Food And Drug Administration Indications Research Articles

Dapsone Use in Dermatology.

Dapsone, initially synthesized for textile dyeing, gained recognition in the 1930s for its antibacterial properties, leading to its utilization in dermatology for leprosy and dermatitis herpetiformis. Despite US Food and Drug Administration (FDA) approval for these conditions, dapsone's off-label uses have expanded, making it a valuable option in various dermatologic conditions. This review seeks to highlight the common uses of dapsone in its FDA indications and off-label indications. Diseases in which dapsone is considered first-line therapy or adjunctive therapy are reviewed, with highlights from the resources included. An overview of dapsone's pharmacokinetics, pharmacodynamics, indications, dosages, and safety profile are also reviewed. Dapsone's versatility and safety profile make it a cost-effective treatment option in dermatology, particularly for patients with limited access to specialized medications. Ongoing clinical trials are also described exploring dapsone's efficacy in novel dermatologic uses. Dapsone has been a valuable adjunctive therapy across various dermatologic conditions for years and evidence for its use continues to expand.

Retrospective study on the efficacy and tolerability of dose modification of PD-1 and PD-L1 inhibitors in hospital-system community outpatient cancer clinics.

Anti-programmed cell death (PD)-1 and anti-PD-L1 medications inhibit the PD-1 and PD-L1 interaction and have been shown to be effective in treating several forms of advanced cancers. Since the approval of these agents, standard dosing protocols have been utilized. However, a small population of patients in the community setting has received dose-modified PD-1 and PD-L1 inhibitors secondary to a lack of tolerability. Data from this study suggests possible benefit with different dosing strategies. The purpose of this retrospective study is to assess the efficacy and tolerability in terms of time to progression and adverse effects in patients receiving dose-modified PD-1 and PD-L1 inhibitors in Food and Drug Administration (FDA)-labeled indications. This single-institution retrospective chart review was conducted in an outpatient community setting on patients with cancer that received nivolumab, pembrolizumab, durvalumab, or atezolizumab for an FDA indication at one of the Houston Methodist Hospital infusion clinic site between September 1, 2017 and September 30, 2019. Data collection included demographics, adverse effects, dosing, treatment delay, and number of immunotherapy cycles administered per patient. This study included 221 patients, who received either nivolumab (n = 81), pembrolizumab (n = 93), atezolizumab (n = 21), or durvalumab (n = 26). There were 11 patients who experienced a dose reduction and 103 patients who experienced a treatment delay. Of the patients with a treatment delay, the median time to progression was 197 days, and for patients with a dose reduction, the median time to progression was 299 days. The results of this study found that the immunotherapy associated adverse effects led to dosing and frequency changes for tolerance with continued therapy. Our data suggests that there could be potential benefits of dose modifications to immunotherapy treatment, but further large studies are needed to assess the efficacy of specific immunotherapy dose modifications on both outcomes and adverse effects.

Antifungal drug price increases in the United States, 2000-2019.

BackgroundAntifungal drugs treat a variety of conditions, ranging from localised dermatologic disease to life‐threatening systemic infections. Some common antifungal drugs experienced large price increases in recent years, however, factors contributing to these price increases are poorly understood. We sought to examine trends in antifungal drug prices and determine underlying drivers of price changes.MethodsAntifungal drug products in the United States were identified using the Food and Drug Administration (FDA) Label database. For each product, we determined the wholesale acquisition cost per unit over time between 2000 and 2019, adjusting for inflation, and examined variables that could impact price: route of administration, number of FDA indications, the quantity of professional guideline recommendations, use for prophylaxis, number of FDA‐approved manufacturers, and whether it was compounded. Price trajectories were clustered into four groups: (1) stable, 2) moderate, (3) high, and (4) extreme price increases.ResultsOf 139 identified drug products, one outlier was removed due to exorbitant price increases. Cluster 1 (n = 31) demonstrated the most stable prices with a 25% mean price increase. Clusters 2 (n = 97), 3 (n = 7), and 4 (n = 3) demonstrated moderate, high, and extreme price increases with 52%, 318%, and 900% mean price increases, respectively. Atypical routes of administration and compounding were over‐represented in clusters 3 and 4. There was no correlation between the number of manufacturers and price changes.ConclusionsAntifungal drugs exhibited large, inflation‐adjusted price increases. Atypical routes of administration and compounding were over‐represented within clusters exhibiting extraordinary price increases. Our data support policies aiming to curb large price increases for medically important drugs.

