for patients and society by providing accessible and affordable medicine taking advantage of the disruptive research and breakthrough innovations on Advanced Therapies for conquering cancer and other chronic and degenerative diseases

Switch from the current focus on scattered, time limited, unconnected EU-funding to a concerted funding program for Advanced Therapies integrating innovative elements supporting a creative and productive ecosystem and to build a true European Research Area on Advanced Therapies as a basis for attracting significant private investment

Take the momentum gained from the FET-Flagship initiative in bringing together stakeholders in the field and go in new directions by supporting sustainable structural elements with sufficient budget according to the achievement of key performance indicators over the next seven years of the Horizon Europe programme as an adequate structural and financial alternative to the cancelled FET-flagship programme:

Coordination and Support Action (CSA): consortium with transparent and dynamic governance that provides support, guidance, education, advice, outreach, networking, and expertise for the large-scale research initiative on Advanced Therapies
Budget: 5-6 Mio €/a (total for 7 years ca. 35-40 Mio €)

Infrastructure: Network of Academic Translational Incubators (Hubs) with adequate infrastructure (competitively selected, co-financing only, basic budget by national/regional funding organisations) as docking points for small teams with breakthrough projects, patients advocates, biotech,
pharma and technology providing industry, policy makers, health insurers and special interest venture funds to accelerate and de-risk the development of Advanced Therapies by:
i) implementing broadly accessible Research and Technology Innovation platforms,
ii) performing high-quality clinical trials,
iii) applying the Refined Translation Tool for de-risking late and costly clinical development
iv) implementing incubators as docking points for start-ups, SME, industry and special interest venture funds
Budget: calls for 15 centers, 3-4 Mio €/a each = 45-50 Mio €/a (total for 7 years ca. 300 Mio €)

Research and Innovation Actions (RIAs): Feed the small teams to generate mission-driven disrupting science and enabling technology interlinked in thematically structured consortia; keep the RIA projects flexible such that they can be halted or extended based on key performance indicators,
for getting new pipeline projects and building-up European Research & Technology Innovation platforms (incl. smart manufacturing 4.0, health-economics, ethics); support docking of successful projects to the innovation Hubs.
Budget: coordinated, annual calls, about 20 consortia x 5-10 Mio €/each(total for 7 years ca. 150 Mio €)

Private Public Partnership and SME support : To bring innovation to the market and patient we need strengthened relationships of academia to industry and investors to support private funding and sustainability for EU-funded projects on Advanced Therapies, including support of SME/industry docked to the programme, notably through the European Innovation Council (EIC) and European Investment Bank (EIB).
Budget: EIC /EIB tools (ca. 150-300 Mio €)

Most urgent
To take the momentum gained from RESTORE´s work during last 2 years, it would be urgent to implement at the beginning of the new Horizon Europe programme a Coordination & Support Action for Advanced Therapies
as outlined above. In addition, the formation of the proposed Hubs is a key element to foster and build a European ecosystem, which attracts private investment to develop Europe as a player and not only a payer in
this emerging field. This requires the support at regional, national, and European level. Recent experiences from the Covid-19 crisis demonstrate the contribution of innovative Hubs, Biotech and Pharma to support the efforts against Covid-19 and how important it is to keep innovative Hubs, Biotech and Pharma in Europe. The offers from overseas for such companies to leave Europe are attractive and can be only competed by a powerful ecosystem.
In addition, RESTORE suggests to adapt the rules for new RIAs in the direction mentioned (keep the RIA projects flexible such that they that can be halted or extended based on key performance indicators).