What Is the Value of Innovative Pharmaceutical Therapies in Oncology and Hematology? A Willingness-to-Pay Study in Bulgaria

ConclusionsWe found a high willingness-to-pay for innovative drugs in oncology and hematology. The wide range of responses observed, however, indirectly implies a lack of consensus on the use of explicit ICER thresholds in Bulgaria.
Source: Value in Health Regional Issues - Category: International Medicine & Public Health Source Type: research

Related Links:

Josep Piulats Uveal melanoma is considered a rare disease but it is the most common intraocular malignancy in adults. Local treatments are effective, but the systemic recurrence rate is unacceptably high. Moreover, once metastasis have developed the prognosis is poor, with a 5-year survival rate of less than 5%, and systemic therapies, including immunotherapy, have rendered poor results. The tumour biology is complex, but angiogenesis is a highly important pathway in these tumours. Vasculogenic mimicry, the ability of melanomas to generate vascular channels independently of endothelial cells, could play an important ro...
Source: Cancers - Category: Cancer & Oncology Authors: Tags: Review Source Type: research
Publication date: Available online 15 June 2019Source: Multiple Sclerosis and Related DisordersAuthor(s): Miljenka-Jelena Jurašić, Vanja Bašić Kes, Iris ZavoreoAbstractA common disease like multiple sclerosis (MS) usually dominates the arena of demyelinating disorders; however, when a red flag such as a first relative family history of neurologic disease other than MS is present, the diagnostics may be more challenging.Recently, we have come across an intriguing clinical and diagnostic dilemma requiring extensive literature search and finally, decision making. Namely, initial presentation in our patient was...
Source: Multiple Sclerosis and Related Disorders - Category: Neurology Source Type: research
The Food and Drug Administration Office of Orphan Products Development is launching web-based educational resources for patients and industry on rare disease topics. The page debuts on International Rare Disease Day Feb. 28; topics include how to interact with FDA.
Source: FDA Consumer Health Information Updates - Category: Consumer Health News Authors: Source Type: news
ConclusionsPersistent oral N-acetylcysteine may be an alternative treatment option for secondary PAP.
Source: Respiratory Medicine Case Reports - Category: Respiratory Medicine Source Type: research
A Chicago biotech claims in a new lawsuit that Cambridge venture capital firm Third Rock Ventures and bluebird bio CEO Nick Leschly worked to hinder its drug development efforts so it could more easily obtain a key part of its intellectual property. Errant Gene Therapeutics is suing Third Rock Ventures and Leschly three years after it sued New York-based Sloan Kettering Memorial Cancer Center over a stalled rare disease trial. In the lawsuit, filed on Friday in Suffolk County Superior Court, Errant…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news
Rare diseases specialist Swedish Orphan Biovitrum (Sobi) has agreed to buy the drug emapalumab and is reorganizing to increase focus on late-stage development in hematology and immunology.Reuters Health Information
Source: Medscape Allergy Headlines - Category: Allergy & Immunology Tags: Family Medicine/Primary Care News Source Type: news
CAMBRIDGE, Mass., June 14, 2019 /PRNewswire/ -- Blueprint Medicines Corporation (NASDAQ: BPMC), a precision therapy company focused on genomically defined cancers, rare diseases and cancer immunotherapy, today announced it has submitted a New Drug...
Source: Drugs.com - New Drug Applications - Category: Drugs & Pharmacology Source Type: news
A Chicago biotech claims in a new lawsuit that Cambridge venture capital firm Third Rock Ventures and bluebird bio CEO Nick Leschly worked to hinder its drug development efforts so it could more easily obtain a key part of its intellectual property. Errant Gene Therapeutics is suing Third Rock Ventures and Leschly three years after it sued New York-based Sloan Kettering Memorial Cancer Center over a stalled rare disease trial. In the lawsuit, filed on Friday in Suffolk County Superior Court, Errant…
Source: bizjournals.com Health Care:Physician Practices headlines - Category: American Health Authors: Source Type: news
Floating-Harbor syndrome (FHS) is a rare syndromic short stature disorder caused by truncating variants in SRCAP. Few Chinese FHS patients had been reported so far and limited knowledge regarding the benefit of g...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
Mucopolysaccharidosis IVA (MPS IVA) is an autosomal recessive lysosomal storage disease due to N-acetylgalactosamine-6-sulfatase (GALNS) deficiency. It results in accumulation of the glycosaminoglycans, kerata...
Source: Orphanet Journal of Rare Diseases - Category: Internal Medicine Authors: Tags: Research Source Type: research
More News: Bulgaria Health | Cancer & Oncology | Children | Hematology | International Medicine & Public Health | Rare Diseases | Study