Qualitative Analysis of Inquiries Received by FDA Regarding Conduct of Clinical Trials during the Covid-19 Public Health Emergency.

BACKGROUND: This report describes the U.S. Food and Drug Administration (FDA) experience in establishing a dedicated mailbox, and in publishing related guidance, to address concerns among interested parties regarding the conduct of clinical trials during the COVID-19 public health emergency (PHE). METHODS: Six hundred and thirty-four mailbox inquiries were received from March 2020 through February 2022. Qualitative methods were used to provide a structured description of, and identify common themes among, these inquiries. RESULTS: Most inquiries came from U.S.-based interested parties, including sponsors, industry trade associations, academic institutions, hospitals, clinics, research sites, trial participants, and individual persons. Approximately one-fifth of questions were related directly to COVID-19 (e.g., proposals for treatment); other inquiries were related to conduct of routine trial-related activities, and concerns were often focused on maintaining compliance with good clinical practice. In March 2020, FDA published a guidance titled Conduct of Clinical Trials of Medical Products During the COVID-19 Public Health Emergency; the document was subsequently revised eight times based in part on issues raised in mailbox inquiries. CONCLUSIONS: The dedicated mailbox enabled expedited communication among invested parties during the COVID-19 PHE; FDA also provided updates of the aforementioned guidance. These efforts supported the continuance of ongoing trials and the initiation of new trials during the PHE in accordance with good clinical practice guidelines, thereby helping to ensure the safety of trial participants while maintaining the quality of trial data. By soliciting and responding to trial-related inquiries and addressing corresponding needs and concerns, FDA improved transparency and communication.

The temporal association between adverse drug reactions and antirheumatic drugs utilisation in Western Australia: a retrospective study from real-world data (1995-2015).

BACKGROUND/OBJECTIVES: Adverse drug reactions (ADRs) can result in morbidity, mortality, and higher healthcare costs. Given the limited information available on ADRs associated with antirheumatic medications, this study aims to analyse and compare ADR reporting for these drugs in the pharmacovigilance datasets of Western Australia (WA) and the United States (US). METHODS: Therapeutic Goods Administration provided WA pharmacovigilance data of selected antirheumatic drugs to from 1995 to 2015. The proportional reporting ratio (PRR) for WA case reports was compared to corresponding USA pharmacovigilance data by assessing the disproportionality of each ADR. clinically significant or true ADRs were determined using the Evans 2001 criteria (n > 2, chi-square > 4, PRR > 2). RESULTS: A total of 232 reports were found in WA, mostly on sixty-nine women aged 45 to 69. Methotrexate, leflunomide, azathioprine, sulfasalazine, and infliximab had the highest reported ADRs, related to gastrointestinal disorders. Patients who used biological agents in WA had 2.7 times the likelihood of reporting true ADRs compared to conventional antirheumatic drugs. The ADR rates in the two datasets were comparable over the study period. CONCLUSIONS: The PRR values of ADRs were consistent between WA and US databases. Methotrexate and infliximab use were commonly associated with ADR reports in WA females, with incidence rates comparable to the US; while patients using biological agents were more likely to report true ADRs than those on conventional antirheumatic drugs in WA.

Women's Health Information-Seeking Experiences and Preferences for Health Communications on FDA-Regulated Products: A Qualitative Study in Urban Area.

A key part of any effort to ensure informed health care decision-making among the public is access to reliable and relevant health-related information. We conducted focus groups with women from three generations across the Baltimore-Washington metropolitan area to explore their information-seeking motivations, perceptions, challenges, and preferences regarding three FDA-regulated products: drugs, vaccines, and medical devices. The youngest generation discussed seeking health information for their children; the other two sought information for their own needs. All participants noted that finding health information appropriate to their reading level was a challenge, as was identifying reliable sources of information. All generations identified in-person and live interactions as their preferred method of communication and health care providers as their preferred source for information. All three generations recognized the usefulness of websites, and the two older generations acknowledged the advantages of brochures. Our findings suggest approaches the FDA could consider to improve communications: (a) supporting in-person and live health information interactions; (b) leveraging the agency's standing with the public to highlight it as a leading source of validated health information; (c) increasing the FDA website's visibility in internet searches and making its navigation easier; and (d) using multi-pronged approaches and media for various audiences.

Challenges and controversies in resectable non-small cell lung cancer: a clinician's perspective.

