scholarly journals Is orphan drug pricing blowing a bubble? The unique situation of orphan drugs and why high prices will likely persist

2013 ◽  
Vol 1 (9) ◽  
pp. 675-679 ◽  
Author(s):  
John-Paul Michaud ◽  
Robin Modi ◽  
M Ian Phillips
Keyword(s):  
Orphan Drug ◽  
Orphan Drugs ◽  
Drug Pricing

OP65 Pharmacoeconomic Evaluation Of Orphan Drugs: Impact Of Extra Criteria?

2019 ◽  
Vol 35 (S1) ◽  
pp. 16-16
Author(s):  
Orla Maguire ◽  
Laura McCullagh ◽  
Cara Usher ◽  
Michael Barry
Keyword(s):  
Cost Effectiveness ◽  
Orphan Drug ◽  
Societal Perspective ◽  
Evaluation Process ◽  
Orphan Drugs ◽  
Pharmacoeconomic Evaluation ◽  
Qaly Gain ◽  
Minor Effect ◽  
Orphan Medicinal Products ◽  
A Minor

IntroductionThere is ongoing debate as to whether conventional pharmacoeconomic evaluation (PE) methods are appropriate for orphan medicinal products (OMPs). The National Centre for Pharmacoeconomics (NCPE) in Ireland has a well-defined process for conducting pharmacoeconomic evaluations of pharmaceuticals, which is the same for OMPs and non-OMPs. The objective of this study was to identify whether supplementary criteria considered in the pharmacoeconomic evaluation of OMPs would affect final reimbursement recommendations.MethodsA literature search was conducted to identify criteria. Orphan drug pharmacoeconomic evaluations completed by the NCPE between January 2015 and December 2017 were identified and supplementary criteria, where feasible, were applied.ResultsFourteen pharmacoeconomic evaluations were included in the study. Three criteria that could feasibly be applied to the NCPE evaluation process were identified, all three of which essentially broadened the economic perspective of the pharmacoeconomic evaluation. Higher cost-effectiveness threshold: Despite being arbitrarily raised from EUR 45,000/QALY to EUR 100,000/QALY, only one orphan drug demonstrated cost-effectiveness at this higher threshold. Weighted QALY gain: here, a weighted gain of between one and three is applied to drugs demonstrating QALY gains between 10 and 30, respectively. No OMPs included in the study showed a QALY gain of more than 10. Thirteen demonstrated QALY gains less than 10 and one could not be evaluated. Societal perspective: six submissions incorporated societal perspective as a scenario analysis. Despite incremental cost-effectiveness ratios (ICERs) being reduced between 4 percent and 58 percent, only two OMPs demonstrated cost-effectiveness at the higher threshold (EUR 100,000/QALY).ConclusionsApplication of supplementary criteria to the pharmacoeconomic evaluation of OMPs had a minor effect on three products assessed. However, for the majority, the final cost-effectiveness outcomes remained the same. The study highlights that other criteria are being considered in the decision to reimburse.

Orphan Drugs: The Question of Products Liability

1985 ◽  
Vol 10 (4) ◽  
pp. 491-513
Author(s):  
Susan F. Scharf
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Rare Diseases ◽  
Tort Law ◽  
Orphan Drugs ◽  
Products Liability ◽  
Liability Law ◽  
Design Defect ◽  
Liability Costs ◽  
Orphan Drug Development

AbstractOrphan drugs, essential for die treatment of persons widi rare diseases, generally are unprofitable for manufacturers to develop and market. While congressional and administrative efforts to promote die development of orphan drugs have met widi modest success, application of products liability doctrine to orphan drug sponsors could subvert those efforts. This Note describes die provisions of die Orphan Drug Act and analyzes products liability law with respect to orphan drug litigation. It argues that die goals of tort law support the imposition of liability for design defect, failure to warn and negligence in testing. Finally, die Note acknowledges diat liability costs create disincentives for orphan drug development and suggests mechanisms for reducing manufacturers’ liability concerns.

