China 
Africa 
Explore chapters and articles related to this topic
The Neurologic Disorders in Film
Published in Eelco F. M. Wijdicks, Neurocinema—The Sequel, 2022
Genetic aberrations may lead to rapid disability and, in some, a major neurodegenerative disease. The promotion of drug development in rare diseases is at the heart of a worldwide collaboration, and many countries have introduced a combination of regulations and policies for orphan drugs in the last two decades. Over 500 rare conditions have been designated.152
Small-Molecule Targeted Therapies
Published in David E. Thurston, Ilona Pysz, Chemistry and Pharmacology of Anticancer Drugs, 2021
Glasdegib (DaurismoTM) (Figure 6.70), developed by Pfizer, is a benzimidazole analogue approved by the FDA in 2018 for use in combination with low-dose cytarabine for the treatment of newly diagnosed acute myeloid leukemia (AML) in patients aged ≥ 75 years or those with comorbidities that preclude use of intensive induction chemotherapy. It was granted orphan drug designation by the European Medicines Agency (EMA) in 2017. Structure of glasdegib (DaurismoTM).
Bayesian Frameworks for Rare Disease Clinical Development Programs
Published in Emmanuel Lesaffre, Gianluca Baio, Bruno Boulanger, Bayesian Methods in Pharmaceutical Research, 2020
Freda Cooner, Forrest Williamson, Bradley P. Carlin
These definitions arise from regional legislative acts. The earliest such regulation was established in United States, the 1983 Orphan Drug Act. The orphan drugs referred to in the act are for rare diseases or conditions, including biological products and antibiotics “orphaned” (abandoned) by drug companies due to their low sales potential, an obvious consequence of disease rarity. Following the establishment of this groundbreaking legislation, rare diseases are now often called “orphan diseases,” and orphan drugs are inclusive of biologic products (though not medical devices). More than a decade later, the second major related legislative action was the 1999 Orphan Regulation adopted by the European Parliament. Both regulations include monetary incentives, marketing exclusivities, and clinical research assistance for rare disease medicinal product development. Other than drug product development, there are other regulations or government entities (e.g. the US Rare Diseases Act in 1992 and the Spanish Rare Diseases Research Institute) that aid patient support groups and fund research projects in rare diseases. Several papers, including Gupta (2012) and Gammie et al. (2015), synthesize details and comparisons across different legislations across nations. In fact, most developed countries and regions now have well-established rare disease or orphan product programs. The US remains one of the leaders and a key player in orphan product development and rare disease research.
Cost-effectiveness of defibrotide for treatment of severe veno-occlusive disease: it is time for evidence based economic evaluations
Published in Journal of Medical Economics, 2021
Approval of Defibrotide by EMA and FDA was facilitated by its orphan drug status. “Orphan drug status” was introduced 30 years ago as an incentive for the pharmaceutical industry to develop novel drugs for rare diseases. The initiative was clearly successful; a multitude of novel drugs has been registered worldwide since. This success is accompanied by a downside. Costs for orphan drugs might bring a solidarity-based health care system to its limits. Prizing of orphan drugs is not based on the costs for research, development and production. It rather follows a sum, representing the willingness in a respective country to pay for the years of life saved. This problem is not restricted to Defibrotide. The concept has become generally accepted, but is now of concern worldwide and intensively discussed: what is a reasonable price for an orphan drug? [13–15]. These arguments are valid on the assumption that the drug will help the patient to gain years of life.
The potential and benefits of repurposing existing drugs to treat rare muscular dystrophies
Published in Expert Opinion on Orphan Drugs, 2018
Hesham M. Ismail, Olivier M. Dorchies, Leonardo Scapozza
Orphan drug designation was put forward to promote drug development for rare diseases and was met successfully. Success of trials for drugs holding an orphan designation are much higher than for other drugs. A major reason behind this has been attributed to the close monitoring and the protocol assistance services that form an integral part of the incentives provided. This close interaction helps setting the expectations of regulatory authorities early on in the process to ensure that they can be met. Others incentives that the ODD brings is the extended market exclusivity, which ignited the search for therapies for mostly overlooked diseases but came at the expense of premium pricing, severely limiting patient access. A reconsideration by the legislative authorities into the matter of pricing has to be taken.
Orphan medicinal products’ access to the Bulgarian pharmaceutical market – challenges and obstacles
Published in Expert Opinion on Orphan Drugs, 2018
Maria Kamusheva, Konstantin Tachkov, Guenka Petrova, Alexandra Savova, Manoela Manova
Up to date, the available data indicates that orphan drugs account for a small percentage of the overall pharmaceutical budget, but it is expected that with increasing reimbursement of orphan medicines their budget impact will be significant [39]. In the last 5 years, the average annual costs of orphan drugs have become much higher than those of non-orphan drugs in the United States of America (U.S.A). The worldwide market for orphan drug sales and share of prescription for the period 2000–2022 is expected to accelerate very fast [23]. The case in Bulgaria is similar. Currently, the budget impact of OMPs in Bulgaria is comparatively low: an average of 1.5% of the total costs for ambulatory pharmacotherapy paid by NHIF and 14% of the total costs for oncology drugs for the period 2013–2016, but with a trend of increase. The share of oncological orphan drugs in Bulgaria and in Latvia is more than half of the total orphan expenditures which demonstrates the significant social burden of oncological diseases in both countries [40]. The need for more oncological medicinal products and their expenditures increase in a relation to the aging of the population.