Daniel S. Mytelka

How to improve R&D productivity: the pharmaceutical industry's grand challenge

The pharmaceutical industry is under growing pressure from a range of environmental issues, including major losses of revenue owing to patent expirations, increasingly cost-constrained healthcare systems and more demanding regulatory requirements. In our view, the key to tackling the challenges such issues pose to both the future viability of the pharmaceutical industry and advances in healthcare is to substantially increase the number and quality of innovative, cost-effective new medicines, without incurring unsustainable R&D costs. However, it is widely acknowledged that trends in industry R&D productivity have been moving in the opposite direction for a number of years. Here, we present a detailed analysis based on comprehensive, recent, industry-wide data to identify the relative contributions of each of the steps in the drug discovery and development process to overall R&D productivity. We then propose specific strategies that could have the most substantial impact in improving R&D productivity.

Healthcare costs, treatment patterns, and resource utilization among pancreatic cancer patients in a managed care population

Abstract Background: Pancreatic adenocarcinoma has few effective treatment options and poor survival. The objective of this study was to characterize treatment patterns and estimate the costs and resource use associated with its treatment in a commercially-insured US population. Methods: In this retrospective claims-based analysis, individuals ≥18 years old with evidence of pancreatic adenocarcinoma between January 1, 2001 and December 31, 2010 were selected from a managed care database. Treatment phase (either initial non-metastatic or metastatic) was determined using a claims-based algorithm. Patients in the pancreatic cancer population were matched 1:3 to a control population. Resource use (events/person-years), treatment patterns, and healthcare costs (per-patient per-month, PPPM) were determined during a variable length follow-up period (from first pancreatic cancer diagnosis to earliest of death, disenrollment, or study end). Results: In this study, 5262 pancreatic cancer patients were matched to 15,786 controls. Rates of office visits, inpatient visits, ER visits, and inpatient stays, and mean total all-cause healthcare costs PPPM (

Estimating the incremental net health benefit of requirements for cardiovascular risk evaluation for diabetes therapies

15,480 vs

Treatment patterns and outcomes among patients with high-intermediate/high-risk diffuse large B-cell lymphoma in the USA

1001) were significantly higher among cancer patients than controls (all p < 0.001). Mean inpatient costs were the single largest cost driver (

Health Care Resource Utilization and Costs among Adult Patients with Advanced Soft Tissue Sarcoma: A Retrospective Medical Record Review in the United Kingdom, Spain, Germany, and France

9917 PPPM). Also, mean total all-cause healthcare costs were significantly higher during the metastatic treatment phase vs the initial treatment phase of non-metastatic disease (

Association of baseline absolute neutrophil counts and survival in patients with metastatic colorectal cancer treated with second-line antiangiogenic therapies: exploratory analyses of the RAISE trial and validation in an electronic medical record data set

21,637 vs

Chemotherapy treatment patterns, costs, and outcomes of patients with gastric cancer in the United States: a retrospective analysis of electronic medical record (EMR) and administrative claims data

10,358, p < 0.001). Conclusions: These results indicate that pancreatic cancer imposes a substantial burden on the US healthcare system, and that treatment of more advanced disease is significantly more costly than initial treatment of non-metastatic disease. Limitations: Additional research is needed to validate the accuracy of the claims-based algorithms used to identify the treatment phase.

Evaluating the Utility of Existing Patient-Reported Outcome Scales in Novel Patient Populations with Pancreatic Cancer, Lung Cancer, and Myeloproliferative Neoplasms Using Medicare Current Beneficiary Survey Data

To evaluate the advantages and disadvantages of pre‐approval requirements for safety data to detect cardiovascular (CV) risk contained in the December 2008 U.S. Food and Drug Administration (FDA) guidance for developing type 2 diabetes drugs compared with the February 2008 FDA draft guidance from the perspective of diabetes population health.

Treatment Patterns and Survival among Adult Patients with Advanced Soft Tissue Sarcoma: A Retrospective Medical Record Review in the United Kingdom, Spain, Germany, and France

Abstract Objective Clinical trials have demonstrated improved outcomes for patients with diffuse large B-cell lymphoma (DLBCL) treated with regimens containing rituximab, but variations in real-world treatment patterns and outcomes have not been studied. The objective of this study was to characterize real-world treatment patterns and outcomes in higher risk DLBCL patients. Methods Patients with an International Prognostic Index score (IPI) ≥3 who received initial rituximab-based therapy from 2005 to 2012 were identified via electronic medical record data from the International Oncology Network. Initial therapy, rates of complete response (CR), post-CR treatments, and outcomes were evaluated. Results Among 257 eligible patients, 75% achieved a CR: 77% (158/206) of patients receiving R-CHOP compared to 71% (36/51) of patients receiving initial therapies other than R-CHOP. Post-CR, 78% of the 158 patients receiving R-CHOP underwent active surveillance; 13% received maintenance rituximab-based treatment; and 6% received radiation therapy. Relapse rates among patients receiving maintenance rituximab, active surveillance, and radiation therapy were 28% (6/21), 19% (24/124), and 0%, (0/10), respectively (P = 0.08). Discussion This study found that active surveillance continues to be the most commonly utilized treatment regimen among DLBCL patients with an IPI score ≥3 achieving a CR on first-line R-CHOP. Other approaches aimed at increasing the time to relapse are being utilized as well, but the clinical benefit of these modalities is unclear. Conclusion Results of this study are consistent with the results from clinical trials and suggest the need for further evaluation of maintenance therapy options for patients at higher risk of relapse.

Baseline absolute neutrophil counts (ANC) and survival in second-line metastatic colorectal cancer (mCRC) patients (pts).

Objective To describe health care resource utilization and costs for patients with advanced soft tissue sarcoma (STS) in the United Kingdom (UK), Spain, Germany, and France. Methods Physicians abstracted data for adult patients with a diagnosis of advanced STS (other than Kaposis sarcoma or gastrointestinal stromal tumor) who received ≥1 lines of systemic therapy. Health care resource utilization related to advanced STS treatment was recorded; associated costs were estimated by applying unit costs. Results A total of 130 physicians provided data for 807 patients (UK: 199; Spain: 203; Germany: 204; and France: 201). The site of care during active treatment varied based on differences in the health care systems of these four countries. Total mean per-patient health care cost in the UK was £19,457; in Spain, €26,814; in Germany, €20,468; and in France, €24,368. Advanced STS-related systemic treatment costs were driven primarily by drug acquisition and administration costs. Treatment-related costs increased during later lines of therapy for all countries except France, where they decreased after first-line therapy. Pain control and antiemetics were the most common supportive care medications. Conclusions This study provides real-world data on resource utilization and estimated costs in advanced STS and could inform policymakers about treatment burden.