scholarly journals Flexible and Expedited Regulatory Review Processes for Innovative Medicines and Regenerative Medical Products in the US, the EU, and Japan

2019 ◽  
Vol 20 (15) ◽  
pp. 3801 ◽  
Author(s):  
Sumimasa Nagai
Keyword(s):  
New Drugs ◽  
European Medicines Agency ◽  
Regulatory Review ◽  
Medical Products ◽  
Gene Therapies ◽  
Advanced Therapy ◽  
The Us ◽  
Innovative Drugs ◽  
Original Feature ◽  
The Eu

Several expedited regulatory review projects for innovative drugs and regenerative medical products have been developed in the US, the EU, and Japan. Each regulatory agency has elaborated an original regulatory framework and adopted regulatory projects developed by the other regulatory agencies. For example, the Food and Drug Administration (FDA) first developed the breakthrough therapy designation, and then the Pharmaceuticals and Medical Devices Agency (PMDA) and European Medicines Agency (EMA) introduced the Sakigake designation and the priority medicines (PRIME) designation, respectively. In addition, the necessity of the product being first development in Japan is the original feature of the Sakigake designation, while actively supporting the development of advanced-therapy medicinal products (ATMPs) by academia or small/medium-sized sponsors is the original feature of the PRIME; these particular features are different from the breakthrough therapy designation in the US. In this review article, flexible and expedited review processes for new drugs, and cell and gene therapies in the US, the EU, and Japan are described. Moreover, all the drugs and regenerative medical products that were granted conditional approval or Sakigake designation in Japan are listed and analyzed herein.

scholarly journals The Biosimilar Landscape: An Overview of Regulatory Approvals by the EMA and FDA

Pharmaceutics ◽  
2020 ◽  
Vol 13 (1) ◽  
pp. 48
Author(s):  
Ioana Gherghescu ◽  
M. Begoña Delgado-Charro
Keyword(s):  
Drug Administration ◽  
Action Plan ◽  
European Medicines Agency ◽  
Patient Access ◽  
Clinical Evaluations ◽  
The Past ◽  
The Us ◽  
Improve Patient ◽  
The Eu

Biosimilar medicines expand the biotherapeutic market and improve patient access. This work looked into the landscape of the European and US biosimilar products, their regulatory authorization, market availability, and clinical evaluation undergone prior to the regulatory approval. European Medicines Agency (EMEA, currently EMA) and Food and Drug Administration (FDA) repositories were searched to identify all biosimilar medicines approved before December 2019. Adalimumab biosimilars, and particularly their clinical evaluations, were used as a case study. In the past 13 years, the EMA has received 65 marketing authorization applications for biosimilar medicines with 55 approved biosimilars available in the EU market. Since the first biosimilar approval in 2015, the FDA has granted 26 approvals for biosimilars with only 11 being currently on the US market. Five adalimumab biosimilars have been approved in the EU and commercialized as eight different medicines through duplicate marketing authorizations. Whilst three of these are FDA-approved, the first adalimumab biosimilar will not be marketed in the US until 2023 due to Humira’s exclusivity period. The EU biosimilar market has developed faster than its US counterpart, as the latter is probably challenged by a series of patents and exclusivity periods protecting the bio-originator medicines, an issue addressed by the US’s latest ‘Biosimilar Action Plan’.

Safety-Related Postmarketing Modifications of Drugs for Hematological Malignancies

2019 ◽  
Vol 143 (1) ◽  
pp. 73-77
Author(s):  
Anat Gafter-Gvili ◽  
Ariadna Tibau ◽  
Pia Raanani ◽  
Daniel Shepshelovich
Keyword(s):  
Hematological Malignancies ◽  
New Drugs ◽  
Priority Review ◽  
Regulatory Review ◽  
Regulatory Pathways ◽  
The Us ◽  
Black Box Warnings ◽  
Accelerated Approval ◽  
Drug Approvals ◽  
Approved Drugs

The prevalence of safety-related postmarketing label modifications of medications for hematological malignancies is unknown. We identified 35 new drugs indicated for hematological malignancies approved by the US Food and Drug Administration between January 1999 and December 2014. Characteristics of supporting trials and safety-related label modifications from approval to December 2017 were collected from drug labels. Regulatory review and approval pathways were also collected. New drug approvals were supported by trials with a median of 167 patients (interquartile range 115–316). All drugs were approved based on surrogate endpoints. Twenty-seven drug approvals (77%) were not supported by randomized controlled trials. All drugs received orphan drug designation, and most were granted fast track designation, priority review, and accelerated approval (83, 74, and 60%, respectively). A total of 28 drugs (80%) had postmarketing safety-related label modifications. Additions to black box warnings, contraindications, warnings and precautions, and common adverse reactions were identified in 31, 11, 77, and 46% of drugs, respectively. Five drugs (14%) were permanently or temporarily withdrawn from the US market. Drugs for hematological malignancies are often approved based on limited evidence through expedited regulatory pathways with incomplete safety profiles. Hematologists should be vigilant for unrecognized side effects when prescribing newly approved drugs.

