The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance: application to diabetes and vascular disease

2011 ◽  
Vol 11 (6) ◽  
pp. 304-307 ◽  
Author(s):  
Kevin V Blake ◽  
Camilla Smeraldi ◽  
Xavier Kurz ◽  
Peter Arlett ◽  
Stella Blackburn ◽  
...  
Keyword(s):  
Drug Utilisation ◽  
Real Life ◽  
European Medicines Agency ◽  
The European Union ◽  
Medicinal Products ◽  
European Network ◽  
Study Protocols ◽  
Real Life Setting ◽  
The Impact ◽  
Support Decision Making

The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) is an initiative led by the European Medicines Agency (EMA) aimed at further strengthening the post-authorisation monitoring of medicinal products in Europe by facilitating the undertaking of multi-centre, independent, studies focusing on safety and on benefit/risk. A key objective of ENCePP is to provide a unique point of access for all involved stakeholders, including industry or regulatory authorities, who are seeking collaboration for the commissioning or the performance of post-authorisation studies. The 2010 EMA regulatory action relating to rosiglitazone included a pharmacoepidemiological drug utilisation study to evaluate the benefit–risk profile in a real-life setting and has also led to the commissioning of an ENCePP study to evaluate the impact of risk-minimisation activities. ENCePP seeks to improve the European Union capacity to conduct such studies and thus support decision making. Application of the ENCePP study concept will result in an increase in trust in medicines and their use. In addition, the ENCePP register of studies will serve as a resource to allow for ready access to study protocols and results, thereby ensuring transparency.

scholarly journals The latest orphan drug designations and the Commission Communication on Regulation (EC) 141/2000

2005 ◽  
Vol 11 (3) ◽  
Author(s):  
Rashmi R Shah
Keyword(s):  
European Union ◽  
Rare Diseases ◽  
European Medicines Agency ◽  
Biological Plausibility ◽  
The European Union ◽  
Medicinal Products ◽  
Market Exclusivity ◽  
Community Regulation ◽  
Orphan Medicinal Products ◽  
Patient Groups

The implementation of Community Regulation on orphan medicinal products in the European Union in April 2000 has resulted in a deluge of applications for designation of medicinal products as orphan for rare diseases. By April 2004, the Committee for Orphan Medicinal Products had already given positive opinion on 63 per cent of the 316 applications considered by them. A significant number of these positive designations have already matured into full marketing authorisations. Three major reasons – failure to meet prevalence or significant benefit criteria or provide evidence of biological plausibility – have equally contributed to either the negative opinion on or the applicants withdrawing the remaining applications. In July 2004, the European Commission issued a communication setting out its position on certain matters relating to the implementation of the designation and market exclusivity provisions. The Commission, the European Medicines Agency (EMEA) and the Committee for Orphan Medicinal Products (COMP) continue to be proactive and provide as much guidance and incentives as practical, engaging themselves with sponsors, patient groups and academia. As experience builds up and issues are clarified, there are expectations that the Community Regulation on orphan medicines will prove to be a spectacular success.

scholarly journals An analysis of characteristics of post-authorisation studies registered on the ENCePP EU PAS Register

F1000Research ◽  
2017 ◽  
Vol 6 ◽  
pp. 1447 ◽  
Author(s):  
Robert Carroll ◽  
Sreeram V. Ramagopalan ◽  
Javier Cid-Ruzafa ◽  
Dimitra Lambrelli ◽  
Laura McDonald
Keyword(s):  
Risk Assessment ◽  
Chart Review ◽  
Drug Utilisation ◽  
Data Capture ◽  
Primary Data ◽  
European Medicines Agency ◽  
Therapeutic Area ◽  
The European Union ◽  
Study Designs ◽  
The Eu

Background: The objective of this study was to investigate the study design characteristics of Post-Authorisation Studies (PAS) requested by the European Medicines Agency which were recorded on the European Union (EU) PAS Register held by the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP). Methods: We undertook a cross-sectional descriptive analysis of all studies registered on the EU PAS Register as of 18th October 2016. Results: We identified a total of 314 studies on the EU PAS Register, including 81 (26%) finalised, 160 (51%) ongoing and 73 (23%) planned. Of those studies identified, 205 (65%) included risk assessment in their scope, 133 (42%) included drug utilisation and 94 (30%) included effectiveness evaluation. Just over half of the studies (175; 56%) used primary data capture, 135 (43%) used secondary data and 4 (1%) used a hybrid design combining both approaches. Risk assessment and effectiveness studies were more likely to use primary data capture (60% and 85% respectively as compared to 39% and 14% respectively for secondary). The converse was true for drug utilisation studies where 59% were secondary vs. 39% for primary. For type 2 diabetes mellitus, database studies were more commonly used (80% vs 3% chart review, 3% hybrid and 13% primary data capture study designs) whereas for studies in oncology, primary data capture were more likely to be used (85% vs 4% chart review, and 11% database study designs). Conclusions: Results of this analysis show that study objectives and therapeutic area influence PAS design in terms of type of data capture used.

