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2.
Patient ; 2020 Jan 16.
Artigo em Inglês | MEDLINE | ID: mdl-31942698

RESUMO

Patient preference studies could provide valuable insights to a National Institute for Health and Care Excellence committee into the preferences patients have for different treatment options, especially if the study sample is representative of the broader patient population. We identify three main uses of patient preference studies along a technology's pathway from drug development to clinical use: in early clinical development to guide the selection of appropriate endpoints, to inform benefit-risk assessments carried out by regulators and to inform reimbursement decisions made by health technology assessment bodies. In the context of the National Institute for Health and Care Excellence's methods and processes, we do not see a role for quantitative patient preference data to be directly incorporated into health economic modelling. Rather, we see a role for patient preference studies to be submitted alongside other types of evidence. Examples where patient preference studies might have added value in health technology assessments include cases where two distinctly different treatment options are being compared, when patients have to decide between multiple treatment options, when technologies have important non-health benefits or when a treatment is indicated for a heterogenous population.

4.
Front Med (Lausanne) ; 6: 73, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-31134200

RESUMO

Aims: To facilitate regulatory learning, we evaluated similarities and differences in evidence requirements between regulatory and health technology assessment (HTA) bodies of Alzheimer's disease (AD) approved products. Methods: The European marketing authorisation application dossiers and European public assessment reports (EPARs) of the licensed AD drugs were screened to identify the phase III randomised controlled trials (RCTs) and outcomes used. We also screened the assessment reports of the National Institute of Health and Care Excellence (NICE, England) and the National Health Care Institute (ZiN, the Netherlands) to identify the studies and outcomes used in HTA assessments. Results: The application dossiers of donepezil, galantamine, rivastigmine, and memantine contained 16 phase III RCTs in total. These trials were also included in HTA assessments except that NICE excluded studies that were not published (n = 2) or trials that included patients with other types of dementia (n = 3). In the regulatory assessments the focus was on cognitive and global outcomes, and to some extent on function. In the HTA assessments of clinical effectiveness other domains were also covered including: function, behaviour and mood, and, occasionally, quality of life. In the economic analyses of NICE the domains cognition, function, and quality of life were included. Conclusion: There was a large overlap in inclusion of trials in regulatory and HTA assessments, although the focus on specific outcomes slightly differed. Understanding the methods and perceptions of both authorities can stimulate regulatory and HTA cross-talk and further alignment, and therefore more rapid patient access to new treatments.

5.
Br J Clin Pharmacol ; 85(7): 1427-1433, 2019 07.
Artigo em Inglês | MEDLINE | ID: mdl-30849187

RESUMO

In 2016, the European Medicines Agency published the conclusions of its pilot on adaptive pathways, with products in early stages of development still building up to their marketing authorisation. Adaptive pathways rests on three principles: iterative development; gathering evidence through real-life use to supplement clinical trial data; and early engagement of patients, payers and health technology assessment bodies in discussions on a medicine's development. While the pilot has now finished, the practical system-wide implications of employing the adaptive pathways approach are not known and further consideration of these three principles is required. In this paper we used the three principles that underpin adaptive pathways to discuss main scientific and European policy developments likely to determine progress on further implementing adaptive pathways in the European setting.

6.
Clin Pharmacol Ther ; 105(3): 684-691, 2019 03.
Artigo em Inglês | MEDLINE | ID: mdl-30300938

RESUMO

This study assessed whether five Health Technology Assessment (HTA) bodies in Europe were more negative about drugs with a Conditional Marketing Authorization (CMA) that are approved without controlled studies compared to CMA drugs that are approved based on controlled studies. The HTA recommendations were categorized into positive, restricted, and negative. A total of 92 HTA recommendations were available for 27 drugs. Thirty of 62 (48%) and 17 of 30 (57%) of the recommendations were negative for drugs with and without controlled studies, respectively. Overall, only 12 (13%) recommendations were positive. In all jurisdictions, recommendations between drugs with and drugs without controlled data were comparable, which suggests that the presence of controlled data is not decisive in HTA evaluations. The small proportion of unrestricted positive recommendations highlights difficulties with recommending the drugs in this cohort, which may be caused by scientific uncertainty or other factors. Earlier collaboration between stakeholders is advised in order to improve patient access.


