THE PROBLEMATIC ASPECTS OF THE HOSPITAL EXEMPTION REGULATION FOR ADVANCED THERAPY MEDICINAL PRODUCTS

2014 ◽  
Vol 21 (1) ◽  
pp. 93-99
Author(s):  
Tatjana Ivaškienė
Keyword(s):  
Industrial Process ◽  
Medical Practitioner ◽  
Pharmaceutical Products ◽  
Medicinal Products ◽  
Member States ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy ◽  
Somatic Cell Therapy ◽  
Special Rules ◽  
Set Up

Advanced therapy medicinal products – new category of biological pharmaceutical products, which covers somatic cell therapy, gene therapy, tissue-engineered products and combined advanced therapy medicinal products. Regulation (EC) No 1394/2007 defined advanced therapy medicinal products and set up special rules for advanced therapy medicinal products intended to be placed on the market in Member States and prepared industrially or manufactured by a method involving an industrial process. The scope of Regulation (EC) No 1394/2007 is not applicable for advanced therapy medicinal products which are prescribed under the exclusive professional responsibility of a medical practitioner, prepared on a non-routine basis according to specific quality standards and used within the same Member State in a hospital, but at the same time it is stressed that Comunity rules related to quality and safety should not be breaked for these products. For this purpose Member States should provide

PP61 Advanced Therapy Medicinal Products Germany: Drugs Or Methods Review?

2019 ◽  
Vol 35 (S1) ◽  
pp. 48-49
Author(s):  
Elvira Müller ◽  
Kurt Neeser ◽  
Ilse-Barbara Oelze
Keyword(s):  
Treatment Method ◽  
Point Of View ◽  
Benefit Assessment ◽  
Grey Zone ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Social Law ◽  
Ambiguous Situation ◽  
Advanced Therapy ◽  
Somatic Cell Therapy

IntroductionAdvanced Therapy Medicinal Products (ATMPs) comprise medicines for human use based on gene therapy, somatic cell therapy or bioprocessed tissue products. ATMPs are pharmaceutically manufactured drugs and mostly subject to central authorization requirements. In terms of social law, it is an ambiguous situation and more heterogeneously dealt with. ATMPs are assigned to method evaluation as well as to the Arzneimittelmarkt-Neuordnungsgesetz (AMNOG) procedure designated for drugs.MethodsGuidelines from Gemeinsame Bundesausschuss (G-BA), Institute for Quality and Efficiency in Health Care (IQWiG) and respective legislation, consultation results and methods/medical devices (MDs) evaluations according to §137h and for drugs according to AMNOG were reviewed and analyzed. Decision criteria and reasoning, assessment outcomes and potential impact on price negotiations were the main aspects for comparison.ResultsATMPs are subject to benefit assessment, with a decision at first on whether to be evaluated as a drug (e.g., Alofisel) or a method/device (e.g., Holoclar). By definition, an ATMP is classified as a treatment method, if the correct administration has at least the same significance for a successful therapy outcome as its mode of action. Depending on the respective decision, an evaluation as method follows or it must undergo the AMNOG process. According to G-BA's and IQWiG's point of view, randomized controlled trials (RCTs) are the “gold standard” for a benefit assessment of new therapies, including ATMPs. However, conduction of RCTs is not always possible for ATMPs which creates a disadvantage in the assessment right from the beginning. Otherwise no distinction is made between drugs and ATMPs in terms of reimbursement modalities. Outcomes based agreements could help overcoming inequalities and lead to quality-oriented reimbursement.ConclusionsATMPs represent a grey zone causing difficulties in classifying them either as method or drug. For individualized therapies evidence beyond RCTs and new reimbursement possibilities should be considered. Until new regulations are in place it is advisable to enter early into respective discussions with authorities.

scholarly journals Advanced Therapy Medicinal Products for the Eye: Definitions and Regulatory Framework

Pharmaceutics ◽  
2021 ◽  
Vol 13 (3) ◽  
pp. 347
Author(s):  
Marina López-Paniagua ◽  
Ana de la Mata ◽  
Sara Galindo ◽  
Francisco Blázquez ◽  
Margarita Calonge ◽  
...  
Keyword(s):  
Legal Framework ◽  
Eye Diseases ◽  
The European Union ◽  
Medicinal Products ◽  
Therapeutic Approaches ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy ◽  
Innovative Products ◽  
Somatic Cell Therapy ◽  
Human Use

Advanced therapy medicinal products (ATMPs) are a group of innovative and complex biological products for human use that comprises somatic cell therapy medicinal products, tissue engineered products, gene therapy medicinal products, and the so-called combined ATMPs that consist of one of the previous three categories combined with one or more medical devices. During the last few years, the development of ATMPs for the treatment of eye diseases has become a fast-growing field as it offers the potential to find novel therapeutic approaches for treating pathologies that today have no cure or are just subjected to symptomatic treatments. Therefore, it is important for all professionals working in this field to be familiar with the regulatory principles associated with these types of innovative products. In this review, we outline the legal framework that regulates the development of ATMPs in the European Union and other international jurisdictions, and the criteria that each type of ATMP must meet to be classified as such. To illustrate each legal definition, ATMPs that have already completed the research and development stages and that are currently used for the treatment of eye diseases are presented as examples.

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.

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.

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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