The Magistral Preparation of Advanced Therapy Medicinal Products (ATMPs)

2020 ◽  
pp. 1-7
Keyword(s):  
Marketing Authorisation ◽  
Health Care Systems ◽  
Human Keratinocytes ◽  
Public Health Care ◽  
Medicinal Products ◽  
Bacteriophage Therapy ◽  
Access To Treatment ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy ◽  
Advanced Therapies

Advanced Therapy Medicinal Products (ATMPs) embody innovative therapies that have created great hope for patients suffering from previously untreatable diseases. Unfortunately, the pharaonic cost to produce and authorise ATMPs is a challenge for both patients and public health care systems, ultimately reducing patients’ access to treatment. Over the last 11 years, only 15 ATMP marketing authorisation applications received a positive draft opinion from the European Medicines Agency’s (EMA’s) Committee for Advanced Therapies (CAT). Moreover, due to poor return on investment, several ATMPs have already been removed from the market. In addition to the centralised procedure to obtain a marketing authorisation, the legislator foresees an alternative route for authorising ATMPs, the so-called “ATMP Hospital Exemption”. However, such ATMPs must be produced on a limited scale, on a non-routine basis. As a result, valuable ATMP therapies that have been used for years in hospitals may disappear. To avoid this, we propose, in this paper, an additional possibility to regularise ATMPs: the “Magistral Preparation of ATMPs”. It is a feasible pathway, which was already proposed for bacteriophage therapy, and which is particularly suitable for personalised therapies and considerably decreases the cost of the final products. We also discuss the practical impact of the ATMP regulation for (for-profit) industries and for (non-profit) hospitals. Two practical examples, the cultured human chondrocytes and the cultured human keratinocytes, are discussed.

Exploratory assessment of the current EU regulatory framework for development of advanced therapies

1969 ◽  
Vol 16 (4) ◽  
Author(s):  
Pawanbir Singh ◽  
Laure Brévignon-Dodin ◽  
Satya P Dash
Keyword(s):  
Regenerative Medicine ◽  
Research And Development ◽  
Regulatory Framework ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Regulatory Strategy ◽  
Technology Changes ◽  
Advanced Therapy ◽  
Advanced Therapies ◽  
The Eu

The Advanced Therapy Medicinal Products (ATMP) Regulation provides a necessary regulatory framework for the commercialisation and use of regenerative medicine-based therapeutic products in the EU. However, concerns have been raised about the appropriateness of the regulatory strategy it has adopted to address different, complex and evolving categories of medicinal products. This article explores some of the potential shortfalls of the ATMP Regulation with regard to facilitating the research and development of advanced therapies in the present and in the future. It concludes that while providing a much needed harmonised regulatory framework for the companies operating in the sector, the new regulation has yet to demonstrate its capacity to keep up with radical technology changes.

scholarly journals Value-based pricing for advanced therapy medicinal products: emerging affordability solutions

2021 ◽  
Author(s):  
Elisabete Gonçalves
Keyword(s):  
High Price ◽  
Value Assessment ◽  
Medicinal Products ◽  
Fair Price ◽  
Advanced Therapy Medicinal Products ◽  
Health Technologies ◽  
Advanced Therapy ◽  
Advanced Therapies ◽  
Managed Entry Agreements ◽  
Access And Affordability

AbstractThe emergence of advanced therapy medicinal products (ATMPs), a disruptive class of health technologies, is generating important challenges in terms of value assessment and their high prices introduce critical access and affordability concerns. The aim of this article is to analyze the challenges of traditional value assessment and price and reimbursement methods in the evaluation of ATMPs and to characterize the current and prospective financing solutions that may ensure patient access and affordability for these health technologies. Standard Health Technology Assessment (HTA) is not designed for ATMPs, and may delay access to these health technologies, thus a broader concept of value is required. As a consequence, value-based pricing methodologies have been gaining terrain to cope with the specific challenges of ATMPs. The pricing and reimbursement framework should ensure the balance between encouragements to innovation and maximization of value for money for payers, through the attribution of a fair price to new health technologies. Early scientific advice by regulatory and HTA bodies to developers is key, as it will contribute to diminish the perspective gap between developers, regulators and payers. The high efficacy/high price dynamic of many advanced therapies will demand novel financing models, both in the EU and US. Managed entry agreements (MEA), with financing being conditional to the submission of additional evidence, associated with methods of leased payments, may offer effective strategies to address the uncertainties caused by the evidence gap associated with ATMPs, ensuring affordable and sustained access.