scholarly journals Shining a light in the black box of orphan drug pricing

2014 ◽  
Vol 9 (1) ◽  
pp. 62 ◽  
Author(s):  
Eline Picavet ◽  
Thomas Morel ◽  
David Cassiman ◽  
Steven Simoens
Keyword(s):  
Orphan Drug ◽  
Black Box ◽  
Drug Pricing

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU

2012 ◽  
Vol 69 (4) ◽  
pp. 1009-1024 ◽  
Author(s):  
Roberta Joppi ◽  
Vittorio Bertele’ ◽  
Silvio Garattini
Keyword(s):  
Orphan Drug ◽  
Orphan Drugs ◽  
Orphan Diseases ◽  
Drug Legislation ◽  
The Eu

scholarly journals The drug development pipeline for glioblastoma - a cross sectional assessment of the FDA Orphan Drug Product designation database

2021 ◽  
Author(s):  
Pascal Johann ◽  
Dominic Lenz ◽  
Markus Ries
Keyword(s):  
Drug Development ◽  
Orphan Drug ◽  
Mechanism Of Action ◽  
Drug Product ◽  
Treatment Options ◽  
Orphan Drugs ◽  
Cross Sectional ◽  
Therapeutic Mechanism ◽  
Development Pipeline ◽  
Insight Into

Abstract Background: Glioblastoma multiforme (GBM) is the most common malignant brain tumor among adult patients and represents an almost universally fatal disease. Novel therapies for GBM are being developed under the orphan drug legislation and the knowledge on the molecular makeup of this disease has been increasing rapidly. However, the clinical outcomes in GBM patients with currently available therapies are still dismal. An insight into the current drug development pipeline for GBM is therefore of particular interest. Objectives: To provide a quantitative clinical-regulatory insight into the status of FDA orphan drug designations for compounds intended to treat GBM. Methods: Quantitative cross-sectional analysis of the U.S. Food and Drug Administration Orphan Drug Product database between 1983 and 2020. STROBE criteria were respected. Results: Four orphan drugs out of 161 (2,4%) orphan drug designations were approved for the treatment for GBM by the FDA between 1983 and 2020. Fourteen orphan drug designations were subsequently withdrawn for unknown reasons. The number of orphan drug designations per year shows a growing trend. In the last decade, the therapeutic mechanism of action of designated compounds intended to treat glioblastoma shifted from cytotoxic drugs (median year of designation 2008) to immunotherapeutic approaches and small molecules (median year of designation 2014 and 2015 respectively) suggesting an increased focus on precision in the therapeutic mechanism of action for compounds the development pipeline. Conclusion: Despite the fact that current pharmacological treatment options in GBM are sparse, the drug development pipeline is steadily growing. In particular, the surge of designated immunotherapies detected in the last years raises the hope that elaborate combination possibilities between classical therapeutic backbones (radiotherapy) and novel, currently experimental therapeutics may help to provide better therapies for this deadly disease in the future.

scholarly journals Billion Dollar Orphans

2019 ◽  
Vol 6 (3) ◽  
pp. 731-768
Author(s):  
John Sheridan
Keyword(s):  
Orphan Drug ◽  
Orphan Drugs ◽  
Federal Courts ◽  
Pharmaceutical Companies ◽  
Orphan Diseases ◽  
Social Purpose

