The new frontier of personalized medicine (a medical model that proposes the personalization of health, with medical decisions, practices, and products tailored to the patient) is represented by Advanced Therapy Medicinal Products (ATMP), which offer opportunities for innovative treatment and are classified into four groups:
Gene therapy drugs: capable of transferring a working copy of a defective gene.
Somatic cell therapy medicines are based on the use of cells or tissues manipulated to the point of treating, preventing, or diagnosing diseases.
Tissue-engineered medicinal products: contain cells or tissues to repair, regenerate, or replace human tissues.
Combined advanced therapy medicinal products: a combination of cell or tissue-based medical devices.
Advanced therapies are becoming not only a hope but a reality of cure for patients suffering from serious diseases who have not yet found a concrete therapeutic answer.
Advanced therapies are the emblem of personalized medicine since they are treatments that start from the patient's cells and therefore act on the genetic cause at the origin of the pathologies, also in some cases even with a single administration the ability to offer benefits to the patient forever.
The costs of this technology are high so that access by patients is often hindered by public health budget limits, the proposal at the Italian level, at least for the moment, is to transform the expenditure items relating to advanced therapies from expenses current at investment expenses (or capital expenditure), as it should be, having these therapies the ability to guarantee results over the years even with a single administration, are often one-shot.
Description of the project
Cittadinanzattiva is engaged in a project to promote at the European level the importance of access to advanced therapies by the highest possible number of patients in the light of the fact that by 2030 there could be as many as 60 advanced therapies that could change the life of 350 thousand patients, but to do this, the accounting system must be changed.
To raise awareness of this need, which is at the same time a great opportunity, CA will organize the following events:
A workshop in Italy and Europe on advanced therapies with pre & post instant surveys.
An event organized in the framework of the European Parliament, in person or online depending on the progress of the pandemic situation.
A call to action aimed at European decision-makers to stimulate decision-makers to redevelop the expenses for advanced therapies, considering them an investment.
The general objective is to promote the importance of advanced therapies articulated in the specific objectives listed below:
Bring the issue of access to the attention of the patient and civic associations and create consensus regarding the "Italian solution", that is, the fact of accounting for expenditure as a multi-year investment and not as a current expenditure (like a drug that runs out of results in the very short term).
Based on the European Charter, stimulate widespread awareness of the rights to personalized treatments, innovation, and access.
Promote empowerment on the issue of advanced therapies.
Strengthen, on the subject, the coalition between Patient and Citizen Associations in Italy and Europe, able to grasp the strategic importance of scientific innovation and able to "push" towards decision-makers to guarantee broader and fairer access.
- Rationale of the initiative
- An overall overview about ATMPs
- Technical Policy Report from the #VITA Working Group “THE ECONOMIC EVALUATION OF ADVANCED THERAPY MEDICINAL PRODUCTS: CHARACTERISTICS AND REASONS FOR A NEW ECONOMIC AND ACCOUNTING APPROACH” (EN – IT).
Our recent press articles (all in IT language)
- Entro il 2030 in arrivo 60 nuove terapie geniche
- Sedicimila terapie avanzate in sperimentazione, 60 in arrivo. La sfida alle malattie (oggi) incurabili
- Terapie avanzate, serve cambiare le politiche di bilancio europee
- Le opportunità e le sfide delle nuove terapie avanzate (1 – 2)
To know more:
- Advanced Therapies Medicinal Products revolution & the respect of the patients’ right to access to care: a call to action
- ATMPs revolution: empowering leaders of EU civic and patients’ advocacy groups
This project was carried out with the unconditional contribution of #VITA which includes:
Bristol Myers Squibb, Gilead Sciences, Janssen,Pfizer, PTC Therapeutics, Roche, Vertex
For further information consult the Linkdin page or don’t hesitate to contact Maddalena D’Urso: