Pertuzumab Panel Discussion Information
Total Page：16
File Type：pdf, Size：1020Kb
Bridging the gap between payer and provider perspectives Volume 18 Special Issue 4 Evidence-Based Oncology AMCP Conference Coverage Also in this issue... Achieving Quality and Containing Cost Implementation SP157 Rationalizing Treatment Through Oncology Pharmacy Management of Cancer Clinical and Coverage Decisions With Predictive Kim Farina, PhD Care Pathways Biomarkers A Successful Model of n 2011, one large pharmacy benefit manager spent more than $25 per member Collaboration Between SP188 How Payers and per year (PMPY) on oncology specialty pharmacy, an increase of $4 PMPY from Oncologists Really I2010 (Table). Spending is forecasted to exceed $40 PMPY by 2014 (Figure 1). Emerg- Payers and Providers Feel About Oncology ing oral cancer drugs are shifting costs from medical benefits to pharmacy benefits. With increasing numbers of expensive specialty pharmacy oncology drugs on the Bruce A. Feinberg, DO; et al Pathways market—and hundreds more in the pipeline—it is clear that improvements in on- cology pharmacy management are needed. espite rising medical costs with- SP 189 Partnering With a Payer At the 2012 Academy of Managed Care Pharmacy Annual Meeting, a panel made in the US healthcare system, to Develop a Value-Based up of a patient advocate, a cancer survivorship specialist, health plan representa- Dquality and outcomes are not Medical Home Pilot tives, an oncologist, and a health services researcher discussed the desired out- improving. Without significant policy comes of oral oncology drug management, practical next steps, and gaps in and reform, the cost-quality imbalance will barriers to implementation. reach unsustainable proportions in Over the last 10 to 20 years, the introduction of new therapeutic agents has the foreseeable future. The rising cost markedly improved overall survival for many types of cancer. In most cases, how- of healthcare in part results from an ever, supporting data have not kept up with these advances, leaving knowledge expanding aging population with an gaps that make coverage and treatment decisions difficult. For many patients, increasing number of life-threatening (continued on page SP150) Pertuzumab Panel Discussion information. for SP148 See page What’s this? Treatment and Cost Implications BeckieOncology Fenrick, Specialty PharmD, Group MBA, Senior of Pertuzumab Director, Medical Operations, Care Providing Access and Controlling Costs Models and Affordability Solutions, Florida Blue, Discusses Companion in the Era of Targeted Therapies Diagnostics for Personalized Medicine in Specialty Pharmacy ertuzumab, a new HER2-positive breast cancer drug, is being launched at a 31% premium to the price of trastuzumab. This is an aggressive strategy Pthat will substantially increase the cost of treating patients with metastatic Healthcare Specialty Group HER2-positive breast cancer, while also aiming to deliver the best outcomes for patients. Pertuzumab will be used as an add-on to trastuzumab to treat patients. Progres- Bruce A. Feinberg, DO sion-free survival was boosted by 6.1 months when the drugs were used in combi- nation in the CLEOPATRA study. diseases. This is further compounded With payers already anxious about the rising costs by a growing arsenal of high-cost ther- in oncology, an 18-month course of the combination apies. In no medical specialty is this Surya Singh, MD, Vice President, treatment is estimated to reach more than $180,000. more apparent than in the area of on- Medical Benefit Management, CVS This cost sets the bar high for the future, as expensive cology. Numerous attempts to reduce Caremark,Pharmacy Addresses Group Methods for targeted drugs are expected to be used increasingly in costs have been attempted, often with Managing Oncology Spending in combination to deliver the best outcomes. limited benefit and brief duration. Be- Specialty Pharmacy To examine these treatment benefits, cost concerns, cause physicians directly or indirectly and potential insurance coverage strategies, AJMC’s control or influence the majority of co-editor-in-chief, Michael E. Chernew, PhD, profes- medical care costs, physician behav- sor, department of healthcare policy, Harvard Medi- ioral changes must occur to bend the cal School, moderated a panel discussion with Lee Michael E. Chernew, PhD healthcare cost curve in a sustainable Newcomer, MD, MHA, senior vice president, oncology, fashion. Experts within academia, Managed Markets Group UnitedHealthcare, and Sandra M. Swain, MD, medical health policy, and business agree that a director, Washington Cancer Institute, Medstar Wash- significant paradigm change in stake- ington Hospital Center. holder collaboration will be necessary A supplement to (continued on page SP151) (continued on