Assessing the Impact of Prior Authorizations on Dermatology Residents.

INTRODUCTION: Prior authorizations (PAs) are administrative tasks commonly required by insurers to approve medications or therapies for patients. Dermatology practices frequently employ coordinators to focus on completing PAs, among other solutions. The degree to which this support is offered in academic centers and, importantly, how much time dermatology residents spend on PAs over educational pursuits is largely unknown. The authors sought to identify the impact of PAs on dermatology residents. METHODS: An IRB-approved (#NCR213814) 13-question survey was distributed nationwide to dermatology residents regarding the impact of PAs on aspects of clinical and scholarly activities.  Results: 150 of 1462 dermatology residents, 10.3%, responded to the survey. 70% of responding residents contribute to obtaining PAs. 58.7% indicated that their program employed a PA coordinator; though, of these, 63.6% still relied on residents for PAs. 84% indicated that for the following month they feared the burden of PAs would lead to a lapse in treatment for patients. 72.7% avoided prescribing certain medications due to PAs. 64% indicated the PA burden impedes their ability to perform scholarly activities. 80.7% indicated the PA burden contributed to burnout or decreased morale. CONCLUSION: Our data highlight that dermatology residents are negatively impacted by the burden of PAs, resulting in reduced time to study, research, and best care for their patients. Dermatology residents and patients would benefit from reducing the burden of PAs, especially on residents by reforms or regulations that reduce dermatologic PAs, or by academic institutions removing these responsibilities from residents as best as possible. Drugs Dermatol. 2024;23(6):485-488.    doi:10.36849/JDD.7617.

Patient administrative burden in the US health care system.

OBJECTIVE: To assess the prevalence of patient administrative tasks and whether they are associated with delayed and/or foregone care. DATA SOURCE: March 2019 Health Reform Monitoring Survey. STUDY DESIGN: We assess the prevalence of five common patient administrative tasks-scheduling, obtaining information, prior authorizations, resolving billing issues, and resolving premium problems-and associated administrative burden, defined as delayed and/or foregone care. Using multivariate logistic models, we examined the association of demographic characteristics with odds of doing tasks and experiencing burdens. Our outcome variables were five common types of administrative tasks as well as composite measures of any task, any delayed care, any foregone care, and any burden (combined delayed/foregone), respectively. DATA COLLECTION: We developed and administered survey questions to a nationally representative sample of insured, nonelderly adults (n = 4155). PRINCIPAL FINDINGS: The survey completion rate was 62%. Seventy-three percent of respondents reported performing at least one administrative task in the past year. About one in three task-doers, or 24.4% of respondents overall, reported delayed or foregone care due to an administrative task: Adjusted for demographics, disability status had the strongest association with administrative tasks (adjusted odds ratio [OR] 2.91, p < 0.001) and burden (adjusted OR 1.66, p < 0.001). Being a woman was associated with doing administrative tasks (adjusted OR 2.19, p < 0.001). Being a college graduate was associated with performing an administrative task (adjusted OR 2.79, p < 0.001), while higher income was associated with fewer subsequent burdens (adjusted OR 0.55, p < 0.01). CONCLUSIONS: Patients frequently do administrative tasks that can create burdens resulting in delayed/foregone care. The prevalence of delayed/foregone care due to administrative tasks is comparable to similar estimates of cost-related barriers to care. Demographic disparities in burden warrant further attention. Enhancing measurement of patient administrative work and associated burdens may identify opportunities for assessing quality, value, and patient experience.

Patient Participation in Clinical Trials of Oncology Drugs and Biologics Preceding Approval by the US Food and Drug Administration.

