How Oncologists Perceive the Availability and Quality of Information Generated From Patient-Reported Outcomes (PROs).

BACKGROUND: Despite increased incorporation of patient-reported outcome (PRO) measures into clinical trials, information generated from PROs remains largely absent from drug labeling and electronic health records, giving rise to concerns that such information is not adequately informing clinical practice. OBJECTIVE: To evaluate oncologists' perceptions concerning the availability and quality of information generated from PRO measures. Additionally, to identify whether an association exists between perceptions of availability and attitudes concerning quality. METHOD: An online, 11-item questionnaire was developed to capture clinician perspectives on the availability and use of PRO data to inform practice. The survey also asked respondents to rate information on the basis of 4 quality metrics: "usefulness," "interpretability," "accessibility," and "scientific rigor." RESULTS: Responses were received from 298 of 1301 invitations sent (22.9% response rate). Perceptions regarding the availability of PRO information differed widely among respondents and did not appear to be linked to practice setting. Ratings of PRO quality were generally consistent, with average ratings for the 4 quality metrics between "satisfactory" and "good." A relationship was observed between ratings of PRO data quality and perceptions of the availability. CONCLUSION: Oncologists' attitudes toward the quality of information generated from PRO measures are favorable but not enthusiastic. These attitudes may improve as the availability of PRO data increases, given the association we observed between oncologists' ratings of the quality of PRO information and their perceptions of its availability.

Building a drug development database: challenges in reliable data availability.

CONTEXT: Policy and legislative efforts to improve the biomedical innovation process must rely on a detailed and thorough analysis of drug development and industry output. OBJECTIVE: As part of our efforts to build a publicly-available database on the characteristics of drug development, we present work undertaken to test methods for compiling data from public sources. These initial steps are designed to explore challenges in data extraction, completeness and reliability. Specifically, filing dates for Investigational New Drugs (IND) applications with the U.S. Food and Drug Administration (FDA) were chosen as the initial objective data element to be collected. MATERIALS AND METHODS: FDA's Drugs@FDA database and the Federal Register (FR) were used to collect IND dates for the 587 New Molecular Entities (NMEs) approved between 1994 and 2014. When available, the following data were captured: approval date, IND number, IND date and source of information. RESULTS: At least one IND date was available for 445 (75.8%) of the 587 NMEs. The Drugs@FDA database provided IND dates for 303 (51.6%) NMEs and the FR contributed with 297 (50.6%) IND dates. Out of the 445 NMEs for which an IND date was obtained, 274 (61.6%) had more than one date reported. DISCUSSION: Key finding of this paper is a considerable inconsistency in reliably available or reported data elements, in this particular case, IND application filing dates as assembled from publicly-available sources. CONCLUSION: Our team will continue to focus on finding ways to collect relevant information to measure impact of drug innovation.

Awareness of nonalcoholic steatohepatitis and associated practice patterns of primary care physicians and specialists.

BACKGROUND: The hepatic manifestation of metabolic syndrome is nonalcoholic fatty liver disease. Patients with nonalcoholic steatohepatitis, the progressive form of nonalcoholic fatty liver disease, have increased risk of fibrosis, cirrhosis and end-stage liver disease. Estimates of prevalence in the United States range from 20-30% for nonalcoholic fatty liver disease and 2-5% for nonalcoholic steatohepatitis; however, physician awareness of these diseases is limited. The purpose of this study was to determine the current level of physician awareness and practices in the diagnosis and management of nonalcoholic fatty liver disease and nonalcoholic steatohepatitis within the United States. METHODS: Physicians were asked to participate in an online, 35-question survey about their awareness of various liver conditions and current practices. RESULTS: Of the 302 responding physicians, 152 were primary care physicians, and 150 were specialists (comprised of gastroenterologists and hepatologists). More specialists than primary care physicians reported that they were aware of the differences between nonalcoholic fatty liver disease and nonalcoholic steatohepatitis (p < 0.001) and that they routinely screened for nonalcoholic fatty liver disease (p < 0.001) and nonalcoholic steatohepatitis (p < 0.001). Almost half of the responding primary care physicians reported being unfamiliar with the nonalcoholic fatty liver disease and nonalcoholic steatohepatitis differences even though they were aware of both, yet 58% of those primary care physicians were treating patients with nonalcoholic fatty liver disease and/or nonalcoholic steatohepatitis. In addition, those primary care physicians who reported being unfamiliar with nonalcoholic steatohepatitis were treating an average of 3.7 patients and reported being as likely as familiar primary care physicians to treat new patients with nonalcoholic steatohepatitis. More than half of the specialists used noninvasive diagnostic test to confirm nonalcoholic steatohepatitis, and 10% of the specialists reported treating patients with drugs not recommended by the current guidelines. CONCLUSIONS: Despite reporting they were not familiar with nonalcoholic steatohepatitis, primary care physicians reported they would likely continue to diagnose and manage patients with nonalcoholic steatohepatitis; therefore, more physician education on the recent practice guideline for nonalcoholic fatty liver disease and nonalcoholic steatohepatitis is needed.