page SP159) The American Journal of Managed Care Digital Media Making PROgress with patient-reported outcomes How PROs were successfully integrated into the Jaka ® (ruxolitinib) drug development program1 JAKAFI endpoints included both biologic and patient-reported outcomes8,9 A novel approach to engage clinicians and FDA TAILORING a PRO tool for myelo brosis COMFORT-I: Percent Change in Spleen Volume in Individual Patients COMFORT-I: Percent Change in TSS in Individual Patients PROs are an important means to demonstrate treatment Myelofi brosis (MF) is a life-threatening, progressive disease From Baseline to Week 24 or Last Observation9,a From Baseline to Week 24 or Last Observation9,a,b bene ts in clinical trials.2,3 Use of a PRO instrument can characterized by splenomegaly, debilitating symptoms and 80 150 evaluate symptoms best judged by the patient, whether cytopenias.5-7 Measures to assess both the splenomegaly 60 100 caused by the disease or treatment toxicity. Assessment and core symptoms of MF were incorporated into the phase III, 40 G 20 G of symptom burden is important because it can be a major double-blind placebo-controlled study, COMFORT-I, for 50 0 indicator of disease severity, progression or improvement. Jakafi . Spleen reduction, as measured by imaging (MRI or CT), -20 0 Incorporating PROs into a clinical trial program provides a was the primary and biologic endpoint, and a reduction in total 35% Reduction -40 50% Improvement symptom score (TSS), the PRO measure, was a key secondary IMPROVEMENT WORSENIN -50 IMPROVEMENT WORSENIN means for evaluating the impact of therapy from the patient’s Change From Baseline (%) Change From Baseline (%) -60 8,9 Upper 50th Percentile Upper 50th Percentile Upper 50th Percentile Upper 50th Percentile perspective and helps patients and clinicians make better- endpoint. The TSS encompassed the following symptoms: -80 -100 informed decisions.4 abdominal discomfort, pain under left ribs, early satiety, pruritus, night sweats and bone/muscle pain.9 Jaka (n = 155) Placebo (n = 153) Jaka (n = 145) Placebo (n = 145) Each bar represents an individual patient’s response. Each bar represents an individual patient’s response. Worsening of TSS is truncated at 150%. To include PROs in the trial, a novel instrument had to be speci cally developed. After patient interviews, advice from clinical experts and extensive input from the FDA, the PROVIDING proof of patient bene t modi ed Myelo brosis Symptom Assessment Form, version MF is progressive, and spleen size and symptoms can become increasingly burdensome to patients over time.5-7 Jaka is 2.0 (modifi ed MFSAF v2.0) was fi nalized as part of the Special proven to decrease total symptom score in patients with intermediate or high-risk MF—this is an important consideration when Protocol Assessment prior to the initiation of COMFORT-I. evaluating and treating patients.9 The FDA approval included patients with intermediate-2 risk and high risk, as well as patients Ultimately, Jaka was approved by the FDA for the treatment with intermediate-1 risk, since intermediate-1 patients may also have symptoms that require treatment. Clinical experience 1,8 of intermediate or high-risk MF. This became Incyte’s rst with Jaka has shown that with the right process, manufacturers can successfully collaborate with regulatory agencies and approved drug and also the rst oncology medicine approved academic experts to develop relevant and validated PRO instruments that can be incorporated into clinical trials.1,8 The approval with symptom data in its label since the FDA ’s draft guidance on of Jakafi marks a signifi cant milestone in which validated PRO instruments can provide symptom data and demonstrate 2,4 PROs was fi nalized in 2009. clinical benefi t. The experience with Jakafi may provide a model for the future use of PROs in marketing applications.8 Indications and Usage during treatment. Thrombocytopenia was generally renal or hepatic impairment [see Dosage and Administration]. a As studied in COMFORT-I, a randomized, double-blind, placebo-controlled phase III Jaka is indicated for treatment of patients with intermediate reversible and was usually managed by reducing the dose Patients should be closely monitored and the dose titrated study with 309 total patients (United States, Canada, Australia). The primary endpoint was the proportion of subjects achieving a ≥35% reduction in spleen volume from or high-risk myelo brosis, including primary myelo brosis, or temporarily withholding Jaka . If clinically indicated, based on safety and ef cacy baseline to Week 24 as measured by MRI or computed tomography (CT). A secondary post–polycythemia vera myelo brosis and post–essential platelet transfusions may be administered • There are no adequate and well-controlled studies of Jakafi endpoint