Risks and Benefits of Cannabis and Cannabinoids in Psychiatry.

The United States is in the midst of rapidly changing laws regarding cannabis. The increasing availability of cannabis for recreational and medical use requires that mental health clinicians be knowledgeable about evidence to be considered when counseling both patients and colleagues. In this review, the authors outline the evidence from randomized double-blind placebo-controlled trials for therapeutic use of cannabinoids for specific medical conditions and the potential side effects associated with acute and chronic cannabis use. Searches of PubMed and PsycInfo were conducted for articles published through July 2021 reporting on "cannabis" or "cannabinoids" or "medicinal cannabis." Additional articles were identified from the reference lists of published reviews. Articles that did not contain the terms "clinical trial" or "therapy" in the title or abstract were not reviewed. A total of 4,431 articles were screened, and 841 articles that met criteria for inclusion were reviewed by two or more authors. There are currently no psychiatric indications approved by the U.S. Food and Drug Administration (FDA) for cannabinoids, and there is limited evidence supporting the therapeutic use of cannabinoids for treatment of psychiatric disorders. To date, evidence supporting cannabinoid prescription beyond the FDA indications is strongest for the management of pain and spasticity. As cannabinoids become more available, the need for an evidence base adequately evaluating their safety and efficacy is increasingly important. There is considerable evidence that cannabinoids have a potential for harm in vulnerable populations such as adolescents and those with psychotic disorders. The current evidence base is insufficient to support the prescription of cannabinoids for the treatment of psychiatric disorders.

Determining Cochlear Implant Candidacy in Adults: Limitations, Expansions, and Opportunities for Improvement.

The safety, efficacy, and success of cochlear implants (CIs) are well established and have led to changes in criteria used by clinicians to determine who should receive a CI. Such changes in clinical decision-making have out-paced the slower-occurring changes that have taken place with regulatory bodies' and insurers' indications. We review the historical development of indications for CIs, including those of the U.S. Food and Drug Administration (FDA), Medicare, Medicaid, and private insurers. We report on expansion to include patients with greater residual hearing, such as those who receive Hybrid and EAS devices, and report on recent FDA approvals that place less emphasis on the patient's best-aided condition and greater emphasis on the ear to be treated. This includes expansion of CIs to patients with single-side deafness and asymmetric hearing loss. We review changes in the test materials used to determine candidacy, including transition from sentences in quiet to sentences in noise to the recent use of monosyllabic words and cognitive screening measures. Importantly, we discuss the recent trend to recommend CIs despite a patient not meeting FDA or insurers' indications (a practice known as “off-label”), which serves as attestation that current indications need to be updated.

