Background: Prescription drugs may be indicated to treat more than one medical condition, and companies may promote more than one indication in the same direct-to-consumer (DTC) ad. This study examined how presenting multiple prescription drug indications in one DTC television ad affects consumers' processing of drug information.
Methods: We conducted two studies with adults diagnosed with diabetes (Study 1, N = 408) or rheumatoid arthritis (Study 2, N = 411). We randomly assigned participants to view one of three television ads: primary indication only (Study 1: diabetic peripheral neuropathy; Study 2: rheumatoid arthritis), primary plus a similar secondary indication (Study 1: fibromyalgia; Study 2: psoriatic arthritis), or primary plus a dissimilar secondary indication (Study 1: generalized anxiety disorder; Study 2: ulcerative colitis).
Results: Remembering and understanding the primary indication was not significantly affected by the presence of a secondary indication (similar or dissimilar). Higher health literacy participants remembered and understood secondary indications.
Conclusions: Including a second indication in DTC television ads does not appear to have detrimental effects and can increase awareness of the second indication for some participants. Industry and regulators should continue to ensure DTC promotion is truthful and non-misleading, irrespective of the number of indications presented.
Introduction: The sales patterns of original drugs after patent expiration in Korea show a relatively high market share and continuous sales growth differently from those in the U.S. and European countries. This study aims to investigates a five-year sales pattern of original drugs after patent expiration in Korea using empirical data.
Methods: Using data from the Ministry of Food and Drug Safety, original drugs whose patents expired in 2012-2018 were extracted. And we used IQVIA data to determine the market share and sales growth rate of 48 original drugs, whose generic drug launched for the first time in the same molecule market, and whose sales data over five years after first generic entry were available. We analyzed the differences by the attribute of variables.
Results: The sales volume of original drugs in the fifth year (Q 20) had an average growth rate of 150.6% compared with that before the first generic drug launched, indicating a continuous growth. The average market share of original drugs in the fifth year (Q 20) decreased to 70.6%, but it was higher than previously reported research results in Korea and other countries. Differences were observed across the category of attribute.
Conclusion: This study demonstrated that while market share of original drugs is decreasing, the sales volume increased continuously until the fifth year, differently from those of other countries. Variations in sales patterns by attributes reflect unique dynamics in Korea.
A Data Monitoring Committee (DMC) plays a pivotal role in monitoring participant safety and efficacy and overseeing the integrity of clinical trials. DMCs accomplish this mission by periodically reviewing accumulating data to assess benefits and harms of interventions in ongoing studies and making subsequent recommendations regarding future clinical trial conduct to the trial sponsor. Reports summarizing data from the clinical trial are prepared for the DMC by statistical and data analysis centers to inform DMC decision-making. In practice however, these reports are often disorganized, complex, and provide overwhelming detail yet insufficient information, that hinders accurate and efficient interpretation of interim data. This review paper delves into the nuances of preparing effective DMC reports, highlighting the importance of simplicity, clarity, and thoughtful relevance in data presentation. We discuss structured approaches for preparing closed reports, which deal with sensitive and sometimes messy interim data, and underscore the use of visual summaries and narrative elements that enhance comprehension and facilitate efficient assessments of trial data. The paper outlines key principles for preparing DMC reports and provides practical guidance on their structure. Ultimately, this guidance seeks to ensure that the data's story is clearly and efficiently conveyed to facilitate the DMC decision-making process.
Introduction: The Saudi Food and Drug Authority (SFDA) conducts inspections in accordance with Good Clinical Practice (GCP) to safeguard clinical trial integrity and protect the rights, safety, and welfare of study participants. These inspections ensure that trials are conducted in compliance with GCP and applicable laws.
Objectives: The study aims to provide a description of GCP inspection findings, analyze their impact on the clinical trial ecosystem, and provide recommendations to improve clinical trial conduction in Saudi Arabia.
Methods: A review was conducted on inspection reports, with two senior independent inspectors examining, collecting, and categorizing the data. Descriptive statistics were used to summarize the categorical variable via frequency distributions.
Results: A total of 131 GCP inspections were performed between 2017 and 2023, totaling 722 observations from 116 (88.5%) inspection visits. The remaining 15 (11.5%) inspection visits recorded no observations. The highest number of visits were conducted in contract research organizations (CRO) (n = 50; 38.2%) with 118 observations, followed by clinical investigator sites (n = 46; 35.1%) with 313 observations, then bioequivalence (BE) centers (n = 33; 25.2%) with 256 observations, and the last 2 (1.5%) visits were conducted in phase I clinical trial units with 35 observations.
Conclusion: This study assesses GCP inspection reports and examines the types of deficiencies and their grades in each area. Observation categories and grades were found to vary by organization type, which indicates the need for specific action plans addressing each organization type separately. This report provided recommendations based on the most common findings to assist researchers and sponsors when conducting clinical trials in Saudi Arabia.
Global harmonization of biosimilar developmental requirements will facilitate development leading to increased patient and societal benefits. However, there are several technical and regulatory hurdles that must be addressed to harmonize the regulatory requirements in different countries and regions. At times, there is a requirement for use of locally sourced reference product, forcing biosimilar developers to repeat analytical or clinical comparability studies against reference product batches sourced from within a given country. While most health authorities no longer require comparative animal toxicology studies of the proposed biosimilar and reference product, these are still required in several countries, forcing biosimilar companies to conduct such studies or risk non-approval of their product. At times, different health authorities request different clinical study designs. In some jurisdictions there is a requirement to generate clinical data in local ethnic populations. Some health authorities require a hybrid label that combines clinical data from the reference biologic and the biosimilar, in the patient leaflet. Recommendations are provided to address each of these hurdles to facilitate global regulatory harmonization of biosimilars. Overcoming these barriers will ultimately increase patient access to these medicines in all regions while providing financial relief to healthcare systems.