The treatment landscape of resectable early-stage non-small cell lung cancer (NSCLC) is transforming due to the approval of novel adjuvant and neoadjuvant systemic treatments. The European Medicines Agency (EMA) recently approved adjuvant osimertinib, adjuvant atezolizumab, adjuvant pembrolizumab, and neoadjuvant nivolumab combined with chemotherapy, and the approval of other agents or new indications may follow soon. Despite encouraging results, many unaddressed questions remain. Moreover, the transformed treatment paradigm in resectable NSCLC can pose major challenges to healthcare systems and magnify existing disparities in care as differences in reimbursement may vary across different European countries. This Viewpoint discusses the challenges and controversies in resectable early-stage NSCLC and how existing inequalities in access to these treatments could be addressed.

New systemic treatment paradigms in resectable non-small cell lung cancer and variations in patient access across Europe.

The treatment landscape of resectable early-stage non-small cell lung cancer (NSCLC) is set to change significantly due to encouraging results from randomized trials evaluating neoadjuvant and adjuvant immunotherapy, as well as adjuvant targeted therapy. As of January 2024, marketing authorization has been granted for four new indications in Europe, and regulatory approvals for other study regimens are expected. Because cost-effectiveness and reimbursement criteria for novel treatments often differ between European countries, access to emerging developments may lead to inequalities due to variations in recommended and available lung cancer care throughout Europe. This Series paper (i) highlights the clinical studies reshaping the treatment landscape in resectable early-stage NSCLC, (ii) compares and contrasts approaches taken by the European Medicines Agency (EMA) for drug approval to that taken by the United States Food and Drug Administration (FDA), and (iii) evaluates the differences in access to emerging treatments from an availability perspective across European countries.

Unpublished trials of alprazolam XR and their influence on its apparent efficacy for panic disorder.

OBJECTIVE: To test for publication bias with alprazolam, the most widely prescribed benzodiazepine, by comparing its efficacy for panic disorder using trial results from (1) the published literature and (2) the US Food and Drug Administration (FDA). METHODS: From FDA reviews, we included data from all phase 2/3 efficacy trials of alprazolam extended-release (Xanax XR) for the treatment of panic disorder. A search for matching publications was performed using PubMed and Google Scholar. Publication bias was examined by comparing: (1) overall trial results (positive or not) according to the FDA v. corresponding publications; (2) effect size (Hedges's g) based on FDA data v. published data. RESULTS: The FDA review showed that five trials were conducted, only one of which (20%) was positive. Of the four not-positive trials, two were published conveying a positive outcome; the other two were not published. Thus, according to the published literature, three trials were conducted and all (100%) were positive. Alprazolam's effect size calculated using FDA data was 0.33 (CI95% 0.07-0.60) v. 0.47 (CI95% 0.30-0.65) using published data, an increase of 0.14, or 42%. CONCLUSIONS: Publication bias substantially inflates the apparent efficacy of alprazolam XR.

Decisions on Non-oncology Breakthrough Therapy Designation Requests in 2017-2019.

BACKGROUND: The US Food and Drug Administration's Breakthrough Therapy Designation (BTD) program is intended to facilitate and expedite development of investigational drugs to address unmet medical needs. The objective of this study is to provide an update on FDA's process for review of BTD requests. METHODS: We reviewed Center for Drug Evaluation and Research (CDER) decisions to grant or deny breakthrough therapy designation requests for non-oncology drugs or biological products ("drugs") from January 1, 2017, through December 31, 2019. Data collection included characteristics of the corresponding drug and condition, reasons for granting or denying breakthrough therapy status, reasons for rescinding or withdrawing breakthrough therapy status after a request was granted (if applicable), and subsequent marketing approval status through 2022. RESULTS: Among 240 requests, 93 (39%) requests were granted and 147 (61%) requests were denied. Granting of requests was more common for conditions where no therapy was available or for orphan diseases. Common reasons for denial included data-related issues, insufficient treatment effect, inadequate study design, endpoint attributes, safety issues, and reliance on post hoc analyses. Among 28 drugs receiving marketing approval as of the end of 2022 for the indication for which BTD was previously granted, 21 (75%) involved a first-in-class mechanism of action. CONCLUSIONS: This analysis describes CDER's decision-making process related to review of requests for breakthrough therapy designations and enhances public awareness regarding efforts to expedite drug development.