605. Factors Underlying Antifungal Price Trends in the United States

BackgroundAntifungal drugs are used to treat conditions ranging from topical dermatologic disease to life-threatening systemic infections. Given their widespread use, understanding factors that affect antifungal pricing will help clinical and policy-related decisions. The purpose of this study was to identify factors associated with price trends among antifungal drugs.MethodsAntifungal drug products available in the United States were identified using the Food and Drug Administration (FDA) Orange Book database. Multiple characteristics related to how a drug may be priced were identified using a variety of sources, including the number of FDA indications (IBM Micromedex), quantity of professional guideline recommendations and use as prophylaxis (Infectious Diseases Society of America guidelines for treatment of fungal infections), and route of administration. Wholesale acquisition cost per unit was identified for each drug from the first date listed through 2019 using the Medi-Span and First Databank databases. Statistical analysis was performed using R (version 3.6.3). Price trajectories over time observed on 138 antifungal drugs were clustered into three groups by the shape of their trajectory using the R package KmlShape (Genolini 2016). Clusters were characterized by stable prices, moderate price increases, and extreme price increases over time. Relationships between cluster membership and drug characteristics were assessed using Fisher’s Exact Test and Likelihood Ratio Tests. All tests were conducted at the 5% level of significance.ResultsThe majority (n=116) of antifungal drugs were characterized by moderate price increase and an additional n=6 exhibited extreme price increases over time. Few (n=16) were stable. Associations were identified between drug characteristics and extreme price increase cluster membership. These included a fewer number of FDA indications (p=0.0028) and atypical route of administration (p=0.0025). No association was identified between cluster membership and quantity of guideline recommendations, or use as prophylaxis.ConclusionNearly 90% of antifungal drugs exhibited at least moderate price increases over time. Extreme price increases were associated with a fewer number of FDA indications and atypical route of administration.Disclosures All Authors: No reported disclosures

Patient Selection in Cervical Disc Arthroplasty.

Patient selection for cervical disc arthroplasty (CDA) in the United States remains a topic of debate among surgeons. Many surgeons base US patient selection for CDA implantation on the Food and Drug Administration (FDA) indications/contraindications. While off-label use does occur, the frequency and extent of off-label use in the US remains largely unknown. Outside the United States, patient selection is notably less stringent; however such data also remain largely unpublished or presented/published with a low level of evidence. Here, we will review the current approved US on-label patient selection criteria for CDA and discuss the rationale and supporting evidence to expand these criteria in the United States. A PubMed literature search was completed using the keywords "cervical disc arthroplasty" and "cervical disc replacement." The articles were evaluated by the authors for patient selection criteria. The current published data do not conclusively prove that the patients excluded from CDA by strict adherence to FDA indications would benefit from CDA surgery over anterior cervical discectomy and fusion. As surgeons, it is a difficult decision regarding when to expand indications to include off-label use of CDA. In our practice, generally CDA patient selection agrees with the FDA indications and contraindications, as there is a lack of level 1 evidence to confirm effectiveness of CDA outside of the current FDA indications. We will likely need more well-constructed studies to include prospective and controlled trials that specifically evaluate the "off-label" applications before US surgeons are convinced to expand indications and insurance companies agree to reimburse.

The African Green Monkey Model of Pneumonic Plague and US Food and Drug Administration Approval of Antimicrobials Under the Animal Rule.

Additional treatment options for pneumonic plague, the most severe form of infection by Yersinia pestis, are needed, as past US Food and Drug Administration (FDA) approvals were not based on clinical trials that meet today's standards, and multiple drugs are sought to counter resistance or use in special populations. Due to the sporadic nature of outbreaks and the low number of pneumonic cases of disease, we sought FDA approval of antimicrobials for treatment under the Animal Efficacy Rule, where efficacy can be demonstrated in 1 or more well-characterized animal models that sufficiently represent human disease. A model was developed in African green monkeys (AGMs) after challenge with a lethal dose of Y. pestis delivered as an aerosol, in 4 independent studies in 3 laboratories. The primary data points were bacteremia (daily), body temperature and heart rate (continuously monitored by telemetry), and survival. In antimicrobial efficacy studies, human-equivalent doses of gentamicin, ciprofloxacin, levofloxacin, and doxycycline were administered upon fever onset for 10 days. Disease in AGMs was similar to case reports of human disease. Fever was determined to be a reliable sign of disease and selected as a treatment trigger. Gentamicin was 60%-80% effective depending on the dose given to animals. Ciprofloxacin and levofloxacin were found to be >90% efficacious. These data were submitted to FDA and plague indications were approved. Doxycycline was less effective. The AGM model of pneumonic plague is reproducible, well-characterized, and mimics human disease. It has been used to support plague indications for fluoroquinolones and to test the efficacy of additional antimicrobials.

