Herrera-Perez, Dianaa; Gill, Jenniferb; Haslam, Alysonc; Crain, Tylerd; Klossner, Quianaa; Prasad, Vinayc,,e Author Information

Background: Having low-income limits one's ability to purchase foods that are high in nutritional value (e.g. vegetables and fruits (V/F)). Higher V/F intake is associated with less diet-related chronic disease. Food pharmacy programs are potential solutions to providing V/F to low-income populations with or at-risk for chronic disease. Aim: This systematic review aimed to determine the effect of food pharmacy programs, including interventions targeting populations at-risk for chronic disease. Methods: We searched Pubmed and Google Scholar databases for studies reporting on food pharmacy interventions and outcomes (hemoglobin A1c, body mass index (BMI), V/F intake, and blood pressure). We calculated pooled mean differences using a random-effects model. Seventeen studies met our inclusion criteria; 13 studies used a pre/post study design, three used a randomized controlled trial, and one was a post-survey only. Results: We found that the pooled mean daily servings of V/F (0.77; 95% CI: 0.30 to 1.24) was higher and BMI (−0.40; 95% CI: −0.50 to −0.31) was lower with food pharmacy interventions We did not find any differences in the pooled mean differences for hemoglobin A1c or systolic blood pressure. Conclusion: Findings posit that food pharmacy programs delivered to primarily low-income individuals with comorbidities may be a promising solution to improving V/F intake and possibly overall diet in these populations.

To ensure the previous progress seen in cancer survival rates continues as we move through the 21st Century it is important to determine future effective policy related to oncology healthcare delivery and funding. Recent successes with, for example, the COVID vaccine response, the decision-making agility exhibited by governments and healthcare systems and the effective use of telehealth and real-world evidence highlight the progress that can be made with pooled efforts and innovative thinking. This shared approach is the basis for the European Beating Cancer Plan which outlines action points for governments and health systems for the period 2021-2025. It fo-cuses on a whole government approach, centred on patients, maximising the potential of new technologies and insights across policy areas including employment, education, transport and taxation, enabling the tackling of cancer drivers in schools, workplaces, research labs, towns and cities and rural communities. Despite the plan there are still concerns that oncology policy has not adequately responded to the pace of innovation and the unique challenges generated by innovative oncological technologies. There needs to be focus on: gaining consensus on the most appropriate methods to assess and price combination therapies and cell and gene ther-apies, developing effective outcome-based payment models for personalised medicine and developing consensus on the ideal approach for multiple indication pricing. Finally, future policy needs to ensure pharmaceutical companies and other research organisations are adequately rewarded for innovation to ensure continued R&D and the development of innovative oncological products.

Abstract Background Identifying ineffective practices that have been used in oncology is important in reducing wasted resources and harm. We sought to examine the prevalence of practices that are being used but have been shown in RCTs to be ineffective (medical reversals) in published oncology studies. Methods We cross-sectionally analyzed studies published in three high-impact oncology medical journals (2009–2018). We abstracted data relating to the frequency and characterization of medical reversals. Results Of the 64 oncology reversals, medications (44%) represented the most common intervention type (39% were targeted). Fourteen (22%) were funded by pharmaceutical/industry only and 56% were funded by an organization other than pharmaceutical/industry. The median number of years that the practice had been in use prior to the reversal study was 9 years (range 1–50 years). Conclusion Here we show that oncology reversals most often involve the administration of medications, have been practiced for years, and are often identified through studies funded by non-industry organizations.

