TITLE

Post-marketing access to orphan drugs: a critical analysis of health technology assessment and reimbursement decision-making considerations

AUTHOR(S)
Iskrov, Georgi; Stefanov, Rumen
PUB. DATE
January 2014
SOURCE
Orphan Drugs: Research & Reviews;2014, Vol. 4, p1
SOURCE TYPE
Academic Journal
DOC. TYPE
Article
ABSTRACT
This study aims to explore the current rationale of post-marketing access to orphan drugs. As access to orphan medicinal products depends on assessment and appraisal by health authorities, this article is focused on health technology assessment (HTA) and reimbursement decision-making considerations for orphan drugs. A critical analysis may identify important factors that could predetermine the combined outcomes of these two processes. Following this objective, an analytical framework was developed, comprising three overlaying issues: to outline what is currently done and what needs to be done in the field of HTA of orphan drugs, to synthesize important variables relevant to the reimbursement decision-making about orphan drugs, and to unveil relationships between theory and practice. Methods for economic evaluation, cost-effectiveness threshold, budget impact, uncertainty of evidence, criteria in reimbursement decision-making, and HTA research agenda are all explored and discussed from an orphan drug perspective. Reimbursement decision-making for orphan drugs is a debate of policy priorities, health system specifics, and societal attitudes. Health authorities need to pursue a multidisciplinary analysis on a range of criteria, ensuring an explicit understanding of the trade-offs for decisions related to eligibility for reimbursement. The only reasonable way to accept a higher valuation of orphan drug benefits is if these are demonstrated empirically. Rarity means that the quality of orphan drug evidence is not the same as for conventional therapies. Closing this gap is another crucial point for the timely access to these products. The generation of evidence goes far beyond pre-market authorization trials and requires transnational cooperation and coordination. Early constructive dialogue among orphan drug stakeholders and elaboration of orphan drug-tailored methodology tools could set the scene for ongoing accumulation of evidence, as well as for proper and timely assessment and appraisal.
ACCESSION #
94752412

 

Related Articles

  • Statistics in clinical research: Important considerations. Barkan, Howard // Annals of Cardiac Anaesthesia;Jan-Mar2015, Vol. 18 Issue 1, p74 

    Statistical analysis is one of the foundations of evidence-based clinical practice, a key in conducting new clinical research and in evaluating and applying prior research. In this paper, we review the choice of statistical procedures, analyses of the associations among variables and techniques...

  • Evidence based practice; keep it simple stupid. Dannaway, Jasan; Maddren, Casey; Mendis, Kumara // Australian Medical Student Journal;2013, Vol. 4 Issue 2, p83 

    Learning and implementing evidence based practice is an expected component of good medical practice. Synthesising evidence in an effective and timely manner is a skill that is growing in importance and relevance. Evidence based practice is widely included in medical school curricula, and...

  • Towards evidence based medicine for paediatricians. Phillips, Bob // Archives of Disease in Childhood -- Education & Practice Edition;9/1/2014, Vol. 99 Issue 9, p878 

    The article shares insight on the steps pediatricians must take in the transition towards evidence-based medicine. Issues discussed are the challenge to the meta-analysis of quantitative studies of therapies that used randomized controlled trial designs such as the use of procalcitonin to guide...

  • Patient-centred decision-making? Biocitizens between evidence-based medicine and self-determination. Winther Jørgensen, Marianne // Evidence & Policy: A Journal of Research, Debate & Practice;Aug2015, Vol. 11 Issue 3, p311 

    Nikolas Rose's work on contemporary biopower centres the 'biocitizen' as a subject governed through freedom and made individually responsible in matters of health. This article argues that a focus on its conditions of possibility will enable a better contextualisation of the biocitizen as merely...

  • Implementing research results in clinical practice- the experiences of healthcare professionals. Kristensen, Nanna; Nymann, Camilla; Konradsen, Hanne // BMC Health Services Research;2/10/2016, Vol. 16, p1 

    Background: In healthcare research, results diffuse only slowly into clinical practice, and there is a need to bridge the gap between research and practice. This study elucidates how healthcare professionals in a hospital setting experience working with the implementation of...

  • KEY OPERATIONAL DRIVERS IN THE MEDICAL TOURISM INDUSTRY. Ferrer, Mario; Medhekar, Anita // International Journal of Accounting Information Science & Leader;Spring2012, Vol. 5 Issue 12, p62 

    High health and insurance cost, long waiting list in developed countries, absence of medical procedures and technology in developing poor countries, in addition low travel costs, state of the art medical technology, good affordable medical facilities, low surgical cost, no waiting period and...

  • The Necessity for Clinical Reasoning in the Era of Evidence-Based Medicine. Sniderman, Allan D.; LaChapelle, Kevin J.; Rachon, Nikodem A.; Furberg, Curt D. // Mayo Clinic Proceedings;Oct2013, Vol. 88 Issue 10, p1108 

    Clinical decisions are increasingly driven by evidence-based recommendations of guideline groups, which aim to be based on the highest quality knowledged randomized clinical trials (RCTs) and meta-analyses. Although RCTs provide the best assessment of the overall value of a therapy, high-quality...

  • Evidence-based clinical management and utilization of new technology in European neurosurgery. Weber, Clemens; Jakola, Asgeir; Gulati, Sasha; Nygaard, Øystein; Solheim, Ole // Acta Neurochirurgica;Apr2013, Vol. 155 Issue 4, p747 

    Background: Evidence-based medicine (EBM) has become one of the pillars of modern patient care. However, neurosurgery has always been an experience-based and technology-driven discipline, and it remains unknown to which extent European neurosurgeons follow high-level evidence-based...

  • Value-based medicine: concepts and application. Jong-Myon Bae // Epidemiology & Health;2015, Vol. 37, p1 

    Global healthcare in the 21st century is characterized by evidence-based medicine (EBM), patient-centered care, and cost effectiveness. EBM involves clinical decisions being made by integrating patient preference with medical treatment evidence and physician experiences. The Center for...

Share

Read the Article

Courtesy of THE LIBRARY OF VIRGINIA

Sorry, but this item is not currently available from your library.

Try another library?
Sign out of this library

Other Topics