Targeted Therapy for Advanced Thyroid Cancer: Kinase Inhibitors and Beyond.

The treatment of advanced thyroid cancer has undergone rapid evolution in the last decade, with multiple kinase inhibitor drug approvals for each subtype of thyroid cancer and a number of other commercially available drugs that have been studied for this indication. Although most of the US Food and Drug Administration (FDA)-approved drugs are antiangiogenic multikinase inhibitors-vandetanib, cabozantinib, sorafenib, lenvatinib-there are two FDA indications that are mutation specific-dabrafenib/trametinib for BRAF-mutated anaplastic thyroid cancer and larotrectinib for NTRK-fusion thyroid cancer. Furthermore, other mutation-specific drugs, immunotherapies, and novel strategies for advanced thyroid cancer are under investigation. Understanding the molecular basis of thyroid cancer, the drugs of interest for treatment of advanced thyroid cancer, and how these drugs can be administered safely and in the appropriate clinical scenario are the topics of this review.

Dabrafenib and trametinib in patients with tumors with BRAF V600E/K mutations: Results from the molecular analysis for therapy choice (MATCH) Arm H.

3002 Background: The NCI-MATCH precision medicine trial assigns patients (pts) with solid tumors, lymphomas, or multiple myeloma with progression on prior treatment to a targeted therapy based on genetic alterations identified in pre-treatment biopsies. Arm H (EAY131-H) evaluated the combination of the BRAF inhibitor (inh) dabrafenib (DAB), and the MEK inh, trametinib (TRM), in pts with BRAF V600E/K mutations. Methods: Pts with melanoma, thyroid, or colorectal cancer were excluded. Pts with NSCLC were excluded after the U.S. Food and Drug Administration (FDA) approved DAB/TRM for this indication. Pts received DAB 150 mg po BID and TRM 2 mg PO daily on 28 day cycles until disease progression or intolerable toxicity; restaging was performed every 2 cycles. The primary endpoint was objective response rate (ORR); secondary endpoints included progression-free survival (PFS), 6-month PFS, and overall survival (OS). Results: A total of 35 pts were enrolled from 1/2016-2/2018; 2 were ineligible (CrCl below criteria; labs out of window). Over 17 distinct tumor histologies were represented. 58% of pts were female, median age was 63 (range 21-85), 94% were Caucasian, and 48% of pts had received at least 3 prior therapies (range 1- 8). The confirmed ORR was 33.3% (90% CI 19.9%, 49.1%), with a median duration of response (DoR) of 12 months (mon). Varied histologies had a DoR of > 12 mon: histiocytic sarcoma, cholangiocarcinoma and mixed adenoneuroendocrine carcinoma of unknown primary, among others. Median PFS was 9.4 mon; the 6 mon PFS rate was 70.6% (90% CI 58.2%-85.5%), and an additional 10 pts had a PFS > 5.5 mon. Median OS has not been reached. At the time of data cutoff (12/2018) 11 pts continue on treatment. Adverse events (AE) were comparable to previously reported profiles of DAB/TRM; no new AEs were identified. The most frequent grade 3 AEs were fatigue, neutropenia, hyponatremia, hypophosphatemia, and urinary tract infection; there was 1 grade 4 sepsis; no grade 5 AEs. Conclusions: In this pre-treated, mixed histology cohort, DAB and TRM showed promising activity outside of currently approved FDA indications warranting further investigations. Correlative analyses are planned. Clinical trial information: NCT02465060.

