The Regulation of Biological and other Innovative Medicinal Products

2021 ◽  
pp. 60-86
Keyword(s):  
Quality Standard ◽  
Medicinal Products ◽  
British Pharmacopoeia ◽  
Biological Products ◽  
Recombinant Cells ◽  
Bacterial Endotoxins ◽  
Advanced Therapy ◽  
Biological Medicinal Product ◽  
Standard Set ◽  
Minimum Quality

This chapter discusses the phrase ‘biological medicinal product’, which is used to refer to products manufactured by biological or biotechnology means or to advanced therapy medicinal products. It examines biological medicinal products that are considered peptides or proteins and are made up of one or more linear sequences of amino acids. It also talks about the compliance of biological products with a minimum quality standard set by the British Pharmacopoeia or European Pharmacopoeia, which includes specific instructions for testing sterility, bacterial endotoxins, microbial limits, volume in container, uniformity of dosage units, and particulate matter. The chapter highlights biological products that are manufactured using recombinant cells and are extracted or made from unaltered tissues or blood that are purified in the same way as recombinant products. It explores the inherent heterogeneity of biological products as it comprises of a mixture of closely related molecules.

scholarly journals Development of an Approach to the Assessment of Changes to Approved Biological Products

2019 ◽  
Vol 19 (2) ◽  
pp. 109-117
Author(s):  
E. V. Petraneva ◽  
I. A. Proskurina ◽  
D. V. Goryachev ◽  
E. L. Kovaleva
Keyword(s):  
Life Cycle Management ◽  
Expert Committee ◽  
Medicinal Products ◽  
Biological Products ◽  
Open Questions ◽  
Before And After ◽  
Biological Medicinal Product ◽  
The Russian Federation ◽  
Data Requirements

The need to strengthen the post-approval regulation of biological products stems from their increasing role in the treatment of serious human diseases. Until recently, there were open questions on the classification of changes and supporting data necessary to confirm the comparability of a biological medicinal product before and after any changes, as well as on the procedures and deadlines for the submission and approval of variations. In October 2017 the WHO Expert Committee on Biological Standardisation developed and published the «Guidelines on procedures and data requirements for changes to approved biotherapeutic products». The WHO recommendations are primarily aimed at resolving the complexities and current problems in the global life cycle management of biotherapeutic products. Guidelines suggest approaches that ensure continued quality, efficacy, and safety of this group of products, as well as continuity in supply and access. The purpose of this paper was to analyse the WHO recommendations on post-approval changes to biotherapeutic products in order to develop harmonised approaches to the assessment of post-approval changes to biological medicinal products in the Russian Federation. The categories of quality changes, supporting data, conditions to be fulfilled, procedures and deadlines set forth in this paper can serve as a basis for further improvement of the national regulatory and methodological framework.

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.

scholarly journals Price and reimbursement of advanced therapeutic medicinal products in Europe: are assessment and appraisal diverging from expert recommendations?

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
Virginia Ronco ◽  
Myriam Dilecce ◽  
Elena Lanati ◽  
Pier Luigi Canonico ◽  
Claudio Jommi
Keyword(s):  
Selection Process ◽  
Grey Literature ◽  
Primary Objective ◽  
Medicinal Products ◽  
Health Authorities ◽  
Advanced Therapy ◽  
Additional Funding ◽  
Managed Entry Agreements ◽  
R Process ◽  
Expert Recommendations

