scholarly journals Comparability: what we can learn from the review of advanced therapy medicinal products

2021 ◽  
Author(s):  
Alexis Cockroft ◽  
Alison Wilson

Publicly available summaries from Marketing Authorization Applications for gene and cell therapy products (advanced therapies) were evaluated to explore data expectations for product characteristics pre and post changes (comparability). Public assessment reports were used to analyze trends in information requests from regulators concerning comparability from current commercial advanced therapies. In the analysis, 12 products approved in the USA and EU were included. Inadequacies were highlighted for comparability data (six products); additional information requests (five products) and major objections were identified relating to comparability (two products, EU). Postapproval authorization obligations were imposed for six products. Comparability data are essential component for regulatory applications and public assessment reports provide a valuable source of insight into regulators’ expectations.

2021 ◽  
Vol 80 (Suppl 1) ◽  
pp. 884.1-884
Author(s):  
D. De Cock ◽  
P. Durez ◽  
V. Badot ◽  
R. Westhovens ◽  
P. Verschueren

Background:Belgium suffered considerably from the COVID19 pandemic with high hospitalisation rates during 2 periods: a first wave in March and April 2020, and a second starting from October until the end of 2020. Measures of lowering social interaction were taken throughout 2020 and intensified during the first and second wave when needed. This pandemic could have influenced the access to care and advanced therapies for patients with Rheumatoid Arthritis (RA). In the electronic platform “Tool for Administrative Reimbursement Drug Information Sharing” (TARDIS), data from all Belgian RA patients on biologic and targeted therapy are collected during the submission of a request for initiation and prolongation of reimbursement for these drugs.Objectives:to investigate the effect of the COVID19 pandemic on the monthly prescription behaviour of a new advanced therapy in 2020 by comparing it to 2019.Methods:Patients were selected for this analysis if they started a new TNFi, B/T cell therapy, IL6 inhibitors or tsDMARD therapy in the TARDIS registry in 2019 or in 2020. Rheumatologists request reimbursement via the online TARDIS tool, which is considered here as a new drug prescription. Prescription behaviour was compared between 2019 and 2020, between bionaive and bioexperienced patients, and between the different drug classesResults:In 2019, 2949 patients were prescribed any new advanced therapy, including 1153 TNFi, 469 B/T cell therapy, 436 IL6 inhibitors and 891 tsDMARDs. In 2020, 2998 patients were prescribed any new advanced therapy including 1233 TNFi, 382 B/T cell therapy, 496 IL6 inhibitors and 887 tsDMARDs.On a monthly basis, on average 246 and 250 new advanced therapies were prescribed in 2019 and 2020 respectively. Monthly deviations from this average in 2019 ranged from -19% to +16%. Monthly deviations from this average in 2020 ranged from -50% to +30%. Figure 1A shows the monthly prescription of new advanced therapies in 2019 and 2020.For bionaive and bioexperienced patients, the same trend can be noted. Monthly deviations in bionaive patients in 2020 ranged from -60% to +38%, compared to -18% to +21% in 2019. Monthly deviations in bioexperienced patients ranged from -40% to +25%, compared to -19% to +17% in 2019.Comparison per drug class in 2020 show similar trends. IL6 inhibitors show a slightly different timeline than other drugs classes with other periods of less or more prescriptions changes compared to the other drugs classes. See Figure 1B.Conclusion:The COVID19 pandemic did affect reimbursement requests for patients starting new advanced therapies in March and April 2020, especially for bionaive patients. The latter half of 2020 was apparently used to catch up with reimbursement requests for patients in need for advanced therapies resulting in similar total numbers of patients treated with advanced therapy in 2019 and 2020. The choice for a particular drug type was not clearly influenced by the pandemic. IL6 inhibitor use did seem to be affected differently by the pandemic, yet caution is warranted as these relatively large differences in proportional changes parallel small differences in actual drug numbers.In sum, the observed effect of the pandemic on initiating advanced therapy during the first wave corresponds with Belgian governmental measures that restricted non-essential care which was less observed in the latter half of 2020.Disclosure of Interests:None declared.


