BackgroundMore than 6,800 rare diseases and conditions have been identified in the US, which affect 25–30 million Americans. In 1983, the US Congress enacted the Orphan Drug Act (ODA) to encourage the development and marketing of drugs to treat rare diseases and conditions. This study analyzed all orphan designations and FDA approvals since 1983 through 2015, discussed the effectiveness of incentives for the development of treatments for rare diseases, and reflected on the ethical imperatives for timely access to orphan drugs.MethodsStudy data were derived from the Food and Drug Administration (FDA) Orange Book and the Office of Orphan Drugs Development. A search was conducted to assess literature on the ethical principles and economic incentives for the development of orphan drugs.ResultsIn the period 1983–2015, the FDA granted 3,647 orphan drug designations and 554 orphan drug approvals. The orphan drug approvals corresponded to 438 different brand names. Cancer was the therapeutic area with the highest number of approvals. The increased number of patients with rare diseases and the growth in the cost of orphan drugs pose a significant economic burden for patients, public programs and private third party payers. Regulatory differences to qualify for orphan designation and various population thresholds employed by the FDA and the European Medicines Agency lead to further unmet health needs for patients with rare diseases and aggravate health inequities. There is no societal consensus on the population and economic thresholds, the drug effectiveness indicator(s), or the societal value to be placed for the approval and reimbursement of orphan drugs.ConclusionOrphan drug development and marketing in the US concentrate in few therapeutic areas. Despite the increase in the number of FDA approved orphan drugs, the unmet needs of patients with rare diseases evidence that the current incentives are not efficiently stimulating orphan drug development. There is need to balance economic incentives to stimulate the development and marketing of orphan drugs without jeopardizing patients’ access to treatment. Thus, aligning pharmaceutical companies’ incentives with societal budgetary constraints is necessary and the ethical imperatives of timely access to orphan drugs need to be agreed upon.
Approximately one in seven approved NMEs were discontinued from the market in the period of 1980-2009. Less than one-quarter (22%) of the total withdrawals were attributed to safety reasons. An ongoing evaluation of new drugs throughout their product life cycle is important to determine their efficacy, safety, and value to society.
Although most commonly used drugs cause adverse effects, some of them with potentially serious consequences, relatively little is known about their economic impact. The purpose of this review is to summarise information describing the cost of treatment of drug-induced adverse effects as an additional cost of pharmaceutical treatment. The focus of this study was limited to the overall economic impact of drug-related morbidity and to the economic analysis of a single class of drugs with different safety profiles. Several studies carried out in the US have investigated adverse drug effects experienced by hospitalised patients and their impact on hospital costs. Patients who developed adverse effects were hospitalised an average of 1.2-3.8 days longer than patients who did not, with additional hospital costs of $US2284-5640 per patient (2000 values). Other research studies in different countries have quantified the incidence and economic consequences of adverse drug effects that occur in the ambulatory setting and that generate hospital admission and emergency department visits. They have shown that preventable adverse effects constitute between 43.3% and 80% of all adverse outcomes leading to emergency visits and hospital admissions, and disproportionately increase healthcare costs. Finally, a recent estimation revealed that in the US the cost of problems linked to drug use in the ambulatory setting exceeded $US177 billion in the year 2000.NSAIDs constitute a widely used class of drugs and they are one of the leading drug classes in causing adverse effects. The acquisition costs of the drugs, as well as the costs for prevention and treatment of adverse effects, determine their cost-effectiveness ratio. Depending on the incidence and severity of adverse effects, the cost per adverse effect avoided ranges from $US215 to $US35 459 (2000 values). According to the contingent valuation methodology, willingness to pay to avoid or reduce the incidence of adverse effects is an indicator of the value individuals associate with the impact of such effects on their well-being. Individuals are willing to pay annually an average of $US240 and $US350, respectively, to avoid vomiting and gastrointestinal distress induced by NSAIDs. Although the results of the different studies reviewed are not strictly comparable because of differences in the severity of adverse effects, the perspective of the analysis, the cost data included and the cost component considered, the data show that, apart from the implications for health, a substantial quantity of resources are used to treat adverse effects.
Background: The Orphan Drug Act (1983) established several incentives to encourage the development of orphan drugs (ODs) to treat rare diseases and conditions. This study analyzed the characteristics of OD designations, approvals, sponsors, and evaluated the effective patent and market exclusivity life of orphan new molecular entities (NMEs) approved in the US between 1983 and 2007.
The emergence of universal electronic prescribing and content knowledge vendors has laid the groundwork for incorporating indications into the CPOE prescribing process. As medication prescribing moves in the direction of inclusion of the indication, it is imperative to design CPOE systems to efficiently and effectively incorporate indications into prescriber workflows and optimize ways this can best be accomplished.
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