scholarly journals Application of Managed Entry Agreements for Innovative Therapies in Different Settings and Combinations: A Feasibility Analysis

2020 ◽  
Vol 17 (22) ◽  
pp. 8309
Author(s):  
Rick A Vreman ◽  
Thomas F Broekhoff ◽  
Hubert GM Leufkens ◽  
Aukje K Mantel-Teeuwisse ◽  
Wim G Goettsch
Keyword(s):  
Target Population ◽  
Decision Makers ◽  
Reimbursement Mechanisms ◽  
Feasibility Assessment ◽  
Innovative Therapy ◽  
Innovative Therapies ◽  
Managed Entry Agreements ◽  
Pricing And Reimbursement ◽  
Evidence Generation ◽  
Financial Outcome

The reimbursement of expensive, innovative therapies poses a challenge to healthcare systems. This study investigated the feasibility of managed entry agreements (MEAs) for innovative therapies in different settings and combinations. First, a systematic literature review included studies describing used or conceptual agreements between payers and manufacturers (i.e., MEAs). Identical and similar MEAs were clustered and data were extracted on their benefits and limitations. A feasibility assessment was performed for each individual MEA based on how it could be applied (financial/outcome-based), on what level (individual patients/target population), in which payment setting (centralized pricing and reimbursement authority yes/no), for what type of therapies (one-time/chronic), within what payment structures, and whether combinations with other MEAs were feasible. The literature search ultimately included 82 papers describing 117 MEAs. After clustering, 15 unique MEAs remained, each describing one or multiple similar agreements. Four of those entailed payment structures, while eleven entailed agreements between payers and manufacturers regarding price, usage, and/or evidence generation. The feasibility assessment indicated that most agreements could be applied throughout the different settings that were assessed and could be applied in different payment structures and in combination with multiple other agreements. The potential to combine multiple agreements leads to a multitude of different reimbursement mechanisms that may manage the price, usage, payment structure, and additional conditions for an innovative therapy. This overview of the feasibility of combinations of MEAs can help decision-makers construct a reimbursement mechanism most suited to their preferences, the type of therapy under evaluation, and the applicable healthcare system.

scholarly journals Scaling up population health interventions from decision to sustainability – a window of opportunity? A qualitative view from policy-makers

2020 ◽  
Vol 18 (1) ◽  
Author(s):  
Karen Lee ◽  
Femke van Nassau ◽  
Anne Grunseit ◽  
Kathleen Conte ◽  
Andrew Milat ◽  
...  
Keyword(s):  
Case Studies ◽  
Good Governance ◽  
Scale Up ◽  
Target Population ◽  
Scaling Up ◽  
Structured Interview ◽  
Health Interventions ◽  
Decision Makers ◽  
Framework Analysis ◽  
Policy Makers

Abstract Background While known efficacious preventive health interventions exist, the current capacity to scale up these interventions is limited. In recent years, much attention has focussed on developing frameworks and methods for scale-up yet, in practice, the pathway for scale-up is seldom linear and may be highly dependent on contextual circumstances. Few studies have examined the process of scaling up from decision to implementation nor examined the sustainability of scaled-up interventions. This study explores decision-makers’ perceptions from real-world scaled-up case studies to examine how scale-up decisions were made and describe enablers of successful scale-up and sustainability. Methods This qualitative study included 29 interviews conducted with purposively sampled key Australian policy-makers, practitioners and researchers experienced in scale-up. Semi-structured interview questions obtained information regarding case studies of scaled-up interventions. The Framework Analysis method was used as the primary method of analysis of the interview data to inductively generate common and divergent themes within qualitative data across cases. Results A total of 31 case studies of public health interventions were described by interview respondents based on their experiences. According to the interviewees’ perceptions, decisions to scale up commonly occurred either opportunistically, when funding became available, or when a deliberate decision was made and funding allocated. The latter scenario was more common when the intervention aligned with specific political or strategic goals. Decisions to scale up were driven by a variety of key actors such as politicians, senior policy-makers and practitioners in the health system. Drivers of a successful scale-up process included good governance, clear leadership, and adequate resourcing and expertise. Establishing accountability structures and appropriate engagement mechanisms to encourage the uptake of interventions were also key enablers. Sustainability was influenced by evidence of impact as well as good acceptability among the general or target population. Conclusions Much like Kingdon’s Multiple Streams Theory of ‘policy windows’, there is a conceptually similar ‘window for scale-up’, driven by a complex interplay of factors such as political need, strategic context, funding and key actors. Researchers and policy-makers need to consider scalability from the outset and prepare for when the window for scale-up opens. Decision-makers need to provide longer term funding for scale-up to facilitate longer term sustainability and build on the resources already invested for the scale-up process.