1880. Does the Label Matter: Initial Use of Newly Approved Antimicrobial Agents in Community Hospitals Without Robust Antimicrobial Stewardship Programs

BackgroundAntimicrobial agents are often used for indications not approved by the Food and Drug Administration (FDA). While this can be appropriate at times, they may be used in settings with no published experience. Judicious use of new agents is critical for conserving their utility. This study characterized initial use after FDA approval of select antimicrobial agents in community hospitals without robust antimicrobial stewardship programs (ASP).MethodsInitial use of systemic antimicrobials approved by the FDA since 2014 was retrospectively reviewed at 6 community hospitals (50–350 beds). Up to 10 charts of first use were reviewed per drug at each hospital. Time from FDA approval to first administration was measured for the following agents: ceftazidime–avibactam, ceftolozane–tazobactam, dalbavancin, isavuconazonium, oritavancin, and peramivir. Clinical indications, prescribing service, and empiric uses were recorded.ResultsMean time from FDA approval to first administration ranged from 12 (tedizolid) to 26 months (dalbavancin). Of frequently used agents (Figure 1), adherence to initial FDA indications ranged from 7% (ceftolozane–tazobactam for complicated urinary tract infection) to 100% (tedizolid for acute bacterial skin and soft-tissue infection). Pneumonia (35%) and osteomyelitis (35%) were the most common off-label indications for ceftazidime–avibactam. Pneumonia was the most common off-label indication for ceftolozane–tazobactam (78%). The most common off-label indications for oritavancin were osteomyelitis (14%) and bacteremia (11%). Infectious Diseases was the main prescribing service for all agents (range 74–95%). Use of Gram-positive agents was mostly empiric whereas Gram-negative agents were targeted against specific pathogens. ConclusionNewly approved antimicrobial agents were used at these six community hospitals within 1–2 years after FDA approval. Agents with primarily Gram-positive activity were more often used for FDA approved indications. Given frequent use of novel Gram-negative agents for pneumonia, there is need for early trials to determine their role for this indication. In the meantime, ASP’s should consider off-label indications such as pneumonia when developing local criteria for use.Disclosures All authors: No reported disclosures.

Trazodone: A Review of Its Pharmacological Properties and Its Off-Label Use in Dogs and Cats

Trazodone is approved by the Food and Drug Administration (FDA) for the treatment of depression and anxiety in humans, it can perform these functions due to its selective serotonin receptor antagonism and reuptake inhibition properties. Trazodone has not been approved for animals by the FDA but it is legally prescribed by veterinarians as an off-label drug for dogs and cats. This review describes trazodone’s mechanism of action, pharmacokinetic properties and adverse effects and summarizes the benefits of trazodone for non-approved FDA indications to reduce anxiety, behavioral stress, sedation, pre-and post-operation stress. It also discusses its cardiac safety profile for dogs and cats. As few clinical trials have been conducted to evaluate trazodone’s efficacy for treatment of the many behavioral and medical conditions in animals, large and randomized controlled clinical trials are still needed to confirm its efficacy in clinical practice.

Guideline Watch (October 2014): Practice Guideline for the Treatment of Patients With Alzheimer's Disease and Other Dementias.

(Reprinted with permission from American Psychiatric Association, http://psychiatryonline.org/guidelines).

Lucentis® (Ranibizumab Ophthalmic Injection) for Diabetic Macular Edema (DME)

