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Rare Diseases Drug Development
Published in Wei Zhang, Fangrong Yan, Feng Chen, Shein-Chung Chow, Advanced Statistics in Regulatory Critical Clinical Initiatives, 2022
Shein-Chung Chow, Shutian Zhang, Wei Zhang
For orphan drug designation, FDA considers using the Mechanism Of Action (MOA) of the drug to determine what distinct disease or condition the drug is intended to treat, diagnose or prevent. Whether a given medical condition constitutes a distinct disease or condition for the purpose of orphan-drug designation depends on a number of factors, assessed cumulatively, including: pathogenesis of the disease or condition; course of the disease or condition; prognosis of the disease or condition; and resistance to treatment. These factors are analyzed in the context of the specific drug for which designation is requested. During the course of reviewing a request for orphan drug designation, equipped with the most current scientific literature about a particular disease or condition, FDA may come to a new understanding about the nature of that disease or condition. Table 10.1 provides a list of some diseases or conditions for which FDA's views on how it categorizes or otherwise understands the disease or condition has evolved. This is not a comprehensive list of orphan disease determinations, but reflective of some of the more common questions we receive. FDA will update this list as appropriate when it makes orphan drug designation determinations that change how we approach the disease or condition in question. For a complete list of orphan drug designations and approvals see the searchable Orphan Products Designation Database.
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
Can Misconduct and Fraud Be Fixed?
Published in Charles E. Dean, The Skeptical Professional's Guide to Rational Prescribing, 2022
Although Pharma paid over $11 billion in fines to settle 103 cases involving off-label or deceptive marketing from 1997 on, the FTC took only one action—against a cancer center. Despite the fines, the FDA has continued to advance the wealth of Pharma during the years 1984–2018,40 by increasing the number of new drug approvals from 34 in 1990–1999 to 41 in 2010–2018. The FDA has also increased the proportion of new drugs approved under the Orphan Drug from 18% in 1984–1995 to 41% in 2008–2018, and has shortened the time for approval of new drugs from 3 years in 1983 to less than 1 year in 2017. The use of Priority Review approvals increased to 81% in 2018. None of this should be surprising, since user fees collected from Pharma increased from an annual mean of $66 million in 1993–1997 to $820 million in 2013–2017. User fees have been shown to account for some 80% of the salaries paid to staffers who approve new drug applications.37 On the face of it, this seems a remarkable conflict of interest.
How the cost-effectiveness results change in the China health policy environment: an economic evaluation of glycopyrrolate/formoterol for the treatment of COPD
Published in Journal of Medical Economics, 2022
Luying Wang, Weili Gu, Xiao Zhang, Shihui Fu, Donger Zhang, Xin Guan, Hongchao Li, Aixia Ma
However, there are still some special circumstances/cases making the price-directed policy well worth consideration. One of the examples is the orphan drugs for rare diseases. Due to the high cost of drug development but small patient pool, the prices of orphan drugs are usually unaffordable33. Rather than bargaining the price into an acceptable range from the NHSA perspective based on the widely used cost-effectiveness threshold, clinical demand, disease burden, clinical efficacy, and disease prevalence should also be evaluated34. Another example is the situation in which “not cost-effective at a zero price”, which is caused by particularly high cost of background treatment or severely low quality-of-life, indicated that healthcare or medical problems such as ethics or fairness could not be solved by pharmacoeconomic evaluation35. Therefore, costs of the treatments and pharmacoeconomic evaluation could mainly reflect the “cost performance” of the intervention and should not be the determinative tools for the government who should care about the public benefit.
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.
Voretigene neparvovec-rzyl for treatment of RPE65-mediated inherited retinal diseases: a model for ocular gene therapy development
Published in Expert Opinion on Biological Therapy, 2020
Thomas A. Ciulla, Rehan M. Hussain, Audina M. Berrocal, Aaron Nagiel
The retina community has generally not been exposed to treatments approved for orphan diseases, which represented another challenge in the development and launch of VN. Historically, rare diseases have been neglected, or ‘orphaned,’ in drug development due in part to the inherent challenges of lengthy and expensive clinical trial operations in small, often geographically dispersed patient populations. In the US, the Orphan Drug Act of 1983 defined orphan diseases as those that affected fewer than 200,000 Americans [60]. The Orphan Drug Act provided sponsors with 7 years of exclusivity, tax credits to defray the cost of development, waived FDA fees, and provided protocol assistance. The European Medicines Agency (EMA) provided similar incentives in 2000, with orphan designation for products addressing life-threatening or debilitating disorders affecting 5 or fewer per 10,000 individuals [61]. There are additional incentives to develop therapies for rare pediatric diseases; in 2017, Spark Therapeutics announced that it received rare pediatric disease designation for VN [62] and was able to sell the associated priority review voucher in 2018, which provided capital to reinvest back into research and development [63].