Importance: Several studies have estimated the financial inputs for successful drug development. Such analyses do not capture the large investment that patient study participants commit to drug development. Objective: To estimate the volume of patients required to achieve a first US Food and Drug Administration (FDA) approval for a new anticancer drug or biologic therapy. Design, Setting, and Participants: This cohort study included a random sample of prelicense oncology drugs and biologics with a trial site in the United States that were launched into clinical efficacy testing between January 1, 2006, and December 31, 2010. Drugs and biologics were identified using ClinicalTrials.gov registration records. Total patient enrollment was captured over an 8-year span, and each intervention was classified based on whether it received FDA approval and was deemed as having intermediate or substantial value according to the American Society of Clinical Oncology Value Framework (ASCO-VF) score. Secondarily, the association between patient numbers and intervention characteristics was tested. Data were analyzed in February 2020. Main Outcomes and Measure: The prespecified primary outcome was the number of patients enrolled in prelicense trials per FDA approval. Results: A total of 120 drugs and biologics were included in our study, with 84 (70.0%) targeted agents, 20 (16.7%) immunotherapies, and 71 (59.2%) novel agents. A total of 13 drugs and biologics (10.8%; 95% CI, 5.3%-16.8%) in our sample gained FDA approval within 8 years, of which 1 (7.7%) was deemed of intermediate value and 3 (23.1%) were deemed of substantial value using ASCO-VF scoring. Overall, 158 810 patients were enrolled in 1335 trials testing these drugs and biologics, 47 913 (30.2%) in trials that led to FDA approval and 110 897 (69.8%) in trials that did not. An estimated 12 217 (95% CI, 7970-22 215) patient study participants contributed to prelicense trials per FDA approval. The estimated number of patients needed to produce a single FDA-approved drug or biologic of intermediate or substantial ASCO-VF clinical value was 39 703 (95% CI, 19 391-177 991). Conclusions and Relevance: The results of this cohort study make visible the substantial patient investment required for prelicense oncology drug development. Such analyses can be used to devise policies that maximize the clinical impact of research on a per-patient basis.

Prior authorization delays biologic initiation and is associated with a risk of asthma exacerbations.

Background: Biologics are effective treatments for patients with severe allergic disease. Impacts of delays in the prior authorization process on clinical outcomes has not been studied. Objective: The objective was to quantify the times for approval and filling of biologics, and whether patients were at risk of exacerbations during this time frame. Methods: The times for insurance approval and pharmacy filling of biologics (omalizumab, benralizumab, mepolizumab, dupilumab) in 80 subjects with severe asthma (n = 60) or urticaria (n = 20) from our clinic were reviewed. We compared the impact of clinical features, insurance, specialty pharmacy on fill times, and quantified exacerbations and prednisone use while awaiting biologic initiation. Results: The mean ± standard deviation (SD) time (days) from submission of a prescription to the first dose available for injection was 44.0 ± 23.2 days. This was composed of the mean ± SD time for insurance approval (21.5 ± 19.6 days) and the mean ± SD time for a specialty pharmacy to fill the medication (22.8 ± 14.1 days). There was no significant difference between the times for diagnosis (asthma versus urticaria), specific biologic, or insurance. The "buy and bill" system was faster than filling via a specialty pharmacy (mean ± SD, 7.3 ± 8.5 days versus 23.3 ± 21.3 days, respectively, p < 0.001). Clinical features of patients with fast versus slow approval times was not significantly different. The subjects with asthma were at high risk of exacerbations and need for prednisone while awaiting initiation of the biologics; 28 of 59 patients (47%) required prednisone, with an mean cumulative dose of 483.2 ± 273.7 mg per person. Conclusion: The prior authorization process for biologics was slow, and the subjects were at high risk of exacerbations during this time. The system needs to be improved to expedite approval and initiation of these medications.

Will prior health insurance authorization for medications continue to hinder hepatitis C treatment delivery in the United States? Perspectives from hepatitis C treatment providers in a large urban healthcare system.

BACKGROUND: The recent introduction of direct acting antivirals for the treatment of hepatitis C virus (HCV) has dramatically improved treatment options for HCV infected patients. However, in the United States (US) treatment uptake has been low and time to initiation of therapy has been long. We sought to examine provider perspectives of facilitators and barriers to HCV treatment delivery. METHODS: From June to August 2019, we conducted in-depth, semi-structured interviews with medical staff providing HCV care as part of a university medical center in Los Angeles, CA. In order to understand the HCV treatment process, we interviewed key staff members providing care to the majority of HCV patients seeking care at the university medical center, including hepatologists and infectious disease specialists as well as key nursing and pharmacy staff. The interviews focused on workload and activities required for HCV treatment initiation for non-cirrhotic, treatment naïve patients. RESULTS: Providers noted that successful HCV treatment delivery was reliant on a care model involving close collaboration between a team of providers, in particular requiring a highly coordinated effort between dedicated nursing and pharmacy staff. The HCV care team overwhelmingly reported that the process of insurance authorization was the greatest obstacle delaying treatment initiation and noted that very few patient level factors served as a barrier to treatment uptake. CONCLUSIONS: In the US, prior authorization for HCV treatment is a requirement for most public and private insurance plans. In an era with access to therapies that allow for a cure-and until revocation of prior authorization for HCV treatment is a reality-implementing strategies that can expedite authorization to accelerate treatment access are critical. Not only will this benefit patients, but it has the potential to help expand treatment to settings that are otherwise too resource strained to successfully deliver HCV care.