Addressing the regulatory and scientific challenges in multiple sclerosis – a statement from the EU regulators

2014 ◽  
Vol 20 (10) ◽  
pp. 1282-1287 ◽  
Author(s):  
Pavel Balabanov ◽  
Manuel Haas ◽  
Andre Elferink ◽  
Serge Bakchine ◽  
Karl Broich
Keyword(s):  
Multiple Sclerosis ◽  
Drug Development ◽  
Guideline Development ◽  
New Drugs ◽  
European Medicines Agency ◽  
Guidance Document ◽  
Flexible Approach ◽  
Official Position ◽  
New Drug Development ◽  
The Eu

Improving and facilitating the process of making new drugs available to patients with multiple sclerosis (MS) requires cooperation among the regulators and other stakeholders. This cooperation will also positively contribute towards developing guidelines of the highest quality in medical, regulatory and scientific aspects. This would be beneficial both in areas that require further guideline development, but also in fields where existing guidance should be adapted to take into account evolution in science. Considering the input from all stakeholders, the European Medicines Agency confirmed its intention to update the relevant guideline and apply a flexible approach towards new drug development strategies in MS. This article is the first official position from the EU regulators, presenting the main changes to be expected in the guidance document.

scholarly journals Association between FDA and EMA expedited approval programs and therapeutic value of new medicines: retrospective cohort study

BMJ ◽  
2020 ◽  
pp. m3434
Author(s):  
Thomas J Hwang ◽  
Joseph S Ross ◽  
Kerstin N Vokinger ◽  
Aaron S Kesselheim
Keyword(s):  
Cohort Study ◽  
Retrospective Cohort Study ◽  
Retrospective Cohort ◽  
New Drugs ◽  
European Medicines Agency ◽  
Health Authorities ◽  
Therapeutic Value ◽  
The Us ◽  
Value Association

AbstractObjectiveTo characterize the therapeutic value of new drugs approved by the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) and the association between these ratings and regulatory approval through expedited programs.DesignRetrospective cohort study.SettingNew drugs approved by the FDA and EMA between 2007 and 2017, with follow-up through 1 April 2020.Data sourcesTherapeutic value was measured using ratings of new drugs by five independent organizations (Prescrire and health authorities of Canada, France, Germany, and Italy).Main outcome measuresProportion of new drugs rated as having high therapeutic value; association between high therapeutic value rating and expedited status.ResultsFrom 2007 through 2017, the FDA and EMA approved 320 and 268 new drugs, respectively, of which 181 (57%) and 39 (15%) qualified for least one expedited program. Among 267 new drugs with a therapeutic value rating, 84 (31%) were rated as having high therapeutic value by at least one organization. Compared with non-expedited drugs, a greater proportion of expedited drugs were rated as having high therapeutic value among both FDA approvals (45% (69/153) v 13% (15/114); P<0.001) and EMA approvals (67% (18/27) v 27% (65/240); P<0.001). The sensitivity and specificity of expedited program for a drug being independently rated as having high therapeutic value were 82% (95% confidence interval 72% to 90%) and 54% (47% to 62%), respectively, for the FDA, compared with 25.3% (16.4% to 36.0%) and 90.2% (85.0% to 94.1%) for the EMA.ConclusionsLess than a third of new drugs approved by the FDA and EMA over the past decade were rated as having high therapeutic value by at least one of five independent organizations. Although expedited drugs were more likely than non-expedited drugs to be highly rated, most expedited drugs approved by the FDA but not the EMA were rated as having low therapeutic value.

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]

Encountering Challenges with the eu Regulation on Advance Therapy Medical Products

2015 ◽  
Vol 22 (5) ◽  
pp. 426-461 ◽  
Author(s):  
Juli Mansnérus
Keyword(s):  
Public Health ◽  
Medical Product ◽  
Internal Market ◽  
Pharmaceutical Companies ◽  
Medical Products ◽  
Advanced Therapy ◽  
Advanced Therapies ◽  
Product Regulation ◽  
Set Up ◽  
The Eu