A retrospective database analysis to assess the impact of zoledronic acid (ZOL) on skeletal-related events (SREs) in solid tumor cancer and multiple myeloma (MM) patients (pts)

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. 9518-9518 ◽  
Author(s):  
S. Thayer ◽  
J. Cooke ◽  
S. Kaura
Keyword(s):  
Risk Reduction ◽  
Health Plan ◽  
Real Life ◽  
Pathologic Fracture ◽  
Mortality Rates ◽  
Cord Compression ◽  
Care Plan ◽  
Bone Lesions ◽  
Real Life Setting ◽  
The Impact

9518 Background: For cancer pts with malignant bone lesions (BM), SREs including pathologic fracture, spinal cord compression, hypercalcemia of malignancy, and radiotherapy and/or surgery to bone are associated with significant morbidity and mortality and reduced quality of life. ZOL is an IV bisphosphonate (BP) proven to reduce and delay incidence of SREs in several tumor types. This study was designed to assess the benefit of long-term ZOL use in a real-life setting. Methods: Claims-based analysis of commercial and Medicare data from a large US managed care plan and a 45 health-plan database was used to evaluate mortality rates, SRE rates, and time from BM to SREs in pts treated with ZOL, pamidronate (PAM), or no IV BP therapy. Pts older than 18 years with solid tumors (breast, prostate, lung, bladder, or renal cell cancers) or MM and BM diagnosed between Jan 2001 and Dec 2006 were included. Continuous enrollment in the health plan for 6 months before the index date and no prior evidence of BM or IV BP use were required. Pts were followed until they left the care plan, switched therapy, or completed the study period. SRE and mortality rates were assessed. Results: The study sample included 28,415 pts with a mean age of 62.5 ± 12.24 years; approx. 25% were treated with ZOL, 8% with PAM, and 67% with no IV BP. Incidences of first and subsequent SREs were higher in the no IV BP group (incidence rate ratio [IRR] = 2.27; 95% CI = 2.03, 2.53 and IRR = 1.3; 95% CI = 1.24, 1.37, respectively) vs the ZOL/PAM groups. Time to first SRE was delayed by approx. 153 days for ZOL vs no IV BP pts. Overall, pts who had greater persistency with ZOL had lower risks of first SRE compared with no IV BP, the greatest risk reduction being observed in the 12- to 18-month persistency cohort, which experienced > 5-fold risk reduction (IRR = 0.19; 95% CI = 0.11, 0.31). The same persistency cohort also had a 27% lower risk of developing a second SRE (IRR = 0.73; 95% CI = 0.62, 0.86) compared with no IV BP. Conclusions: This study showed that in cancer pts with BM, persistence with ZOL resulted in reduced risk of developing first and subsequent SREs. Pts with longer persistence with ZOL achieved better outcomes. [Table: see text]

Impact on OS of 2nd and 3rd generation TKI in EGFR mt+ and ALK+ patients: Results of the NOWEL network.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e20560-e20560 ◽  
Author(s):  
Julia Roeper ◽  
Maria Netchaeva ◽  
Anne Christina Lueers ◽  
Ursula Stropiep ◽  
Cora Hallas ◽  
...  
Keyword(s):  
Real Life ◽  
Stage Iv ◽  
Cancer Centers ◽  
Real Life Setting ◽  
Effective Choice ◽  
Detection Of Mutations ◽  
The Impact ◽  
Choice Of Therapy ◽  
Generation Sequencing

e20560 Background: Available clinical research data shows that early mutation testing for patients with NSCLC stage IV could lead to an effective choice of therapy for patients with proven mutations. Targeted therapies achieve a higher ORR, PFS, OS and a better quality of life than chemotherapy in mt+ patients. With the advent of 2nd and 3rd generation TKI´s effective in 1st generation TKI resistant tumors, we wanted to study the impact of these drugs on the outcome of patients in a real life setting in 3 lung cancer centers. Methods: 1383 patients from the three cancer centers diagnosed with NSCLC stage IV (UICC 7) were examined. Methods for the detection of mutations included Sanger Sequencing, hybridization based COBAS testing as well as hybrid cage next generation sequencing. Results: 880/1383 (64%) consecutive patients with non-squamous cell NSCLC from the cancer centers were studied for the presence of tumor mutations, especially for EGFR and ALK mutations. The EGFR mutation rate was 16.6% (141/880), and the ALK-translocation rate 3.8% (24/635). Median OS in EGFR mt+ patients was 31 (n = 78) vs. 32 (n = 38) vs. 16 (n = 14) months respectively (center 1 vs. center 2 vs. center 3). Median OS in ALK mt+ patients was 25 (n = 17) months in center 1 and 11 (n = 5) months in center 2 (p < 0.05). Use of 3rd generation TKI Osimertinib (n = 17) lead to a significantly higher OS (n = 17, median OS 67 mo) than the use of only 1st and 2nd generation TKI (n = 113, median OS 24 mo, p < 0.000). Similarly, use of 2nd and 3rd generation ALKi impacted significantly on median OS: Crizotinib alone n = 7, 17 months, Crizotinib followed by Ceritinib and/or Brigatinib (n = 9) median OS not reached, p < 0.001. Conclusions: Smalldifferences in OS were observed, depending on the treatment centers, but the use of multiple EGFR and ALK-I impacted highly significantly on the outcome of patients with EGFR and ALK-alterations in a real life setting.

