Regulatory Aspects for Approval of Advanced Therapy Medicinal Products in the EU

Fürst-Ladani, Shayesteh; Bührer, Anja; Fürst, Walter; Schober-Ladani, Nathalie

doi:10.1007/164_2023_648

Cited by 2 publications

(1 citation statement)

References 18 publications

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“…Whilst regulatory policies are being adopted in Europe to facilitate the accelerated approval of ATMPs (Fürst-Ladani et al, 2023), the complexities of the existing pathways are often seen as a barrier by therapy developers (Pizevska et al, 2022). However, if marketing authorization is successfully obtained, gaining access to a market where there was previously unmet need can set up the product as the market leader, develop economies of scale, and potentially establish it as the new standard of care ("first-mover advantage").…”

Section: Introductionmentioning

confidence: 99%

Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries

Rejon-Parrilla,

Espin,

Garner

et al. 2023

Front. Pharmacol.

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Introduction: Advanced Therapy Medicinal Products are a type of therapies that, in some cases, hold great potential for patients without an effective current therapeutic approach but they also present multiple challenges to payers. While there are many theoretical papers on pricing and reimbursement (P&R) options, original empirical research is very scarce. This paper aims to provide a comprehensive international review of regulatory and P&R decisions taken for all ATMPs with centralized European marketing authorization in March 2022.Methods: A survey was distributed in July 2022 to representatives of 46 countries.Results: Responses were received from 20 countries out of 46 (43.5%). 14 countries reimbursed at least one ATMP. Six countries in this survey reimbursed no ATMPs.Conclusion: Access to ATMPs is uneven across the countries included in this study. This arises from regulatory differences, commercial decisions by marketing authorization holders, and the divergent assessment processes and criteria applied by payers. Moving towards greater equality of access will require cooperation between countries and stakeholders, for example, through the WHO Regional Office for Europe’s Access to Novel Medicines Platform.

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Section: Introductionmentioning

confidence: 99%

Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries

Rejon-Parrilla,

Espin,

Garner

et al. 2023

Front. Pharmacol.

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Update on the Clinical Applications of Mesenchymal Stem Cells

Khalid Ahmed

2023

J Stem Cell Ther Transplant

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Mesenchymal stem cells are heterogenous adult multipotent stromal cells that can be isolated from various sources including bone marrow, peripheral blood, umbilical cord blood, dental pulp, and adipose tissue. They have certain regenerative, anti-inflammatory, immunomodulatory, immunosuppressive, antimicrobial, and other properties that enable them to have several therapeutic and clinical applications including treatment of various autoimmune disorders; role in hematopoietic stem cell transplantation and regenerative medicine; treatment of skin, pulmonary and cardiovascular disorders; treatment of neurological and eye diseases; as well as treatment of various infections and their complications. Different factors including donor age, biological source, route of administration, and signaling pathways have an impact on the functions and consequently the clinical applications of mesenchymal stromal cells. The products of mesenchymal stem cells such as extracellular vesicles and exosomes reproduce the biological effects and most of the therapeutic actions of the parent stem cells. Genetic engineering and the use of specific mesenchymal stromal cell products have improved their clinical efficacy and decreased their adverse effects. However, despite the recent progress in the use of mesenchymal stem cells, the clinical application of these cells in the treatment of several diseases still faces real challenges that need to be resolved. The current status of mesenchymal stem cells and the controversies related to their clinical utilization in various disease conditions will be thoroughly discussed in this review.

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Regulatory Aspects for Approval of Advanced Therapy Medicinal Products in the EU

Cited by 2 publications

References 18 publications

Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries

Pricing and reimbursement mechanisms for advanced therapy medicinal products in 20 countries

Update on the Clinical Applications of Mesenchymal Stem Cells

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