European collaboration on relative effectiveness assessments: What is needed to be successful?

Kleijnen, Sarah; Toenders, Wil; Groot, Folkert de; Huić, Mirjana; George, Elisabeth; Wieseler, Beate; Pavlovic, Mira; Bucsics, Anna; Siviero, P.D.; Graaff, M. van der; Rdzany, Rafał; Kristensen, Finn Børlum; Goettsch, Wim

doi:10.1016/j.healthpol.2015.01.018

Cited by 22 publications

(24 citation statements)

References 12 publications

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“…Other methodological issues should be considered in the specific field of RDs and ODs, such as the use of evidence provided by real-world data [31] , the acceptance of intermediate or surrogate outcomes, and the adoption of off-label drugs or best supportive care as comparators. Different authors analysed possible barriers and critical success factors for the implementation of cross-border assessments in Europe and reported that autonomy in decision-making of healthcare coverage is a politically sensitive issue [56,57] . Some of the identified success factors include the early agreement on topic selection and scope definition for assessment, a methodological framework flexible enough to fit demands/capacities from individual countries, voluntary use at the national level of the common assessment framework, the necessity to build trust among participating countries, and the need of relying on transparent and high-quality processes to get valid outputs.…”

Section: Comprehensive Value Assessmentmentioning

confidence: 99%

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Mincarone

Leo²,

Sabina³

et al. 2017

Public Health Genomics

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The pricing and reimbursement policies for pharmaceuticals are relevant to balance timely and equitable access for all patients, financial sustainability, and reward for valuable innovation. The proliferation of high-cost specialty medicines is particularly true in rare diseases (RDs) where the pricing mechanism is characterised by a lack of transparency. This work provides an overall picture of current strategies for the definition of the reimbursed prices of orphan drugs (ODs) and highlights some potential improvements. Current strategies and suggestions are presented along 4 dimensions: (1) comprehensive value assessment, (2) early dialogs among relevant stakeholders, (3) innovative reimbursement approaches, and (4) societal participation in producing ODs. Comprehensive value assessment could be achieved by clarifying the approach of distributive justice to adopt, ensuring a representative participation of stakeholders, and with a broad consideration of value-bearing factors. With respect to early dialogs, cross-border cooperation can be determinant to companies and agencies. The cost-benefit ratio of early dialogs needs to be demonstrated and the “regulatory capture” effect should be monitored. Innovative reimbursement approaches were developed to balance the need for evidence-based decisions with the timely access to innovative drugs. The societal participation in producing ODs needs to be recognised in a collaborating framework where adaptive agreements can be developed with mutual satisfaction. Such agreements could also impact on coverage and reimbursement decisions as additional elements for the determination of a comprehensive societal value of ODs. Further research is needed to investigate the highlighted open challenges so that RDs will not remain, in practical terms, orphan diseases.

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Section: Comprehensive Value Assessmentmentioning

confidence: 99%

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Mincarone

Leo²,

Sabina³

et al. 2017

Public Health Genomics

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“…Also, some steps have been taken to establish internationally recognized good practices in HTA (16). Consensus has been reached around the practical tools and methods in the field of HTA in Europe (17) including the HTA Core Model (18) and rapid relative effectiveness assessments of new pharmaceuticals to be used for European collaboration (19)(20)(21). Continuous benchmarking of performance will be of great value to capture changes in the system.…”

Section: Benchmarking Hta Agencies: Improving Timeliness and Transparmentioning

confidence: 99%

Benchmarking health technology assessment agencies—methodological challenges and recommendations

Wang

Lipska

McAuslane

et al. 2020

Int J Technol Assess Health Care

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Objectives The objectives of the study were to establish a benchmarking tool to collect metrics to enable increased clarity regarding the differences and similarities across health technology assessment (HTA) agencies, to assess performance within and across HTA agencies, identify areas in the HTA processes in which time is spent and to enable ongoing performance improvement. Methods Common steps and milestones in the HTA process were identified for meaningful benchmarking among agencies. A benchmarking tool consisting of eighty-six questions providing information on HTA agency organizational aspects and information on individual new medicine review timelines and outcomes was developed with the input of HTA agencies and validated in a pilot study. Data on 109 HTA reviews from five HTA agencies were analyzed to demonstrate the utility of this tool. Results This study developed an HTA benchmarking methodology, comparative metrics showed considerable differences among the median timelines from assessment and appraisal to final HTA recommendation for the five agencies included in this analysis; these results were interpreted in conjunction with agency characteristics. Conclusions It is feasible to find consensus among HTA agencies regarding the common milestones of the review process to map jurisdiction-specific processes against agreed metrics. Data on characteristics of agencies such as their scope and remit enabled results to be interpreted in the appropriate local context. This benchmarking tool has promising potential utility to improve the transparency of the review process and to facilitate both quality assurance and performance improvement in HTA agencies.

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“…The benefits of establishing European Reference networks for rare diseases has been widely recognised [93], notably by overcoming the limited experience of professionals confronted with rare disease and by improving access for EU citizens to treatment requiring a particular concentration of resources or expertise. The future European collaboration on Relative Effectiveness Assessments, which is currently launching pilots within EUNetHTA’s Joint Action 3 [94] and planning to be formalised in 2019, is a key step forward towards this needed collaboration.…”

Section: Principles On Omp European Co-ordinationmentioning

confidence: 99%

Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

Annemans

Aymé

Cam

et al. 2017

Orphanet J Rare Dis

102

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Rare diseases are an important public health issue with high unmet need. The introduction of the EU Regulation on orphan medicinal products (OMP) has been successful in stimulating investment in the research and development of OMPs. Despite this advancement, patients do not have universal access to these new medicines. There are many factors that affect OMP uptake, but one of the most important is the difficulty of making pricing and reimbursement (P&R) decisions in rare diseases. Until now, there has been little consensus on the most appropriate assessment criteria, perspective or appraisal process. This paper proposes nine principles to help improve the consistency of OMP P&R assessment in Europe and ensure that value assessment, pricing and funding processes reflect the specificities of rare diseases and contribute to both the sustainability of healthcare systems and the sustainability of innovation in this field. These recommendations are the output of the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL), a collaboration between rare disease experts, patient representatives, academics, health technology assessment (HTA) practitioners, politicians and industry representatives. ORPH-VAL reached its recommendations through careful consideration of existing OMP P&R literature and through a wide consultation with expert stakeholders, including payers, regulators and patients. The principles cover four areas: OMP decision criteria, OMP decision process, OMP sustainable funding systems and European co-ordination. This paper also presents a guide to the core elements of value relevant to OMPs that should be consistently considered in all OMP appraisals. The principles outlined in this paper may be helpful in drawing together an emerging consensus on this topic and identifying areas where consistency in payer approach could be achievable and beneficial. All stakeholders have an obligation to work together to ensure that the promise of OMP’s is realised.Electronic supplementary materialThe online version of this article (doi:10.1186/s13023-017-0601-9) contains supplementary material, which is available to authorized users.

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European collaboration on relative effectiveness assessments: What is needed to be successful?

Cited by 22 publications

References 12 publications

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Reimbursed Price of Orphan Drugs: Current Strategies and Potential Improvements

Benchmarking health technology assessment agencies—methodological challenges and recommendations

Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL)

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