Guidelines for pharmacotherapy in Alzheimer's disease - A primer on FDA-approved drugs.

The growing prevalence of dementia makes it important for us to better understand its pathophysiology and treatment modalities, to improve the quality of life of patients and caregivers. Alzheimer's disease (AD), a neurodegenerative disease, is the most common form of amnestic dementia in the geriatric population. Pathophysiology of AD is widely attributed to aggregation of amyloid-beta (Aß) plaques and hyperphosphorylation of tau proteins. Initial treatment modalities aimed to increase brain perfusion in a non-specific manner. Subsequent therapy focused on rectifying neurotransmitter imbalance in the brain. Newer drugs modify the progression of the disease by acting against aggregated Aß plaques. However, not all drugs used in therapy of AD have been granted approval by the United States Food and Drug Administration (FDA). This review categorizes and summarizes the FDA-approved drugs in the treatment of AD in a manner that would make it a convenient reference for researchers and practicing physicians alike. Drugs that mitigate symptoms of dementia may be categorized into mitigators of Behavioral and Psychological Symptoms of Dementia (BPSD), and mitigators of cognitive decline. BPSD mitigators include brexpiprazole, an atypical antipsychotic with a once-daily dosage suited to treat agitation in dementia patients, and suvorexant, an orexin receptor antagonist used to treat sleep disturbances. Cognitive decline mitigators include cholinesterase inhibitors such as donepezil, rivastigmine, and galantamine and glutamate inhibitors such as memantine. Donepezil is the most commonly prescribed drug. It is cheap, well-tolerated, and may be prescribed orally once daily, or as a transdermal patch once weekly. It increases ACh levels, enhances oligodendrocyte differentiation and also protects against Aß toxicity. However, regular cardiac monitoring is required due to reports of cardiac conduction side effects. Rivastigmine requires a twice-daily oral dosage or once-daily replacement of transdermal patch. It has fewer cardiac side effects than donepezil, but local application-site reactions have been noted. Galantamine, in addition to improving cognitive symptoms in a short span of time, also delays the development of BPSDs and has minimal drug-drug interactions by virtue of having multiple metabolic pathways. However, cardiac conduction disturbances must be closely monitored for. Memantine, a glutamate regulator, acts as an anti-Parkinsonian agent and an antidepressant, in addition to improving cognition and neuroprotection, and requires a once-daily dosage in the form of immediate-release or sustained-release oral tablets. Disease-modifying drugs such as aducanumab and lecanemab reduce the Aß burden. Both act by binding with fibrillary conformations of Aß plaques in the brain. These drugs have a risk of causing amyloid-related imaging abnormalities, especially in persons with ApoE4 gene. Aducanumab is administered once every 4 weeks and lecanemab once every 2 weeks. The decision on the choice of the drug must be made after considering the availability of drug, compliance of patient (once-daily vs. multiple doses daily), cost, specific comorbidities, and the risk-benefit ratio for the particular patient. Other non-pharmacological treatment modalities must also be adopted to have a holistic approach toward the treatment of AD.

United States Food and Drug Administration Regulation of Human Cells, Tissues, and Gene Therapies.

Research and development of gene therapies and cell- or tissue-based therapies has experienced exponential growth in recent decades and the potential for these products to treat diverse, often rare, clinical indications is promising. The Office of Therapeutic Products (OTP) in the Center for Biologics Evaluation and Research (CBER) at the United States Food and Drug Administration (US FDA) is responsible for the regulation of these products, among others, throughout the entire product lifecycle. This chapter provides an overview of the science- and data-driven approach to US FDA regulatory oversight of cell and gene therapy (CGT) products to ensure their safety and efficacy.

Adverse Events of Abiraterone Acetate vs Enzalutamide.

INTRODUCTION: This study aimed to highlight the comprehensive differences in adverse events between abiraterone and enzalutamide based on a big data data set. METHODS: We downloaded adverse event data sets of abiraterone and enzalutamide from the Food and Drug Administration Adverse Event Reporting System database. We used the Medical Dictionary for Regulatory Activities to treat each adverse event as a preferred term and grouped it into the System Organ Class. Logistic regression analyses were performed to compare abiraterone and enzalutamide. RESULTS: In total, we extracted 59,680 data sets. After exclusion by criteria, we included 26,015 reports on enzalutamide and 7,507 on abiraterone. Enzalutamide and abiraterone presented different toxicity profiles in most System Organ Classes. Overall, the reporting odds ratio indicated a higher incidence rate of serious adverse events for abiraterone than enzalutamide. CONCLUSIONS: In conclusion, our findings suggest that both drugs present a discrete and nonoverlapping toxicity profile that varies by System Organ Class and patient age. This data set confirms, for the most part, what has been reported in clinical trials as well as true real-world reports.