Treatment outcomes in men with metastatic castration-resistant prostate cancer (mCRPC) and cardiovascular disorders or diabetes: The Prostate Cancer Registry.

2017 ◽  
Vol 35 (15_suppl) ◽  
pp. e16537-e16537
Author(s):  
Nicolaas Lumen ◽  
Simon Chowdhury ◽  
Alison J. Birtle ◽  
Anders Bjartell ◽  
Luis Costa ◽  
...  
Keyword(s):  
Prostate Cancer ◽  
Cancer Registry ◽  
Real Life ◽  
Routine Care ◽  
Castration Resistant Prostate Cancer ◽  
Real Life Setting ◽  
Cardiac Disorders ◽  
High Level ◽  
A Prospective Study ◽  
The Impact

e16537 Background: The Prostate Cancer Registry is a prospective study of >3000 men with mCRPC in routine care. These patients (pts) have a high level of comorbidities which are often underrepresented in RCTs. Methods: Data were collected from pts with mCRPC irrespective of treatment (tx). Outcomes in pts with comorbidities were assessed. Results: At study entry, 16.6% of pts had a cardiac disorder, 9.7% hypertension only, 17.7% diabetes. Characteristics, first txs and outcomes are shown in the table below. Conclusions: In this real-life setting, AAP, ENZ, or DOC tx was feasible in pts with cardiac disorders, hypertension or diabetes; proportions treated with each drug were similar to the overall mCRPC population. Regardless of tx PFS was shorter than TTP, which may reflect the impact of pts’ comorbidities in real life. Pts with cardiac disorders had poorer outcomes vs the overall population. Clinical trial information: NCT02236637. [Table: see text]

Pathways to faster access to medicines

2018 ◽  
Vol 56 (8) ◽  
pp. 93-96 ◽  
Keyword(s):  
Treatment Options ◽  
Clinical Trial Data ◽  
Access To Medicines ◽  
Trial Data ◽  
European Medicines Agency ◽  
The European Union ◽  
Life Threatening ◽  
The Uk ◽  
New Treatments ◽  
The Impact

Before a medicine can be marketed in the UK, marketing authorisation approval is needed from the European Medicines Agency (EMA) or the Medicines and Healthcare products Regulatory Agency (MHRA). However, the time it takes to appraise a medicine is considered by some to delay access to new treatments for people with serious or life-threatening conditions who have no other treatment options. Also, the standard regulatory process may be less suitable for medicines for rare conditions in which it is difficult to gather a large amount of clinical trial data. Here we look at a range of new regulatory and access pathways that have been developed to respond to these challenges and consider some of their potential pitfalls. In a future article we will review the impact that the UK’s departure from the European Union (EU) will have on licensing processes.

scholarly journals Theoretical foundation of diversification decisions: Opportunism or financial benefits

2011 ◽  
Vol 8 (2) ◽  
pp. 37-45
Author(s):  
Raffaele Staglianò ◽  
Maurizio La Rocca
Keyword(s):  
Corporate Governance ◽  
Firm Value ◽  
Theoretical Foundation ◽  
Real Life ◽  
Empirical Models ◽  
Relevant Aspect ◽  
Firm Diversification ◽  
Real Life Setting ◽  
Financial Benefits ◽  
The Impact

The impact of firm diversification on firm value has received considerable attention from economists. However, there is no consensus on the direction of this relationship. It may be that theoretical and empirical models do not capture several complexities of real-life setting, that affect the motivation to diversify. This article surveys recent studies that extend traditional frameworks to incorporate relevant aspect of corporate governance topics

scholarly journals Live biotherapeutic products: the importance of a defined regulatory framework

