Rare diseases and pediatrics: how can the incentives be reshaped?
Who participated and what was said at the orkshop "Rare diseases and pediatrics: therapeutic innovation and national and European investment attraction measures", promoted by the High School of Economics and Management of Health Systems (Altems) – Faculty of Economics of Catholic University with the participation of Farmindustria.
Rare diseases are those pathologies that affect no more than 5 people for every 10 thousand inhabitants. In Europe there are between 25 and 30 million people affected by rare diseases , 2 million in Italy alone. The future of research was the main topic of the workshop " Rare diseases and pediatrics: therapeutic innovation and national and European investment attraction measures ", promoted by the High School of Economics and Management of Health Systems (Altems) – Faculty of Economics of the Catholic University with the participation of Farmindustria.
THE SIMPLIFICATION OF THE EUROPEAN REGULATORY FRAMEWORK
During the workshop, the need to simplify the regulatory framework emerged to ensure that all European states have homogeneous access to medicines to treat rare diseases. "In the European Union – said Professor Cicchetti, director of ALTEMS – the evolution of the reference Regulations, the search for a balance between the competences of the regulatory agencies and that of the bodies responsible for the Health Technology Assessment is engaging European institutions, authorities regulations, parliaments and national governments in a discussion which, however, struggles to find a definitive meeting point ". In January 2022, the Clinical Trials Regulation entered into force “which aims to harmonize the processes of presentation, evaluation and supervision of clinical trials throughout the EU – continues prof. Cicchetti -. In particular, the Information System of Clinical Trials (CTIS) is active which will make it possible to rationalize the processes, guaranteeing the EU to continue to attract clinical research. Italy is therefore in a new European competition with other countries that requires serious reflection on the state of the art and on the actions to be taken to ensure competitiveness for the country system ".
SCACCABAROZZI (PRES. FARMINDUSTRIA): "IN THE LAST 20 YEARS MORE THAN 2500 AUTHORIZATIONS FOR ORPHAN DRUGS"
According to the latest analysis by Iqvia, in Italy, between 2017 and 2020, the average time taken at European level between the marketing authorization and access to the orphan drug is 482 days . A figure below the EU average. Even if our country is positioned, for example, after Germany (102 days), Denmark (249), Austria (261), the Netherlands (380) and England (441 days). In Italy 75% of orphan drugs approved in Europe are already available against 37% of the European average. Furthermore, the analysis of the Italian Medicines Agency shows that the authorization procedure for orphan drugs lasted an average of about 400 days in 2020 , down from about 450 in 2018. "Over 2,500 orphan drug designations and more of 200 medicines for rare diseases have obtained authorization for marketing in the EU in the last 20 years ”, says Massimo Scaccabarozzi , President of Farmindustria. " With a tripled number of clinical trials, again in Europe, between 2016 and 2021 . With a share of 26% of the total number of trials in 2021. And Italy has had a higher increase than the continental one, going from 119 trials in progress in 2016 to 749 in 2021, which represent 32% of overall clinical trials.