Assuntos
Aprovação de Drogas/métodos , Medicina Baseada em Evidências/métodos , Reembolso de Seguro de Saúde , Avaliação da Tecnologia Biomédica/métodos , Europa (Continente) , Medicina Baseada em Evidências/normas , Humanos , Reembolso de Seguro de Saúde/normas , Estudos Retrospectivos , Avaliação da Tecnologia Biomédica/normas
7.
Clin Pharmacol Ther ; 105(5): 1148-1155, 2019 May.
Artigo em Inglês | MEDLINE | ID: mdl-29901216

RESUMO

Medicines Adaptive Pathways to Patients (MAPPs) seeks to foster access to novel beneficial treatments for the right patient groups at the earliest appropriate time in the product life-span, in a sustainable fashion. We summarize the MAPPs engagement process and critical questions to be asked at each milestone of the product life-span. These considerations are of relevance for regulatory and access pathways that strive to address the "evidence vs. access" conundrum.

8.
J Alzheimers Dis ; 67(2): 495-501, 2019.
Artigo em Inglês | MEDLINE | ID: mdl-30584137

RESUMO

ROADMAP is a public-private advisory partnership to evaluate the usability of multiple data sources, including real-world evidence, in the decision-making process for new treatments in Alzheimer's disease, and to advance key concepts in disease and pharmacoeconomic modeling. ROADMAP identified key disease and patient outcomes for stakeholders to make informed funding and treatment decisions, provided advice on data integration methods and standards, and developed conceptual cost-effectiveness and disease models designed in part to assess whether early treatment provides long-term benefit.

9.
CNS Drugs ; 32(12): 1085-1090, 2018 12.
Artigo em Inglês | MEDLINE | ID: mdl-30467744

RESUMO

Although there are a growing number of well-reported, late-stage clinical trial failures in Alzheimer's disease, the introduction of a disease-modifying therapy within the next 5 years may be anticipated. These treatments are likely to target Alzheimer's disease in the earlier disease stages, unlike drugs that are currently available that treat symptoms of moderate-to-severe dementia. Therefore, there is a need to establish a consensus on regulatory and health technology assessment requirements for Alzheimer's disease, as a new drug will need to undergo regulatory and health technology assessments before it becomes available to patients. This article reports the discussions and activities of the regulatory and health technology assessment expert advisory group of the 2-year ROADMAP (real-world outcomes across the Alzheimer's disease spectrum: a multimodal data access platform) project. The expert advisory group discussions identified a lack of consensus on validated outcomes in the earliest Alzheimer's disease stages, the need for filling gaps between outcomes used across clinical trials and real-world settings, and the role that real-world evidence might have in characterising the impact of a possible disease-modifying therapy on caregivers, resource use and long-term outcomes.


Assuntos
Doença de Alzheimer/tratamento farmacológico , Antipsicóticos/uso terapêutico , Avaliação da Tecnologia Biomédica , Resultado do Tratamento , Progressão da Doença , Feminino , Humanos , Masculino , Modelos Econométricos
11.
Front Pharmacol ; 9: 280, 2018.
Artigo em Inglês | MEDLINE | ID: mdl-29636692

RESUMO

As per the EMA definition, adaptive pathways is a scientific concept for the development of medicines which seeks to facilitate patient access to promising medicines addressing high unmet need through a prospectively planned approach in a sustainable way. This review reports the findings of activities undertaken by the ADAPT-SMART consortium to identify enablers and explore the suitability of managed entry agreements for adaptive pathways products in Europe. We found that during 2006-2016 outcomes-based managed entry agreements were not commonly used for products with a conditional marketing authorization or authorized under exceptional circumstances. The barriers and enablers to develop workable managed entry agreements models for adaptive pathways products were discussed through interviews and a multi-stakeholder workshop with a number of recommendations made in this paper.

