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Article ; Online: Financing and Reimbursement of Approved Advanced Therapies in Several European Countries.

Iglesias-López, Carolina / Agustí, Antònia / Vallano, Antoni / Obach, Mercè

Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research

2023 Volume 26, Issue 6, Page(s) 841–853

Abstract: Objectives: The uncertainty in the cost-benefit of advanced therapy medicinal products (ATMPs) is a current challenge for their reimbursement in health systems. This study aimed to provide a comparative analysis of the National Health Authorities (NHAs) ...

Abstract	Objectives: The uncertainty in the cost-benefit of advanced therapy medicinal products (ATMPs) is a current challenge for their reimbursement in health systems. This study aimed to provide a comparative analysis of the National Health Authorities (NHAs) reimbursement recommendations issued in different European countries. Methods: The NHA reimbursement recommendations for the approved ATMPs were compared among 8 European Union (EU) Countries (EU8: Ireland, England/Wales, Scotland, The Netherlands, France, Germany, Spain, and Italy). The search was carried out until December 31, 2021. Results: A total of 19 approved ATMPs and 76 appraisal reports were analyzed. The majority of the ATMPs were reimbursed, although with uncertainty in added therapeutic value. No relationship between the type of the European Medicines Agency approval and reimbursement was found. Managed entry agreements, such as payment by results, were necessary to ensure market access. The main issue during the evaluation was to base the cost-effectiveness analyses on assumptions because of the limited long-term data. The estimated incremental cost-effectiveness ratio among countries reveals high variability. Overall, the median time to NHA recommendation for the EU8 is in the range of 9 to 17 months. Conclusions: Transparent, harmonized, and systematic assessments across the EU NHAs in terms of cost-effectiveness, added therapeutic value, and grade of innovativeness are needed. This could lead to a more aligned access, increasing the EU market attractiveness and raising public fairness in terms of patient access and pricing.
MeSH term(s)	Humans ; Europe ; France ; Germany ; European Union ; Cost-Benefit Analysis
Language	English
Publishing date	2023-01-13
Publishing country	United States
Document type	Journal Article
ZDB-ID	1471745-1
ISSN	1524-4733 ; 1098-3015
ISSN (online)	1524-4733
ISSN	1098-3015
DOI	10.1016/j.jval.2022.12.014
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Article ; Online: Temporary derogation from European environmental legislation for clinical trials of genetically modified organisms for coronavirus disease 2019.

Iglesias-Lopez, Carolina

Cytotherapy

2020 Volume 23, Issue 1, Page(s) 10–11

Abstract: Attempts to streamline environmental procedures for those products containing or consisting of genetically modified organisms (GMOs) among the European Union (EU) Member States are ongoing but still need to be further developed. These procedures can be ... ...

Abstract	Attempts to streamline environmental procedures for those products containing or consisting of genetically modified organisms (GMOs) among the European Union (EU) Member States are ongoing but still need to be further developed. These procedures can be complex, resource-intensive and time-consuming. Some candidate vaccines currently under development for COVID-19 include genetically modified viruses, which may be considered GMOs. Given the public health emergency caused by the COVID-19 outbreak, on July 15, 2020, the European Parliament approved a temporary derogation of the European environmental requirements to facilitate that those clinical trials with GMOs intended to treat or prevent COVID-19 can start as soon as possible in Europe. This measure has been very controversial, since it could entail risks to human health and the environment, and could be seen as unfair for other products targeting unmet medical needs. With the adoption of this measure, the bottlenecks and obstacles for the development of innovative GMO-based medicines in the EU that the environmental legislation entails have become even more evident.
MeSH term(s)	COVID-19/epidemiology ; COVID-19/prevention & control ; COVID-19/virology ; COVID-19 Vaccines/genetics ; Clinical Trials as Topic/legislation & jurisprudence ; Europe/epidemiology ; European Union ; Humans ; Organisms, Genetically Modified ; Pandemics/prevention & control ; Risk Assessment ; SARS-CoV-2/genetics ; SARS-CoV-2/immunology
Chemical Substances	COVID-19 Vaccines
Keywords	covid19
Language	English
Publishing date	2020-09-26
Publishing country	England
Document type	Journal Article
ZDB-ID	2039821-9
ISSN	1477-2566 ; 1465-3249
ISSN (online)	1477-2566
ISSN	1465-3249
DOI	10.1016/j.jcyt.2020.09.005
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Article: Temporary derogation from European environmental legislation for clinical trials of genetically modified organisms for coronavirus disease 2019

Iglesias-Lopez, Carolina

Cytotherapy

Keywords	covid19
Publisher	WHO
Document type	Article
Note	WHO #Covidence: #795221
Database	COVID19

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Article: Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States.