The emergence of personalized RNA therapeutics, tailored to individual patients' genetic profiles, offers new hope for treating both common and rare diseases. This review explores regulatory aspects of N-of-1 and N-of-few approaches, providing promising treatments for ultra- or nano-rare diseases that lack established therapies. These diseases present unique challenges, as patients may represent the sole individual or a small group worldwide with a specific mutation, necessitating personalized approaches to treatment development, validation, and approval. While progress is promising, the regulatory landscape remains nascent, raising challenges in ensuring safety and industry sustainability. Artificial intelligence (AI) and automated systems, coupled with real-world evidence (RWE) monitoring, offer significant potential to address these challenges by optimizing development, manufacturing, and regulatory compliance. Drawing parallels from other regulatory domains, this review presents a design envelope framework, integrated with AI tools, to streamline the approval process and enhance the adaptability of RNA-based treatments. Case studies of individualized RNA-based treatments highlight successes and setbacks, underscoring the need for regulatory alignment. Collaborative efforts from stakeholders and regulatory authorities are essential to refine this framework for real-world application. Overall, this review emphasizes the transformative potential of personalized RNA therapeutics in advancing precision medicine.
Importance: Racially and ethnically diverse, equitable representation among clinical trial participants is important for enhancing the drug development process and promoting equitable healthcare outcomes.
Objective: To understand the barriers and drivers for inclusive clinical trials, focusing on the attitudes, perceptions, experiences, and challenges faced by underrepresented populations.
Design: An online questionnaire was administered online from April to June 2023 and involved 12,017 respondents from 54 countries. This survey utilized a convenience sampling strategy. Statistical analysis was performed to compare responses among racial and ethnic groups.
Setting: The study was conducted globally. Survey respondents were recruited through various patient recruitment organizations, patient advocacy groups, and contract research organizations.
Respondents: Adults 18 years or older who received an email or had online access were eligible to participate. Racial and ethnic composition included White (81%), Hispanic/Latino (15%), Black/African American (6%), Asian (6%), and other ethnicities.
Exposure(s): Respondents were asked about their perceptions, concerns and experiences related to clinical research access and participation.
Main outcome(s) and measure(s): Key outcomes included barriers to clinical research participation, factors influencing trust in pharmaceutical companies and past experiences.
Results: Barriers to clinical research participation varied among ethnic groups. Asian respondents cited concerns about time off work (22%) and time required to participate (19%) more frequently as compared to White respondents (7% and 7%, respectively; p < 0.05). Hispanics expressed higher concerns about time off work (15%) and receiving placebo (10%) as compared to Non-Hispanics (8% and 5%, respectively, p < 0.05). Black and Hispanic respondents placed higher importance on diversity in staff compared to White and non-Hispanic respondents (B: 32%; W: 12%; Hispanic: 22%; Non-Hispanic: 13% p < 0.05). Black, Asian, and Hispanic respondents reported higher levels of disruption in participation related to technology use (Black: 31%; Hispanic: 30%; Asian: 29%) and completing study requirements at home (Black: 32%; Hispanic: 30%; Asian: 26%) as compared to White (13%, 15%; p < 0.05%) and non-Hispanic respondents (14%, 17%; p < 0.05).
Conclusions: The findings highlight the need to address barriers to diversity in clinical trials and improve trial experiences of underrepresented communities, facilitating design of more inclusive and patient-centered trials.
This paper describes the planning of a patient preference study for evaluating device features for the subcutaneous (SC) delivery of high dose/large volume (e.g., > 2 ml) of drugs. Multiple sources, including qualitative patient interviews, the involvement of patient partners, and solicitation of advice from the US Food and Drugs Administration (FDA), were used to refine the attributes and levels in the development of a preference study protocol to investigate what Multiple Sclerosis (MS) patients consider important regarding medical device features for high dose SC administration.
In November 2007, a black box warning was mandated for rosiglitazone in type 2 diabetes mellitus (T2DM) based on an increased risk of ischemic cardiovascular (CV) events. The Food and Drug Administration (FDA) issued a directive that a CV outcomes trial must be done for any new diabetes drug to demonstrate no CV harm. Therefore, the Empagliflozin Cardiovascular Outcome Event Trial in Type 2 Diabetes Mellitus Patients (EMPA-REG OUTCOME) trial was started in 2011 alongside 13 additional randomized clinical trials (RCTs) of empagliflozin in T2DM. The results of EMPA-REG OUTCOME set the stage for later RCTs in heart failure. Results from these clinical trials have changed the outlook for patients both with and without T2DM and with and without heart failure. A Program Data Monitoring Committee (DMC) with the same core members was utilized for these trials between 2011 and 2024. This committee is likely to be one of the longest serving DMCs since it served 28 trials with empagliflozin between 2011 and 2024. The committee encountered several important challenges which are discussed in this article. Moreover, the committee provides several important take-home messages which we hope will be of value in discussing issues in creating, developing and running DMCs in the future. These include: 1. Whether and when to be blinded and unblinded; 2. How to proceed when the primary endpoint shows no evidence of benefit, but there is evidence for a mortality benefit; 3. Development of presentation of data using figures and boxplots for rapid review of adverse events and laboratory data to assess clinical challenges; 4. How to manage a catastrophic serious adverse event; 5. Suggestions for an ideal structure of the report for the DMC closed session; and 6. The relation between the DMC, sponsor and Contract Research Organization. Our experience emphasizes the value of continuity with the same members serving over a 13-year period.
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