THE REVIEW OF THE REGULATION OF DRUGS FOR RARE DISEASES
The change in the European legislation governing the production of drugs for rare diseases, also through incentives, was one of the hot topics of the debate. " The revision of the regulation on orphan drugs intends to capitalize on the results of the legislation of the last 20 years ," he said. Tiziana Beghin , Head of M5S delegation, European Parliament -. The current incentive provides for 10 years of market exclusivity for recognized products, the revision aims to remodel this aspect. At the same time , the Commission is considering restricting the definition of rare diseases to below the current threshold of 5 per 10,000 people ”. Thanks to the incentives present in the EU, “ orphan drugs, as well as pediatric ones, have benefited from a framework capable of promoting and attracting investments from the private and public sector with certainly significant results – said Professor Cicchetti -. In light of the new regulatory and institutional scenarios and the new opportunities offered by research, it is essential to initiate a dialogue between all stakeholders on the best ways to strengthen the current reference framework and further promote the innovation necessary to satisfy still many therapeutic needs that they found no answer. The expected impact from the evolution of the sector is potentially enormous: both at an economic and social level and for the repercussions on the fundamental rights of patients. "
INCENTIVES AND MARKETING CERTAINTY: FRANCO LOCATELLI'S PROPOSAL
Franco Locatelli , President of the Superior Health Council, in his speech focuses attention on a very interesting issue that concerns precisely the regulation of the incentives made available to pharmaceutical companies. “ Between 27 and 36 million people are affected by rare diseases in Europe, 3 to 4% of newborns suffer from a rare disease. In 2021, 251 authorizations for the marketing of drugs were requested, only 17 arrived – underlines Locatelli -. AIFA was the first regulatory agency to activate a series of calls for independent research, due to the possibility for academic institutions to develop projects that otherwise would never have found a response. The big investment must be to improve pediatric research and increase the possibility of drugs. We need to think of innovative approaches to avoid redundancies and delays in authorization must be avoided ”. The professor. Locatelli then reports the case of a drug against thalassemia , the experimental phase was carried out in Europe but then commercialization was not carried out. “In Italy we have about 7 thousand people with thalassemia. It has been found that through “ginadition therapy” 90% of them stop transfusing. Too bad that the company that developed the ginadition therapy trial for thalassemia has decided to stop commercial activities in our country in a sudden and not in the least way announced – said prof. Locatelli -. The reimbursements guaranteed by the European agencies were unable to cover production costs . The negotiations took place in Germany and France, two countries put together do not reach 300 thalassemia and in Italy a negotiation has never been opened with AIFA and through no fault of AIFA. The experimentation that led to phase 3 was largely carried out in Europe. Are we convinced that we shouldn't think about introducing a commercial constraint if we come to experiment on our continent? ".
INCENTIVES FOR THE PRODUCTION OF DRUGS FOR RARE DISEASES: THE WORD TO COMPANIES
On the subject of incentives to pharmaceutical companies for research on rare diseases, Dr. Lucia Aleotti, shareholder and board member of Menarini, spoke. “When we talk about incentives for orphan drugs we are talking about incentives aimed at the pharmaceutical industry to make the industry find it interesting to invest in certain research areas – says Dr. Aleotti -. We know that the current incentive structure has actually achieved its goal. When you want to redirect incentives you need to involve industries to understand if the new regulations are incentives or not. Today the picture works because it gives certainties, companies know that by investing in some pathologies you can get to the registration of your drugs. When this picture changes, it is necessary to deal with reality and ask companies if they have an incentive to invest in other sectors. The risk is that some areas are left uncovered and not fill the gap where there is a lack ".
THE CONCERNS OF PHARMACEUTICAL COMPANIES FOR A COMPETITION AT THE LOWER
At the end of 2022 the European Commission will decide on the new pharmaceutical legislation for orphan and pediatric drugs. “There are shadows on this: the challenge is that research and development are a global challenge – recalled Marcello Cattani Coordinator of the Rare Diseases Farmindustria Group -. In our continent, 80% of the drugs resulting from innovative research and development come from the USA. Shadows are linked to regulatory protection and research incentives. The Italian industry is strong on research: there are about 800 studies on rare diseases . In summary, Farmindustria is close to the world of innovation and the right to equity of European countries. The PNRR represents a further development channel also with a view to collaboration between industries and academia ". Ugo Di Francesco, CEO of Chiesi Farmaceutica, spoke on the risk of global competition to the downside . “There is growing global and European competition on orphan drugs – says the manager of Chiesi Farmaceutica -. On the part of all of us there is the risk that the generation of innovation can be renegotiated downwards . These are complex issues that require a complex solution and that risk taking innovation further and further away from Europe and Italy. Access for rare disease patients can also range from 3 months to 3 years , increasing inequalities. Access to therapeutic treatments should depend on need and not on geographic origin. All together we should work to guarantee patients quick and homogeneous access in Europe ”.
This is a machine translation from Italian language of a post published on Start Magazine at the URL https://www.startmag.it/sanita/malattie-rare-e-pediatria-come-rimodulare-gli-incentivi/ on Wed, 25 May 2022 11:30:21 +0000.