In 1983, the US Congress enacted the Orphan Drug Act to incentivize pharmaceutical companies to develop drugs, vaccines, and diagnostic agents for rare diseases.1Sarpatwari A. Beall R.F. Abdurrob A. He M. Kesselheim A.S. Evaluating the impact of the Orphan Drug Act's seven-year market exclusivity period.Health Aff (Millwood). 2018; 37: 732-737Crossref PubMed Scopus (33) Google Scholar The Act offers 7 years of marketing exclusivity, a 25% tax credit (50% before 2017) on qualified research and development costs, and research subsidies for orphan diseases, defined as conditions that affect less than 200,000 people in the United States. Since its passage, the number of orphan drugs has risen dramatically. In 2018, 58% of all new drug approvals had a rare disease indication.1Sarpatwari A. Beall R.F. Abdurrob A. He M. Kesselheim A.S. Evaluating the impact of the Orphan Drug Act's seven-year market exclusivity period.Health Aff (Millwood). 2018; 37: 732-737Crossref PubMed Scopus (33) Google Scholar One overlooked issue is that approval of an orphan drug may alter the frequency of the disease. Practitioners may be more vigilant to test for, screen for, or diagnose a condition with an approved therapeutic option. In many cases, increased diagnostic enthusiasm may be fueled by corporate sponsors through educational programs, conference sponsorships, and direct outreach to patients. If diseases are diagnosed more frequently after approval, then pivotal trial data may misestimate therapeutic efficacy. This situation occurs for the simple reason that all trial data apply to populations only as they were identified and enrolled in the study. If novel diagnostic criteria or increased vigilance is later applied, it is possible that population characteristics shift, disease severity lessens, and the efficacy of the therapy is altered. In this article, we discuss the implications of 3 rare disease awareness campaigns supported by pharmaceutical companies and consider potential corrective steps. Consider the case of transthyretin amyloidosis (ATTR) cardiomyopathy (CM), a rare condition in which amyloid deposits build up in the heart, increasing wall thickness and impairing function, often leading to heart failure and arrhythmias.2Maurer M.S. Schwartz J.H. Gundapaneni B. et al.ATTR-ACT Study InvestigatorsTafamidis treatment for patients with transthyretin amyloid cardiomyopathy.N Engl J Med. 2018; 379: 1007-1016Crossref PubMed Scopus (1262) Google Scholar In May 2019, tafamidis was approved for ATTR-CM, the first drug to receive approval by the US Food and Drug Administration (FDA) for the condition.3Witteles R.M. Bokhari S. Damy T. et al.Screening for transthyretin amyloid cardiomyopathy in everyday practice.JACC Heart Fail. 2019; 7: 709-716Crossref PubMed Scopus (176) Google Scholar The approval was based on a randomized trial in which patients’ diagnoses of ATTR were confirmed by biopsy of cardiac and noncardiac sites and their CM diagnoses by echocardiography that revealed intraventricular wall thickness exceeding 12 mm, a history of heart failure, and at least one prior hospitalization due to heart failure.2Maurer M.S. Schwartz J.H. Gundapaneni B. et al.ATTR-ACT Study InvestigatorsTafamidis treatment for patients with transthyretin amyloid cardiomyopathy.N Engl J Med. 2018; 379: 1007-1016Crossref PubMed Scopus (1262) Google Scholar These specific inclusion criteria are critical in defining a cohort of patients in whom a common diagnosis—CM—is far more likely to be due to amyloid deposits. Three months after the drug’s approval, experts in the field advocated for screening through a publication titled “Screening for Transthyretin Amyloid Cardiomyopathy in Everyday Practice.”3Witteles R.M. Bokhari S. Damy T. et al.Screening for transthyretin amyloid cardiomyopathy in everyday practice.JACC Heart Fail. 2019; 7: 709-716Crossref PubMed Scopus (176) Google Scholar The authors determined that ATTR-CM was underdiagnosed and that early treatment was essential for appropriate patient care, based on discussions at an expert scientific meeting funded by Pfizer Inc. However, prior to the push to expand diagnostic testing, ATTR-CM was preferentially diagnosed in patients with unexplained CM. In the pivotal trial leading to approval, patients were excluded if their heart failure was thought to be driven by another process.2Maurer M.S. Schwartz J.H. Gundapaneni B. et al.ATTR-ACT Study InvestigatorsTafamidis treatment for patients with transthyretin amyloid cardiomyopathy.N Engl J Med. 2018; 379: 1007-1016Crossref PubMed Scopus (1262) Google Scholar Moreover, many older patients with CM may have incidental amyloidosis, but cardiac dysfunction may be sequelae of multifactorial processes. As such, screening for ATTR-CM has the potential to make a rare disease more common and change the characteristics of patients who receive the treatment. This result may improve outcomes—identify more eligible patients who benefit—or worsen outcomes—lead to the treatment of individuals whose CM is multifactorial, who do not derive commensurate gain. A second case concerns spinal muscular atrophy (SMA), a rare genetic neuromuscular disease that causes muscle wasting in both infants and adults. The severity of the disease differs depending on SMA type. Spinal muscular atrophy type 0 (SMA0) is the most severe, while SMA3 and SMA4 are less severe, having no effect on life expectancy.4Bodamer O.A. Spinal muscular atrophy.in: Dashe J. UpToDate. UpToDate, Waltham, MA2020www.uptodate.comDate accessed: June 1, 2020Google Scholar In 2016, the FDA approved nusinersen, the first drug indicated for the treatment of children and adults with all SMA types.4Bodamer O.A. Spinal muscular atrophy.in: Dashe J. UpToDate. UpToDate, Waltham, MA2020www.uptodate.comDate accessed: June 1, 2020Google Scholar The approval was based on interim results from the ENDEAR (Efficacy and Safety of Nusinersen in Infants With Spinal Muscular Atrophy) trial, a randomized controlled trial that found that among infants with SMA1, those treated with nusinersen were significantly more likely to have a motor milestone response (defined as improvement in at least one category of the Hammersmith Infant Neurological Examination and more categories with improvements than categories with worsening) compared with those in the control group.5Finkel R.S. Mercuri E. Darras B.T. et al.ENDEAR Study GroupNusinersen versus sham control in infantile-onset spinal muscular atrophy.N Engl J Med. 2017; 377: 1723-1732Crossref PubMed Scopus (1312) Google Scholar Although the trial only investigated nusinersen’s effect on patients with SMA1, the FDA approved the drug for all SMA types. Currently, 20 states have adopted and