The Major Role of Clinicians in the Discovery of Off-Label Drug Therapies

Objective: To determine, through a review of the medical literature and author contact, the role of clinicians in the discovery of off label use of Food and Drug Administration approved prescription drugs. Data Sources: The literature was accessed through MEDLINE (1999-December 2003). Additional sources accessed included the U.S. Patent Office and Micromedex, Thompson Scientific and Healthcare, Inc. Data Synthesis: A survey of new therapeutic uses for New Molecular Entity drugs approved in 1998 was conducted for the subsequent 5 years of commercial availability. During that time period, a total of 144 new applications were identified in a computerized search of the literature for the 29 new drugs approved in 1998. Literature and patent searches were conducted to identify the first report of each new application. Authors of the seminal articles were contacted via survey and telephone interview to determine whether they were in fact the originators of the new applications. If they were, examinations of article contents and author surveys were used to explore whether each new application was discovered via clinical practice that was independent of pharmaceutical company or university research (field discovery) or whether the discovery was made by or with the involvement of pharmaceutical firm or university researchers (central discovery). Conclusions: Post-NDA discoveries of new, off-label uses for new drugs were present in 22 of the 29 drugs in our sample. We found that 59% (85/144) of the drug therapy innovations in our sample were discovered by practicing clinicians via field discovery. The major role of clinicians in the discovery of new, off-label drug therapies has not been previously documented or explored. We propose that this finding has important regulatory and health policy implications

Reducing Medical Costs and Improving Quality via Self-Management Tools

The authors consider the advantages and limitations of self-management tools used for treating chronic illness

Beyond the EPR: Complementary roles of the hospital-wide electronic health record and clinical departmental systems

<p>Abstract</p> <p>Background</p> <p>Many hospital departments have implemented small clinical departmental systems (CDSs) to collect and use patient data for documentation as well as for other department-specific purposes. As hospitals are implementing institution-wide electronic patient records (EPRs), the EPR is thought to be integrated with, and gradually substitute the smaller systems. Many EPR systems however fail to support important clinical workflows. Also, successful integration of systems has proven hard to achieve. As a result, CDSs are still in widespread use. This study was conducted to see which tasks are supported by CDSs and to compare this to the support offered by the EPR.</p> <p>Methods</p> <p>Semi-structured interviews with users of 16 clinicians using 15 different clinical departmental systems (CDS) at a Medium-sized University hospital in Norway. Inductive analysis of transcriptions from the audio taped interviews.</p> <p>Results</p> <p>The roles of CDSs were complementary to those of the hospital-wide EPR system. The use of structured patient data was a characteristic feature. This facilitated quality development and supervision, tasks that were poorly supported by the EPR system. The structuring of the data also improved filtering of information to better support clinical decision-making. Because of the high value of the structured patient data, the users put much effort in maintaining their integrity and representativeness. Employees from the departments were also engaged in the funding, development, implementation and maintenance of the systems.</p> <p>Conclusion</p> <p>Clinical departmental systems are vital to the activities of a clinical hospital department. The development, implementation and clinical use of such systems can be seen as bottom-up, user-driven innovations.</p

Pharmacotherapy of Schizophrenic Patients: Preponderance of Off-Label Drug Use

Multiple drug class combinations are often prescribed for the treatment of schizophrenia, although antipsychotic monotherapy reflects FDA labeling and scientific justification for combinations is highly variable. This study was performed to gain current data regarding drug treatment of schizophrenia as practiced in the community and to assess the frequencies of off-label drug class combinations. 200 DSM IV-diagnosed schizophrenic patients recruited from community treatment sources participated in this cross-sectional study of community based schizophrenic patients. Drug class categories include First and Second Generation Antipsychotic drugs (FGA and SGA, respectively), mood stabilizers, antidepressants and anti-anxiety drugs. 25.5% of patients received antipsychotic monotherapy; 70% of patients received an antipsychotic and another drug class. A total of 42.5% of patients received more than one antipsychotic drug. The most common drug class combination was antipsychotic and a mood stabilizer. Stepwise linear discriminant function analysis identified the diagnosis of schizoaffective schizophrenia, history of having physically hurt someone and high scores on the General Portion of the PANSS rating scale predicted the combined use of an antipsychotic drug and a mood stabilizer. “Real world” pharmacotherapy of schizophrenia has developed its own established practice that is predominantly off-label and may have outstripped current data support. The economic implications for public sector payers are substantial as well as for the revenue of the pharmaceutical industry, whose promotion of off-label drug use is an increasingly problematic. These data are consistent with the recognition of the therapeutic limitations of both first and second generation antipsychotic drugs

Market failure in the diffusion of cliniciandeveloped innovations : The case of off-label drug discoveries

Medical doctors occasionally discover potentially valuable new off-label uses for drugs during their clinical practice. They apply these to help their own patients, but often have minimal incentives to invest in diffusing them further. Thus, the benefits that other clinicians might obtain are to some extent an externality from the perspective of the discoverer. This represents a form of market failure: effort invested in diffusion could lower adoption costs for many, but few innovators will invest that effort and social welfare will be accordingly reduced. In this study we explore for empirical evidence for the market failure just described, and do find evidence for it. In a sample of US clinicians, diffusion efforts increase the diffusion of generally valuable discoveries, but innovating clinicians typically invest little to support diffusion. We conclude with a discussion of how such a market failure could be addressed