Association of Infectious Disease Physician Approval of Peripherally Inserted Central Catheter With Appropriateness and Complications.

Importance: Peripherally inserted central catheters (PICCs) are frequently used to deliver intravenous antimicrobial therapy. However, inappropriate PICC use may lead to patient harm. Objective: To evaluate whether infectious disease physician approval prior to PICC placement for intravenous antimicrobials is associated with more appropriate device use and fewer complications. Design, Setting, and Participants: This cohort study of 21 653 PICCs placed for a primary indication of intravenous antimicrobial therapy between January 1, 2015, and July 26, 2019, was conducted in 42 hospitals participating in a quality collaborative across Michigan among hospitalized medical patients. Main Outcomes and Measures: Appropriateness of PICCs was defined according to the Michigan Appropriateness Guide for Intravenous Catheters as a composite measure of (1) single-lumen catheter use, (2) avoiding use of PICCs for 5 days or less, and (3) avoiding use of PICCs for patients with chronic kidney disease (defined as an estimated glomerular filtration rate <45 mL/min/1.73 m2). Complications related to PICCs included catheter occlusion, deep vein thrombosis, and central line-associated bloodstream infection. The association between infectious disease physician approval, device appropriateness, and catheter complications was assessed using multivariable models, adjusted for patient comorbidities and hospital clustering. Results were expressed as odds ratios with 95% CIs. Results: A total of 21 653 PICCs were placed for intravenous antimicrobials (11 960 PICCs were placed in men [55.2%]; median age, 64.5 years [interquartile range, 53.4-75.4 years]); 10 238 PICCs (47.3%) were approved by an infectious disease physician prior to placement. Compared with PICCs with no documented approval, PICCs with approval by an infectious disease physician were more likely to be appropriately used (72.7% [7446 of 10 238] appropriate with approval vs 45.4% [5180 of 11 415] appropriate without approval; odds ratio, 3.53; 95% CI, 3.29-3.79; P < .001). Furthermore, approval was associated with lower odds of a PICC-related complication (6.5% [665 of 10 238] with approval vs 11.3% [1292 of 11 415] without approval; odds ratio, 0.55; 95% CI, 0.50-0.61). Conclusions and Relevance: This cohort study suggests that, when PICCs were placed for intravenous antimicrobial therapy, infectious disease physician approval of PICC insertion was associated with more appropriate device use and fewer complications. Policies aimed at ensuring infectious disease physician approval prior to PICC placement for antimicrobials may improve patient safety.

Administrative Burden and Costs of Prior Authorizations in a Dermatology Department.

Importance: Insurance companies use prior authorizations (PAs) to address inappropriate prescribing or unnecessary variations in care, most often for expensive medications. Prior authorizations negatively affect patient care and add costs and administrative burden to dermatology offices. Objective: To quantify the administrative burden and costs of dermatology PAs. Design, Setting, and Participants: The University of Utah Department of Dermatology employs 2 full-time and 8 part-time PA staff. In this cross-sectional study at a large academic department spanning 11 clinical locations, these staff itemized all PA-related encounters over a 30-day period in September 2016. Staff salary and benefits were publicly available. Data were analyzed between December 2018 and August 2019. Main Outcomes and Measures: Proportion of visits requiring PAs, median administrative time to finalize a PA (either approval or denial after appeal), and median cost per PA type. Results: In September 2016, 626 PAs were generated from 9512 patient encounters. Staff spent 169.7 hours directly handling PAs, costing a median of $6.72 per PA. Biologic PAs cost a median of $15.80 each and took as long as 31 business days to complete. The costliest PA equaled 106% of the associated visit's Medicare reimbursement rate. Approval rates were 99.6% for procedures, 78.9% for biologics, and 58.2% for other medications. After appeal, 5 of 23 (21.7%) previously denied PAs were subsequently approved. Conclusions and Relevance: Prior authorizations are costly to dermatology practices and their value appears limited for some requests. Fewer unnecessary PAs and appeals might increase practice efficiency and improve patient outcomes.