Iglesias-López, Carolina / Agustí, Antonia / Obach, Mercè / Vallano, Antonio

Frontiers in pharmacology

2019 Volume 10, Page(s) 921

Abstract: Advanced therapy medicinal products (ATMPs) are a fast-growing field of innovative therapies. The European Union (EU) and the United States (US) are fostering their development. For both regions, ATMPs fall under the regulatory framework of biological ... ...

Abstract	Advanced therapy medicinal products (ATMPs) are a fast-growing field of innovative therapies. The European Union (EU) and the United States (US) are fostering their development. For both regions, ATMPs fall under the regulatory framework of biological products, which determines the legal basis for their development. Sub-classifications of advanced therapies are different between regions, while in EU, there are four major groups, i.e., gene therapy, somatic cell therapy, tissue-engineered therapies, and combined advanced therapies; in US, the sub-classification covers two major groups of products, i.e., gene therapy and cellular therapy. The inclusion criteria that define a gene therapy are equivalent in both regions, and the exclusion criteria are directly related to the indications of the product. In the EU, there is a clear differentiation between cell- and tissue-based products regarding their classification as advanced therapies or coverage by other legal frameworks, whereas in US, there is a broader classification about whether or not these products can be categorized as biologic products. Both in EU and in US, in order to classify a cell- or a tissue-based product as an advanced therapy, it must be ensured that the processing of the cells implies a manipulation that alters their biological characteristics, although the term of manipulation in US differentiates between structural and non-structural cells and tissues. The regulatory terminology used to define ATMPs and their sub-classification reveals some differences between EU and US.
Language	English
Publishing date	2019-08-30
Publishing country	Switzerland
Document type	Journal Article
ZDB-ID	2587355-6
ISSN	1663-9812
ISSN	1663-9812
DOI	10.3389/fphar.2019.00921
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Article: Current landscape of clinical development and approval of advanced therapies.

Iglesias-Lopez, Carolina / Agustí, Antonia / Vallano, Antoni / Obach, Merce

Molecular therapy. Methods & clinical development

2021 Volume 23, Page(s) 606–618

Abstract: Advanced therapy medicinal products (ATMPs) are innovative therapies that mainly target orphan diseases and high unmet medical needs. The uncertainty about the product's benefit-risk balance at the time of approval, the limitations of nonclinical ... ...

Abstract	Advanced therapy medicinal products (ATMPs) are innovative therapies that mainly target orphan diseases and high unmet medical needs. The uncertainty about the product's benefit-risk balance at the time of approval, the limitations of nonclinical development, and the complex quality aspects of those highly individualized advanced therapies are playing a key role in the clinical development, approval, and post-marketing setting for these therapies. This article reviews the current landscape of clinical development of advanced therapies, its challenges, and some of the efforts several stakeholders are conducting to move forward within this field. Progressive iteration of the science, methodologically sound clinical developments, establishing new standards for ATMPs development with the aim to ensure consistency in clinical development, and the reproducibility of knowledge is required, not only to increase the evidence generation for approval but to set principles to achieve translational success in this field.
Language	English
Publishing date	2021-11-11
Publishing country	United States
Document type	Journal Article ; Review
ZDB-ID	2872938-9
ISSN	2329-0501 ; 2329-0501
ISSN (online)	2329-0501
ISSN	2329-0501
DOI	10.1016/j.omtm.2021.11.003
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Article: Methodological Characteristics of Clinical Trials Supporting the Marketing Authorisation of Advanced Therapies in the European Union.

Iglesias-Lopez, Carolina / Agustí, Antònia / Vallano, Antonio / Obach, Merce

Frontiers in pharmacology

2021 Volume 12, Page(s) 773712

Abstract: Several advanced therapy medicinal products (ATMPs) have been approved in the European Union (EU). The aim of this study is to analyse the methodological features of the clinical trials (CT) that supported the marketing authorization (MA) of the approved ...