12.
Pharmacoepidemiol Drug Saf ; 26(12): 1442-1450, 2017 Dec.
Artigo em Inglês | MEDLINE | ID: mdl-28345151

RESUMO

PURPOSE: Regulatory agencies and other stakeholders increasingly rely on data collected through registries to support their decision-making. Data from registries are a cornerstone of post-marketing surveillance for monitoring the use of medicines in clinical practice. This study was aimed at gaining further insight into the European Medicines Agency's (EMA) requests for new registries and registry studies using existing registries and to review the experience gained in their conduct. METHODS: European Public Assessment Reports were consulted to identify products for which a request for a registry was made as a condition of the marketing authorisation. All centrally authorised products that received a positive opinion of the EMA Committee for Medicinal Products for Human Use between 1 January 2005 and 31 December 2013 were included. Data regarding registry design and experiences were collected from EMA electronic record keeping systems. RESULTS: Of 392 products that received a positive Committee for Medicinal Products for Human Use opinion during 2005-2013, 31 registries were requested for 30 products in total. Sixty-five percent were product registries whereas 35% were disease registries and 71% of the registries had a primary safety objective. Most commonly reported issues with registries were delayed time to start and low patient accrual rates. CONCLUSIONS: The delays found in getting new registries up and running support the need to improve the timeliness of data collection in the post-marketing setting. Methodological challenges met in conducting this study highlighted the need for a clarification of definitions and epidemiological concepts around patient registries. The results will inform the EMA Patient Registry initiative to support use of existing patient registries for the post-authorisation benefit-risk monitoring of medicinal products. © 2017 Commonwealth of Australia. Pharmacoepidemiology & Drug Safety © 2017 John Wiley & Sons, Ltd.


Assuntos
Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/epidemiologia , Vigilância de Produtos Comercializados/métodos , Sistema de Registros , Bases de Dados Factuais , Aprovação de Drogas , Indústria Farmacêutica , Europa (Continente)/epidemiologia , União Europeia , Humanos , Legislação de Medicamentos , Farmacovigilância , Estudos Retrospectivos
13.
Pharmacoepidemiol Drug Saf ; 25(9): 1004-14, 2016 09.
Artigo em Inglês | MEDLINE | ID: mdl-27146035

RESUMO

PURPOSE: The aim of this study was to determine the outcomes and timing within the product life cycle of all benefit-risk reassessment procedures for marketed products that were completed by the committee for medicinal product for human use during 2001-2012. METHODS: A cohort of all referral procedures for benefit-risk reassessment (Article 20, Article 31, Article 36, Article 107 procedures) for which committee for medicinal product for human use issued an opinion between 1 January 2001 and 31 December 2012 was created. The European Medicines Agency website and the Dutch Medicines Evaluation Board website were used to collect all data. RESULTS: There were a total of 73 benefit-risk reassessments during the study period; 61 reassessments for a single product and 12 reassessments for multiple products or an entire product class. Nineteen reassessments resulted in the recommendation to remove the product from the market. On average, a benefit-risk reassessment was performed 18.7 years after the product was first marketed. Seventeen products were marketed 5 years or less when the reassessment procedure was completed; six of these products were subsequently removed from the market. CONCLUSIONS: The majority of all benefit-risk reassessments that were performed during the study period did not result in removing the product from the market, but rather, in confirming the positive benefit-risk of the product, conditional to changes to the product's marketing authorisation. About half of all products that were removed from the market during the 2000s had been marketed for more than 20 years. Copyright © 2016 John Wiley & Sons, Ltd.


