scholarly journals Perspectives, Expectations, and Concerns of European Patient Advocates on Advanced Therapy Medicinal Products

2021 ◽  
Vol 8 ◽  
Author(s):  
Stefano Benvenuti ◽  
Chiuhui Mary Wang ◽  
Simona Borroni
Keyword(s):  
Medicinal Products ◽  
Structured Interviews ◽  
Advanced Therapy Medicinal Products ◽  
Geographical Concentration ◽  
Advanced Therapy ◽  
Wide Range ◽  
Reimbursement Scheme ◽  
Patient Advocates ◽  
Degree Of Convergence ◽  
Whole Life Cycle

This paper presents the results of a qualitative study based on semi-structured interviews of 10 expert patient advocates on several different issues around Advanced Therapy Medicinal Products (ATMPs). The interviews were conducted between February and May 2020 based on a guideline with a list of 8 topics that covered concerns about safety and ethics, access problems and limitations, pricing of ATMPs and educational needs for patient communities. Overall, the interviewees expressed a high degree of convergence of opinions on most of the topics and especially on the identification of the reasons for concern. Conversely, when asked about possible solutions, quite a wide range of solutions were proposed, although with many common points. However, it highlights that the debate is still in its infancy and that there are not yet consolidated positions across the whole community. A general concern emerging from all the interviews is the potential limitation of access to approved ATMPs, both due to the high prices and to the geographical concentration of treatment centers. However, patients recognize the value of a model with a limited number of specialized clinical centers administering these therapies. On the ethical side, patients do not show particular concern as long as ATMPs and the underlying technology is used to treat severe diseases. Finally, patients are asking for both more education on ATMPs as well as for a more continuous involvement of patient representatives in the whole “life-cycle” of a new ATMP, from the development phase to the authorization, from the definition of the reimbursement scheme to the collection of Real Word Data on safety and long-term efficacy of the treatment.

scholarly journals Is the Conjunctiva a Potential Target for Advanced Therapy Medicinal Products?

Pharmaceutics ◽  
2021 ◽  
Vol 13 (8) ◽  
pp. 1140
Author(s):  
Yolanda Diebold ◽  
Laura García-Posadas
Keyword(s):  
Tissue Engineering ◽  
Gene Therapy ◽  
Cell Therapy ◽  
Ocular Surface ◽  
Ocular Tissue ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Potential Target ◽  
Advanced Therapy ◽  
Wide Range

The conjunctiva is a complex ocular tissue that provides mechanical, sensory, and immune protection for the ocular surface. It is affected by many diseases through different pathological mechanisms. If a disease is not treated and conjunctival function is not fully restored, the whole ocular surface and, therefore, sight is at risk. Different therapeutic approaches have been proposed, but there are still unsolved conjunctival alterations that require more sophisticated therapeutic options. Advanced therapy medicinal products (ATMPs) comprise a wide range of products that includes cell therapy, tissue engineering, and gene therapy. To the best of our knowledge, there is no commercialized ATMP specifically for conjunctival treatment yet. However, the conjunctiva can be a potential target for ATMPs for different reasons. In this review, we provide an overview of the advances in experimental phases of potential ATMPs that primarily target the conjunctiva. Important advances have been achieved through the techniques of cell therapy and tissue engineering, whereas the use of gene therapy in the conjunctiva is still marginal. Undoubtedly, future research in this field will lead to achieving commercially available ATMPs for the conjunctiva, which may provide better treatments for patients.

scholarly journals Advanced Therapy Medicinal Products for Eye Diseases: Goals and Challenges

Pharmaceutics ◽  
2021 ◽  
Vol 13 (11) ◽  
pp. 1819
Author(s):  
Yolanda Diebold ◽  
Laura García-Posadas
Keyword(s):  
Eye Diseases ◽  
Medicinal Products ◽  
Advanced Therapy Medicinal Products ◽  
Advanced Therapy ◽  
Wide Range

Advanced therapy medicinal products (ATMPs) are a novel class of medicines with enormous potential to improve treatments for a wide range of diseases, including those affecting eye structures [...]

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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