Objective: To review the efficacy, safety and pharmacologic parameters of ranibizumab (Lucentis®) intravitreal ophthalmic injection versus alternative agents in the management of diabetic macular edema (DME). Data sources: Information was reviewed from: MEDLINE (January 2000-December 2015), both manufacturers of ranibizumab (Genentech and Novartis), and the United States (U.S.) Food and Drug Administration (FDA). The internet was searched using the following key terms: avastin, diabetic macular edema (DME), eye, injection, intravitreal, lucentis, microvascular, ophthalmic, pharmacologic, ranibizumab, and retinopathy. Study selection and data extraction: All English-language articles and abstracts were evaluated for inclusion. Appropriate, randomized controlled trials in DME are included in this review. Data synthesis: The safety and efficacy of ranibizumab intravitreal ophthalmic injection (0.3 mg every 28-days) versus (0.5 mg every 28-days) versus placebo was evaluated in two randomized, double-masked studies in patients with DME over 24-36 months. Patients were between 21-91 years old (mean age 62); all patients were eligible for additional types of treatment, if needed, beginning month three of the studies [i.e., macular laser treatment or pan retinal photocoagulation (PRP)]. At month 24, compared to monthly ranibizumab 0.3 mg, no additional benefit was observed with monthly treatment with the ranibizumab 0.5 mg dose; therefore, outcomes measured in months 24-36 were ranibizumab 0.3 mg vs. placebo and based on changes in visual acuity. They were defined as: a. Gain of greater than or equal (>) to15 letters b. Loss of less than (<) 15 letters visual acuity and c. Mean change in visual acuity. Conclusions: Ranibizumab (Lucentis®) intravitreal ophthalmic injection 0.3 mg given every 28 days was shown to be safe and effective for the management of diabetic macular edema (DME) and is the first and only FDA approved pharmacological choice. Ranibizumab received its fourth FDA indication for treatment of serious eye diseases in February, 2015. It is now approved for the treatment of diabetic retinopathy in those with DME.

Use of Integrase Inhibitors in HIV-Infected Children and Adolescents.

Resistance to antiretroviral drugs is an increasingly prevalent challenge affecting both the adult and pediatric HIV-infected populations. Though data on the safety, pharmacokinetics, and efficacy of newer antiretroviral agents in children typically lags behind adult data, newer agents are becoming available for use in HIV-infected children who are failing to respond to or are experiencing toxicities with traditional antiretroviral regimens. Integrase strand transfer inhibitors are one such new class of antiretrovirals. Raltegravir has been US Food and Drug Administration (FDA) approved for use in patients over the age of 4 weeks. Elvitegravir is a second member of this class, and has the potential for use in children but does not yet have a Pediatric FDA indication. Dolutegravir, a second-generation integrase inhibitor, is approved for those older than 12 years. This review summarizes the use of integrase inhibitors in children and adolescents, and highlights the results of recent clinical trials.

Antipsychotic medication prescribing in children enrolled in Medicaid.

Although the increase in treatment of children and adolescents with antipsychotic medications has been well documented, much less is known about the factors related to the use of these agents and how closely the treatment follows best practice recommendations. Prescribers of each antipsychotic medication prescription issued for a Medicaid-insured child in Vermont aged <18 years were sent a prior authorization survey that assessed several domains including the clinical indication, other treatments, metabolic monitoring, prescriber specialty, and prescription origin. These variables were combined to categorize prescriptions as following indications approved by the US Food and Drug Administration (FDA) and best practice guidelines. The response rate of the survey was 80%, with 677 surveys from 147 prescribers available for analysis; more than one-half of the respondents were primary care clinicians. Overall, the clinical indication for an antipsychotic prescription followed best practice guidelines 91.7% of the time, with overall best practice guidelines followed at a rate of 50.1%. An FDA indication was followed in 27.2% of cases. Psychiatrists were significantly more likely to follow best practice guidelines than nonpsychiatrists. Antipsychotic medications were typically used only after other pharmacologic and nonpharmacologic treatments were ineffective, although previous treatment with cognitive-behavioral therapy was uncommon (15.5%). Metabolic monitoring that included serial laboratory tests was reported in 57.2% of cases. Current prescribing patterns of antipsychotic medications for children and adolescents follow best practice guidelines approximately one-half of the time, with nonadherence often related to lack of metabolic monitoring.

Ceftaroline Fosamil for the Treatment of Community-Acquired Pneumonia: from FOCUS to CAPTURE.