Abstract	Several advanced therapy medicinal products (ATMPs) have been approved in the European Union (EU). The aim of this study is to analyse the methodological features of the clinical trials (CT) that supported the marketing authorization (MA) of the approved ATMPs in the EU. A systematic review of the characteristics of pivotal CT of ATMPs approved in the EU until January 31st, 2021 was carried out. A total of 17 ATMPs were approved and 23 CT were conducted to support the MA (median, 1, range, 1-3). Of those studies, 8 (34.78%) were non-controlled and 7 (30.43%) used historical controls. Only 7 (30.4%) were placebo or active-controlled studies. Among all CT, 21 (91.3%) were open-label and 13 (56.52%) had a single-arm design. To evaluate the primary endpoint, 18 (78.26%) studies used an intermediate and single variable. The median (IQR) number of patients enrolled in the studies was 75 (22-118). To date, ATMPs' approval in the EU is mainly supported by uncontrolled, single-arm pivotal CT. Although there is a trend toward an adaptive or a life cycle approach, a switch to more robust clinical trial designs is expected to better define the benefit and the therapeutic added value of ATMPs.
Language	English
Publishing date	2021-11-29
Publishing country	Switzerland
Document type	Journal Article ; Review
ZDB-ID	2587355-6
ISSN	1663-9812
ISSN	1663-9812
DOI	10.3389/fphar.2021.773712
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Article ; Online: Comparison of regulatory pathways for the approval of advanced therapies in the European Union and the United States.

Iglesias-Lopez, Carolina / Obach, Mercè / Vallano, Antonio / Agustí, Antonia

Cytotherapy

2021 Volume 23, Issue 3, Page(s) 261–274

Abstract: Background aims: Regulatory agencies in the European Union (EU) and in the United States of America (USA) have adapted and launched regulatory pathways to accelerate patient access to innovative therapies, such as advanced therapy medicinal products ( ... ...

Abstract	Background aims: Regulatory agencies in the European Union (EU) and in the United States of America (USA) have adapted and launched regulatory pathways to accelerate patient access to innovative therapies, such as advanced therapy medicinal products (ATMPs). The aim of this study is to analyze similarities and differences between regulatory pathways followed by the approved ATMPs in both regions. Methods: A retrospective analysis of the ATMPs approved by EU and US regulatory agencies was carried out until May 31, 2020. Data were collected on the features and timing of orphan drug designation (ODD), scientific advice (SA), expedited program designation (EP), marketing authorization application (MAA) and marketing authorization (MA) for both regions. Results: In the EU, a total of fifteen ATMPs were approved (eight gene therapies, three somatic cell therapies, three tissue-engineered products and one combined ATMP), whereas in the USA, a total of nine were approved (five gene therapies and four cell therapies); seven of these were authorized in both regions. No statistical differences were found in the mean time between having the ODD or EP granted and the start of the pivotal clinical trial or MAA in the EU and USA, although the USA required less time for MAA assessment than the EU (mean difference, 5.44, P = 0.012). The MAA assessment was shorter for those products with a PRIME or breakthrough designation.. No differences were found in the percentage of ATMPs with expedited MAA assessment between the EU and the USA (33.3% versus 55.5%, respectively, P = 0.285) or in the time required for the MAA expedited review (mean difference 4.41, P = 0.105). Approximately half of the products in both regions required an Advisory Committee during the MAA review, and 60% required an oral explanation in the EU. More than half of the approved ATMPs (67% and 55.55% in the EU and the USA, respectively) were granted an ODD, 70% by submitting preliminary clinical data in the EU. The mean number of SA and protocol assistance per product conducted by the European Medicines Agency was 1.71 and 3.75, respectively, and only 13% included parallel advice with health technology assessment bodies. A total of 53.33% of the products conducted the first SA after the pivotal clinical study had started, reporting more protocol amendments. Finally, of the seven ATMPs authorized in both regions, the type of MA differed for only two ATMPs (28.6%), and four out of eight products non-commercialized in the USA had a non-standard MA in the EU. Conclusions: The current approved ATMPs mainly target orphan diseases. Although EU and US regulatory procedures may differ, the main regulatory milestones reached by the approved ATMPs are similar in both regions, with the exception of the time for MAA evaluation, the number of authorized products in the regions and the type of authorization for some products. More global regulatory convergence might further simplify and expedite current ATMP development in these regions.
MeSH term(s)	Cell- and Tissue-Based Therapy ; Drug Approval ; European Union ; Genetic Therapy ; Humans ; Retrospective Studies ; Therapies, Investigational ; United States
Language	English
Publishing date	2021-01-19
Publishing country	England
Document type	Journal Article
ZDB-ID	2039821-9
ISSN	1477-2566 ; 1465-3249
ISSN (online)	1477-2566
ISSN	1465-3249
DOI	10.1016/j.jcyt.2020.11.008
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Article ; Online: Regulatory and clinical development to support the approval of advanced therapies medicinal products in Japan.