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.

Economic guidelines for oncology products: Adaptation of the Canadian Agency for Drugs and Technologies in Health (CADTH) technology assessment guidance document

2009 ◽  
Vol 27 (15_suppl) ◽  
pp. e17572-e17572
Author(s):  
N. Mittmann ◽  
W. K. Evans ◽  
A. Rocchi ◽  
C. J. Longo ◽  
H. J. Au ◽  
...  
Keyword(s):  
Working Group ◽  
Progression Free Survival ◽  
Target Population ◽  
Decision Makers ◽  
Phase Iii ◽  
Economic Evaluations ◽  
Guidance Document ◽  
Health Technologies ◽  
Intermediate Outcomes

e17572 Background: Economic evaluations (EE) are routinely used by decision-makers in Canada. CADTH's “Guidelines for the Economic Evaluation of Health Technologies: Canada” Third edition, 2006, provide guidance on the conduct of EEs for all therapeutic products. The consistency and quality of oncology EEs are variable and therapeutics in the cancer care environment presented unique challenges in decision making. Several chapters of the CADTH document adequately defined methods for the conduct of an oncology EE. However, some chapters required more specific guidance to improve the quality of oncology EEs. The goal was to provide direction on methods for the conduct of high quality EEs in oncology. Methods: The Working Group on Economic Analysis, NCIC CTG and CADTH jointly initiated this project and formed a working group (WG) of oncologists, health economists, decision makers and economic analysts. The WG identified CADTH chapters where oncology-specific guidance would be required. In-person and teleconference meetings provided content and structure for the document. Formal reviews by external academic experts, cancer agencies, patient groups and the pharmaceutical industry were conducted. Feedback was reviewed by the WG and incorporated as appropriate. Results: Chapters requiring guidance included: target population, comparators, perspective, effectiveness, modeling, type of evaluation, valuing health, time horizon, costs and resources, sensitivity analysis and equity. Guidance included clarity around CADTH methodology and recommendations for oncology products. For example for the effectiveness chapter, there was guidance around the use of intermediate outcomes (progression free survival vs. overall survival) and type of evidence (phase II vs. phase III). Overall recommendations for chapters will be presented. Conclusions: The oncology adapted economic guidelines provide specific guidance on the conduct of EEs for oncology products and will be published as an addendum to CADTH's third edition document. Their use should lead to more consistent application of EE methodologies for anti-cancer drugs and higher quality information for decision-makers at a national and perhaps international level. No significant financial relationships to disclose.

Exploring consumer doubt towards local new products innovation and purchase intention

2016 ◽  
Vol 12 (1) ◽  
pp. 2-17 ◽  
Author(s):  
Mehdi Afzali ◽  
Elsadig Musa Ahmed
Keyword(s):  
Information Seeking ◽  
Purchase Intention ◽  
Target Population ◽  
Decision Makers ◽  
Aesthetic Design ◽  
Content Type ◽  
Two Factors ◽  
Innovative Products ◽  
Survey Results ◽  
The Relationship

Purpose – The purpose of this paper is to develop a scale to find the relationship between consumer doubt, skepticism, familiarity, information seeking, value for money and aesthetic design with customers’ purchase intention. Design/methodology/approach – This study focussed on students of Malaysian university of multimedia in Melaka campus and used questionnaires to obtain the relevant data. Convenience random sampling method is used whereby 200 questionnaires were distributed among the target population and exactly 200 completed answers were collected. Findings – The survey results show that aesthetic design and information seeking of a product has a positive significant relationship with customers’ purchase intention. Respondents reported a consideration on these two factors and it is revealed that the scale used in this study is reliable and valid kind of measurement to assess customers’ purchase intention. Practical implications – To minimize the innovating failure among launched new innovative products, managers and decision makers should focus on variables used in this study. By focussing on aesthetic design and information seeking they can overcome some of the problems cause failure. Originality/value – This research focus on customers’ purchase intention to buy a Malaysian-made innovative new product and their lack of confidence and trust if the product satisfy their needs. The scale in this study show that this research is valid and it gives new perception toward purchase intention and innovation.

