Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers

Eichler, Hans‐Georg; Bloechl-Daum, Brigitte; Abadie, Eric; Barnett, David; König, Franz; Pearson, Steven D.

doi:10.1038/nrd3079

Cited by 134 publications

(139 citation statements)

References 41 publications

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“…This paper will review the link between clinical trial data, the cost-effectiveness studies for new cancer drugs, coverage decisions informed by such studies, uptake and use of the drugs, and evidence of impact on outcome in the relevant patient population. The inclusion of impact on outcome in the analysis reflects the shift in focus from efficacy to relative effectiveness in assessing the value of new medicines [6]. Value for patients is created when anticancer drugs are chosen that provide maximum difference in the chance of benefit and harm, the overall objective for personalized cancer medicine [7].…”

Section: Introductionmentioning

confidence: 99%

Cost effectiveness in practice and its effect on clinical outcomes

Ramsey

Wilking

2014

Journal of Cancer Policy

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a b s t r a c tThe value of new cancer drugs is maximized when they are used for the right patient in the right way in clinical practice. Clinical trials conducted during drug development are the most important source of information to predict value at the time a drug is introduced in practice. Regulatory approval is an indication of value, which lately has been complemented with an assessment of clinical value for decisions about reimbursement, using the methodology of health technology assessment (HTA). Formal cost-effectiveness studies are an important part of this methodology, aiming to assist decisions about value for money in health care spending. The question is if the addition of a complementary HTA and cost-effectiveness study increases the value realized by the drug in practice compared to how it would be used without these assessments.We review the issues involved in providing an answer by using the introduction of new targeted therapies for metastatic renal cell cancer (mRCC). Specifically, we examine the link between clinical trial data and estimations of cost-effectiveness at drug launch, reimbursement decisions, uptake and use in different countries and evidence about impact on outcome in patient populations for which the new drugs are indicated.We conclude that there is a weak link between the assessments used at drug launch and the value created in clinical practice. We suggest measures that are necessary for the achievement of evidencebased and cost-effective cancer care in clinical practice.

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Section: Introductionmentioning

confidence: 99%

Cost effectiveness in practice and its effect on clinical outcomes

Ramsey

Wilking

2014

Journal of Cancer Policy

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“…Examples of these HTA innovations include proposals for early scientific dialogue (3), adaptive pathways (17), better use of real-world data (5), and encouraging more innovative product listing agreements (18). Innovators have also innovated, through improved evidence production for payers, organizational changes that better consider payer needs, and coalitions intended to standardize evidence collection and measurement (5).…”

Section: Hta At a Crossroadsmentioning

confidence: 99%

“…Beyond an increasing realization that patients need to be involved in early and ongoing scientific dialogue, in addition to assessments of a technology at any specific time, is work to develop and implement "fast track" regulatory approaches and calls for "adaptive licensing" (18). This has led to the recognition that all regulatory and HTA bodies need to work together, and with patients, if these approaches are to achieve their goals of giving patients faster and more appropriate access to promising new technologies (17).…”

Section: Re-thinking Scientific Dialogue and Stakeholder Engagement Tmentioning

confidence: 99%

Changing Health Technology Assessment Paradigms?

Husereau¹,

Henshall

Sampietro-Colom³

et al. 2016

Int J Technol Assess Health Care

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Objectives: Health technology assessment (HTA) has to innovate to best support changing health system environments and to help provide access to valuable innovation under fiscal constraint. Methods: Issues associated with changing HTA paradigms were identified through scoping and explored through deliberation at a meeting of industry and HTA leaders. Results: Five broad areas of change (engagement, scientific dialogue, research prioritization, adaptive approaches, and real world data) were identified. The meeting focused on two themes derived from these: re-thinking scientific dialogue and multi-stakeholder engagement, and re-thinking value, affordability, and access. Earlier and ongoing engagement to steer the innovation process and help achieve appropriate use across the technology lifecycle was perceived as important but would be resource intensive and would require priority setting. Patients need to be involved throughout, and particularly at the early stages. Further discussion is needed on the type of body best suited to convening the dialogue required. There was agreement that HTA must continue to assess value, but views differed on the role that HTA should play in assessing affordability and on appropriate responses to challenges around affordability. Enhanced horizon scanning could play an important role in preparing for significant future investments. Conclusions: Early and ongoing multi-stakeholder engagement and revisiting approaches to valuing innovation are required. Questions remain as to the most appropriate role for HTA bodies. Changing HTA paradigms extend HTA's traditional remit of being responsive to decision-makers demands to being more proactive and considering whole system value.Keywords: Health technology assessment, Patient participation, Stakeholder engagement, Financial management, Value-based purchasing Often, the purpose of health technology assessment (HTA) is to inform decisions about the coverage/reimbursement and use of new and existing health interventions, typically within health systems with limited resources to acquire and deliver them (1). HTA should be tailored to the types of decisions it is informing and reflect the needs and realities of the health and innovation systems within which it is operating. This is reflected in a growing recognition that it should take account of the lifecycle of health system innovation to be more impactful (2;3).In recent years, there have been fundamental changes in approaches to health system delivery, influenced by increasing public demand and patient influence, limited resources, new governance models, developments in information and communication technology, and new service delivery models seeking to improve quality and efficiency (4). Commercial innovators have similarly created new approaches to technology development, exploiting scientific advances, and working more closely with health system regulators and administrators to develop the evidence of safety and effectiveness that they seek (5).The Health Technology Assessment internatio...

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“…al., 2012, el asesoramiento conjunto entre las ARM e IETS (Wonder et. al., 2013), y el diseño y conducción de los ensayos clínicos comparativos (Eichler et. al., 2010).…”

Section: Ilustración 2 Tensiones En La Relación Entre Arm Iets Y Adcunclassified

La articulación en los sistemas de priorización

Cañón¹,

Vaca

Giedión³

et al. 2016

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Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers

Cited by 134 publications

References 41 publications

Cost effectiveness in practice and its effect on clinical outcomes

Cost effectiveness in practice and its effect on clinical outcomes

Changing Health Technology Assessment Paradigms?

La articulación en los sistemas de priorización

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