The increasing number and high prices of orphan drugs have triggered concern among patients, payers, and policymakers about the affordability of new drugs approved using the incentives set by the Orphan Drug Act (ODA) of 1983. This study evaluated the factors associated to the differences in the treatment cost of new orphan and non-orphan drugs approved by the FDA from 2017 to 2021. A generalized linear model (GLM) with the Gamma log-link analysis was used to ascertain the association of drug characteristics with the treatment costs of orphan and non-orphan drugs. The results of the study showed that the median and interquartile range (IQR) drug cost was USD 218,872 (IQR = USD 23,105) for orphan drugs and USD 12,798 (IQR = USD 57,940) for non-orphan drugs (p < 0.001). Higher market entry prices were associated with biologics (108%; p < 0.001), orphan status (177%; p < 0.001), US sponsor companies (48%; p = 0.035), chronic use (1083%; p < 0.001), treatment intent (163%; p = 0.004), and indications for oncology (624%; p < 0.001) or genetic disorders (624%; p < 0.001). Higher market entry treatment cost for newly approved drugs were associated with biologics, orphan status, US sponsor companies, chronic use, therapeutic intent, and indications for oncology or genetic disorders.

Background: In Saudi Arabia, the government has implemented compulsory premarital screening and consultations for high-risk and positive sickle cell results (traits = AS gene and disease = SS gene). However, despite these measures being in place since 2004, there are still cases of children being born with sickle cell disease. This study aims to evaluate the costs associated with the government’s mandatory premarital screening for sickle cell anemia, compare these expenses with those incurred due to high-risk marriages and analyze the government’s healthcare spending on sickle cell anemia management. Method: A decision tree model was conceptualized for the purpose of this study to identify the possible paths from the premarital screening (PMS) procedure. A total of 300,000 cases were processed through this decision tree model. Results: The annual management costs for children with the probability of having sickle cell disease are estimated to be USD 10,746,450 in the screening arm and USD 40,488,000 in the no-screening arm. These costs vary depending on the genetic combination of the parents. For individuals with the SS/SS gene combination, the estimated cost is USD 8,137,800 per year. When parents have the SS/AS gene combination, the estimated cost is USD 2,071,950 annually. For those with the sickle cell trait combination (AS/AS), the cost is estimated to be USD 536,700 per year. A direct comparison shows a modeled PMS incremental cost is estimated at USD 29,741,550, which is approximately a 73% reduction in healthcare costs. Conclusions: The premarital screening for sickle cell disease is not only cost-saving but also shows the potential for significantly reducing healthcare spending related to sickle cell disease in the future.

Introduction: As the pharmaceutical advertising landscape evolves with digital advancements, this study examines public awareness and perceptions of medication advertisements in Saudi Arabia. It focuses on the effects of regulatory frameworks and evaluates how they influence public understanding and attitudes toward these advertisements. Method: A cross-sectional study was conducted using an electronic survey in Saudi Arabia in December 2023. The survey was distributed on social media platforms and reached a diverse sample of 440 participants. It covered public perception and attitudes toward drug advertisements, knowledge of regulatory laws, and preferences regarding advertising mediums. Results: Out of the 440 participants in the study, who were primarily employees with bachelor’s degrees, there was a clear awareness of drug advertisements. The average age of the group was 33 years, and a significant portion (71.1%) held a bachelor’s degree, with 51.1% being employed. The findings revealed that 25.5% of participants frequently noticed drug ads, while 22.7% saw them very often. Although many found the ads informative, there were significant concerns about unrealistic expectations and the risk of overmedication; 89.8% believed the ads set unrealistic expectations about the effectiveness of medications. Additionally, 60.7% thought that celebrity endorsements might mislead the audience, and 91.1% felt that ads should provide more detailed information about potential risks and side effects. Regarding preferred advertising platforms, mobile apps and websites were favored (47%), followed closely by social media (46.4%). A striking 93.2% of participants believed that drug ads on social media should be subject to stricter regulations, and 96.4% wanted more proactive monitoring of online advertising. Many also reported using other sources, such as medical review sites, to verify medication information. Conclusions: Pharmaceutical advertising in Saudi Arabia must balance ethical transparency with educational value. The influence of digital platforms underscores the necessity for stricter regulation and accurate information dissemination. A collaborative approach is essential to align advertising practices with public health interests and regulatory standards.

Medication safety relies on effective pharmacovigilance (PV), but inadequate professional awareness and training contribute to under-reporting of adverse d