Cost-effectiveness of tafamidis therapy for transthyretin amyloid cardiomyopathy
DS Kazi, BK Bellows, SJ Baron, C Shen, DJ Cohen… - Circulation, 2020 - Am Heart Assoc
Background: In patients with transthyretin amyloid cardiomyopathy, tafamidis reduces all-
cause mortality and cardiovascular hospitalizations and slows decline in quality of life …
cause mortality and cardiovascular hospitalizations and slows decline in quality of life …
FDA approval, clinical trial evidence, efficacy, epidemiology, and price for non-orphan and ultra-rare, rare, and common orphan cancer drug indications: cross …
T Michaeli, H Jürges, DT Michaeli - bmj, 2023 - bmj.com
Objective To analyze the US Food and Drug Administration (FDA) approval, trials, unmet
needs, benefit, and pricing of ultra-rare (< 6600 affected US citizens), rare (6600-200 000 …
needs, benefit, and pricing of ultra-rare (< 6600 affected US citizens), rare (6600-200 000 …
An international review of health technology assessment approaches to prescription drugs and their ethical principles
LZ Rand, AS Kesselheim - The Journal of Law, Medicine & …, 2020 - journals.sagepub.com
In many countries, health technology assessment (HTA) organizations determine the
economic value of new drugs and make recommendations regarding appropriate pricing …
economic value of new drugs and make recommendations regarding appropriate pricing …
Cost effectiveness of caplacizumab in acquired thrombotic thrombocytopenic purpura
Acquired thrombotic thrombocytopenic purpura (TTP) is a life-threatening disease
characterized by thrombotic microangiopathy leading to end-organ damage. The standard of …
characterized by thrombotic microangiopathy leading to end-organ damage. The standard of …
New business models for pharmaceutical research and development as a global public good: considerations for the WHO European Region
Public concern has been increasing about the high prices of novel medicines, limits on
availability and access, and the strain on health-care budgets across all countries …
availability and access, and the strain on health-care budgets across all countries …
Spending For Orphan Indications Among Top-Selling Orphan Drugs Approved To Treat Common Diseases: Study estimates the proportion of spending in the US on …
The Orphan Drug Act of 1983 provides benefits to promote the development of treatments for
rare diseases that have limited sales potential. Policy makers have questioned whether this …
rare diseases that have limited sales potential. Policy makers have questioned whether this …
1696MO Partial orphan cancer drugs: FDA approval, clinical benefit, trials, epidemiology, price, beneficiaries, and spending
DT Michaeli, CT Michaeli - Annals of Oncology, 2023 - annalsofoncology.org
Background The Orphan Drug Act (ODA) incentivizes drug development for rare diseases
with limited sales potential. Yet, orphan drugs frequently turn into multi-billion-dollar …
with limited sales potential. Yet, orphan drugs frequently turn into multi-billion-dollar …
Estimating the Size of the US Market for New Antibiotics with Activity against Carbapenem-Resistant Enterobacteriaceae
CJ Clancy, MH Nguyen - Antimicrobial Agents and chemotherapy, 2019 - Am Soc Microbiol
New antibiotics with activity against carbapenem-resistant Enterobacteriaceae (CRE)
improve outcomes of CRE-infected patients. However, companies developing these drugs …
improve outcomes of CRE-infected patients. However, companies developing these drugs …
Five-year sales for newly marketed prescription drugs with and without initial orphan drug act designation
Methods| We identified newly marketed brand-name drugs using SSR Health, which
contains data on more than 1200 brand-name drugs made by public companies. We …
contains data on more than 1200 brand-name drugs made by public companies. We …
Cost of exempting sole orphan drugs from Medicare negotiation
Importance The Inflation Reduction Act (IRA) requires Medicare to negotiate prices for some
high-spending drugs but exempts drugs approved solely for the treatment of a single rare …
high-spending drugs but exempts drugs approved solely for the treatment of a single rare …