Press Release N°62/2021 Advanced therapies: a new management model to quickly reach the patient's bedside; the on-going alliance between the ISS and ASSOBIOTEC-Federchimica

ISS, November 24th 2021 -

Brusaferro (ISS): "Supporting innovation means promoting fair and effective clinical transfer"

Palmisano - ASSOBIOTEC - Federchimica: "A permanent Task Force including all the players in the Health System is essential". The Association also presents its new position paper Cell&Gene

More than 2,600 clinical trials are in progress worldwide, of which 240 in Phase 3 (158 carried out by the bio-pharmaceutical industry and 85 by academia, governments or other institutions)

24 November 2021 - Constant dialogue between patients, clinicians and institutions to strengthen the alliance and create a common network. This is the focus of the webinar "Advanced therapies in Italy: today and tomorrow", organized by the Istituto Superiore di Sanità – ISS (Italian National Institute of Health) and Assobiotec-Federchimica "which was held today at the ISS. The goal is to improve interventions in each of the steps that lead to new therapies: from preclinical to clinical development through to authorization and administration to patients.

"Only a strong alliance among research bodies, institutions and innovative companies - says Silvio Brusaferro president of the ISS - can keep the level of innovation high in Italy. While the development of innovative and sustainable treatments (often also sparked by academic research) is the responsibility of the manufacturing companies, the task of the institutions is to ensure the development of effective and safe therapies and to facilitate their availability to patients. The challenge of the future is to offer all patients the opportunity to be treated fairly and sustainably by bridging regional discrepancies ”.

Today's webinar aims at promoting dialogue among the various stakeholders so as to successfully address the important challenge of access to innovation, with particular reference to gene therapies. This challenge requires the adoption of new models in each stage of the development of these products and a stronger cooperation among all the actors involved so as to favour a methodologically rigorous development of Advanced Therapies and, above all, a timely transfer to the patient’s bedside of the fruits of research in the name of sustainability and equity.

"At last year's meeting there was agreement on the fact that advanced therapies represent an important frontier and a significant resource for the future, but they are faced with an equally important challenge which is that of access to innovation” - explains Patrizia Popoli, Director of the ISS Research Centre for the preclinical and clinical assessment of drugs. “After 14 months, the challenge is still there, although some progress has been made. Patients suffering from serious diseases for which there are no satisfactory therapies have the right to receive potentially effective treatments as quickly as possible. Bringing this to fruition is, therefore, a duty for companies and institutions. The latter, in particular, have a number of difficult tasks, such as: providing regulatory support to the companies, guaranteeing the development of sufficiently effective and safe drugs, adopting assessment strategies that are suited to the complexity of advanced therapies while maintaining the necessary methodological rigor, and finally, identifying and implementing organizational measures for the transfer of the discoveries to clinicians".

Just a year ago, on the occasion of the first webinar organized jointly by the ISS and Assobiotec-Federchimica on Advanced Therapies, it was found that there are a dozen therapies available in Italy. In the last 12 months, 4 new ATMPs have been approved by the EMA. Today, 14 are already on the market in Europe and they have been approved for 17 indications. Seven are reimbursable by AIFA, while six advanced therapies are in the process of being assessed. The data from the latest ATMP Forum Report also tell us that new trials are underway for about twenty disorders and that the therapeutic areas mostly involved are oncology and haematology. This snapshot clearly shows that the number of these therapies is expected to grow rapidly. A trend also confirmed by data from Alliance Regenerative Medicines which speak of more than 2,600 trials worldwide, of which more than 240 are already in phase 3 (158 carried out by the bio-pharmaceutical industry and 85 by academia, governments or other institutions). In the next 10 years, therefore, many new therapies will be available in clinical practice for the treatment of disorders that while still being niche pathologies are certainly not as rare as they used to be.

Riccardo Palmisano, President of Assobiotec-Federchimica states, "To win the challenge of rapid access to innovation, there needs to be a permanent alliance between research institutions, innovative companies and regulatory bodies. For this we propose to strengthen the already fruitful collaboration that was started last year with the Round Table which included the ISS, the Ministry of Health, AIFA and the Regions, through the establishment of a permanent Task Force that meets periodically. The goal is clear: to bring to light and remove the many obstacles that we still meet today along all the different stages of development of these therapies. The progress of science is unstoppable and all the players in the Health System must work together to make sure that patients receive the utmost benefits".

"When the Cell & Gene working group, specifically dedicated to Advanced Therapies, was set up a dozen years ago in Assobiotec-Federchimica”, adds Luigi Boano, Vice President of the Industrial Association, “many people probably thought it was a premature leap forward. Instead, we were fully aware of the fact that in the future these therapies would represent a radar for the treatment of various diseases with a fatal outcome: rare diseases, tumours ... Here, today, we are publishing a new, updated position document, in which recommendations are analysed and proposed for a correct, effective, sustainable and safe management of the various steps of the ATMP management process, with a view to guaranteeing the availability of these drugs for all those patients who can benefit from them. A document that, we hope, will be a valid tool for greater efficiency of the system in the management of these drugs".

A CLOSER LOOK

What are Advanced Therapies (ATMPs)
Advanced therapies, which include cell, gene and tissue engineering therapies, and which in Europe are called ATMP (Advanced Therapy Medicinal Products) are the protagonists of a revolution in the medical field. For the first time in the history of medicine it has been possible to develop personalized drugs based on biological material designed to heal the patient in a single administration.
Unlike the small molecules obtained from chemical synthesis and the biotechnological macromolecules such as the monoclonal antibodies and recombinant proteins, ATMPs are made up of cells or tissues (engineered if anything at all) or of nucleic acids. These drugs, innovative by definition, are proving their effectiveness not so much in treating the symptoms, but the disease itself, by intervening directly on the causes and offering new healing prospects for pathologies that until now had no therapeutic solution.

The 14 Advanced Therapies approved by EMA