2020 ◽  
pp. 1-7

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.


Author(s):  
Matthias Renner ◽  
Brigitte Anliker ◽  
Egbert Flory ◽  
Jürgen Scherer ◽  
Martina Schüßler-Lenz ◽  
...  

2017 ◽  
Vol 33 (S1) ◽  
pp. 54-55
Author(s):  
Grace Hampson ◽  
Adrian Towse

INTRODUCTION:There is considerable excitement around the development of regenerative medicines (or advanced therapy medicinal products, ATMPs), with the expectation that they may bring substantial clinical gains and offer cures for previous debilitating and fatal diseases. However, high costs mean that Health Technology Assessment (HTA) and reimbursement decisions are challenging for payers and manufacturers, even when the therapies are expected to offer good value for money.In Europe, seven ATMPs have market authorization, yet only one has achieved national level reimbursement. Statistics such as these put HTA bodies under pressure to review their methods and consider how these can apply to regenerative medicines.METHODS:We present a review of one example, from the United Kingdom's National Institute for Health and Care Excellence (NICE), who commissioned an external organization to undertake a mock appraisal of a hypothetical ATMP using standard methods. The therapeutic area chosen for the mock appraisal was chimeric antigen receptor (CAR) T-cell therapy for treating relapsed or refractory B-cell acute lymphoblastic leukaemia.RESULTS:The role of uncertainty was a key consideration within the report, yet we found that the presentation of uncertainty within the mock appraisal was misleading for decision makers.We found that the exercise represents a thorough mock HTA of CAR T-cell therapy. However, it focused on testing whether ATMPs could fit into the existing HTA pathway for conventional medicines, rather than seeking to identify the most suitable approach for assessing regenerative medicines. We suggest the latter would have been a more relevant question for the mock appraisal.CONCLUSIONS:Any significant departures from the usual HTA process must be based on solid economic rationale if we are to ensure efficient allocation of resources. Thus, in order for regenerative medicines to be given ‘special treatment,’ it must be demonstrated that societal preferences, or the full extent of health (or non-health) benefits, are not being realised for this group of treatments through existing HTA methods.


Pharmaceutics ◽  
2021 ◽  
Vol 13 (8) ◽  
pp. 1140
Author(s):  
Yolanda Diebold ◽  
Laura García-Posadas

The conjunctiva is a complex ocular tissue that provides mechanical, sensory, and immune protection for the ocular surface. It is affected by many diseases through different pathological mechanisms. If a disease is not treated and conjunctival function is not fully restored, the whole ocular surface and, therefore, sight is at risk. Different therapeutic approaches have been proposed, but there are still unsolved conjunctival alterations that require more sophisticated therapeutic options. Advanced therapy medicinal products (ATMPs) comprise a wide range of products that includes cell therapy, tissue engineering, and gene therapy. To the best of our knowledge, there is no commercialized ATMP specifically for conjunctival treatment yet. However, the conjunctiva can be a potential target for ATMPs for different reasons. In this review, we provide an overview of the advances in experimental phases of potential ATMPs that primarily target the conjunctiva. Important advances have been achieved through the techniques of cell therapy and tissue engineering, whereas the use of gene therapy in the conjunctiva is still marginal. Undoubtedly, future research in this field will lead to achieving commercially available ATMPs for the conjunctiva, which may provide better treatments for patients.


1969 ◽  
Vol 16 (4) ◽  
Author(s):  
Pawanbir Singh ◽  
Laure Brévignon-Dodin ◽  
Satya P Dash

The Advanced Therapy Medicinal Products (ATMP) Regulation provides a necessary regulatory framework for the commercialisation and use of regenerative medicine-based therapeutic products in the EU. However, concerns have been raised about the appropriateness of the regulatory strategy it has adopted to address different, complex and evolving categories of medicinal products. This article explores some of the potential shortfalls of the ATMP Regulation with regard to facilitating the research and development of advanced therapies in the present and in the future. It concludes that while providing a much needed harmonised regulatory framework for the companies operating in the sector, the new regulation has yet to demonstrate its capacity to keep up with radical technology changes.


