Real World Evidence

Real world data (RWD) and real world evidence (RWE) are playing an increasingly important role in health care decision-making. The 21st Century Cures Act, passed in 2016, places focus on the use of these types of data to support regulatory decision making including evaluating post-market safety, monitoring adverse events and to making regulatory decisions for medical products. In addition, the health care community is more frequently using RWD and RWE to support coverage decisions and to develop guidelines and decision support tools for use in clinical practice.

Many medical product manufacturers look to RWD and RWE to support clinical trial designs (e.g., large simple trials, pragmatic clinical trials) and observational studies to generate cost effective, innovative, and new treatment approaches.

The Importance of Real World Data

Exponent’s scientists design, collect, analyze and critically evaluate RWD. At every point in the medical product total product life cycle, there is a need for more information to support decision-making. Understanding and identifying the best source of RWD to address specific needs for stakeholders with a variety of research needs can be a complicated question. There are many sources of RWD available but not all of them can withstand a thorough review of quality and be deemed fit to be used as RWE. Thoughtful and appropriate use of RWE by leveraging existing data sources can minimize additional burden on patients and save time and money, therefore providing answers that can move products to market faster or identify and solve problems earlier.

Our experience with regulators, product development, user requirements, performance and process monitoring, and database design can help clients create tools for data collection that will stand up to regulatory scrutiny and also provide ongoing collection of RWD. After medical products reach the market, our broad experience with public and commercial claims and medical records databases, registries, and surveillance data make us particularly qualified to address questions of data quality and appropriateness.

With our team of scientists and statisticians, Exponent works with clients to explore the nuances of the research questions and identify datasets or sources that have both the quantity and quality of information to address regulatory and market access questions. Not only do we consider whether a data source seems capable of providing useful information, but we can review other uses of similar data to recommend insights about its perceived value by the target audience. For new data sources or uses, our analytics and regulatory consultants can help clients review and prepare the data to support its use for a particular purpose.
RWD Capabilities

RWD has grown exponentially over the last decades through widespread adoption of electronic health records, and pharmacy and payer databases. Technology has increasingly put real-time RWD in the hands of patients and healthcare professionals. Wearables, implantables, and home health monitoring devices with internet connectivity are commonplace and likely to increase. Many devices are capable of generating enormous amounts of clinical RWD but manufacturers and healthcare professionals often struggle with how to identify quality data and where and when to use RWD and RWE.

How Exponent Can Help

Exponent’s consultants provide a range and depth of expertise in epidemiology, toxicology, regulation, statistics, and health economics and outcomes research to assist our clients in the identification, design, and development of materials that help them achieve a competitive market edge, including:

  • Economic and clinical value analyses
  • Identification of high quality RWD and RWE for specific needs
  • Review of specific industry case studies where RWE has succeeded and failed
  • Data analytics, including payer database and electronic health record analytics
  • Scientific white paper development and peer-reviewed publication
  • Market access and reimbursement strategies including evidence generation and product value demonstration 
  • Portable modeling tools for one-on-one discussions with stakeholders
  • Medical device outcomes assessment.

Additionally, Exponent works with clients to communicate results to the target audience. Our team has been involved with preparing, and presenting findings to regulators, as well as presenting in public forums and publishing in peer-reviewed journals.

Relevant Publications

Market Access, Marketing, and Medical Affairs:


Sackman, J.E., Abrams, M.A., Numerof, R.E. The Impact of the Affordable Care Act (“Obamacare”) on Innovation in Biopharma. BioPharm International, January 2014.

Sackman, J.E., Citrin, L. How to Address Cost Outliers. Healthcare Financing Management Association (HFMA) Magazine. March 2014

Agarwal, S.J., Delhougne, G.V., Citrin, L., Sackman, J.E., Senagore, A.J. The Impact of Minimally Invasive Surgery On Complex DRG Assignments. Managed Care. March 2014.

Sackman, J.E. Kuchnreuther, M. Navigating Emerging Markets: India. BioPharm International May 2014.

Sackman, J.E., Rothenberg, S. Market Access – Global Challenges for Oncology Drugs. PM360, May 2014

Sackman J.E., Abrams, M.A. Population Health 101. Hospitals & Health Networks (H&H) Magazine. July 2014.

Sackman J.E., Buseman, C. Payment Reform: Essential Elements for Going at Risk. Healthcare Financial Management Association (HFMA). October 2014.

Sackman, J.E. Navigating Emerging Markets: Eastern Europe. BioPharm International February 2013.

Sackman, J.E.. Real World Data: Medical Device and Diagnostics Perspectives. Eye for Pharma. March 2013.

Sackman, J.E. Navigating Emerging Markets: Latin America. BioPharm International. April 2013.

Sackman, J.E. Navigating Emerging Markets: ASEAN. BioPharm International. June 2013.

Sackman, J.E. Navigating Emerging Markets: South Korea. BioPharm International. August 2013

Sackman, J.E., Kuchenreuter, M. Changing Payer Perspectives on Market Access Across Europe’s G-5. eye for pharma. October 2013.

Numerof, R.E., Sackman J.E. Economic and Clinical Value as Marketing Strategy: What Payer Changes Mean for Your Business. eye for pharma. October 2013.

Sackman, J.E. Navigating Emerging Markets: Middle East and North Africa. BioPharm International. October 2013.

Sackman, J.E. Kuchenreuther, M. Navigating Emerging Markets: Israel BioPharm International December 2013.

