VP74 Orphan Black Box: Explanatory Principles

2019 ◽  
Vol 35 (S1) ◽  
pp. 92-93
Author(s):  
George Wang ◽  
Richard Macaulay
Keyword(s):  
Market Access ◽  
Economic Value ◽  
Orphan Drugs ◽  
Regulatory Approval ◽  
Marketing Authorization ◽  
European Medicines Agency ◽  
Reimbursement Mechanisms ◽  
Regulatory Pathways ◽  
Small Patient ◽  
Pricing And Reimbursement

IntroductionOrphan legislations over the past thirty years have successfully increased the number of drugs receiving marketing authorization for rare diseases. However, for a therapy to be accessible to most patients, it requires not only marketing authorization, but market access via public reimbursement. In many major markets, the pricing and reimbursement of new therapies is based on an assessment by a national Health Technology Assessment (HTA) body, for which economic value is typically a key consideration. This research evaluates the outcome of HTAs of orphan drugs in Europe.MethodsHTA decision data (to 31/08/2017) was extracted from Gemeinsame Bundesausschuss (G-BA), Haute Autorité de Santé (HAS), National Institute for Health and Care Excellence (NICE), Pharmaceutical Benefits Advisory Committee (PBAC), and Scottish Medicines Consortium (SMC) websites. EC-approval data was extracted from the European Medicines Agency (to 31/08/2017).ResultsOnly a small minority of drugs for orphan diseases received full recommendations for their licensed indication(s) by NICE (3/35, 9%), SMC (8/66, 12%) and PBAC (1/44, 2%). 37% (26/70) of drugs assessed received positive HTA outcome by HAS (ASMR I-III). In Germany, all approved orphan drugs (100/100) received automatic additional benefit post regulatory approval by G-BA.ConclusionsThere have been significant challenges for manufacturers in converting regulatory approval of orphan drugs into commercial success and optimised market access. Attaining positive HTA appraisals for these drugs, which have been approved under expedited regulatory pathways on a less than fully mature dataset, whilst also having high prices, due to small patient populations, limiting commercial returns, may necessitate increased utilisation of alternative reimbursement mechanisms.

scholarly journals PRO72 MARKET ACCESS, PRICING AND REIMBURSEMENT PROCESSES FOR ORPHAN DRUGS IN THE EU-5 COUNTRIES: A COMPARATIVE ASSESSMENT

2019 ◽  
Vol 22 ◽  
pp. S854
Author(s):  
L. Vollmer ◽  
M. Prada ◽  
F. Benazet ◽  
I. Berard ◽  
N. López ◽  
...  
Keyword(s):  
Market Access ◽  
Orphan Drugs ◽  
Comparative Assessment ◽  
Access Pricing ◽  
Pricing And Reimbursement ◽  
The Eu

PP30 Do Conditional Regulatory Pathways Affect Health Technology Assessment Recommendations?

2019 ◽  
Vol 35 (S1) ◽  
pp. 42-42
Author(s):  
Jesmine Cai ◽  
Tina Wang ◽  
Neil McAuslane ◽  
Lawrence Liberti
Keyword(s):  
Health Care ◽  
Health Technology Assessment ◽  
Technology Assessment ◽  
Health Technology ◽  
Regulatory Approval ◽  
European Medicines Agency ◽  
Regulatory Pathways ◽  
Medical Need ◽  
Timely Access ◽  
Active Substances

IntroductionIn an effort to expedite the approval of drugs treating serious illnesses or addressing unmet medical need, conditional approvals have been used by the European Medicines Agency. In this study, the effects of conditional approvals were investigated in terms of health technology assessment (HTA) recommendations and timing in Europe.MethodsFirst HTA recommendations of new active substances (NASs) issued between 2015 and 2017 were collected from the National Institute for Health and Care Excellence (England), Haute Autorité de Santé (France), Institute for Quality and Efficiency in Health Care (Germany), Scottish Medicine Consortium (Scotland) and Tandvårds-Läkemedelförmånsverket (Sweden). The HTA recommendations were then classified into the following categories: positive, positive with restrictions, negative and multiple and if the regulatory approval pathway had been standard or conditional.ResultsOf this cohort of NASs that received an HTA recommendation, eight of 56 in England, 12 of 83 in France, 11 of 77 in Germany, nine of 58 in Scotland and four of 49 in Sweden were approved via a conditional review. Generally, except in England, there were a higher proportion of positive first recommendations for conditional approvals when compared to standard approvals, with Germany showing the largest proportional difference (43 percent) between the two pathways and also a faster time to recommendation. This may relate to the proportion of conditional assessments that were orphan medicines. With the exception of Germany, the time taken from regulatory approval to first HTA recommendation for products with conditional approvals is higher than those for standard approvals, with the largest difference seen in Sweden (241 days longer).ConclusionsConditionally approved NASs showed a variable HTA outcome; although there was generally a higher proportion of positive recommendations thus enabling more likely access in conditional approvals, the timing from regulatory approval to HTA recommendation was longer compared with standard approvals. This warrants a better understanding of the factors and uncertainties underlying these recommendations, supporting timely access of NASs with conditional approval.

