NDLI: Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement

Please wait, while we are loading the content...

Volume: 4 Issue: 1

Feasibility and attractiveness of indication value-based pricing in key EU countries

What makes a compliant Phase III and pre-launch patient advocacy strategy?

The quality of life of Spanish patients with Huntington's disease measured with H-QoL-I and EQ-5D

Perceptions and factors affecting pharmaceutical market access: results from a literature review and survey of stakeholders in different settings

A price comparison of recently launched proprietary pharmaceuticals in the UK and the US

Risk of discontinuation of Advanced Therapy Medicinal Products clinical trials

Cost-effectiveness analysis of the direct and indirect impact of intranasal live attenuated influenza vaccination strategies in children: alternative country profiles

Cost-effectiveness of ocriplasmin for the treatment of vitreomacular traction and macular hole

Validation of disease states in schizophrenia: comparison of cluster analysis between US and European populations

Advanced therapy medicinal products: current and future perspectives

The value of innovation under value-based pricing

Drug pricing reform in China: analysis of piloted approaches and potential impact of the reform

Demonstrating the value of medicines: evolution of value equation and stakeholder perception of uncertainties

A training paradigm to enhance performance and safe use of an innovative neuroendovascular device

Challenges in striving to simultaneously achieve multiple resource allocation goals: the pan-Canadian Oncology Drug Review (pCODR) example

Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement

Determining the value of medical technologies to treat ultra-rare disorders: a consensus statement

Content Provider	Taylor & Francis Online
Author	Nord, Erik Richardson, Jeff Schlander, Michael Tolley, Keith Postma, Maarten Persson, Ulf Garattini, Silvio Solà-Morales, Oriol De Kolominsky-Rabas, Peter Simoens, Steven Toumi, Mondher
Abstract	BackgroundIn most jurisdictions, policies have been adopted to encourage the development of treatments for rare or orphan diseases. While successful as assessed against their primary objective, these policies have prompted concerns among payers about the economic burden that might be caused by an annual cost per patient in some cases exceeding 100,000 Euro. At the same time, many drugs for rare disorders do not meet conventional standards for cost-effectiveness or ‘value for money’. Owing to the fixed (volume-independent) cost of research and development, this issue is becoming increasingly serious with decreasing prevalence of a given disorder.MethodsIn order to critically appraise the problems posed by the systematic valuation of interventions for ultra-rare disorders (URDs), an international group of clinical and health economic experts was convened in conjunction with the Annual European ISPOR Congress in Berlin, Germany, in November 2012. Following this meeting and during subsequent deliberations, the group achieved a consensus on the specific challenges and potential ways forward.ResultsThe group concluded that the complexities of research and development for new treatments for URDs may require conditional approval and reimbursement policies, such as managed entry schemes and coverage with evidence development agreements, but should not use as justification surrogate end point improvement only. As a prerequisite for value assessment, the demonstration of a minimum significant clinical benefit should be expected within a reasonable time frame. As to the health economic evaluation of interventions for URDs, the currently prevailing logic of cost-effectiveness (using benchmarks for the maximum allowable incremental cost per quality-adjusted life year gained) was considered deficient as it does not capture well-established social preferences regarding health care resource allocation.ConclusionModified approaches or alternative paradigms to establish the ‘value for money’ conferred by interventions for URDs should be developed with high priority.
File Format	PDF HTM / HTML
ISSN	20016689
DOI	10.3402/jmahp.v4.33039
Journal	Journal of Market Access & Health Policy
Volume Number	4
Issue Number	1
Language	English
Publisher	Routledge
Publisher Date	2016-10-27
Access Restriction	Open
Subject Keyword	Orphan drugs Health technology assessment Economic evaluation Cost-effectiveness Social cost value analysis Multicriteria decision making
Content Type	Text
Resource Type	Article
Subject	Health Informatics Health Policy

Central Library (ISO-9001:2015 Certified)
Indian Institute of Technology Kharagpur
Kharagpur, West Bengal, India | PIN - 721302

See location in the Map
03222 282435
Mail: support@ndl.gov.in

Sl.	Authority	Responsibilities	Communication Details
1	Ministry of Education (GoI), Department of Higher Education	Sanctioning Authority	https://www.education.gov.in/ict-initiatives
2	Indian Institute of Technology Kharagpur	Host Institute of the Project: The host institute of the project is responsible for providing infrastructure support and hosting the project	https://www.iitkgp.ac.in
3	National Digital Library of India Office, Indian Institute of Technology Kharagpur	The administrative and infrastructural headquarters of the project	Dr. B. Sutradhar bsutra@ndl.gov.in
4	Project PI / Joint PI	Principal Investigator and Joint Principal Investigators of the project	Dr. B. Sutradhar bsutra@ndl.gov.in Prof. Saswat Chakrabarti will be added soon
5	Website/Portal (Helpdesk)	Queries regarding NDLI and its services	support@ndl.gov.in
6	Contents and Copyright Issues	Queries related to content curation and copyright issues	content@ndl.gov.in
7	National Digital Library of India Club (NDLI Club)	Queries related to NDLI Club formation, support, user awareness program, seminar/symposium, collaboration, social media, promotion, and outreach	clubsupport@ndl.gov.in
8	Digital Preservation Centre (DPC)	Assistance with digitizing and archiving copyright-free printed books	dpc@ndl.gov.in
9	IDR Setup or Support	Queries related to establishment and support of Institutional Digital Repository (IDR) and IDR workshops	idr@ndl.gov.in