European Committee on Antimicrobial Susceptibility Testing-Recommended Rapid Antimicrobial Susceptibility Testing of Escherichia coli, Klebsiella pneumoniae, and Staphylococcus aureus From Positive Blood Culture Bottles.

Background: Early diagnosis and treatment are important for a good prognosis of bloodstream infections. The European Committee on Antimicrobial Susceptibility Testing (EUCAST) recommends rapid antimicrobial susceptibility testing (RAST) based on the disk diffusion methodology for 4, 6, and 8 hours of incubation. We evaluated EUCAST-RAST of Escherichia coli, Klebsiella pneumoniae, and Staphylococcus aureus from positive blood culture bottles. Methods: Twenty strains of E. coli, K. pneumoniae, and S. aureus were tested using EUCAST-RAST. Ten antimicrobial agents against E. coli and K. pneumoniae and four agents against S. aureus were tested. The diameter of the inhibition zone (mm) was compared with the minimal inhibitory concentration (µg/mL) obtained using the Sensititre AST system (TREK Diagnostic Systems, East Grinstead, UK). Results: For E. coli, the percentage of total categorical agreement (CA) was 69.5% at 4 hours, and 87% at 8 hours. For K. pneumoniae, the total CA was 89% at 4 hours, and 95.5% at 6 hours. For S. aureus, the total CA was 100% after 4 hours. Discrepancies were observed mainly for E. coli with ß-lactam antimicrobial agents, and the numbers of errors decreased over time. Conclusions: EUCAST-RAST for K. pneumoniae and S. aureus met the United States Food and Drug Administration criteria at 6 and 4 hours, respectively, whereas that for E. coli did not meet the criteria for up to 8 hours. RAST can shorten the turn-around testing time by more than one day; therefore, if applied accurately according to laboratory conditions, antimicrobial agent results can be reported faster.

Animal use and opportunities for reduction in carcinogenicity studies supporting approved new drug applications in the U.S., 2015-2019.

A minimum of 65,341 rats and mice were used in 109 carcinogenicity studies conducted for new drug applications approved by the U.S. Food and Drug Administration from 2015 through 2019. By analyzing how these animals were used, we compared the potential for reducing animal use of implementing existing international guidelines and recommendations. The greatest reduction, 18.7%, would result from evaluating exposure by microsampling blood in main studies to replace toxicokinetics satellites, which used three-fold more mice than rats. A similar reduction, 17.3%, would result from replacing 33 long-term studies in mice with short-term studies in transgenic mice. Based on histopathology findings in chronic studies, 15 long-term studies in rats could have been waived, using 8410 fewer rats. Simply using single, rather than dual, negative control groups would result in a 7.8% reduction, and eliminating positive control groups would use 640 fewer transgenic mice. Combined, an estimated 46% reduction would be achieved, using approximately 29,876 fewer animals. The publication of an addendum to the main carcinogenicity testing guideline promises to decrease the number of long-term studies conducted in rats and mice and presents opportunity to promote full harmonization and implementation of related recommendations that would further dramatically reduce animal use.

Regenerative medicine strategies for hair growth and regeneration: A narrative review of literature.

Hair loss, or alopecia, is associated with several psychosocial and medical comorbidities, and it remains an economic burden to individuals and the society. Alopecia is attributable to varied mechanisms and features a multifactorial predisposition, and the available conventional medical interventions have several limitations. Thus, several therapeutic strategies for alopecia in regenerative medicine are currently being explored, with increasing evidence suggesting that mesenchymal stem cell (MSC) implantation, MSC-derived secretome treatment, and blood-derived platelet-rich plasma therapies are potential treatment options. In this review, we searched the Cochrane Library, MEDLINE (PubMed), EMBASE, and Scopus using various combinations of terms, such as "stem cell," "alopecia," "hair loss," "Androgenetic alopecia," "male-pattern hair loss," "female-pattern hair loss," "regenerative hair growth," "cell therapy," "mesenchymal stem cells," "MSC-derived extracellular vesicles," "MSC-derived exosomes," and "platelet-rich plasma" and summarized the most promising regenerative treatments for alopecia. Moreover, further opportunities of improving efficacy and innovative strategies for promoting clinical application were discussed.

