Long-term follow-up of cancer patients treated with gene therapy medicinal products.

European Union requirements are discussed for the long-term follow-up of advanced therapy medicinal products, as well as how they can be applied to cancer patients treated with gene therapy medicinal products in the context of clinical trials, as described in a specific guideline issued by Gene Therapy Working Party at the European Medicine Agency.

Breastfeeding promotion and support: a quality improvement study.

BACKGROUND: Breastfeeding success is determined by early skin to skin contact, early initiation of breastfeeding, rooming-in, baby-led breastfeeding, creation of a favorable environment, specific training of health professionals, and continuity of care. OBJECTIVE: To investigate the women's satisfaction regarding the care and support received in the first days after childbirth. MATERIAL AND METHODS: A questionnaire of 24 items was administered to mothers before discharge, from May to September 2019 at the University Hospital of Modena. RESULTS: The predictive variables of exclusive breastfeeding were the delivery mode, age at birth and parity. The multivariate analysis showed that a high satisfaction score was associated with vaginal birth (OR=2.63, p=0.005), rooming-in during the hospitalization (OR=8.64, p<0.001), the skin to skin contact (OR=6.61, p=0.001) and the first latch-on within 1 hour after birth (OR=3.00, p=0.02). CONCLUSIONS: Mothers' satisfaction is one of the important factors of positive experience during hospital stay and of better health outcomes.

Breastfeeding pathologies: analysis of prevalence, risk and protective factors.

BACKGROUND AND AIM OF THE STUDY: Breastfeeding is essential for the health of mothers and newborns, and it is recommended by WHO-UNICEF as the sole source of nutrition and protection for the first 6 months of life and beyond. In order to fully promote this practice, it is important to recognize early conditions that can lead to pathological breastfeeding. AIM: The study aims to analyze the prevalence and the possible risk or protective factors concerning the pathology of breastfeeding. METHODS: For this observational study were consulted the medical records and the files of the Breastfeeding clinic of 1065 puerperal women, of the University Hospital of Modena, from January to August 2016. The data were processed  with the SPSS Software. RESULTS: In our study population, 532 (50%) puerperal women presented a breastfeeding-related disease, of which 330 (31%) had a disease affecting the mother (breast engorgement, fissures, a-/hypo-galactia, discontinuation of breastfeeding, galactocele, mastitis and candidiasis), 105 (9.9%) of the newborn (inadequate suction, neonatal jaundice, pathological weight loss, need for admission to NICU) and 97 (9.1%) of both the mother and the newborn. DISCUSSIONS: It is evident from the results that the predicting factors of pathology in breastfeeding are present in pregnant women who give birth in an early gestational age and with high age, birth rate and nationality. CONCLUSIONS: staff training courses are essential to respond to WHO-UNICEF recommendations and to improve the continuity of care for the mother-child dyad.

ATMPs for Cancer Immunotherapy: A Regulatory Overview.

This chapter discusses European regulatory requirements for development of advanced therapy medicinal products (ATMP) for cancer immunotherapy approaches, describing the framework for clinical trials and for marketing authorization.Regulatory critical issues and challenges for developing ATMP are also discussed, with focus on potency determination, long-term follow-up, comparability, and insertional mutagenesis issues. Some of the most critical features of GMP application to ATMP are also described.

Clinical development of gene therapy needs a tailored approach: a regulatory perspective from the European Union.

Gene therapy is a rapidly evolving field that needs an integrated approach, as acknowledged in the concept article on the revision of the guideline on gene transfer medicinal products. The first gene therapy application for marketing authorization was approved in the International Conference on Harmonisation (ICH) region in 2012, the product being Alipogene tiparvovec. The regulatory process for this product has been commented on extensively, highlighting the challenges posed by such a novel technology. Here, as current or previous members of the Committee for Advanced Therapies, we share our perspectives and views on gene therapy as a treatment modality based on current common understanding and regulatory experience of gene therapy products in the European Union to date. It is our view that a tailored approach is needed for a given gene therapy product in order to achieve successful marketing authorization.

The committee for advanced therapies' of the European Medicines Agency reflection paper on management of clinical risks deriving from insertional mutagenesis.

In the European Union, the Committee for Advanced Therapies of the European Medicines Agency takes the lead in the scientific assessment for marketing authorization applications for advanced therapy medicinal products, which include gene therapy medicinal products, somatic cell therapy medicinal products, and tissue-engineered products. The Committee for Advanced Therapies also takes the lead in defining the scientific framework for the quality, nonclinical and clinical development of such products. This reflection paper represents the Committee's current thinking on management of clinical risks deriving from insertional mutagenesis. A multidisciplinary approach to insertional mutagenesis is provided. This reflection paper has been adopted by the committee in its April 2013 meeting.

Translational research on advanced therapies.

Fostering translational research of advanced therapies has become a major priority of both scientific community and national governments. Advanced therapy medicinal products (ATMP) are a new medicinal product category comprising gene therapy and cell-based medicinal products as well as tissue engineered medicinal products. ATMP development opens novel avenues for therapeutic approaches in numerous diseases, including cancer and neurodegenerative and cardiovascular diseases. However, there are important bottlenecks for their development due to the complexity of the regulatory framework, the high costs and the needs for good manufacturing practice (GMP) facilities and new end-points for clinical experimentation. Thus, a strategic cooperation between different stakeholders (academia, industry and experts in regulatory issues) is strongly needed. Recently, a great importance has been given to research infrastructures dedicated to foster translational medicine of advanced therapies. Some ongoing European initiatives in this field are presented and their potential impact is discussed.

Challenges with advanced therapy medicinal products and how to meet them.

Advanced therapy medicinal products (ATMPs), which include gene therapy medicinal products, somatic cell therapy medicinal products and tissue-engineered products, are at the cutting edge of innovation and offer a major hope for various diseases for which there are limited or no therapeutic options. They have therefore been subject to considerable interest and debate. Following the European regulation on ATMPs, a consolidated regulatory framework for these innovative medicines has recently been established. Central to this framework is the Committee for Advanced Therapies (CAT) at the European Medicines Agency (EMA), comprising a multidisciplinary scientific expert committee, representing all EU member states and European Free Trade Association countries, as well as patient and medical associations. In this article, the CAT discusses some of the typical issues raised by developers of ATMPs, and highlights the opportunities for such companies and research groups to approach the EMA and the CAT as a regulatory advisor during development.

Regulatory considerations for translating gene therapy: a European Union perspective.

A preclinical study on a gene therapy approach for treatment of the severe muscle weakness associated with a variety of neuromuscular disorders provides a forum to discuss the translational challenges of gene therapy from a regulatory point of view. In this Perspective, the findings are considered from the view of European regulatory requirements for first clinical use.