2020 ◽  
Vol 52 (9) ◽  
pp. 1397-1406 ◽  
Author(s):  
Magali Cordaillat-Simmons ◽  
Alice Rouanet ◽  
Bruno Pot
Keyword(s):  
Health Benefit ◽  
The United States ◽  
Regulatory Framework ◽  
European Medicines Agency ◽  
The European Union ◽  
Technical Requirements ◽  
Regulatory Constraints ◽  
Wide Range ◽  
The Impact ◽  
Intended Use

Abstract Probiotics have been defined as “Live microorganisms that when administered in adequate amounts confer a health benefit on the host”. This definition covers a wide range of applications, target populations and (combinations of) microorganisms. Improved knowledge on the importance of the microbiota in terms of health and disease has further diversified the potential scope of a probiotic intervention, whether intended to reach the market as a food, a food supplement or a drug, depending on the intended use. However, the increased interest in the clinical application of probiotics may require specific attention given their administration in a diseased population. In addition to safety, the impact of the type of product, in terms of quality, production method and, e.g., the acceptance of side effects, is now part of the current regulatory constraints for developers. In the European Union, foods are regulated by the European Food Safety Authority and drugs by the European Medicines Agency; in the United States, the Food and Drug Administration (FDA) deals with both categories. More recently, the FDA has defined a new “live biotherapeutic products” (LBP) category, clarifying pharmaceutical expectations. Since 2019, the quality requirements for this category of drug products have also been clarified by the European Pharmacopoeia (Ph. Eur.). Similar to all products intended to prevent or treat diseases, LBPs will have to be registered as medicinal products to reach the market in the US and in Europe. In this area, regulatory authorities and the pharmaceutical industry will routinely use guidelines of the “International Council for Harmonization of Technical Requirements for Pharmaceuticals for Human Use” (ICH). Although ICH guidelines are not legally binding, they provide very important recommendations, recognized by almost all drug authorities in the world. In this review, we discuss some aspects of this regulatory framework, especially focusing on products with an intended use in a diseased or vulnerable target population.

DTCA of Prescription Medicines in the European Union: Is There Still a Need for a Ban?

2010 ◽  
Vol 17 (5) ◽  
pp. 471-484 ◽  
Author(s):  
Mareen Poser
Keyword(s):  
European Union ◽  
European Commission ◽  
General Public ◽  
Information Provision ◽  
The United States ◽  
The European Union ◽  
Medicinal Products ◽  
Prescription Medicines ◽  
Direct To Consumer Advertising ◽  
The Impact

AbstractThe pharmaceutical sector is one of the main markets in the European Union. The consumption of medicines is high and steadily increasing. However, the pharmaceutical market is subject to a wider range of restrictions than almost any other sector. The restrictions mainly apply to information provision and advertising practice within the community. One of the main features in pharmaceutical regulation is the ban on direct-to-consumer advertising (DTCA) of prescription medicines. However, an abolition of the ban is controversial in the European Community, especially as the pharmaceutical industry keeps pleading for its ability to use the highly effective marketing strategy of DTCA to promote prescription medicines to the general public. Such advertising is only allowed in two jurisdictions in the world, New Zealand and the United States. In both systems the impact of DTCA on the consumer and the economy has been subject to research. The outcome of these studies is outlined in this article. Since the European Commission has provided a new proposal to amend the current information practice regarding prescription medicines in 2008 (European Commission, Proposal for a Directive of the European Parliament and of the Council Amending, as Regards Information to the General Public on Medicinal Products Subject to Medical Prescription, Directive 2001/83/EC on the Community Code Relating to Medicinal Products for Human Use, COM/2008/0663 final) it is time to examine different options to regulate the provision of information and discuss the need for a ban of DTCA.

scholarly journals Biosimilars: the science of extrapolation

Blood ◽  
2014 ◽  
Vol 124 (22) ◽  
pp. 3191-3196 ◽  
Author(s):  
Martina Weise ◽  
Pekka Kurki ◽  
Elena Wolff-Holz ◽  
Marie-Christine Bielsky ◽  
Christian K. Schneider
Keyword(s):  
Decision Making ◽  
European Union ◽  
Clinical Trials ◽  
Safety Data ◽  
Real Life ◽  
Medical Community ◽  
The European Union ◽  
Efficacy And Safety ◽  
Medicinal Products ◽  
Regulatory Decision

Abstract Despite the establishment of a specific approval pathway, the issuance of detailed scientific guidelines for the development of similar biological medicinal products (so-called “biosimilars”) and the approval of several biosimilars in the European Union, acceptance of biosimilars in the medical community continues to be low. This is especially true in therapeutic indications for which no specific clinical trials with the biosimilar have been performed and that have been licensed based on extrapolation of efficacy and safety data from other indications. This article addresses the concerns frequently raised in the medical community about the use of biosimilars in such extrapolated indications and explains the underlying scientific and regulatory decision making including some real-life examples from recently licensed biosimilars.

Sign in / Sign up

Export Citation Format

Share Document