United States Food and Drug Administration's authorization of reduced exposure claims for IQOS®: implications for regulation in Latin America.

Philip Morris International has used the July 7, 2020 United States Food and Drug Administration's (US FDA) modified risk tobacco product order for IQOS®, which authorized certain reduced exposure marketing claims, as a corporate strategy to promote and normalize its heated tobacco products in Latin America. The modified risk tobacco product orders are based on the US's unique regulatory system that is not, and should not be, replicated anywhere else in the world. Philip Morris International's global public relations campaign largely ignored the FDA's rejection of reduced risk claims for IQOS and other key FDA findings that are important for policy-makers, regulators, and consumers - including tobacco users and Philip Morris International's customers - to understand the risks associated with the product. In Latin America in particular, Philip Morris International has used media outlets to promote this misleading information to the public. This company has also used the FDA ruling to lobby regulators in Latin America to relax regulations on IQOS in the region. As tobacco companies rapidly introduce new tobacco products in low- and middle-income countries, public health advocates and Parties to the World Health Organization (WHO) Framework Convention on Tobacco Control (FCTC) should take measures to prevent the promotion of misleading statements about heated tobacco products, including IQOS. As Latin American countries are at different stages in their regulation of heated tobacco products, governments should adhere to their WHO FCTC obligations and the recommendations of the Conference of the Parties by entirely prohibiting the sale of heated tobacco products or strictly applying to heated tobacco products all the relevant tobacco demand-reduction policies based on the WHO FCTC (making sure to capture both heated cigarettes and heating devices).

Philip Morris International ha empleado el dictamen que la Administración de Alimentos y Medicamentos (FDA) de Estados Unidos emitió el 7 de julio del 2020 sobre IQOS como producto de tabaco de riesgo modificado ­que la autorizó a usar ciertas declaraciones relativas a una exposición reducida al comercializar el producto­ como estrategia corporativa para promover y normalizar sus productos de tabaco calentado en América Latina. Los dictámenes sobre productos de tabaco de riesgo modificado se fundamentan en el sistema regulatorio único de Estados Unidos, que no se replica ni debería ser replicado en ningún otro lugar del mundo. La campaña mundial de relaciones públicas de Philip Morris International omitió en gran medida que la FDA rechazó los argumentos de que IQOS implica un riesgo reducido y otros hallazgos clave de la FDA que son importantes para que los responsables de las políticas, los reguladores y los consumidores, incluidos los consumidores de tabaco y los clientes de Philip Morris International, comprendan los riesgos asociados con el producto. En América Latina en particular, Philip Morris International ha utilizado los medios de comunicación para difundir esta información engañosa. Esta compañía también ha utilizado el fallo de la FDA para presionar a los reguladores en América Latina con el objetivo de que flexibilicen las regulaciones sobre IQOS en la Región. A medida que las compañías tabacaleras introducen con celeridad nuevos productos de tabaco en países de ingresos bajos y medianos, los defensores de la salud pública y los Estados Parte del Convenio Marco para el Control del Tabaco de la Organización Mundial de la Salud (CMCT de la OMS) deben tomar medidas para evitar la difusión de declaraciones engañosas sobre los productos de tabaco calentado, como IQOS. Dado que los países latinoamericanos se encuentran en diferentes etapas en la regulación de los productos de tabaco calentado, los gobiernos deben cumplir con sus obligaciones estipuladas en el CMCT de la OMS y las recomendaciones de la Conferencia de las Partes mediante la prohibición total de la venta de productos de tabaco calentado o la aplicación estricta a los productos de tabaco calentado de todas las políticas pertinentes sobre la reducción de la demanda de tabaco basadas en el CMCT de la OMS (y asegurarse de abarcar tanto los cigarrillos calentados como los dispositivos de calentamiento).