implemented newborn SMA screening programs, and more have developed pilot programs with intent to implement. Cure SMA, a nonprofit group, has been advocating and lobbying for newborn screening since the approval of nusinersen in 2016. The organization has close ties to Biogen, the manufacturer of nusinersen. By screening for SMA, it is likely that the population of patients given the disease label and treated with nusinersen is different than those included in pivotal trials. After all, what parent would choose to omit therapy for their child when told they are suffering from a progressive neurologic ailment? In December 2014, the FDA approved ruxolitinib for patients with polycythemia vera (PV) and an enlarged spleen whose red blood cell counts insufficiently respond to standard treatment.6FDA approves Jakafi (ruxolitinib) for the treatment of patients with uncontrolled polycythemia vera [press release]. Incyte Corporation; December 4.https://investor.incyte.com/press-releases/press-releases/2014/FDA-Approves-Jakafi-ruxolitinib-for-the-Treatment-of-Patients-with-Uncontrolled-Polycythemia-Vera/default.aspxDate: 2014Date accessed: June 1, 2020Google Scholar Polycythemia vera is a rare hematologic malignancy with an annual incidence of 1 in 36,000 to 100,000 persons.6FDA approves Jakafi (ruxolitinib) for the treatment of patients with uncontrolled polycythemia vera [press release]. Incyte Corporation; December 4.https://investor.incyte.com/press-releases/press-releases/2014/FDA-Approves-Jakafi-ruxolitinib-for-the-Treatment-of-Patients-with-Uncontrolled-Polycythemia-Vera/default.aspxDate: 2014Date accessed: June 1, 2020Google Scholar According to the World Health Organization, a diagnosis of PV requires 3 major criteria—elevated hemoglobin level, a bone marrow biopsy specimen showing hypercellularity, and the presence of a JAK2 mutation—or 2 of these major criteria and 1 minor criterion, eg, low erythropoietin levels.7World Health Organization (WHO) diagnostic criteria for primary myelofibrosis (PMF), polycythemia vera (PV), and essential thrombocythemia (ET). Incyte Corporation, 2016https://www.mpnconnect.com/pdf/who-diagnostic-criteria-mf-pv-et.pdfDate accessed: June 1, 2020Google Scholar However, these criteria are not highly sensitive or specific to PV and can present because of other health issues. Hemoglobin levels may fluctuate, and there may be a population of healthy people with JAK2 mutations, now recognized as clonal hematopoiesis. In 2017, Incyte Corporation teamed up with the daytime TV drama “General Hospital” to create an episode to bring PV awareness to the general public.8Mailankody S. Prasad V. Pharmaceutical marketing for rare diseases: regulating drug company promotion in an era of unprecedented advertisement.JAMA. 2017; 317: 2479-2480Crossref PubMed Scopus (9) Google Scholar In this case, the awareness campaign may have encouraged patients and health care professionals to consider PV. Given the disease’s flexible diagnostic criteria, it is possible that some patients diagnosed as having the condition may have been unrecognizable previously. When disease campaigns change the scope of patient populations, the impact could be positive or negative. Provision of a disease-modifying agent to more people who may benefit could improve health outcomes in the population. However, patients who deviate from trial populations could derive less or no therapeutic benefit. For instance, older patients with multifactorial CM and incidental amyloid deposits may not benefit from tafamidis, nor may patients with asymptomatic SMA, who would be subject to intrathecal infusions and its attendant risks. This situation is particularly true for rare disease treatment in which the pivotal trials are more likely to measure surrogate outcomes rather than clinical outcomes and be more likely to have more cases of serious adverse events.9Kesselheim A.S. Myers J.A. Avorn J. Characteristics of clinical trials to support approval of orphan vs nonorphan drugs for cancer.JAMA. 2011; 305: 2320-2326Crossref PubMed Scopus (176) Google Scholar Moreover, health care professionals may be blind to therapeutic inefficacy, eg, a patient with SMA who advances through motor milestones would be declared a success of therapy even though improvement may have occurred in the absence of therapy. The costs of these treatments also cannot be ignored. Ruxolitinib can cost between $91,000 and $143,000 annually. At a price of $225,000 per year, tafamidis is currently the most expensive cardiovascular drug in the United States,10Confidio. 3rd Quarter 2019 Pipeline Report.https://confidio.com/wp-content/uploads/2019/06/CON-Q3-2019-Pipeline-Report.pdfDate accessed: June 1, 2020Google Scholar while nusinersen is priced at $750,000 for the first year of treatment and $375,000 each year thereafter and is prescribed as a lifetime treatment.10Confidio. 3rd Quarter 2019 Pipeline Report.https://confidio.com/wp-content/uploads/2019/06/CON-Q3-2019-Pipeline-Report.pdfDate accessed: June 1, 2020Google Scholar The high costs of these drugs will not only limit access and add a financial burden to the US health care system but may also create a barrier for comparative efficacy trials on more affordable treatments.11Gurwitz J.H. Maurer M.S. Tafamidis—a pricey therapy for a not-so-rare condition.JAMA Cardiol. 2020; 5: 247-248Crossref PubMed Scopus (30) Google Scholar To address these challenges, we suggest that the FDA take 2 actions. First, the agency should impose risk evaluation and mitigation strategy programs on rare disease treatments requiring physicians and patients to attest to the indication for which the drugs were approved. Second, the incidence of orphan diseases before and after FDA approval should be monitored. If sizable deviations are observed, the FDA should be given the regulatory authority to demand confirmatory studies in the new population. Tafamidis may be a lifesaving drug based on a diagnosis made in 2014, but what is its effect in 2021 if the diagnosis is made more frequently or loosely? Disease awareness in orphan diseases makes us revisit the longstanding question in medicine: Does efficacy translate into effectiveness in a shifting medical landscape? The current incentivization for rare drug development may have unintended consequences. Industry-funded campaigns to promote disease awareness may contribute to broader diagnoses and inappropriate treatment. By creating a risk evaluation and mitigation strategy program for rare disease treatments and monitoring the incidence of orphan diseases before and after regulatory approvals, the FDA can better regulate the orphan drug market and ensure that patients are receiving appropriate, effective treatments.