Ceftaroline fosamil (ceftaroline hereafter) is the latest addition to the armamentarium for the treatment of patients with community-acquired pneumonia (CAP). It is currently approved by the Food and Drug Administration (FDA) for community-acquired bacterial pneumonia (CABP), which is a recent FDA indication that centers on individuals with documented bacterial pneumonias that arise in the community setting. The purpose of this review is to summarize and discuss the major findings from the Phase III CAP clinical trials as well as the clinical experience with ceftaroline among patients with CAP in the “Ceftaroline Assessment Program and Teflaro® Utilization Registry” (CAPTURE). In its two Phase III CAP trials, ceftaroline was compared to ceftriaxone among adults with radiographically confirmed CAP requiring hospitalization who were classified as Pneumonia Outcomes Research Team (PORT) risk class III or IV. Among patients with CAP, clinical success at test of cure was 84.3% vs 77.7% (difference 6.6%, 95% confidence interval [CI]: 1.6–11.8%) in those treated with ceftaroline and ceftriaxone, respectively, across the two Phase III clinical trials. Among patients with a culture-confirmed CABP, day 4 response rates were numerically higher, albeit non-significant, among patients that received ceftaroline vs. ceftriaxone (69.5% for ceftaroline vs. 59.4% for ceftriaxone, difference 10.1%, 95% CI, −0.6% to 20.6%). The efficacy of ceftaroline is supported by real-world observational data from CAPTURE for patients with both CAP and CABP. In addition, the CAPTURE program afforded an opportunity to assess the outcomes of patients who were excluded or limited in the original Phase III trials in a non-comparative fashion. These underrepresented patient populations with CAP included: patients that received prior antibiotics, patients in the ICU, patients with severe renal dysfunction, and those with methicillin-resistant Staphylococcus aureus (MRSA) isolated from respiratory or blood culture. As CAPTURE is a retrospective, non-comparator convenience sample registry, all the findings need to be interpreted with caution.Electronic supplementary materialThe online version of this article (doi:10.1007/s40121-014-0036-8) contains supplementary material, which is available to authorized users.

Toward Personalized Medicine in the Pharmacotherapy of Alcohol Use Disorder: Targeting Patient Genes and Patient Goals

Toward Personalized Medicine in the Pharmacotherapy of Alcohol Use Disorder: Targeting Patient Genes and Patient Goals

Use of Administrative Data to Identify Off-Label Use of Second-Generation Antipsychotics in a Medicaid Population

OBJECTIVE The use of second-generation antipsychotics for conditions not approved by the U.S. Food and Drug Administration (FDA) is a prevalent phenomenon with important implications. The objective of this study was to determine the accuracy of administrative claims for identifying off-label use of second-generation antipsychotics in a Medicaid population in 2009. METHODS The authors estimated the sensitivity, specificity, positive predictive values (PPV), and negative predictive values of Medicaid claims data for detecting off-label use of second-generation antipsychotics in the electronic health records of 788 patients. Separate estimates were calculated for patients without schizophrenia and bipolar disorder, the two most long-standing FDA indications for use of second-generation antipsychotics, and for a subset of patients using a second-generation antipsychotic with indications for treatment-resistant depression. RESULTS Medicaid claims determined a lack of schizophrenia and bipolar disorder in the medical record with a sensitivity of 72% and a specificity of 85%. The prevalence of identifying neither diagnosis was 83%, which was associated with a predictive ability (PPV) of 96%. Among those using a second-generation antipsychotic with an indication for treatment-resistant depression, the sensitivity, specificity, and PPV of Medicaid claims for identifying off-label use were 41%, 86%, and 87%, respectively. CONCLUSIONS Medicaid claims data had high predictive ability for identifying users of second-generation antipsychotics who did not have documentation of schizophrenia or bipolar disorder in the medical record. The predictive utility of the claims was diminished when the analyses were limited to patients using a second-generation antipsychotic with an indication for treatment-resistant depression.

Commentary

Commentary