scholarly journals Role of the EMA specific marketing authorization procedures for early access on the time to patient access in Bulgaria

Pharmacia ◽  
2021 ◽  
Vol 68 (2) ◽  
pp. 421-425
Author(s):  
Ivo Tsekov ◽  
Maria Dimitrova ◽  
Yulian Voynikov
Keyword(s):  
Market Access ◽  
Marketing Authorization ◽  
Patient Access ◽  
The European Union ◽  
Legal Requirements ◽  
Time To Market ◽  
The Public ◽  
Early Access ◽  
Pricing And Reimbursement

Despite the early access procedures for marketing authorization (MA) valid throughout the European Union still in the most of the Member states patient access to innovative medicines depends on cost-effectiveness, budget impact assessment and negotiations for price discount with the public payers. Retrospective analysis on the availability and time to market access of medicines authorized under the European medicines agency’s specific procedures for early access shows that despite the shortening of the time to market access after 2013, for most medicines still exceeds 365 days. This is due to the fact that requirements for pricing and reimbursement across EU is fixed to some degree and medicines with MA for early access are subject to the same legal requirements as the medicines with standard centralized marketing authorization. Some specific national legal requirements for pricing and reimbursement decisions, population of interest and manufactures intentions to enter certain markets should also be considered.

Strategies of stratification: Regulating market access in the era of personalized medicine

2021 ◽  
pp. 030631272110055
Author(s):  
Sarah Wadmann ◽  
Amalie Martinus Hauge
Keyword(s):  
Personalized Medicine ◽  
Patient Population ◽  
Science And Technology Studies ◽  
Market Access ◽  
European Medicines Agency ◽  
The Political ◽  
Market Regulation ◽  
Publicly Funded ◽  
Pharmaceutical Markets ◽  
Danish Healthcare System

Personalized medicine raises the stakes of pharmaceutical market regulation. Drawing on pragmatist valuation studies and science and technology studies literature on personalized medicine and pharmaceutical markets, this article demonstrates how complex negotiations about the value of a pharmaceutical can constitute a market in various ways, while also shaping the concerned patient populations. Tracing the path of a pharmacogenetic treatment, Spinraza, from its approval by the European Medicines Agency to its adoption in the publicly funded Danish healthcare system, we show how the market was formatted through particular stratifications of the patient population. We conceptualize these seemingly technical moves as strategies of stratification, that is, the application of techniques to assemble and divide data – and what data are meant to represent – into groups delineated by certain characteristics. We argue that stakeholders’ use of strategies of stratification has important implications not only for market access, but also for the delineation of diseases and patient populations. Hence, it is crucial to make intelligible the mutual constitution of pharmaceutical markets and patient populations and the political efforts of delineating and connecting the two.

scholarly journals A Decade of Marketing Authorization Applications of Anticancer Drugs in the European Union: An Analysis of Procedural Timelines

2021 ◽  
Author(s):  
Marjolein Garsen ◽  
Maaike Steenhof ◽  
Alex Zwiers
Keyword(s):  
Anticancer Drugs ◽  
Marketing Authorization ◽  
European Medicines Agency ◽  
Company Size ◽  
Scientific Advice ◽  
The European Union ◽  
Small Companies ◽  
Procedure Time ◽  
Total Procedure Time ◽  
Total Procedure

Abstract Background Cancer is a serious global health problem and a major cause of death. The European Medicines Agency (EMA) has established several regulatory initiatives to expedite the development and authorization of drugs to ensure timely access of patients. In this study, we analyzed the procedural timelines of marketing authorization applications for anticancer drugs in the EU, with a specific focus to special regulatory programs, scientific advice and company size. Methods Anticancer drugs that received an opinion from the EMA between January 2010 and December 2019 were included in the study. Public assessment reports were used to obtain publicly available information of the drugs. Results We identified 96 applications for new anticancer drugs. 34 applications were granted access to at least one expedited program offered by the EMA. Total procedure time was reduced from average 370 to 200–215 days when accelerated assessment was granted. Granting of a conditional marketing authorization or an orphan designation, as well as having scientific advice, only mildly affected total procedure time. Average total procedure time of small companies was much longer compared with medium-sized and large companies (483 versus 356 days), which was caused by an increased clock stop time. Conclusion Total procedure time for anticancer is mainly affected by the granting of accelerated assessment, which reduced the total procedure time, and company size, where total procedure time is much longer for small companies. Small companies are advised to have, and especially adhere to scientific advice to reduce procedure time and increase the chance of success.

scholarly journals Genetic regulation of shoot architecture in cucumber

2021 ◽  
Vol 8 (1) ◽  
Author(s):  
Xiaofeng Liu ◽  
Jiacai Chen ◽  
Xiaolan Zhang
Keyword(s):  
Leaf Shape ◽  
Economic Value ◽  
Genetic Regulation ◽  
Strong Impact ◽  
Crop Species ◽  
Visual Appearance ◽  
Regulatory Pathways ◽  
Architectural Features ◽  
Shoot Architecture ◽  
Plant Form