scholarly journals Systematic Review: Allogenic Use of Stromal Vascular Fraction (SVF) and Decellularized Extracellular Matrices (ECM) as Advanced Therapy Medicinal Products (ATMP) in Tissue Regeneration

2020 ◽  
Vol 21 (14) ◽  
pp. 4982 ◽  
Author(s):  
Pietro Gentile ◽  
Aris Sterodimas ◽  
Jacopo Pizzicannella ◽  
Laura Dionisi ◽  
Domenico De Fazio ◽  
...  
Keyword(s):  
Systematic Review ◽  
Meta Analysis ◽  
Stromal Vascular Fraction ◽  
Lateral Epicondylitis ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy ◽  
Meta Analyses ◽  
Clinical Experiments

Stromal vascular fraction (SVF) containing adipose stem cells (ASCs) has been used for many years in regenerative plastic surgery for autologous applications, without any focus on their potential allogenic role. Allogenic SVF transplants could be based on the possibility to use decellularized extracellular matrix (ECM) as a scaffold from a donor then re-cellularized by ASCs of the recipient, in order to develop the advanced therapy medicinal products (ATMP) in fully personalized clinical approaches. A systematic review of this field has been realized in accordance with the Preferred Reporting for Items for Systematic Reviews and Meta-Analyses-Protocols (PRISMA-P) guidelines. Multistep research of the PubMed, Embase, MEDLINE, Pre-MEDLINE, PsycINFO, CINAHL, Clinicaltrials.gov, Scopus database, and Cochrane databases has been conducted to identify articles and investigations on human allogenic ASCs transplant for clinical use. Of the 341 articles identified, 313 were initially assessed for eligibility on the basis of the abstract. Of these, only 29 met all the predetermined criteria for inclusion according to the PICOS (patients, intervention, comparator, outcomes, and study design) approach, and 19 have been included in quantitative synthesis (meta-analysis). Ninety-one percent of the studies previously screened (284 papers) were focused on the in vitro results and pre-clinical experiments. The allogenic use regarded the treatment of perianal fistulas, diabetic foot ulcers, knee osteoarthritis, acute respiratory distress syndrome, refractory rheumatoid arthritis, pediatrics disease, fecal incontinence, ischemic heart disease, autoimmune encephalomyelitis, lateral epicondylitis, and soft tissue defects. The information analyzed suggested the safety and efficacy of allogenic ASCs and ECM transplants without major side effects.

Recent EU Regulatory Law Developments for the Pharmaceutical Industry

2012 ◽  
Vol 3 (3) ◽  
pp. 421-424
Author(s):  
Audrey Chenesseau ◽  
Anna Pavlou
Keyword(s):  
Pharmaceutical Industry ◽  
Data Protection ◽  
Pharmaceutical Companies ◽  
Medicinal Products ◽  
News Report ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy ◽  
Regulatory Law ◽  
Pricing And Reimbursement

The present Flash News Report sets outs recent EU regulatory law developments (as of 25 June 2012) which pharmaceutical companies should be aware of in the areas of Pharmacovigilance, Information on Medicines, Pricing and Reimbursement, EU Data Protection and Advanced Therapy Medicinal Products.

Nanomaterials in Medical Devices: Regulations' Review and Future Perspectives

2021 ◽  
pp. 1910-1917
Author(s):  
Karolina Jagiello ◽  
Anita Sosnowska ◽  
Alicja Mikolajczyk ◽  
Tomasz Puzyn
Keyword(s):  
Risk Assessment ◽  
Risk Management ◽  
Risk Analysis ◽  
Medical Devices ◽  
Future Perspectives ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Probability Of Occurrence ◽  
Advanced Therapy ◽  
Related Harm

This article gives a brief description of the existing regulations related to biomaterials safety that need to be considered before it is introduced into EU market. According to these regulations, the risk analysis should include two characteristics: probability of occurrence of harm, and severity. Identified user-related harm should be reduced by managing the risk. Additionally, the review presents an overview of engineered biomaterials (EBMs), which in combination with nanoscale components (NPs) have shown promises in Advanced Therapy Medicinal Products (ATMP) and Medical Devices (MD). In this article, recent challenges, objectives and perspectives in risk assessment and risk management of ATMP and MD composed of nanobiomaterials were also highlighted.

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