Evaluating the effect of prior authorizations in patients with complex dermatologic conditions.

BACKGROUND: In dermatology, prior authorizations can delay treatment, decrease patient adherence, and deter providers from advocating for their patients. Patients with complex dermatologic conditions, often requiring off-label treatments, may face particularly significant insurance barriers. OBJECTIVE: Evaluate the effect of prior authorizations in patients with complex dermatologic conditions. METHODS: This prospective cohort study assessed patients treated by a dermatologist during 5 months who specialized in complex dermatology. Patients included were older than 18 years, treated at V.P.W.'s rheumatology-dermatology clinic, and prescribed a medication or ordered a diagnostic procedure that elicited an insurance prior authorization. Data on prior authorization outcome, administrative time, and delay to treatment were collected. RESULTS: Of 51 prior authorizations, 51% were initially denied, with systemic medications more likely denied than topical ones (P < .001). Total administrative time spent on 50 prior authorizations tracked was 62.5 hours (median time per prior authorization 30 minutes [interquartile range 17-105 minutes]). Time to access treatment was tracked for 80% of prior authorizations; median delay was 12 days [interquartile range 5.5-23 days]. LIMITATIONS: Single-center, single-provider patient panel. CONCLUSION: Patients with complex dermatologic conditions face a significant barrier to care because of prior authorizations. The administrative burden for provider practices to address these prior authorizations is substantial and may warrant a streamlined system in collaboration with insurers.

Effectiveness of mandatory peer review to reduce antipsychotic prescriptions for Medicaid-insured children.

BACKGROUND: Prior authorization of prescription medications is a policy tool that can potentially impact care quality and patient safety. OBJECTIVE: To examine the effectiveness of a mandatory peer-review program in reducing antipsychotic prescriptions among Medicaid-insured children, accounting for secular trends that affected antipsychotic prescribing nationally. DATA SOURCE: Medicaid Analytical eXtracts (MAX) with administrative claims for health services provided between January 2006 and December 2011. STUDY DESIGN: This retrospective, observational study examined prescription claims records from Washington State (Washington) and compared them to a synthetic control drawing from 20 potential donor states that had not implemented any antipsychotic prior authorization program or mandatory peer review for Medicaid-insured children during the study period. This method provided a means to control for secular trends by simulating the antipsychotic use trajectory that the program state would have been expected to experience in the absence of the policy implementation. PRINCIPAL FINDINGS: Before the policy implementation, antipsychotic use prevalence closely tracked those of the synthetic control (6.17 per 1000 in Washington vs. 6.21 in the synthetic control group). Within two years after the policy was implemented, prevalence decreased to 4.04 in Washington and remained stable in the synthetic control group (6.47), corresponding to an approximately 38% decline. CONCLUSION: Prior authorization program designs and implementations vary widely. This mandatory peer-review program, with an authorization window and two-stage rollout, was effective in moving population level statistics toward safe and judicious use of antipsychotic medications in children.

Association of Formulary Prior Authorization Policies With Buprenorphine-Naloxone Prescriptions and Hospital and Emergency Department Use Among Medicare Beneficiaries.