Self-reported physician practices in pulmonary arterial hypertension: Diagnosis, assessment, and referral.

BACKGROUND: Numerous clinical trials have contributed to rapid advancements in the diagnosis and management of pulmonary arterial hypertension (PAH), yet patients often do not undergo right heart catheterization (RHC) with vasoreactivity testing and may receive a delayed or incorrect diagnosis. Efforts to improve standards of care include the designation of Pulmonary Hypertension Association (PHA)-Accredited PH Care Centers (PHCCs). This study evaluated current practices in the diagnosis and assessment of PAH. METHODS: A survey of 167 physicians who had ≥1 claim for PAH in the past 3 months was conducted. RESULTS: Of 167 respondents, 15% were affiliated with a PHCC, 40% had referred ≥1 patient with diagnosed PAH, and 79% had ≥1 patient referred to them by another physician who they then newly diagnosed with PAH. More than half (52%) reported having ≥1 patient who was previously misdiagnosed with PAH referred to them by another physician. RHC and vasoreactivity testing, respectively, were performed in 43% and 33% of patients with PAH who respondents referred to another physician, 86% and 67% of patients newly diagnosed by respondents, and 84% and 57% of patients who respondents considered accurately diagnosed prior to being referred to them. Respondents affiliated with a PHCC were more likely to try to refer to another physician affiliated with a PHCC, and to perform RHC and vasoreactivity testing. CONCLUSIONS: Self-reported clinical practices often deviated from established guidelines. Future research should focus on both clinical efficacy and ways to encourage clinicians to bring their practices in line with well-supported, evidence-based recommendations.

Idiopathic pulmonary fibrosis: Physicians' perceptions of patient treatment with recently approved drugs.

Idiopathic pulmonary fibrosis (IPF) is a rare, chronic and ultimately fatal disease for which only palliative treatments existed until recently. Between 2011 and 2015, two new drugs, pirfenidone and nintedanib, were approved in the US and Europe for the treatment of IPF, providing hope for patients. The objectives of our work were to understand physicians' expected use of these new treatments in the US and Europe, and to estimate their potential. To achieve this goal, we conducted surveys amongst US and European Union (EU) pulmonologists caring for patients with IPF. There was a significant difference between EU and US physicians in the treatment of patients with mild disease with pirfenidone; the EU physicians anticipated using pirfenidone for 57% of their patients with mild disease, whereas the US pulmonologists anticipated using it for 34% of their patients (p = 0.01). Regarding patients with severe disease, the US pulmonologists anticipated treating 74% with either pirfenidone (46%) or nintedanib (28%), whereas the EU pulmonologists treated 28% with pirfenidone and anticipated treating 20% with nintedanib. These findings suggest treatment with pirfenidone and nintedanib based on disease severity may vary between US and EU physicians, which may affect patient outcomes.

Improving pharmaceutical innovation by building a more comprehensive database on drug development and use.

New drugs and biologics have had a tremendous impact on the treatment of many diseases. However, available measures suggest that pharmaceutical innovation has remained relatively flat, despite substantial growth in research and development spending. We review recent literature on pharmaceutical innovation to identify limitations in measuring and assessing innovation, and we describe the framework and collaborative approach we are using to develop more comprehensive, publicly available metrics for innovation. Our research teams at the Brookings Institution and Deerfield Institute are collaborating with experts from multiple areas of drug development and regulatory review to identify and collect comprehensive data elements related to key development and regulatory characteristics for each new molecular entity approved over the past several decades in the United States and the European Union. Subsequent phases of our effort will add data on downstream product use and patient outcomes and will also include drugs that have failed or been abandoned in development. Such a database will enable researchers to better analyze the drivers of drug innovation, trends in the output of new medicines, and the effect of policy efforts designed to improve innovation.