Assuntos
Aprovação de Drogas , Vigilância de Produtos Comercializados/estatística & dados numéricos , Medição de Risco/métodos , Retirada de Medicamento Baseada em Segurança/estatística & dados numéricos , Europa (Continente) , Humanos , Países Baixos , Vigilância de Produtos Comercializados/métodos , Estudos Retrospectivos , Fatores de Tempo
14.
Eur J Clin Pharmacol ; 71(10): 1237-44, 2015 Oct.
Artigo em Inglês | MEDLINE | ID: mdl-26204969

RESUMO

PURPOSE: The aim of this study is to provide a comprehensive overview of the outcomes of marketing authorisation applications via the mutual recognition and decentralised procedures (MRP/DCP) and assess determinants of licensing failure during CMDh referral procedures. METHODS: All MRP/DCP procedures to the Co-ordination group for Mutual recognition and Decentralised procedures-human (CMDh) during the period from January 2006 to December 2013 were analysed. Reasons for starting referral procedures were scored. In addition, a survey under pharmaceutical companies was performed to estimate the frequency of licensing failure prior to CMDh referrals. RESULTS: During the study period, 10392 MRP/DCP procedures were finalized. Three hundred seventy-seven (3.6%) resulted in a referral procedure, of which 70 (19%) resulted in licensing failure, defined as refusal or withdrawal of the application. The frequency of CMDh referrals decreased from 14.5% in 2006 to 1.6% in 2013. Of all referrals, 272 (72%) were resolved through consensus within the CMDh, the remaining 105 (28%) were resolved at the level of the CHMP. Most referrals were started because of objections raised about the clinical development program. Study design issues and objections about the demonstration of equivalence were most likely to result in licensing failure. An estimated 11% of all MRP/DCP procedures resulted in licensing failure prior to CMDh referral. CONCLUSION: Whereas the absolute number of MRP/DCP procedures resulting in a referral has reduced substantially over the past years, no specific time trend could be observed regarding the frequency of referrals resulting in licensing failure. Increased knowledge at the level of companies and regulators has reduced the frequency of late-stage failure of marketing applications via the MRP/DCP.


Assuntos
Aprovação de Drogas/organização & administração , Aprovação de Drogas/estatística & dados numéricos , Drogas em Investigação , União Europeia , Política , Aprovação de Drogas/legislação & jurisprudência , Medicamentos Genéricos , Humanos , Marketing , Fatores de Tempo
15.
Drug Saf ; 38(5): 437-53, 2015 May.
Artigo em Inglês | MEDLINE | ID: mdl-25822400

RESUMO

Adverse drug reactions (ADRs) cause considerable mortality and morbidity but no recent reviews are currently available for the European region. Therefore, we performed a review of all epidemiological studies quantifying ADRs in a European setting that were published between 1 January 2000 and 3 September 2014. Included studies assessed the number of patients who were admitted to hospital due to an ADR, studies that assessed the number of patients who developed an ADR during hospitalization, and studies that measured ADRs in the outpatient setting. In total, 47 articles were included in the final review. The median percentage of hospital admissions due to an ADR was 3.5 %, based on 22 studies, and the median percentage of patients who experienced an ADR during hospitalization was 10.1 %, based on 13 studies. Only five studies were found that assessed ADRs occurring in the outpatient setting. These results indicate that the occurrence of ADRs in the European hospital setting-both ADRs that result in hospitalization and ADRs that occur during the hospital stay-is significant. Furthermore, the limited number of studies that were performed in the outpatient setting identify a lack of information regarding the epidemiology of ADRs in this setting.


Assuntos
Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/epidemiologia , Causalidade , Efeitos Psicossociais da Doença , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/mortalidade , Efeitos Colaterais e Reações Adversas Relacionados a Medicamentos/terapia , Europa (Continente)/epidemiologia , Hospitalização , Humanos , Estudos Observacionais como Assunto , Prevalência
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