Importance: Prior authorization requirements may be a barrier to accessing medications for opioid use disorder treatment and may, therefore, be associated with poor health care outcomes. Objective: To determine the association of prior authorization with use of buprenorphine-naloxone and health care outcomes. Design, Setting, and Participants: This comparative interrupted time series analysis examined enrollment and insurance claims data from Medicare beneficiaries with an opioid use disorder diagnosis or who filled a prescription for an opioid use disorder medication between 2012 and 2017. Over this period, 775 874 members were in 1479 Part D plans that always required prior authorization, 113 286 members were in 206 plans that removed prior authorization, 189 461 members were in 489 plans that never required prior authorization, and 619 919 members were in 485 plans that added prior authorization. Data analysis was performed from April 2019 to February 2020. Exposures: Removal or addition of prior authorization and new prescriptions filled for buprenorphine-naloxone. Main Outcomes and Measures: Buprenorphine-naloxone use, inpatient admissions, emergency department visits, and prescription drug and medical expenditures. Results: The study population in 2012 included 949 206 Medicare beneficiaries (mean [SD] age, 57 [15] years; 550 445 women [58%]). Removal of prior authorization was associated with an increase of 17.9 prescriptions (95% CI, 1.1 to 34.7 prescriptions) filled for buprenorphine-naloxone per plan per year, which is a doubling of the number of prescriptions, on average. Each prescription filled was associated with statistically significant decreases in adverse health care outcomes: substance use disorder-related inpatient admissions decreased by 0.1 admission per plan per year (95% CI, -0.2 to -0.1 admission per plan per year), and substance use disorder-related emergency department visits decreased by 0.1 visit per plan per year (95% CI, -0.13 to -0.03 visit per plan per year) (all P < .001). Combining these results, removal of prior authorization was associated with a reduction in substance use disorder-related inpatient admissions by 2.0 admissions per plan per year (95% CI, -4.3 to -0.1 admissions per plan per year) and substance use disorder-related emergency department visits by 1.4 visits per plan per year (95% CI, -3.2 to -0.1 visits per plan per year). Conclusions and Relevance: Removing prior authorization for buprenorphine-naloxone was associated with an increase in the medication use and decreases in health care utilization and expenditures.

Medications requiring prior authorization across health insurance plans.

OBJECTIVE: To determine the amount of variation in numbers and types of medications requiring prior authorization (PA) by insurance plan and type. METHODS: Most health insurance companies require PA for medications to ensure safe and effective use and contain costs. We generated 4 lists of medications that required PA during 2017 for commercial, marketplace, Medicaid, and Medicare plans. We aggregated medications according to the generic medication name equivalent using codes and medication names. We compared these medications to assess how many of the medications required PA by 1, 2, 3, or all 4 of the insurance plans. We counted all prescription orders written for a patient age 18 years or older with health plan insurance during 2017 for any of the medications that appeared on the health plan's PA lists by querying the electronic health record. RESULTS: PA was required for 600 unique medications in 2017 across the 4 plans. Of 691,457 prescription orders written for 114,159 members, 31,631 (5%) were written for 1 of the 600 medications that required PA by at least 1 insurance plan. There were 12,540 medication orders (written for 6,642 members) that potentially required PA. The marketplace plan required PA for the greatest number of medications (440), followed by the Medicare (272), commercial (271), and Medicaid (72) plans. The most commonly prescribed classes of medications for which PA was required by at least 1 plan were antihyperlipidemics (22% of orders potentially requiring PA), narcotic analgesics (13%), hypnotics (12%), antidiabetic medications (9%), and antidepressants (9%). For only 25% of medications (151 of 600) was PA required by at least 3 plans, and for only 5% (32 of 600) was PA required by all 4 insurance types. CONCLUSION: Medications requiring PA can differ within a single health insurance company, but this variation may be unavoidable due to external factors.

Treatment Delays Associated With Prior Authorization for Infusible Medications: A Cohort Study.

OBJECTIVE: Prior authorizations (PAs) are commonly used by health payers as cost-containment strategies for expensive medications, including infused biologics. There is scarce data about the effect of PA requirements on patient-oriented outcomes. METHODS: We included patients for whom an infusible medication was prescribed for a rheumatologic condition. The exposures of interest were a PA requirement and whether or not the PA was denied. The primary outcome was the difference in days from medication request to infusion. Secondary outcomes included the proportion of denied PAs and differences in glucocorticoid exposure following a PA request. RESULTS: Of the 225 patients, the infusible medications of 160 (71%) required a PA. PAs were associated with a greater number of days to infusion compared to cases in which no authorization was required (median 31 days [interquartile range (IQR) 15-60 days] versus median 27 days [IQR 13-41 days]; P = 0.045), especially among the 33 patients (21%) whose PA was denied initially (median 50 days [IQR 31-76 days] versus median 27 days [IQR 13-41 days]; P < 0.001). PA denials were associated with greater prednisone-equivalent glucocorticoid exposure in the 3 months following the request than when a PA was not required (median 605 mg [IQR 0-1,575] versus median 160 mg [IQR 0-675]; P = 0.01). Twenty-seven of the 33 PA requests that were initially denied (82%) were eventually approved. Thus, 96% of all PAs were ultimately approved. CONCLUSION: PA requirements are associated with treatment delays and denials are associated with greater glucocorticoid exposure. Because the great majority of PA requests are ultimately approved, the value of PA requirements and their impact on patient safety should be reevaluated.