This Comment examines the extent to which Congress empowered the FDA to address the increase in petitions and the general accessibility of orphan drug remedies. Specifically, this Comment seeks to understand why the FDA’s interpretation of the purpose of the ODA seems to conflict with the statutory intent as interpreted by federal courts. This Comment considers a statute’s ultimate goal or social purpose to be the purpose of the statute, whereas the express mechanisms by which Congress seeks to bring about these goals is best understood as the statute’s intent. To understand the FDA and judiciary’s differing interpretations of the ODA, this Comment analyzes the language of the Statute, recent ODA litigation, FDA’s promulgated regulations, as well as recent response to pharmaceutical companies’ increase in designation requests for orphan drugs. Ultimately, this Comment strives to determine whether or not the ODA can effectively achieve the goals Congress set forth in 1983.This Comment conducts a statutory analysis of the ODA and closely examines how courts, the FDA, and litigant pharmaceutical companies interpret the Statute differently. This Comment argues that Congress’s intent in passing the ODA was to create lucrative incentives for the development of drugs for orphan diseases. But, Congress’s purpose in drafting the ODA was to ensure the drugs became available to patients. The incentives serve as a tool to achieve the purpose of the ODA: to treat patients suffering from rare diseases.This Comment concludes that to better effectuate this purpose, Congress must amend the ODA or pass other legislation empowering the FDA to promulgate regulations that alter the schedule and administration of the ODA’s lucrative “basket of goodies.”

scholarly journals ORPHAN DRUG POLICY

2021 ◽  
pp. 1-2
Author(s):  
ROSHANI GOEL
Keyword(s):  
Orphan Drug ◽  
Secondary Data ◽  
Orphan Drugs ◽  
World Market ◽  
Pharmaceutical Products ◽  
Pharmaceutical Companies ◽  
Infrastructure Development ◽  
Wide Range ◽  
Drug Policies ◽  
Government Data

Pharmaceutical industry is crucial for mankind and contributes in some way in socio economic development of the society through jobs, community welfare, and supply chains. Indian pharmaceutical companies are one of the largest markets in the world, estimated 10% of global production and 2% of world market. The industry is has shown significant growth in infrastructure development, producing wide range of pharmaceutical products with new technical advancements. The country is famous for producing and providing pharmaceutical products at much cheaper prices than the US and EU. However, the country lacks in investment in research on rare disease or orphan drugs. This paper tries to highlight the ways in which the market in orphan drugs can grow in India with the help of international partners. Objective: The objective of the study was to look at existing orphan drug policies and how we can aim at making it more equitable in India. Methods: Research is completely based on secondary data from online journals and government data. The study is analytical in its approach and is descriptive in nature. Results: Pharmaceutical companies invest less in Orphan drugs as they do not mark an assured profit with present investment. Conclusions: To maintain the interest of Indian pharmaceutical companies it is suggested that, Indian companies should work in collaboration with countries which lead in orphan drug markets.

scholarly journals Excluding Orphan Drugs from the 340B Drug Discount Program: the Impact on 18 Critical Access Hospitals

2012 ◽  
Vol 3 (1) ◽  
Author(s):  
Madeline Carpinelli Wallack ◽  
Todd Sorensen
Keyword(s):  
Federal Government ◽  
Orphan Drug ◽  
Orphan Drugs ◽  
Safety Net ◽  
Original Research ◽  
Critical Access Hospitals ◽  
Patient Protection ◽  
High Prevalence ◽  
Safety Net Providers ◽  
The Impact

Purpose: The 340B Drug Pricing Program is a federal program designed to reduce the amount that safety net providers spend on outpatient drugs. The Patient Protection and Affordable Health Care Act of 2010 extended eligibility for 340B to critical access hospitals (CAHs) for all drugs except those designated as "orphan." Because this policy is unprecedented, this study quantifies the gross financial impact that this exemption has on a group of CAHs. Methods: Drug spending for 2010 from 18 CAHs in Minnesota and Wisconsin are reviewed to identify the prevalence of orphan drug purchases and to calculate the price differentials between the 340B price and the hospitals' current cost. Results: The 18 CAHs' purchases of orphan drugs comprise an average of 44% of the total annual drug budgets, but only 5% of units purchased, thus representing a very high proportion of their expenditures. In the aggregate, the 18 hospitals would have saved $3.1 million ($171,000 average per hospital) had purchases of drugs with orphan designations been made at the 340B price. Because CAH claims for Medicare are reimbursed on a cost-basis, the Federal government is losing an opportunity for savings. Conclusion: The high prevalence of orphan drug use and considerable potential for cost reduction through the 340B program demonstrate the loss of benefit to the hospitals, Federal government and the states.   Type: Original Research

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