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1.
Value Health ; 26(1): 138-150, 2023 01.
Artigo em Inglês | MEDLINE | ID: mdl-36031480

RESUMO

OBJECTIVES: Advanced therapy medicinal products (ATMPs) are drugs for human use for the treatment of chronic, degenerative, or life-threatening diseases that are based on genes, tissues, or cells. This article aimed to identify and critically review published economic analyses of ATMPs. METHODS: A systematic review of economic analyses of ATMPs was undertaken. Study characteristics, design, sources of data, resources and unit costs, modeling and extrapolation methods, study results, and sensitivity analyses were assessed. RESULTS: A total of 46 economic analyses of ATMP (from 45 articles) were included; 4 were cell therapy medicinal products, 33 gene therapy medicinal products, and 9 tissue-engineered products. 30 therapies had commercial marketing approval; 39 studies were cost-utility analysis, 5 were cost-effectiveness analysis, and 2 were cost only studies. Four studies predicted that the ATMP offered a step change in the management of the condition and 10 studies estimated that the ATMP would offer a lower mean cost. CONCLUSIONS: Comparison with historical controls, pooling of data, and use of techniques such as mixture cure fraction models should be used cautiously. Sensitivity analyses should be used across a plausible range of prices. Clinical studies need to be designed to align with health technology assessment requirements, including generic quality of life, and payers should aim for clarity of criteria. Regulators and national payers should aim for compatibility of registers to allow interchange of data. Given the increasing reliance on industry-funded economic analyses, careful critical review is recommended.


Assuntos
Marketing , Qualidade de Vida , Humanos , Análise Custo-Benefício
2.
Health Policy ; 126(12): 1248-1255, 2022 12.
Artigo em Inglês | MEDLINE | ID: mdl-36283859

RESUMO

Advanced therapy medicinal products (ATMPs) are a fast-growing field of medicine with wide potential application. Nevertheless, so far, only 19 have obtained European Union (EU) marketing authorisation and only 13 of these have translated successfully into clinical practice. This study conducts an umbrella review to identify the main barriers for the evaluation of ATMPs and their translation into clinical practice across the development lifecycle. 71 systematic reviews were included, of which 50 dealt primarily with effectiveness and safety, 13 with translation from pre-clinical to human subjects. Others dealt with economic issues and translation from health technology assessment to market access. The literature highlights the importance of synergistic research groups or networks that collaborate across the in-vitro science, preclinical and clinical investigation phases, and the role of private investor capital and public-private collaborations. Most ATMPs reviewed seem to have a favourable safety profile although considerable uncertainties remain. Randomised controlled trials are not always feasible in these patient groups. Greater sharing of data is recommended, both at preclinical and post-marketing real world evidence. There are considerable variations between EU countries in how they regulate hospital exemption for ATMPs, and this can lead to inequitable access for patients.


Assuntos
Marketing , Humanos , União Europeia
3.
Recent Pat Biotechnol ; 16(3): 243-255, 2022 08 03.
Artigo em Inglês | MEDLINE | ID: mdl-35240977

RESUMO

Extracellular vesicles derived from mesenchymal stromal cells (MSC-EVs) are gaining interest for medical purposes. The promising therapeutic effects exhibited in both preclinical and clinical studies have suggested that they may become an alternative for certain applications to cell-based therapies, which are subjected to stricter regulations. The commercial exploitation of these candidates requires a proper patent strategy from both the industry and public research organizations. Here, we performed a global patent literature analysis to identify key players and therapeutic applications in the field. Our results showed an increasing rate of patent publications since 2009, with Asia (specifically China) leading the patenting activity. The therapeutic use of MSC-EVs within patent literature covers a wide range of diseases, in which "Dermal and Wounds," "Neurology" and "Cardiovascular" are the main therapeutic areas. Moreover, most of these patents include "productby- process" claims, since the therapeutic effects of MSC-EVs could be influenced by their manufacturing process. Our results followed scientific and clinical literature trends.


Assuntos
Vesículas Extracelulares , Células-Tronco Mesenquimais , Ásia , Terapia Baseada em Transplante de Células e Tecidos , Patentes como Assunto
4.
J Nanobiotechnology ; 18(1): 174, 2020 Nov 23.
Artigo em Inglês | MEDLINE | ID: mdl-33228673

RESUMO

BACKGROUND: Treatment of patients affected by severe burns is challenging, especially due to the high risk of Pseudomonas infection. In the present work, we have generated a novel model of bioartificial human dermis substitute by tissue engineering to treat infected wounds using fibrin-agarose biomaterials functionalized with nanostructured lipid carriers (NLCs) loaded with two anti-Pseudomonas antibiotics: sodium colistimethate (SCM) and amikacin (AMK). RESULTS: Results show that the novel tissue-like substitutes have strong antibacterial effect on Pseudomonas cultures, directly proportional to the NLC concentration. Free DNA quantification, WST-1 and Caspase 7 immunohistochemical assays in the functionalized dermis substitute demonstrated that neither cell viability nor cell proliferation were affected by functionalization in most study groups. Furthermore, immunohistochemistry for PCNA and KI67 and histochemistry for collagen and proteoglycans revealed that cells proliferated and were metabolically active in the functionalized tissue with no differences with controls. When functionalized tissues were biomechanically characterized, we found that NLCs were able to improve some of the major biomechanical properties of these artificial tissues, although this strongly depended on the type and concentration of NLCs. CONCLUSIONS: These results suggest that functionalization of fibrin-agarose human dermal substitutes with antibiotic-loaded NLCs is able to improve the antibacterial and biomechanical properties of these substitutes with no detectable side effects. This opens the door to future clinical use of functionalized tissues.


Assuntos
Antibacterianos , Lipídeos/química , Nanoestruturas , Pele Artificial , Engenharia Tecidual/métodos , Amicacina/química , Amicacina/farmacologia , Antibacterianos/química , Antibacterianos/farmacologia , Materiais Biocompatíveis/química , Proliferação de Células/efeitos dos fármacos , Sobrevivência Celular/efeitos dos fármacos , Células Cultivadas , Colistina/análogos & derivados , Colistina/química , Colistina/farmacologia , Portadores de Fármacos/química , Portadores de Fármacos/toxicidade , Fibroblastos/citologia , Humanos , Nanoestruturas/química , Nanoestruturas/toxicidade
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