The macroeconomic benefits of Tennessee APRNs having full practice authority.

BACKGROUND: To-date, advocacy efforts to advance full practice authority for APRNs have primarily stressed arguments based on evidence on the cost effectiveness and quality of APRN-provided care, as well as the improved care access and patient satisfaction these providers offer. PURPOSE: The economic impact analysis forecasts the additional job and economic output associated with granting Tennessee APRNs full practice authority. METHODS: The IMPLAN software and a variety of data inputs were used to estimate the direct, indirect, and induced economic impact on jobs, labor income, value-added benefits, total output, and tax revenues. FINDINGS: From a 2017 baseline, the cumulative impact of granting Tennessee APRNs full practice authority is a net gain of 25,536 jobs and $3.2 billion in economic impact. DISCUSSION: Granting Tennessee APRNs full practice authority would confer substantial economic benefits and employment opportunities to the state.

The effectiveness of formulary restriction and preauthorization at an academic medical center.

The impact of formulary restriction and preauthorization (FRPA) on prescribing trends was examined over a 5-year period at an academic medical center. Ordinary least squares regression was used to identify hospital units demonstrating statistically significant trends in prescription of restricted agents. Significant decreases in restricted drug use were seen on 2 of 7 medicine units subject to FRPA, whereas a significant increase was seen in 1 of 4 surgical units subject to FRPA.

Insurance Approval for Proton Beam Therapy and its Impact on Delays in Treatment.

PURPOSE: Prior authorization (PA) has been widely implemented for proton beam therapy (PBT). We sought to determine the association between PA determination and patient characteristics, practice guidelines, and potential treatment delays. METHODS AND MATERIALS: A single-institution retrospective analysis was performed of all patients considered for PBT between 2015 and 2018 at a National Cancer Institute-designated Comprehensive Cancer Center. Differences in treatment start times and denial rates over time were compared, and multivariable logistic regression was used to identify predictors of initial denial. RESULTS: A total of 444 patients were considered for PBT, including 396 adult and 48 pediatric patients. The American Society for Radiation Oncology model policy supported PBT coverage for 77% of the cohort. Of adult patients requiring PA, 64% were initially denied and 32% remained denied after appeal. In patients considered for reirradiation or randomized phase 3 trial enrollment, initial denial rates were 57% and 64%, respectively. Insurance coverage was not related to diagnosis, reirradiation, trial enrollment, or the American Society for Radiation Oncology model policy guidelines, but it was related to insurance category on multivariable analysis (P < .001). Over a 3-year timespan, initial denial rates increased from 55% to 74% (P = .034). PA delayed treatment start by an average of 3 weeks (and up to 4 months) for those requiring appeal (P < .001) and resulted in 19% of denied patients abandoning radiation treatment altogether. Of pediatric patients, 9% were initially denied, all of whom were approved after appeal, and PA requirement did not delay treatment start (P = .47). CONCLUSIONS: PA requirements in adults represent a significant burden in initiating PBT and cause significant delays in patient care. Insurance approval is arbitrary and has become more restrictive over time, discordant with national clinical practice guidelines. Payors and providers should seek to streamline coverage policies in alignment with established guidelines to ensure appropriate and timely patient care.

The Insurance Approval Process for Proton Radiation Therapy: A Significant Barrier to Patient Care.

