Pnrr and rare diseases. An opportunity not to be missed

by Andrea Piccioli

What I would like to emphasize here is the importance of funds for rare diseases (50 million euros), which are all too often allocated due to their small number in terms of incidence and prevalence, certainly not in terms of the total number people (millions of people)) forced to live with it

02 MAY

Extraordinary time requires extraordinary resources. And there is no doubt that this is the period we are experiencing, as the possibilities for financing the EU “NextgenerationEu” are just as extraordinary.

An ambitious development and modernization program, thanks to which Italy was able to present its reform and investment package for the years 2021-2026, or the National Recovery and Resilience Plan (PNRR), divided into six missions: digitization, innovation, competitiveness, culture and tourism; green revolution and ecological change; sustainable mobility infrastructure; education and research; inclusion and cohesion; health.

It is in connection with this last point that, as the director of the largest public health research body in Italy, the Istituto Superiore di Sanità, I would like to focus on the investment of over € 15 billion between creating and strengthening proximity networks on the one hand and innovation and digitization of the National Health Service (NHS) on the other hand. Goals that the pandemic has proven to be as urgent as ever must be achieved. I would say that it is essential to improve aid to citizens and patients from a qualitative and temporal point of view.

To this end, the network of intermediate territorial structures, such as community centers and hospitals, such as all proximity services, including home care and the development of telemedicine services, is therefore very valuable – and its implementation is welcomed. For the same purpose, renewal and modernization of existing technological and digital structures is all the more necessary.

Again in connection with mission 6 of the PNNR, significant resources were made available by the call launched a few days ago by our Ministry of Health: over 262 million euros for the benefit of biomedical research.

A loan of Rs 50 crore to be set aside for “proof of concept“That is to say, for projects aimed at promoting the transition from the knowledge developed in the laboratory to its development at the industrial level; an additional 50 million for projects on rare diseases (excluding rare cancers for which it is envisaged an additional warning); 162.070 million focused on chronic non-communicable diseases for studies of risk factors and prevention; etiopathogenesis and disease mechanisms.Two-year projects whose time and access details are specified in the Communication itself.

What I would like to emphasize here, also as the head of the Newborn Screening Coordination Center (CCSN) based in the ISS, is the importance of funds for rare diseases, which are all too often allocated due to their small number in terms of incidence. and prevalence certainly not in relation to the total number of people (millions) who are forced to live with it.

I am pleased to read in the Communication that the efforts that can be funded are numerous and range from the basic research area to the organization of models for the care of patients.

They range from the creation and improvement of biobanks, valuable tools for the collection and storage of clinical data, including samples collected for neonatal screening to be used for research purposes, to support centers specializing in the development of advanced therapies (gene and cell therapies and tissues). and by reuse of drugs, i.e. repositioning of molecules used for other pathologies. From the development of new drug delivery systems to reaching out to organs and tissues not covered by current treatments, to the creation of consortia between research and industry to the development of new molecules. From building a network of specialists with different skills to developing ways to involve patients, with attention to the transition from pediatric to adult life and to acute / emergency situations.

In short, the field is large and the initiative includes a second half. In fact, the intention is to launch an additional tender by 2023 for a similar amount (over 262 million), to allocate a total of more than 524 million euros. Again divided into proof of concept projects (100 million), research programs / projects on tumors and rare diseases (100 million), projects on diseases with a major impact on health (324,140 million).

A breath of fresh air for Italian research, an opportunity that must definitely be seized.

Andrea Piccioli
Director of the Higher Department of Health

Editorial of “RaraMente” Newsletter by Iss

02 May 2022
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