EU market authorisation strategy: lessons from the first 22 ATMP submitted to the EMA

2019 ◽  
Vol 5 (6) ◽  
pp. 759-791 ◽  
Author(s):  
Christopher A Bravery ◽  
Oliver Ball ◽  
Sarah Robinson
Keyword(s):  
Market Authorisation

scholarly journals EU FP7 research funding for an orphan drug (Orfadin®) and vaccine (Hep C) development: a success and a failure?

2021 ◽  
Vol 14 (1) ◽  
Author(s):  
L. Schmidt ◽  
O. Sehic ◽  
C. Wild
Keyword(s):  
Hepatitis C ◽  
Late Stage ◽  
Research Funding ◽  
Early Stage ◽  
Basic Research ◽  
Pharmaceutical Products ◽  
Clinical Development ◽  
Clinical Indication ◽  
Market Authorisation ◽  
Risk Capital

Abstract Background We considered the extent of the contribution of publicly funded research to the late-stage clinical development of pharmaceuticals and medicinal products, based on the European Commission (EC) FP7 research funding programme. Using two EC FP7-HEALTH case study examples—representing two types of outcomes—we then estimated wider public and charitable research funding contributions. Methods Using the publicly available database of FP7-HEALTH funded projects, we identified awards relating to late-stage clinical development according to the systematic application of inclusion and exclusion criteria, classified them according to product type and clinical indication, and calculated total EC funding amounts. We then identified two case studies representing extreme outcomes: failure to proceed with the product (hepatitis C vaccine) and successful market authorisation (Orfadin® for alkaptonuria). Total public and philanthropic research funding contributions to these products were then estimated using publicly available information on funding. Results 12.3% (120/977) of all EC FP7-HEALTH awards related to the funding of late-stage clinical research, totalling € 686,871,399. Pharmaceutical products and vaccines together accounted for 84% of these late-stage clinical development research awards and 70% of its funding. The hepatitis C vaccine received total European Community (FP7 and its predecessor, EC Framework VI) funding of €13,183,813; total public and charitable research funding for this product development was estimated at € 77,060,102. The industry sponsor does not consider further development of this product viable; this now represents public risk investment. FP7 funding for the late-stage development of Orfadin® for alkaptonuria was so important that the trials it funded formed the basis for market authorisation, but it is not clear whether the price of the treatment (over €20,000 per patient per year) adequately reflects the substantial public funding contribution. Conclusions Public and charitable research funding plays an essential role, not just in early stage basic research, but also in the late-stage clinical development of products prior to market authorisation. In addition, it provides risk capital for failed products. Within this context, we consider further discussions about a public return on investment and its reflection in pricing policies and decisions justified.

scholarly journals Raising the bar for market authorisation of new drugs

BMJ ◽  
2012 ◽  
Vol 345 (aug07 1) ◽  
pp. e4261-e4261 ◽  
Author(s):  
H. Naci ◽  
J. Cylus ◽  
S. Vandoros ◽  
A. Sato ◽  
K. Perampaladas
Keyword(s):  
New Drugs ◽  
Market Authorisation

Time intervals between U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) new cancer therapy approvals.

2021 ◽  
Vol 39 (15_suppl) ◽  
pp. 1575-1575
Author(s):  
Mark Lythgoe ◽  
Jonathan Krell ◽  
Jeremy Lyle Warner ◽  
Aakash Desai ◽  
Ali Raza Khaki
Keyword(s):  
Kinase Inhibitors ◽  
European Medicines Agency ◽  
Cancer Therapies ◽  
Market Authorisation ◽  
Regulatory Review ◽  
Median Delay ◽  
Therapeutic Class ◽  
The Us ◽  
Accelerated Approval ◽  
Tumour Group

1575 Background: Novel therapies are transforming cancer care. Regulatory review and approval are essential to deliver safe and efficacious innovations to patients. Studies prior to 2010 describe quicker approval decisions for new oncology drug registrations with the FDA compared to the EMA (median delay 238 days). Both regulatory agencies have subsequently improved procedures to expedite approval times. We compared regulatory market authorisation dates at the FDA and EMA for new oncology therapies from 2010-2020. Methods: New oncology therapeutic approvals between 2010-2020 were identified from the FDA and EMA regulatory databases. We analysed only initial approvals (not supplementary licenses) for active anti-cancer therapies (excluding biosimilars and supportive drugs). The delay in regulatory approval between the FDA and EMA was calculated in calendar days. We further analysed therapies by therapeutic class, evaluating for significant differences. Results: We identified 108 new therapy registrations during the study period. 104 (96.3%) therapies were approved by the FDA and 90 (83.3%) had EMA market authorisation. 4 (3.7%) drugs were not FDA registered, including 3 unsuccessful applications and 1 which sought licensing in a different indication. 18 (16.5%) drugs were not EMA registered, including 9 (8.8%) which did not pursue EMA licensing, 3 (2.9%) withdrawn licensing applications, 3 (2.9%) sought licensing in different tumour group/indication, 1 (0.9%) rejected application and 2 (1.9%) with applications under review at submission date. Of the 86 drugs approved by both agencies, 80 were approved first by the FDA and 6 by the EMA. The median delay in approval between the FDA and EMA was 227 days (IQR:124-354 days). Table shows approvals by therapeutic class. The shortest median time difference for approval was for monoclonal antibodies (171 days) with the longest for kinase inhibitors (281 days). Conclusions: This study shows more new oncology therapies are approved by the FDA than the EMA. Patients in the US typically have access to approved therapies earlier than in Europe. From 2010 to 2020 the median delay between FDA and EMA approval was 227 days, falling by 11 days compared to 2003-10, [non-statistically significant]. Such lengthy delays could exceed the life expectancy of many patients with advanced cancer. Innovations for accelerated approval at both the FDA (e.g. Project Orbis) and EMA (e.g., PRIME) have potential to lead to faster approval.[Table: see text]

