PRO’s. Keep this acronym in mind: it stands for Patient Reported Outcomes. And in practice it highlights the importance of evaluation of patients on therapieswith particular attention also to undesirable effects. More and more elements like this become basic in the study path of a drug. Also because they are tools for improving treatment paths and in some way intervening on the sustainability of the healthcare system.
The PRO’s are just one element of the clinical researchthe engine of scientific progress and innovation, an asset and heritage of the individual and society. Clinical studies directly concern patients and citizens, both because participation in a study allows those living with a pathology to access innovative therapeutic options in advance and safely, more effective than those already in use, and because the value of research translates to health, social and economic benefits for all patients and for the entire community, with an improvement in global health.
Promoting awareness among citizens of the value of research and overcoming the resistance that hinders patient participation in clinical trials are the objectives of the national awareness and information campaign on the value of research and clinical trials “We are the research. All together, from science to cure“, promoted by the EUPATI APS Expert Patient Academy (AdPEE) together with an authoritative and broad coalition of 47 patient associations, academies and healthcare companies, scientific societies and research centers, with the patronage of Fondazione Policlinico Universitario Agostino Gemelli IRCCS – Catholic University of the Sacred Heart, its Clinical Trial Center and the University Hospital of Alessandria.
How much research is worth in Italy
Research is the human and economic capital of a State. New and integrated strategic levers are needed to enhance clinical research in Italy: attraction of economic capital and investments, training of staff, retaining and attracting young talents.
“According to AIFA’s Report on clinical trials of medicines in Italy 2023, from 2000 to 2022, over 15,400 clinical trials in the country – comments Onofrio Mastandrea, who is part of the Farmindustria Council. In 2023, pharmaceutical companies allocated 2 billion euros to R&D, 7% of total investments in the country. However, critical issues remain: the reduction in clinical trials carried out in Europe, from 18% of the global total in 2013 to 9% in 2023, has led to 60,000 fewer patients accessing a trial and therefore innovative therapies. However, the collaboration between the Ministry of Health, AIFA and stakeholders in the sector has the aim of improving Italy’s attractiveness for clinical research. Investment in human capital is fundamental: the ability to train, retain and attract young talents who will constitute the future of scientific research, also in the pharmaceutical industry”.
Profit and non-profit studies
Clinical research represents an opportunity for patients, as it allows them to access the most advanced therapies and contributes to the development of better treatments for all. Italy has always had a leadership role in clinical research, above all due to the excellent quality of scientific production, recognized internationally. Unfortunately, in our country financing for scientific research they are insufficient. Yet, demonstrating the safety and effectiveness of an innovative drug means bringing better treatments onto the market in a relatively short time than those already available. Undoubtedly, clinical research is a “hot” sector that should be strengthened to make Italy more attractive for hosting clinical trials.
“In Italy, in 80% of cases, the studies are ‘profit’, i.e. supported by pharmaceutical companies and only 20% are ‘non-profit’, i.e. so-called academic studies – recalls Giampaolo Tortora, Director of the Comprehensive Cancer Center, Fondazione Policlinico University ‘A. Gemelli’ IRCCS of Rome. The lack of unsponsored studies it is a serious damage, because the testing of new drugs brings advantages from a scientific and economic point of view. It has been shown, in fact, that for every euro invested in research, the profit for the National Health Service is equal to 3 euros and, in the case of clinical trials for oncology drugs, even 3.35 euros”.
The stages of the research
Scientific research is not an exclusive process. Its goals and successes are the consequence of a process shared by doctors, scientists, researchers and patients, united in the common goal of improving the health of the community. There pre-clinical testing it is essential to ensure that investigational drugs have an acceptable safety profile and a good chance of being effective.
Clinical studies on humans are the tools on which the actual development of a new drug is based. Their objective is to verify and evaluate the safety, absolute priority, and effectiveness of a new molecule through phases 1, 2, 3 and 4 (pharmacovigilance), before it becomes a drug available to patients.
“The process is very slow, gradual and can last up to ten years; healthy subjects or patients participating in a clinical study must always express their consent – explains Dominique Van Doorne, Treasurer and Scientific Director of the EUPATI APS Expert Patient Academy (AdPEE). Without good patient participation in clinical trials there would be no research. The idea of the ‘guinea pig’ patient is obsolete and outdatedtoday patients involved in clinical trials are followed with great attention and rigor. The Ethics Committees are the bodies that control and guarantee that the research is carried out in compliance with the participants and the international rules dictated by the Declaration of Helsinki and the Good Clinical Practices, or GCP”.
How much is the expert patient worth
The expert and aware patient is a central figure in clinical research, because he knows the disease from personal experience and is capable of representing the interests of patients with the skills acquired. The patient makes the difference, not only because he makes his knowledge available to the scientific community, but because he contributes to defining the most important clinical endpoints from the patient’s point of view, and not as the clinician or researcher perceives them, and to evaluate valuable data on quality of life with the disease.
“Being an informed patient means understanding the value of the research and how safe it is, it also allows you to understand the importance of active participation and the need to remain in the study until its conclusion – underlines Paola Kruger, Scientific Director of the EUPATI Italy Course and EUPATI Expert Patient . Correct information it is the basis for being able to consciously participate in clinical research. Training, however, gives the patient the opportunity to be involved – from the beginning – in the design of the project, in the development of the clinical protocol and subsequently in the collection of data generated by the patients”.
What the campaign proposes
The initiative proposes a journey behind the scenes of clinical research to let patients, caregivers and public opinion know how the complex and rigorous path that leads from the laboratory works new therapeutic options at the bed of the person being cared for.
“The EUPATI APS Expert Patient Academy (AdPEE) has always believed it was important to address citizens’ concerns regarding clinical trials – reports Nicola Merlin, President of the EUPATI APS Expert Patient Academy (AdPEE). We believe it is essential to combat fake news and highlight the value of participation in clinical trials (both for the advancement of science and for the potential benefit of the participants themselves), involve patients so that they can be active protagonists in decisions regarding their health and treatments. This campaign intends to build a ‘bridge’ of trust between the scientific community and public opinion: “research is us” is an important step towards greater collaboration and mutual alliance, key elements for the progress of science and public health” .