Abstract Background Progression in tumor assessments is often detected at a follow‐up appointment rather than when actual change in progression has occurred, which can bias PFS outcomes. Aim We sought to evaluate the frequency of tumor assessment scans in clinical trials of anti‐cancer interventions and to compare this to recommended (National Comprehensive Cancer Network) and real‐world frequencies of tumor assessments. Methods In a cross‐sectional analysis, we searched for articles published in the three top oncology journals between July 2017 and June 2020. We included articles that were RCTs of patients that had unresectable or metastatic solid tumors and used an intervention that was designed to be anti‐tumor. We abstracted median PFS survival for each group, the PFS hazard ratio, frequency of tumor assessment scans, tumor type, intervention type, and information regarding the study. Results We found that, in the 182 comparisons (163 articles), less frequent tumor assessment (occurring more than 9 weeks between assessments) was associated with higher median PFS values for both the intervention group (p < .0001) and the control group (p < .0001). PFS hazard ratios for studies scanning for tumors every 10 or more weeks were no different than for studies scanning for tumors more frequently (p = .88). Data on the frequency of tumor assessments in the real world is sparse. Conclusion We found that less frequent tumor assessment frequency was associated with longer median PFS in both intervention and control groups of clinical oncology trials but was not associated with differences in PFS hazard ratios. Future research is needed to compare real world to trial assessment.

