The battle against time that affects the entire world population anxious to have effective treatments and vaccines against the new Coronavirus, for those suffering from serious illnesses a daily reality known long before this epidemic. They know very well, for example, patients with a tumor for whom receiving a new drug quickly can make a fundamental difference, especially for those with a advanced cancer or those who have already tried all the effective standard therapies but have not benefited from them. In these situations it can be a great advantage to be able to participate in an experimentation with an innovative treatment that is not yet on the market and that will be (if it proves effective) even after several years.
No economic costs for the sick
At what price? Economic nobody, the sick pay nothing – he replies Giordano Beretta, national president of the Italian Association of Medical Oncology (Aiom) and head of Medical Oncology at Humanitas Gavazzeni in Bergamo -. And with all possible safeguards: before reaching human tests, a new molecule is initially studied in the laboratory and on guinea pigs. Before the trial starts on patients, it must also be approved by an ethics committee and the green light generally comes only if there are strong data on safety for toxicity (i.e. you take care to ascertain before the possible side effects are not too heavy ) and only if there are good reasons to think the treatment is effective. Certainly that the new drug is superior to the standard treatment cannot be guaranteed, nor is it possible to know whether it will help the individual patient.
How to participate
To be enrolled in a study you must have characteristics that are written in the protocol approved by the ethics committee, where precise inclusion and exclusion criteria are indicated: it is not enough to have a type of tumor (for example breast), but they are taken into account considering many different and complex biological characteristics (cancer subtype, genetic mutations, etc.) and clinical characteristics (general conditions of the patient, therapies already carried out, any other pathologies present and so on). How do you participate? Not the patient who has to worry about looking for it, but the oncologist who should propose it to him, after having inquired about any experiments indicated in his case, stresses Beretta. At any time, then, a patient can decide to leave a study and will be guaranteed a another treatment suitable for your case: if there are no more effective ones to fight the cancer, palliative care and supportive therapies are always available to control the symptoms and improve the quality of life.
Facilitations: early access to the new treatment
And experimentation is not the only option to be able to receive treatments not yet available in clinical practice – he recalls Francesco Perrone, director of the Clinical Trials Unit of the National Cancer Institute Pascale of Naples and member of the national board of Aiom -. If a new drug has completed the experimental process, but has not yet been approved by AIFA and therefore not reimbursed by the National Health Service, it can still be accessed through the “early access” methods, which translated from English means early access. It is an important opportunity that concerns safe treatments with proven efficacy as they have already received a favorable opinion from the European authorities. But we need to streamline the bureaucratic process, which is still too long and complex, and better prepare the oncologists (for this reason Aiom has activated special courses) who in a recent survey highlighted their difficulties in being able to administer treatments to their patients by exploiting the various early access strategies adds Perrone. The procedures for requesting drugs should be simplified and response times by Aifa shortened, but these systems offer in different ways an opportunity to access, promptly and at the expense of the National Health Service or the manufacturers, to treatments potentially useful for seriously ill patients. which have no therapeutic alternatives.
Any rules that can help
There are particular rules that allow and regulate the prescription of drugs before Aifa’s go-ahead – explains Massimo Di Maio, Aiom national secretary and director of Oncology at the Mauritian Order Hospital of Turin -. The first is law 648/1996 which allows the dispensing of a drug, in the course of clinical trials or with a different therapeutic indication, having been included in a list approved by AIFA. Then there is the Aifa Fund of 5% (law 326/2003) which allows the use of orphan drugs for the treatment of rare diseases or treatments awaiting commercialization. There is law 94/1998 which authorizes the “off label” use (outside the ordinary rules) of particular treatments under the responsibility of a doctor and, finally, there are the nominal uses that are allowed thanks to the free supply of the drug from part of the pharmaceutical companies. Finally, there is the legislation on compassionate use (Ministerial Decree 8 May 2003 and Ministerial Decree 7 September 2017.
What compassionate use means
It is possible to resort to the therapeutic use of a medicinal product undergoing clinical trials (so-called compassionate use) for the treatment of patients with serious illnesses, rare diseases, rare cancers or in conditions of life-threatening disease, for whom valid therapeutic alternatives are not available or who cannot be included in a clinical trial or, for the purposes of therapeutic continuity, for patients already treated with clinical benefit in the context of a completed clinical trial explains Di Maio. Compassionate use means the free supply by the pharmaceutical company of medicines not yet authorized on the Italian territory undergoing clinical trials, but also of medicines with marketing authorization for indications other than those authorized. The request is made by a doctor for nominal use (i.e. for a single patient) or in the context of therapeutic use programs, who submits it to the evaluation of the ethics committee of the hospital, after having received the ok from the manufacturer concludes Di Maio.
May 23, 2021 (change May 23, 2021 | 19:37)
© REPRODUCTION RESERVED