▼Romosozumab for osteoporosis

2021 ◽  
pp. dtb-2021-000027
Keyword(s):  
High Risk ◽  
Postmenopausal Women ◽  
Brand Name ◽  
Market Authorisation ◽  
Antiresorptive Therapy ◽  
Severe Osteoporosis ◽  
Subcutaneous Injections ◽  
Risk Of Fracture ◽  
Authorisation Holder ◽  
Additional Monitoring

AbstractGeneric name: RomosozumabBrand name: EvenityFormulation: 105 mg solution for injection in a pre-filled penMarket Authorisation holder: UCB Pharma LimitedIndication: treatment of severe osteoporosis in postmenopausal women at high risk of fractureDose: 210 mg romosozumab (administered as two subcutaneous injections of 105 mg each) once a month for 12 months. It is recommended that patients begin antiresorptive therapy after completing treatment with romosozumab.Cost: £427.75 for two pre-filled pens each containing 105 mg romosozumabClassification: Prescription only medicine (POM) subject to additional monitoring (▼)

Shackles for Bees? The ECJ's Judgment on GMO-Contaminated Honey

2012 ◽  
Vol 3 (1) ◽  
pp. 123-129 ◽  
Author(s):  
Matthias Lamping
Keyword(s):  
Marketing Authorisation ◽  
Genetically Modified Organisms ◽  
Genetically Modified ◽  
European Court Of Justice ◽  
Market Authorisation ◽  
Court Of Justice ◽  
European Court ◽  
Considerable Impact ◽  
Natural Component

Case C-442/09, Bablok and Others (not yet reported)An old German proverb says: What the farmer doesn't know he doesn't eat. In the case of the Bablok decision delivered by the European Court of Justice on 6 September 2011, it is not the farmers, but the judges who seem to be wary about the unknown. According to their judgement, substances derived from genetically modified plants require market authorisation to be placed on the market as food, even if the substance itself is not fertile anymore. Since the Court takes the view that pollen is an ingredient of honey rather than a natural component, honey contaminated with pollen from genetically modified organisms will fall within the classification of foodstuffs requiring marketing authorisation. So whenever a bee collects pollen from a genetically modified plant, this can make the entire honey harvest unmarketable. Not even the slightest contamination will be tolerated, irrespective of whether it was intentional. Because the prohibition to put unauthorised honey on the market applies abstractly, regardless of whether there is a concrete risk for the health of consumers, the judgement will have considerable impact on the coexistence of conventional, ecological and genetically-modified farming.

Proposed Standards and Regulatory Mechanisms for New Homeopathic Drug Development

2021 ◽  
Vol 34 (04) ◽  
pp. 283-290
Author(s):  
Gitanjali Talele ◽  
Rajesh Shah
Keyword(s):  
Drug Discovery ◽  
Drug Development ◽  
Regulatory Mechanism ◽  
New Drugs ◽  
Regulatory Mechanisms ◽  
Regulatory Body ◽  
Market Authorisation ◽  
New Drug ◽  
Homeopathic Drug ◽  
Basic Proposition

Abstract Introduction Researchers working with new insights and new targets in new drug discovery in the homeopathy space observe that the path of drug-development and market authorisation has been less travelled and the pathway is not yet well-mapped. The need of the time is to define clear guidelines and regulatory mechanisms to facilitate the process of new drug discovery. Overview The article is about the proposed methods for identifying the new homeopathic substances for therapeutic use. An overview of the current regulations for drug development in India is discussed in this article. Method of new drug development in homeopathy, standards and regulatory mechanism for approval of new drugs are proposed with few illustrations and references. An introductory plan, based on the perspective and experience of researcher, practitioner, academician and inventor for drug discovery is proposed. Discussion An urgent need for setting up the guidelines for new drug discovery has been identified and a basic proposition is made for the same, suggesting practical, pragmatic and achievable measures, and independent regulatory body to encourage drug development and research.

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