Despite both enthusiasm and concern regarding data sharing from clinical trials, few studies have documented the outputs of data sharing. The FDA has a considerable collection of raw data from drugs for their marketing authorization. An examination of data sharing with the FDA provides a snapshot into studies that this may facilitate.

International Journal of CancerVolume 149, Issue 9 p. 1723-1724 LETTER TO THE EDITOR After COVID-19, telemedicine may be used in addition to usual care and not in lieu of: Implications for health systems Jennifer Gill, Jennifer Gill Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California, USASearch for more papers by this authorVinay Prasad, Corresponding Author Vinay Prasad [email protected] @VPrasadMDMPH Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California, USA Department of Medicine, University of California San Francisco, San Francisco, California, USA Correspondence Vinay Prasad, University of California San Francisco, 550 16th Street, San Francisco, CA 94158, USA. Email: [email protected]Search for more papers by this author Jennifer Gill, Jennifer Gill Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California, USASearch for more papers by this authorVinay Prasad, Corresponding Author Vinay Prasad [email protected] @VPrasadMDMPH Department of Epidemiology and Biostatistics, University of California San Francisco, San Francisco, California, USA Department of Medicine, University of California San Francisco, San Francisco, California, USA Correspondence Vinay Prasad, University of California San Francisco, 550 16th Street, San Francisco, CA 94158, USA. Email: [email protected]Search for more papers by this author First published: 27 July 2021 https://doi.org/10.1002/ijc.33752 Funding information: Arnold Ventures Read the full textAboutPDF ToolsRequest permissionExport citationAdd to favoritesTrack citation ShareShare Give accessShare full text accessShare full-text accessPlease review our Terms and Conditions of Use and check box below to share full-text version of article.I have read and accept the Wiley Online Library Terms and Conditions of UseShareable LinkUse the link below to share a full-text version of this article with your friends and colleagues. Learn more.Copy URL Share a linkShare onEmailFacebookTwitterLinkedInRedditWechat No abstract is available for this article. REFERENCES 1Olver I, Carey M, Bryant J, Boyes A, Evans T, Sanson-Fisher R. Second opinions in medical oncology. BMC Palliat Care. 2020; 19(1): 112-112. https://doi.org/10.1186/s12904-020-00619-9 10.1186/s12904-020-00619-9 PubMedWeb of Science®Google Scholar 2Ruetters D, Keinki C, Schroth S, Liebl P, Huebner J. Is there evidence for a better health care for cancer patients after a second opinion? A systematic review. J Cancer Res Clin Oncol. 2016; 142(7): 1521-1528. https://doi.org/10.1007/s00432-015-2099-7 10.1007/s00432-015-2099-7 PubMedWeb of Science®Google Scholar 3Lehmann V, Smets EMA, de Jong M, de Vos FYF, Stouthard JM, Hillen MA. Patient-provider communication during second opinion consultations in oncology. Patient Educ Couns. 2021. https://doi.org/10.1016/j.pec.2021.03.011 10.1016/j.pec.2021.03.011 Web of Science®Google Scholar 4Stavert RR, Lott JP. The bystander effect in medical care. N Engl J Med. 2013; 368(1): 8-9. https://doi.org/10.1056/nejmp1210501 10.1056/NEJMp1210501 CASPubMedWeb of Science®Google Scholar 5Johnston KJ, Wen H, Joynt Maddox KE. Lack of access to specialists associated with mortality and preventable hospitalizations of rural medicare beneficiaries. Health Affairs. 2019; 38(12): 1993-2002. https://doi.org/10.1377/hlthaff.2019.00838 10.1377/hlthaff.2019.00838 PubMedWeb of Science®Google Scholar 6Heifetz LJ, Christensen SD, de Vere-White RW, Meyers FJ. A model for rural oncology. J Oncol Pract. 2011; 7(3): 168-171. https://doi.org/10.1200/jop.2010.000167 10.1200/JOP.2010.000167 PubMedGoogle Scholar Volume149, Issue91 November 2021Pages 1723-1724 This article also appears in:World Cancer Day 2022 ReferencesRelatedInformation

This Viewpoint advocates for more clinical trial data regarding the use of pembrolizumab for patients with bacillus Calmette-Guerin-unresponsibe, non-muscle-invasive bladder cancer.