Author(s):  
Elisabete Gonçalves

AbstractThe emergence of advanced therapy medicinal products (ATMPs), a disruptive class of health technologies, is generating important challenges in terms of value assessment and their high prices introduce critical access and affordability concerns. The aim of this article is to analyze the challenges of traditional value assessment and price and reimbursement methods in the evaluation of ATMPs and to characterize the current and prospective financing solutions that may ensure patient access and affordability for these health technologies. Standard Health Technology Assessment (HTA) is not designed for ATMPs, and may delay access to these health technologies, thus a broader concept of value is required. As a consequence, value-based pricing methodologies have been gaining terrain to cope with the specific challenges of ATMPs. The pricing and reimbursement framework should ensure the balance between encouragements to innovation and maximization of value for money for payers, through the attribution of a fair price to new health technologies. Early scientific advice by regulatory and HTA bodies to developers is key, as it will contribute to diminish the perspective gap between developers, regulators and payers. The high efficacy/high price dynamic of many advanced therapies will demand novel financing models, both in the EU and US. Managed entry agreements (MEA), with financing being conditional to the submission of additional evidence, associated with methods of leased payments, may offer effective strategies to address the uncertainties caused by the evidence gap associated with ATMPs, ensuring affordable and sustained access.


2017 ◽  
Author(s):  
M. Karagyaur ◽  
P. Makarevich ◽  
E. Shevchenko ◽  
D. Stambolsky ◽  
N. Kalinina ◽  
...  

2019 ◽  
Vol 20 (3) ◽  
pp. 251-264 ◽  
Author(s):  
Yinlu Feng ◽  
Zifei Yin ◽  
Daniel Zhang ◽  
Arun Srivastava ◽  
Chen Ling

The success of gene and cell therapy in clinic during the past two decades as well as our expanding ability to manipulate these biomaterials are leading to new therapeutic options for a wide range of inherited and acquired diseases. Combining conventional therapies with this emerging field is a promising strategy to treat those previously-thought untreatable diseases. Traditional Chinese medicine (TCM) has evolved for thousands of years in China and still plays an important role in human health. As part of the active ingredients of TCM, proteins and peptides have attracted long-term enthusiasm of researchers. More recently, they have been utilized in gene and cell therapy, resulting in promising novel strategies to treat both cancer and non-cancer diseases. This manuscript presents a critical review on this field, accompanied with perspectives on the challenges and new directions for future research in this emerging frontier.


2020 ◽  
Vol 15 ◽  
Author(s):  
Geeta Aggarwal ◽  
Manju Nagpal ◽  
Ameya Sharma ◽  
Vivek Puri ◽  
Gitika Arora Dhingra

Background: Biopharmaceuticals such as Biologic medicinal products have been in clinical use over the past three decades and have benefited towards the therapy of degenerative and critical metabolic diseases. It is forecasted that market of biologics will be going to increase at a rate of 20% per year, and by 2025, more than ˃ 50% of new drug approvals may be biological products. The increasing utilization of the biologics necessitates for cost control, especially for innovators products that have enjoyed a lengthy period of exclusive use. As the first wave of biopharmaceuticals is expired or set to expire, it has led to various opportunities for the expansion of bio-similars i.e. copied versions of original biologics with same biologic activity. Development of biosimilars is expected to promote market competition, meet worldwide demand, sustain the healthcare systems and maintain the incentives for innovation. Methods: Appraisal of published articles from peer reviewed journals, PubMed literature, latest news and guidelines from European Medicine Agency, US Food Drug Administration (FDA) and India are used to identify data for review. Results: Main insight into the quality requirements concerning biologics, current status of regulation of biosimilars and upcoming challenges lying ahead for the upgrading of marketing authorization of bio-similars has been incorporated. Compiled literature on therapeutic status, regulatory guidelines and the emerging trends and opportunities of biosimilars has been thoroughly stated. Conclusion: Updates on biosimilars will support to investigate the possible impact of bio-similars on healthcare market.


Sign in / Sign up

Export Citation Format

Share Document