scholarly journals The use of innovative payment mechanisms for gene therapies in Europe and the USA

2021 ◽  
Author(s):  
Jesper Jørgensen ◽  
Panos Kefalas
Keyword(s):  
Healthcare System ◽  
Therapeutic Potential ◽  
Decision Makers ◽  
Data Uncertainty ◽  
Supporting Evidence ◽  
Reimbursement Mechanisms ◽  
Gene Therapies ◽  
Payment Schemes ◽  
The Usa

Innovative reimbursement mechanisms have long been considered potential solutions to the data uncertainty associated with one-off, high-value gene therapies that have long-term therapeutic potential, combined with limited supporting evidence at launch. The launches of increasing numbers of such gene therapies in Europe and the USA in the past 5 years provide valuable exemplars of how innovative reimbursement mechanisms are used by healthcare system decision makers in practice. This review details the use of such reimbursement schemes for recently launched gene therapies in key European countries and the USA, and shows that they are more widespread in Europe than in the USA. Although innovative payment schemes are increasingly used across countries, differences in healthcare system structures (e.g., single- vs multi-payer systems) and willingness to pay mean that decision makers in different countries have different incentives to manage uncertainties around long-term, real-world product value.

Development of the Point-of-Care Key Evidence Tool (POCKET): a checklist for multi-dimensional evidence generation in point-of-care tests

2019 ◽  
Vol 57 (6) ◽  
pp. 845-855 ◽  
Author(s):  
Jeremy R. Huddy ◽  
Melody Ni ◽  
Shivani Misra ◽  
Stella Mavroveli ◽  
James Barlow ◽  
...  
Keyword(s):  
Lead Time ◽  
Point Of Care ◽  
Structured Interview ◽  
Interview Study ◽  
Decision Makers ◽  
Face Validity ◽  
Stakeholder Workshop ◽  
Point Of Care Tests ◽  
Evidence Generation ◽  
Expert Workshop

Abstract Background This study aimed to develop the Point-of-Care Key Evidence Tool (POCKET); a multi-dimensional checklist to guide the evaluation of point-of-care tests (POCTs) incorporating validity, utility, usability, cost-effectiveness and patient experience. The motivation for this was to improve the efficiency of evidence generation in POCTs and reduce the lead-time for the adoption of novel POCTs. Methods A mixed qualitative and quantitative approach was applied. Following a literature search, a three round Delphi process was undertaken incorporating a semi-structured interview study and two questionnaire rounds. Participants included clinicians, laboratory personnel, commissioners, regulators (including members of National Institute for Health and Care Excellence [NICE] committees), patients, industry representatives and methodologists. Qualitative data were analysed based on grounded theory. The final tool was revised at an expert stakeholder workshop. Results Forty-three participants were interviewed within the semi-structured interview study, 32 participated in the questionnaire rounds and nine stakeholders attended the expert workshop. The final version of the POCKET checklist contains 65 different evidence requirements grouped into seven themes. Face validity, content validity and usability has been demonstrated. There exists a shortfall in the evidence that industry and research methodologists believe should be generated regarding POCTs and what is actually required by policy and decision makers to promote implementation into current healthcare pathways. Conclusions This study has led to the development of POCKET, a checklist for evidence generation and synthesis in POCTs. This aims to guide industry and researchers to the evidence that is required by decision makers to facilitate POCT adoption so that the benefits they can bring to patients can be effectively realised.

Overseeing Innovative Therapy without Mistaking it for Research: A Function-Based Model Based on Old Truths, New Capacities, and Lessons from Stem Cells

2010 ◽  
Vol 38 (2) ◽  
pp. 286-302 ◽  
Author(s):  
Patrick L. Taylor
Keyword(s):  
Stem Cells ◽  
Clinical Research ◽  
Basic Science ◽  
Basic Research ◽  
Standard Of Care ◽  
The Body ◽  
Medical Interventions ◽  
Innovative Therapy ◽  
New Knowledge ◽  
Innovative Therapies