PURPOSE: Proton therapy is increasingly prescribed for cancer treatment, given its potential for improvements in clinical outcomes and toxicity reduction; however, insurance coverage continues to be a barrier to patient access. This study examined insurance approval and appeal outcomes at a large-volume proton therapy center to clarify the process and identify areas for improvement. METHODS AND MATERIALS: In 2013 to 2016, 1753 patients with thoracic or head and neck cancer were considered for proton therapy; 903 (553 thoracic, 350 head and neck) entered the insurance process. Rates of and times to approval and successful appeal after initial denial were calculated. Clinical factors were evaluated for association with insurance outcomes via logistic regression. RESULTS: Approval rates by Medicare (n = 538) and private insurance (n = 365) were 91% and 30% on initial request, at a median 3 days and 14 days from inquiry to determination. Of the 306 patients initially denied coverage, 276 appealed the decision, and denial was overturned for 189 patients (68%; median time, 21 days from initial inquiry). On multivariable analysis, Medicare (odds ratio [OR], 14.20; P < .001) was the strongest predictor of initial approval. Approval rates decreased from 2013 to 2014 versus 2015 to 2016 (OR 0.54; P = .001). For patients who appealed denial, multivariable analysis found no associations between approval and trial enrollment or tumor type. Submission of a comparison treatment plan (proton vs photon) indicating dosimetric advantage to normal tissues was associated with decreased likelihood of approval (OR 0.43; P = .006), as was a prescribed dose of ≥66 Gy (OR 0.48; P = .019). CONCLUSIONS: Despite an 87% ultimate approval rate for proton therapy, the insurance process is a resource-intensive barrier to patient access associated with significant time delays to cancer treatment. These findings, plus the lack of clinical correlates with insurance outcomes, highlight a need for increased efficiency, transparency, and collaboration among stakeholders to promote timely patient care and research.

Coverage of Novel Therapeutic Agents by Medicare Prescription Drug Plans Following FDA Approval.

BACKGROUND: Regulatory approval of novel therapies by the FDA does not guarantee insurance coverage requisite for most clinical use. In the United States, the largest health insurance payer is the Centers for Medicare & Medicaid Services (CMS), which provides Part D prescription drug benefits to over 43 million Americans. While the FDA and CMS have implemented policies to improve the availability of novel therapies to patients, the time required to secure Medicare prescription drug benefit coverage-and accompanying restrictions-has not been previously described. OBJECTIVE: To characterize Medicare prescription drug plan coverage of novel therapeutic agents approved by the FDA between 2006 and 2012. METHODS: This is a cross-sectional study of drug coverage using Medicare Part D prescription drug benefit plan data from 2007 to 2015. Drug coverage was defined as inclusion of a drug on a plan formulary, evaluated at 1 and 3 years after FDA approval. For covered drugs, coverage was categorized as unrestrictive or restrictive, which was defined as requiring step therapy or prior authorization. Median coverage was estimated at 1 and 3 years after FDA approval, overall, and compared with a number of drug characteristics, including year of approval, CMS-protected class status, biologics versus small molecules, therapeutic area, orphan drug status, FDA priority review, and FDA-accelerated approval. RESULTS: Among 144 novel therapeutic agents approved by the FDA between 2006 and 2012, 14% (20 of 144) were biologics; 40% (57 of 144) were included in a CMS-protected class; 31% (45 of 144) were approved under an orphan drug designation; 42% (60 of 144) received priority review; and 11% (16 of 144) received accelerated approval. The proportion of novel therapeutics covered by at least 1 Medicare prescription drug plan was 90% (129 of 144) and 97% (140 of 144) at 1 year and 3 years after approval, respectively. At 3 years after approval, 28% (40 of 144) of novel therapeutics were covered by all plans. Novel therapeutic agents were covered by a median of 61% (interquartile range [IQR] = 39%-90%) of plans at 1 year and 79% (IQR = 57%-100%) at 3 years (P < 0.001). When novel therapeutics were covered, many plans restricted coverage through prior authorization or step therapy requirements. The median proportion of unrestrictive coverage was 29% (IQR = 13%-54%) at 3 years. Several drug characteristics, including therapeutic area, FDA priority review, FDA-accelerated approval, and CMS-protected drug class, were associated with higher rates of coverage, whereas year of approval, drug type, and orphan drug status were not. CONCLUSIONS: Most Medicare prescription drug plans covered the majority of novel therapeutics in the year following FDA approval, although access was often restricted through prior authorization or step therapy and was dependent on plan choice. DISCLOSURES: Funding for this study was contributed by a student research grant awarded to Shaw and provided by the Yale School of Medicine Office of Student Research under National Institutes of Health training grant award T35DK104689. Ross reports research grants to Yale University from the U.S. Food and Drug Administration (U01FD005938, U01FD004585), Medtronic, Johnson & Johnson, Centers for Medicare & Medicaid Services (HHSM-500-2013-13018I), Blue Cross-Blue Shield Association, Laura and John Arnold Foundation, Agency for Healthcare Research and Quality (R01HS022882), and National Institutes of Health (R01HS025164), unrelated to this study. Dhruva has nothing to disclose. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.