Carolina, Iglesias-Lopez / Antònia, Agustí / Mercè, Obach / Antonio, Vallano

Expert opinion on biological therapy

2022 Volume 22, Issue 7, Page(s) 831–842

Abstract: Introduction: A new category of products, i.e. regenerative medicine products (RPs), has been defined for advanced therapies medicinal products in Japan, as well as a legislative and regulatory framework to promote their clinical development.: Areas ... ...

Abstract	Introduction: A new category of products, i.e. regenerative medicine products (RPs), has been defined for advanced therapies medicinal products in Japan, as well as a legislative and regulatory framework to promote their clinical development. Areas covered: This review analyses the most relevant features of the regulatory strategies and clinical development that led RPs to their approval in Japan. Expert opinion: As of 31
MeSH term(s)	Drug Approval ; European Union ; Humans ; Japan ; Regenerative Medicine ; United States
Language	English
Publishing date	2022-07-04
Publishing country	England
Document type	Journal Article ; Review
ZDB-ID	2052501-1
ISSN	1744-7682 ; 1471-2598
ISSN (online)	1744-7682
ISSN	1471-2598
DOI	10.1080/14712598.2022.2093637
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Article: Corrigendum: Regulatory Framework for Advanced Therapy Medicinal Products in Europe and United States.

Iglesias-Lopez, Carolina / Agustí, Antonia / Obach, Mercè / Vallano, Antonio

Frontiers in pharmacology

2020 Volume 11, Page(s) 766

Abstract: This corrects the article DOI: 10.3389/fphar.2019.00921.]. ...

Abstract	[This corrects the article DOI: 10.3389/fphar.2019.00921.].
Language	English
Publishing date	2020-05-25
Publishing country	Switzerland
Document type	Published Erratum
ZDB-ID	2587355-6
ISSN	1663-9812
ISSN	1663-9812
DOI	10.3389/fphar.2020.00766
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Article: ATM Variant as a Cause of Hereditary Cutaneous Melanoma in a Spanish Family: Case Report.

Lendinez-Sanchez, Gonzalo / Diaz-Redondo, Tamara / Campos, Marcos Iglesias / Porta Pelayo, Javier / Porta Pelayo, José María / Muriel-López, Carolina

Case reports in oncology

2024 Volume 17, Issue 1, Page(s) 386–391

Abstract: Introduction: Ataxia-Telangiectasia Mutated (ATM) is a cancer predisposition gene; carriers of germline pathogenic variants have an increased risk of developing malignancies, including breast, prostate, pancreatic, and ovarian cancer. Most ATM variants ... ...

Abstract	Introduction: Ataxia-Telangiectasia Mutated (ATM) is a cancer predisposition gene; carriers of germline pathogenic variants have an increased risk of developing malignancies, including breast, prostate, pancreatic, and ovarian cancer. Most ATM variants are of uncertain significance. Findings from genome-wide association studies (GWAS) suggest that ATM may be a low-risk melanoma susceptibility locus. Case report: We report the case of a Hispanic family whose members who have presented cutaneous melanoma have been found to be carriers for the ATM pathogenic variant c.3747-1G>C (rs730881364), one of whom was diagnosed at 24 years old. Discussion: We describe for the first time the possible clinical association between ATM (c.3747-1G>C) and familial melanoma. In silico splice site analysis predicts that this alteration will weaken the native splice acceptor site and will result in the creation or strengthening of a novel splice acceptor site, assuming a variant that entails loss of functionality that is probably pathogenic and related to oncogenesis. However, we cannot exclude that cutaneous melanoma in both members and at an early age is the result of chance, environmental interaction, other uncontrolled external factors, or the interaction of other genetic alterations other than the ATM variant described in this study.
Language	English
Publishing date	2024-02-27
Publishing country	Switzerland
Document type	Case Reports
ZDB-ID	2458961-5
ISSN	1662-6575
ISSN	1662-6575
DOI	10.1159/000536105
Database	MEDical Literature Analysis and Retrieval System OnLINE

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