A Philip Morris International utilizou a decisão de 7 de julho de 2020 da Administração de Alimentos e Fármacos dos Estados Unidos (United States Food and Drug Administration, FDA), que caracterizou o IQOS como produto de tabaco com risco modificado e que permitiu o uso de determinadas alegações de exposição reduzida no marketing do produto, como estratégia corporativa para promover e normalizar seus produtos de tabaco aquecido na América Latina. As decisões relativas aos produtos de tabaco com risco modificado se baseiam no singular sistema regulatório dos EUA, que não é e não deve ser reproduzido em nenhum outro lugar do mundo. A campanha global de relações públicas da Philip Morris International ignorou em grande parte a rejeição da FDA às afirmações de risco reduzido do IQOS e outros achados fundamentais da FDA, que são informações importantes para formuladores de políticas, órgãos regulamentadores e consumidores ­ incluindo usuários de tabaco e clientes da Philip Morris International ­ entenderem os riscos associados ao produto. A Philip Morris International tem usado a mídia para veicular essa informação enganosa ao público, principalmente na América Latina. A empresa também usou a decisão da FDA para pressionar órgãos regulamentadores na América Latina a flexibilizarem a regulamentação do IQOS na região. Conforme as empresas de tabaco introduzem rapidamente novos produtos em países de baixa e média renda, os ativistas de saúde pública e as Partes da Convenção-Quadro para Controle do Tabaco (CQCT) da Organização Mundial da Saúde (OMS) devem tomar providências para prevenir a promoção de alegações enganosas sobre produtos de tabaco aquecido, incluindo o IQOS. Como os países da América Latina estão em diferentes estágios da regulamentação de produtos de tabaco aquecido, os governos devem cumprir suas obrigações com a CQCT da OMS e seguir as recomendações da Conferência das Partes, proibindo totalmente a venda de produtos de tabaco aquecido ou aplicando rigorosamente aos produtos de tabaco aquecido todas as políticas relevantes de redução da demanda por tabaco, com base na CQCT da OMS (certificando-se de abranger tanto os cigarros aquecidos quanto os dispositivos de aquecimento).

Small-molecule inhibitors, immune checkpoint inhibitors, and more: FDA-approved novel therapeutic drugs for solid tumors from 1991 to 2021.

The United States Food and Drug Administration (US FDA) has always been a forerunner in drug evaluation and supervision. Over the past 31 years, 1050 drugs (excluding vaccines, cell-based therapies, and gene therapy products) have been approved as new molecular entities (NMEs) or biologics license applications (BLAs). A total of 228 of these 1050 drugs were identified as cancer therapeutics or cancer-related drugs, and 120 of them were classified as therapeutic drugs for solid tumors according to their initial indications. These drugs have evolved from small molecules with broad-spectrum antitumor properties in the early stage to monoclonal antibodies (mAbs) and antibody‒drug conjugates (ADCs) with a more precise targeting effect during the most recent decade. These drugs have extended indications for other malignancies, constituting a cancer treatment system for monotherapy or combined therapy. However, the available targets are still mainly limited to receptor tyrosine kinases (RTKs), restricting the development of antitumor drugs. In this review, these 120 drugs are summarized and classified according to the initial indications, characteristics, or functions. Additionally, RTK-targeted therapies and immune checkpoint-based immunotherapies are also discussed. Our analysis of existing challenges and potential opportunities in drug development may advance solid tumor treatment in the future.

Pharmacists' Knowledge and Perceptions of FDA Approval Standards and the Breakthrough Therapy Designation.

The "breakthrough therapy" designation (BTD) is a recent mechanism implemented by the United States Food and Drug Administration (FDA) to expedite access to drugs that address unmet needs. The purpose of this study is to describe pharmacists' knowledge of FDA drug-approval standards and knowledge and perceptions of the BTD. Pharmacists engaged in advanced clinical practice were identified through membership profiles of a professional pharmacy organization. Eligible participants were then sent a questionnaire to assess knowledge of FDA approval standards and the BTD. A total of 226 pharmacists responded. The majority of respondents were women (70.2%) and had completed post-graduate training (85.8%). Over half correctly answered at least two of three questions on FDA approval standards (58.1%) and the BTD (78.1%). Only 24.1% of respondents identified as being familiar with the BTD. The majority of pharmacists (62.8%) were certain that FDA-approved "breakthrough" drugs represented a major advance over currently approved therapies and most (88.5%) preferred the drug designated as "breakthrough" in a hypothetical scenario. In conclusion, pharmacists were able to correctly answer questions about FDA approval standards and the BTD. However, they were unfamiliar with the implications of a BTD and may overestimate the benefit demonstrated by these drugs. Future research should identify knowledge gaps in pharmacist understanding of regulatory mechanisms designed to expedite drug approval.