AbstractCucumber (Cucumis sativus L.) is an important vegetable crop species with great economic value. Shoot architecture determines the visual appearance of plants and has a strong impact on crop management and yield. Unlike most model plant species, cucumber undergoes vegetative growth and reproductive growth simultaneously, in which leaves are produced from the shoot apical meristem and flowers are generated from leaf axils, during the majority of its life, a feature representative of the Cucurbitaceae family. Despite substantial advances achieved in understanding the regulation of plant form in Arabidopsis thaliana, rice, and maize, our understanding of the mechanisms controlling shoot architecture in Cucurbitaceae crop species is still limited. In this review, we focus on recent progress on elucidating the genetic regulatory pathways underlying the determinant/indeterminant growth habit, leaf shape, branch outgrowth, tendril identity, and vine length determination in cucumber. We also discuss the potential of applying biotechnology tools and resources for the generation of ideal plant types with desired architectural features to improve cucumber productivity and cultivation efficiency.

scholarly journals IS THE EUNETHTA HTA CORE MODEL® FIT FOR PURPOSE? EVALUATION FROM AN INDUSTRY PERSPECTIVE

2018 ◽  
Vol 34 (5) ◽  
pp. 458-463 ◽  
Author(s):  
Marlene Gyldmark ◽  
Kristian Lampe ◽  
Jörg Ruof ◽  
Johannes Pöhlmann ◽  
Ansgar Hebborn ◽  
...  
Keyword(s):  
Technology Use ◽  
Market Access ◽  
Economic Value ◽  
Clinical Effectiveness ◽  
Health Technology ◽  
Core Model ◽  
European Network ◽  
Survey Results ◽  
Fit For Purpose ◽  
Evidence Generation

Objectives:The HTA Core Model® was developed to improve the transferability of health technology assessment (HTA) between settings. The model has been used by HTA agencies but is also of interest to manufacturers, for improving internal evidence generation and communicating with other HTA stakeholders. To establish if the model is fit for purpose from an industry perspective, the pharmaceutical company Roche, collaborating with the European Network for HTA (EUnetHTA), conducted an assessment of the model.Methods:A questionnaire was developed to evaluate all assessment elements in the HTA Core Model v2.0 for their usefulness in meeting payers’ evidence needs and demonstrating value. The questionnaire was completed by country affiliate teams working in evidence generation and reimbursement submissions for pharmaceuticals. Survey results were discussed in workshops to ensure consistency and alignment between teams.Results:The questionnaire was completed by six teams. An additional team from global pricing and market access participated in workshops. Model domains pertaining to the health problem and current technology use, technology description, clinical effectiveness, and economic value were considered most important because they meet payers’ evidence needs. Overall, the model was considered useful to improve the efficiency of HTA evidence generation, share evidence internally, and communicate value to payers and HTA agencies.Conclusions:From an industry perspective, the HTA Core Model provides a useful framework and common terminology for efficient generation of transferable HTA evidence. The timeliness, efficiency, and transparency of HTA processes could be improved by a more standardized approach to HTA across settings.

scholarly journals Key Regulatory Pathways, MicroRNAs, and Target Genes Participate in Adventitious Root Formation of Acer Rubrum L

2021 ◽  
Author(s):  
Wenpeng Zhu ◽  
Manyu Zhang ◽  
Jianyi Li ◽  
Hewen Zhao ◽  
Kezhong Zhang ◽  
...  
Keyword(s):  
Transcriptional Activation ◽  
High Throughput Sequencing ◽  
Target Genes ◽  
Seedling Survival ◽  
Expression Patterns ◽  
Economic Value ◽  
Acer Rubrum ◽  
Pcr Analysis ◽  
Asexual Propagation ◽  
Regulatory Pathways

Abstract BackgroundAcer rubrum L. is a colorful ornamental tree with great economic value. Because this tree is difficult to root under natural conditions and the seedling survival rate is low, vegetative propagation methods are often used. Because the formation of adventitious roots (ARs) is essential for the survival of asexual propagation of A. rubrum, it is necessary to investigate the molecular regulatory mechanisms in the formation of ARs of A. ruburm. To address this knowledge gap, we sequenced the transcriptome and sRNA of the A. rubrum variety ‘Autumn Fantasy’ using high-throughput sequencing and explored changes in gene and microRNA (miRNA) expression in response to exogenous auxin treatment. ResultsWe identified 82,468 differentially expressed genes between the treated and untreated ARs, as well as 48 known and 95 novel miRNAs. We also identified 172 target genes of the known miRNAs using degradome sequencing. Two regulatory pathways (ubiquitin mediated proteolysis and plant hormone signal transduction), Ar-miR160a and the target gene ArARF10 were shown to be involved in the auxin response. We further investigated the expression patterns and regulatory roles of ArARF10 through subcellular localization, transcriptional activation, plant transformation, qRT-PCR analysis, and GUS staining. ConclusionsDifferential expression patterns indicated the Ar-miR160a-ArARF10 interaction might play a significant role in the regulation of AR formation in A. rubrum. Our study provided new insights into mechanisms underlying the regulation of AR formation in A. rubrum.

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