We assessed the frequency that oncology drugs approved by the U.S. Food and Drug Administration (FDA) based on a single-arm study when there is already evidence of existing and available treatments. For this, we conducted a retrospective cross-sectional analysis of FDA-approved oncology drugs based on a single-arm study. All FDA announcements for all oncology drugs approved from May 2014 through June 2019 on a single-arm trial were included. We then performed a systematic search in PubMed, looking for studies on other drugs for the same indication as the FDA drug approval. For the 60 indications, we found 38 instances (63%) of existing therapies being used for the same indication. Of those, we found that 20 drugs were approved based upon a response rate lower than response rates of existing therapies in the same indication. Among oncology drugs that were FDA-approved based on a single-arm study, we found evidence of existing, available therapies being used for the same indication as the FDA-approved drug in the majority of indications (63%), and in one-third of all indications, the response rates for existing therapies were numerically better than the FDA-approved drug. These results suggest that there are inconsistencies in the standards set for oncology drug approvals, and many uncontrolled trials leading to drug approvals could have contemporary controls for which equipoise exists.

BACKGROUND:Noninferiority (NI) trials should help identify interventions that offer some benefit (eg, lower financial costs, more tolerable, or less invasive) without sacrificing noticeable effectiveness, and researchers should adhere to appropriate standards in the conduct and reporting of methods. This study describes the characteristics of a systematic sampling of NI studies from an updated search of recent published oncology trials.METHODS:We performed a cross-sectional analysis of NI research published between 2014 and 2018 in the top 3 medical journals and top 3 oncology journals. We estimated the percentage of NI trials in oncology that report informative details of study, such as justification for conducting NI trial, justification of NI margin, analysis population, and alpha level.RESULTS:There were 94 NI studies and 104 comparisons, and 59.6% (n=62) of comparisons declared NI. The median NI margin of comparisons reporting an odds or hazard ratio was 1.3 (1.05-3.2; n=64). Twenty-three percent (n=22) of studies did not provide a justification for conducting a NI study; 54.3% (n=51) of studies did not provide a justification of the margin they used in their study. Only approximately 46% (n=43) of comparisons used both an intention-to-treat (ITT) and per-protocol (PP) analysis, and 37.3% (n=35) of studies used a one-sided alpha level of >.025. There is notable variation in key elements of the conduct and reporting of NI trials, including the NI margin, the alpha level, and the population analyzed. Furthermore, a high number of studies do not provide justification for conducting a NI study or the margin used for determining NI.CONCLUSIONS:These results suggest that there is room for improvement in the reporting and conduct of NI trials in oncology.

IMPORTANCE Immunotherapy checkpoint inhibitors have generated considerable interest because of durable responses in a number of hitherto intractable tumor types. OBJECTIVE To estimate the percentage of patients with cancer in the United States who are eligible for and respond to checkpoint inhibitor drugs approved for oncology indications by the US Food and Drug Administration (FDA). DESIGN, SETTING, AND PARTICIPANTS Retrospective cross-sectional study performed from June 2018 through October 2018 using publicly available data to determine (1) demographic characteristics of patients with advanced or metastatic cancer, (2) FDA data on checkpoint inhibitors approved from January 2011 through August 2018, (3) measures of response from drug labels, and (4) published reports estimating the frequency of various inclusion criteria. MAIN OUTCOMES AND MEASURES The estimated percentages of US patients with cancer who are eligible for and who respond to immunotherapy checkpoint inhibitor drugs, by year. RESULTS Six checkpoint inhibitor drugs were approved for 14 indications between March 25, 2011, and August 17, 2018. The estimated percentage of patients with cancer who were eligible for checkpoint inhibitor drugs increased from 1.54%(95% CI, 1.51%-1.57%) in 2011 to 43.63%(95% CI, 43.51%-43.75%) in 2018. The percentage of patients with cancer estimated to respond to checkpoint inhibitor drugs was 0.14%(95% CI, 0.13%-0.15%) in 2011 when ipilimumab was approved for unresectable or metastatic melanoma and increased to 5.86%(95% CI, 5.80%-5.92%) by 2015. By 2018, the estimated percentage of responders increased to 12.46%(95% CI, 12.37%-12.54%). CONCLUSIONS AND RELEVANCE The estimated percentages of patients who are eligible for and who respond to checkpoint inhibitor drugs are higher than reported estimates for drugs approved for genome-driven oncology but remain modest. Future research should explore biomarkers to maximize the benefit of immunotherapy among patients receiving it.

This study uses public employee salary data from 14 US public medical schools and the Open Payments database from the US Centers for Medicare & Medicaid Services to examine the ratio of academic oncologists' reported salaries to their general payments from the medical industry.