This article aims at analysing how well the Advanced Therapy Medical Product Regulation (ec) No. 1394/2007 (atmp Regulation) meets the needs of small and medium-sized enterprises (smes), academia and public tissue establishments developing advanced therapy medical products (atmps). Benefits and shortcomings of the atmp Regulation are identified, and possible amendments are proposed to accelerate the translation of research into advanced therapies and to facilitate the commercialisation of atmps whilst ensuring safety. It was set up as a lex specialis to ensure the free movement of atmps within the eu in order to facilitate their access to the internal market and to foster the competitiveness of European pharmaceutical companies, while guaranteeing the highest level protection of public health. Since the adoption of the atmp Regulation in late 2008, only 5 atmps have been granted marketing authorisations thus far. Hence, there is a need to analyse whether the atmp Regulation meets its objectives.

scholarly journals Moving somatic gene editing to the clinic: routes to market access and reimbursement in Europe

2021 ◽  
Author(s):  
Tessel Rigter ◽  
David Klein ◽  
Stephanie S. Weinreich ◽  
Martina C. Cornel
Keyword(s):  
Gene Editing ◽  
Current System ◽  
European Medicines Agency ◽  
Great Promise ◽  
Commercial Development ◽  
Advanced Therapy Medicinal Product ◽  
Gene Therapies ◽  
Market Authorization ◽  
Advanced Therapy ◽  
Somatic Gene

AbstractSomatic gene editing (SGE) holds great promise for making genetic therapy possible for many monogenic conditions very soon. Is our current system of European market authorization and reimbursement ready for the expected tsunami of gene therapies? At a recent workshop of the Netherlands ZonMw consortium on ethical, legal, and social implications of personalized medicine, we discussed the current possibilities for bringing new gene therapies to the clinic. In Europe, it is not yet clear whether the route via the European medicines agency as an advanced therapy medicinal product is the most appropriate for evaluation of highly personalized SGE applications, although this may optimally guarantee safety and effectiveness. Compassionate use may ensure faster access than the centralized procedure but does not stimulate the commercial development of products. Prescription to named patients may only provide adequate access for single patients. Temporary authorization of use may allow access to medication half a year before formal market authorization has been granted, but may also have large budget impacts. Magistral compounding under a hospital exemption may be an attractive solution for rare, tailor-made applications at an acceptable price. To approve local experimental use of a therapy on a case-by-case basis may be fast, but does not guarantee optimal safety, effectiveness, and broad implementation. We argue that alternative routes should be considered for products developed for a market of large groups of patients versus unique personalized treatments. A balance between scientific evidence for safety and effectiveness, affordability, and fast access may demand a range of alternative solutions.

scholarly journals The AIFA time-to-reimbursement: a comparison between the last two committees from 2015 to 2020

2020 ◽  
Vol 7 (1) ◽  
pp. 109-114
Author(s):  
Paola Raimondo ◽  
Giorgio Casilli ◽  
Martina Isernia ◽  
Dario Lidonnici ◽  
Roberto Ravasio ◽  
...  
Keyword(s):  
Time Delay ◽  
Average Duration ◽  
Economic Assessment ◽  
New Drugs ◽  
Economic Conditions ◽  
European Medicines Agency ◽  
Official Journal ◽  
Managed Entry Agreements ◽  
R Process ◽  
Innovative Drugs

Objective. To compare the time-to-reimbursement of the last two committees of the Italian Medicines Agency (AIFA), respectively appointed in 2015 and in 2018. Methods. The analysis was run through a specific internal database created by MA-Provider. The database was populated with information regarding European Medicines Agency (EMA) approved new drugs, including each step of the Italian Price and Reimbursement (P&R) process reported in the monthly outcomes of Technical Scientific Committee (CTS) and Price and Reimbursement Committee (CPR) meetings from September 2015 to April 2020. Results. The 2015 and the 2018 committees have reimbursed respectively 39 and 28 drugs by comparing their initial 19 months of activity. Significant differences have been observed in negotiated economic conditions, in particular an increase in the number of drugs with confidential discount (2018-committee: 96.4% vs 2015-committee: 64.1%; p = 0.003) and a reduction in the application of Managed Entry Agreements (MEAs) (2018-committee: 10.7% vs 2015-committee: 33.3%; p = 0.036). The average duration of the P&R procedure managed by the 2018-committee has increased by 45 days compared to the 2015-committee (287 days vs 242 days; p = 0.071) and this trend of delay is associated to the active scientific/economic assessment phase by CTS and CPR (particularly by the latter) and not to administrative phases (e.g. Official Journal publications). Conclusions. The observed differences between committees may be explained by the higher number of oncological and/or innovative drugs assessed by the 2018-committee (regarding the time delay, probably linked to greater difficulties in finding a win-win agreement able to satisfy both AIFA and Pharmaceutical Company).

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