Innovative therapy is the name we give to novel medical interventions, radically different from the standard of care, provided in order to benefit a patient, rather than to acquire new knowledge. They are paradigmshifting, not incremental, responses to serious patient problems that standard medical care inadequately addresses. Innovative therapies are often devised by clinicians, not basic science researchers; they do not follow the linear model of basic research, to translation, to clinical research, to application. Instead, they come from thinking backwards from the patient’s circumstances, and forward from deep knowledge of how the body functions, to challenge the limits of current mechanisms for effecting cures.

scholarly journals Generic Feasibility Assessment: Helping to Choose the Nuclear Piece of the Net Zero Jigsaw

Energies ◽  
2021 ◽  
Vol 14 (5) ◽  
pp. 1229
Author(s):  
William Bodel ◽  
Kevin Hesketh ◽  
Grace McGlynn ◽  
Juan Matthews ◽  
Gregg Butler
Keyword(s):  
Carbon Dioxide Emissions ◽  
Energy Use ◽  
Climate Change Policy ◽  
Electrical Energy ◽  
Decision Makers ◽  
Low Carbon ◽  
Feasibility Assessment ◽  
Change Policy ◽  
Net Zero ◽  
The Uk

The United Kingdom has declared a climate change policy of 100% reduction in carbon dioxide emissions by 2050. Efforts thus far have been limited solely to electricity generation methods. While progress has been admirable, effort now must be directed at the nation’s non-electrical energy use. Nuclear energy is an essential part of any energy future, since it is low-carbon, firm and supplies synchronous electricity; however the nation’s nuclear strategy to date has been erratic, costly and lacking in strategic oversight. A multitude of reactor designs are on offer for potential uptake, and decision-makers must have clarity of vision on what these systems must deliver before forming a strategy. Choosing between these systems, given the uncharted energy future faced by the UK is a daunting prospect. Generic feasibility assessment offers a tool for decision-makers to assist them in selecting the most suitable nuclear system for chosen future conditions. Generic feasibility assessment offers an alternative to traditional multi-attribute decision analyses, which can be confusing to even committed stakeholders when large numbers of attributes are weighted and compiled. Generic feasibility assessment forms part of a toolkit which will be of utility in achieving net zero by 2050, given the short time that remains.

scholarly journals Exploring the opportunities for alignment of regulatory postauthorization requirements and data required for performance-based managed entry agreements

2021 ◽  
Vol 37 (1) ◽  
Author(s):  
Hans-Georg Eichler ◽  
Roisin Adams ◽  
Einar Andreassen ◽  
Peter Arlett ◽  
Marc van de Casteele ◽  
...  
Keyword(s):  
Data Quality ◽  
Data Sharing ◽  
Patient Population ◽  
Time Horizon ◽  
Public Information ◽  
European Medicines Agency ◽  
Data Generation ◽  
Patient Access ◽  
Managed Entry Agreements ◽  
Pricing And Reimbursement

Abstract Performance-based managed entry agreements (PB-MEAs) might allow patient access to new medicines, but practical hurdles make competent authorities for pricing and reimbursement (CAPR) reluctant to implement PB-MEAs. We explored if the feasibility of PB-MEAs might improve by better aligning regulatory postauthorization requirements with the data generation of PB-MEAs and by active collaboration and data sharing. Reviewers from seven CAPRs provided structured assessments of the information available at the European Medicines Agency (EMA) Web site on regulatory postauthorization requirements for fifteen recently authorized products. The reviewers judged to what extent regulatory postauthorization studies could help implement PB-MEAs by addressing uncertainty gaps. Study domains assessed were: patient population, intervention, comparators, outcomes, time horizon, anticipated data quality, and anticipated robustness of analysis. Reviewers shared general comments about PB-MEAs for each product and on cooperation with other CAPRs. Reviewers rated regulatory postauthorization requirements at least partly helpful for most products and across domains except the comparator domain. One quarter of responses indicated that public information provided by the EMA was insufficient to support the implementation of PB-MEAs. Few PB-MEAs were in place for these products, but the potential for implementation of PB-MEAs or collaboration across CAPRs was seen as more favorable. Responses helped delineate a set of conditions where PB-MEAs may help reduce uncertainty. In conclusion, PB-MEAs are not a preferred option for CAPRs, but we identified conditions where PB-MEAs might be worth considering. The complexities of implementing PB-MEAs remain a hurdle, but collaboration across silos and more transparency on postauthorization studies could help overcome some barriers.

Sign in / Sign up

Export Citation Format

Share Document