ABSTRACT
BACKGROUND: Single patient Investigational New Drug (IND) applications are one mechanism through which experimental therapies are accessed for children with cancer. The landscape of use, outcomes, and toxicity from single patient INDs remains unknown in pediatric oncology. METHODS: We performed a retrospective analysis of all single patient INDs requested and prescribed at a single institution between 1/1/2007 and 5/1/2019. We report aggregate data from the US Food and Drug Administration (FDA) on single patient IND applications over the final two years of the study (2017-2019). We report an overview of all IND applications, as well as clinical descriptions of patients, treatments, outcomes, and toxicity. RESULTS: Over the 2-year period, the FDA approved all 171 submitted single patient IND requests for pediatric oncology. We identified 56 requests from our center during the 12-year study period, and all were approved (median time from FDA submission to approval: 1 day (range 0-12)). 71% of requests were based on disease histology. Lack of pediatric clinical trial (65%) was the most common reason for use. 48 approved requests were ultimately administered. The median duration of treatment was 84 days (range: 4-1590), with 3 patients remaining on treatment at time of analysis. Only 7% discontinued treatment due to toxicity. Three-year overall survival was 50% (95% CI, 35-64). CONCLUSIONS: Single patient INDs in pediatric oncology were universally approved in our national and single-center analysis. In our cohort, single patient INDs were primarily utilized based on disease histology, rather than genomics, for agents that lacked a clinical trial.
ABSTRACT
OBJECTIVES: A preliminary electronic pain assessment program known as Pain Assessment Interview Network, Clinical Advisory System (painCAS), was implemented in 2 pain centers over the course of 10 months to understand the tool's impact on opioid risk assessment documentation and clinical workflow. The program contains validated electronic versions of screeners for opioid misuse risk (SOAPP-R and Current Opioid Misuse Measure). METHODS: Charts of patients with an initial and 2 follow-up visits were randomly selected for review of presence of opioid risk assessments before and after implementation of the electronic assessment program. Clinical and administrative staff members were interviewed to gain their perceptions of the impact of the program. RESULTS: Significant increases were observed in the documentation of opioid risk assessments between the baseline patient chart reviews before implementation of the program (n = 66) and the postintervention patient chart reviews after the implementation of the program (n = 39), for both initial and follow-up clinic visits (P < 0.001). Specific benefits of the program identified by 7 clinicians and 8 administrators included ease of use, reduced paperwork, completion of the assessment before the clinic visit, and incorporation of information directly into an electronic medical record (EMR). Perceived barriers to implementation included poor patient compliance, changes in administration workflow, and difficulties associated with patients with no email addresses, and limited computer skills. CONCLUSIONS: Implementation of an opioid risk electronic pain assessment program significantly increased the likelihood that a risk assessment would be included in the medical record, which has implications for improvement of quality of care.