Abstract Background Advanced therapy medicinal products (ATMPs) represent an important cornerstone for innovation in healthcare. However, uncertainty on the value, the high average cost per patient and their one-shot nature has raised a debate on their assessment and appraisal process for pricing and reimbursement (P&R) purposes. This debate led experts providing for recommendations on this topic. Our primary objective is to investigate the ATMPs P&R process in the main five European countries and to understand if this process is consistent with published P&R expert recommendations. We also investigated the current ATMP pipelines to understand if future ATMPs will create challenges for their P&R process. Methods P&R framework for ATMPs in the European Major five (EU5) countries was investigated through a literature search on PubMed, institutional websites of National Health Authorities and grey literature. The ATMPs pipeline database was populated from a clinical trial database (clinicaltrials.gov), relying on inclusion and exclusion criteria retrieved from the literature. Results Reimbursement status of ATMPs is different across the EU5 countries, with the exception of CAR-Ts which are reimbursed in all countries. Standard P&R process in place for other medicinal products is extended to ATMPs, with the exception of some cases in Germany. List prices, where available, are high and, tend to be aligned across countries. Outcome-based Managed Entry Agreements (MEAs) have been extensively used for ATMPs. Extra-funds for hospitals managing ATMPs were provided only in Germany and, as additional fund per episode, in France. The accreditation process of hospitals for ATMPs management was in most countries managed by the national authorities. As far as ATMPs pipeline is concerned, ATMPs in development are mostly targeting non-rare diseases. Conclusions Expert recommendations for ATMPs P&R were partially applied: the role of outcome-based MEAs has increased and the selection process of the centres authorized to use these treatments has been enhanced; additional funding for ATMPs management to accredited centres has not been completely considered and annuity payment and broader perspective in cost considerations are far from being put in place. These recommendations should be considered for future P&R negotiations to pursue rational resource allocation and deal with budget constraints.

Standard minimi di qualitŕ: un approccio teorico

2012 ◽  
pp. 29-41
Author(s):  
Grassi Iacopo
Keyword(s):  
High Standard ◽  
Policy Instrument ◽  
Quality Standard ◽  
Average Quality ◽  
Proposed Model ◽  
Positive Externality ◽  
Experience Good ◽  
The Government ◽  
Minimum Quality

At least since Akerlof (1970), asymmetric information in the case of experience goods has been a central issue in the economic literature. This paper studies regulation in markets where the quality of the experience good is never completely verifiable by consumers even after purchase. In the proposed model firms can decide the quality of the good: always producing a high quality good creates a positive externality in the market, but it causes an incentive to the firms to deviate and produce low quality goods. The main policy instrument for the government, in order to maximize Social Welfare, is to fix a minimum quality standard, but imposing a too high standard might, in some cases, lower the average quality of the good in the market.

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.

Experimental Assessment of Strength Parameters of River Sand for Sandcrete Block Production

2021 ◽  
Vol 53 ◽  
pp. 67-75
Author(s):  
Babatunde Ogunbayo ◽  
Clinton Aigbavboa ◽  
Opeoluwa Akinradewo
Keyword(s):  
Compressive Strength ◽  
Quality Standard ◽  
Sieve Analysis ◽  
Building Structures ◽  
Strength Parameters ◽  
River Sand ◽  
Sieve Analyses ◽  
Aggregate Material ◽  
Average Compressive Strength ◽  
Minimum Quality

Sandcrete block is a vital building material used in the construction of building structures. The sandcrete blocks are produced by different manufacturers using river sand obtained from different locations as aggregate material without recourse to the minimum quality standard for the blocks produced. The study assessed the strength parameters of river sand used as an aggregate material in block production to determine its quality and suitability in relation to the strength of block produced. Three (3) block manufacturing sites in Nigeria were visited and 27 (twenty-seven) blocks of size 450 mm x 225 mm x 225 mm were selected randomly from the sites. The properties of the river sand was analyzed through sieve analyses, bulk density, silt content and water absorption while the compressive strength of the blocks was also tested. The result of sieve analysis of the river sand used in block production for this study all satisfied the particle size requirements of BS EN 933-1:1997 for general construction work including block production. The result of the study also shows that blocks produced with the river sand after 28days have an average compressive strength of 1.23 N/mm2 (SW), 1.54 N/mm2 (SE) and 1.95N/mm2 (NE). The study, therefore, concluded and recommended that regulatory and professional bodies in partnership with relevant associations should organize seminars for producers of sandcrete blocks on the best practices involved in producing quality sandcrete blocks.

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