Centers for Medicare & Medicaid Services, Medicare Data:

Kurtz SM, Lau EC, Ong KL, Adler EM, Kolisek FR, Manley MT. Which clinical and patient factors influence the national economic burden of hospital readmissions after total joint arthroplasty? Clin Orthop Relat Res 2017 Jan 20. [Epub ahead of print]

S, Shu AC, Ong KL, Baykal D, Lau E, Malkani AL. Complications after revision total hip arthroplasty in the Medicare population. J Arthroplasty 2017;32(6):1954-8.

Kurtz SM, Lau EC, Ong KL, Adler EM, Kolisek FR, Manley MT. Has health care reform legislation reduced the economic burden of hospital readmissions following primary total joint arthroplasty? J Arthroplasty 2017 Jun 8. [Epub ahead of print].

Cochran AR, Ong KL, Lau E, Mont MA, Malkani AL. Risk of reinfection after treatment of infected total knee arthroplasty. J Arthroplasty 2016;31(9 Suppl):156-61.

Schmier JK, Hulme-Lowe CK, Covert DW, Lau EC. An updated estimate of costs of endophthalmitis following cataract surgery among Medicare patients: 2010-2014. Clinical Ophthalmology 2016;10:2121-27.

Kurtz SM, Lau EC, Ong KL, Adler EM, Kolisek FR, Manley MT. Hospital, patient, and clinical factors influence 30- and 90-day readmission after primary total hip arthroplasty. J Arthroplasty 2016;31(10):2130-8.

Ong KL, Anderson AF, Niazi F, Fierlinger AL, Kurtz SM, Altman RD. Hyaluronic acid injections in Medicare knee osteoarthritis patients are associated with longer time to knee arthroplasty. J Arthroplasty 2016;31(8):1667-73.

Koenig KM, Ong KL, Lau EC, Vail TP, Berry DJ, Rubash HE, Kurtz S, Bozic KJ. The use of hyaluronic acid and corticosteroid injections among Medicare patients with knee osteoarthritis. J Arthroplasty 2016;31(2):351-5.

Schmier JK, Covert DW, Hulme-Lowe C, Mullins A, Mahlis E. Treatment costs of cystoid macular edema among patients following cataract surgery. Clinical Ophthalmology 2016;10:477-83.

Kurtz SM, Lau E, Ong KL, Katz JN, Bozic KJ. Universal health insurance coverage in Massachusetts did not change the trajectory of arthroplasty use or costs. Clin Orthop Relat Res 2016;474(5):1090-8.

Goel A, Lau EC, Ong KL, Berry DJ, Malkani AL. Dislocation rates following primary total hip arthroplasty have plateaued in the Medicare population. J Arthroplasty 2015;30(5):743-6.

Ong KL, Lotke PA, Lau E, Manley MT, Kurtz SM. Prevalence and costs of rehabilitation and physical therapy after primary TJA. J Arthroplasty 2015;30(7):1121-6.

Lovald ST, Ong KL, Malkani AL, Lau EC, Schmier JK, Kurtz SM, et al. Complications, mortality, and costs for outpatient and short-stay total knee arthroplasty patients in comparison to standardstay patients. J Arthroplasty 2014; 29(3):510–5.

Lovald S, Ong K, Lau E, Schmier J, Bozic K, Kurtz S. Mortality, cost, and downstream disease of total hip arthroplasty patients in the Medicare population. J Arthroplasty 2014; 29(1):242–6.

Ong K, Auerbach JD, Lau E, Schmier J, Ochoa JA. Perioperative outcomes, complications, and costs associated with lumbar spinal fusion in older patients with spinal stenosis and spondylolisthesis. Neurosurgical Focus 2014; 36(5):E5.

Lovald ST, Ong KL, Lau EC, Schmier JK, Bozic KJ, Kurtz SM. Mortality, cost, and health outcomes of total knee arthroplasty in Medicare patients. J Arthroplasty 2013; 28(3):449–54.

Ong KL, Lau E, Kemner JE, Kurtz SM. Two-year cost comparison of vertebroplasty and kyphoplasty for the treatment of vertebral compression fractures: are initial surgical costs misleading? Osteoporos Int 2013;24(4):1437-45.

Edidin AA, Ong KL, Lau E, Schmier JK, Kemner JE, Kurtz SM. Cost-effectiveness analysis of treatments for vertebral compression fractures. Applied Health Economics and Health Policy 2012;10(4):273–284.

Schmier JK, Covert DW, Lau EC. Patterns and costs associated with progression of age-related macular degeneration. American Journal of Ophthalmology 2012; 154(4):675–681.

Kurtz SM, Lau E, Watson H, Schmier J, Parvizi J. Economic burden of periprosthetic joint infection in the United States. J Arthroplasty 2012; 27(8 suppl):61–5.

Kurtz SM, Lau E, Ong KL, Carreon L, Watson H, Albert T, Glassman S. Infection risk for primary and revision instrumented lumbar spine fusion in the Medicare population. J Neurosurg Spine 2012;17(4):342-7.

Kurtz SM, Lau E, Ianuzzi A, Schmier J, Lanman T, Isaza J, Albert T. National revision burden for lumbar total disc replacement in the United States: Epidemiologic and economic perspectives. Spine 2010; 35(6):690–6.

Ong KL, Lau E, Suggs J, Kurtz SM, Manley MT. Risk of subsequent revision after primary and revision total joint arthroplasty. Clin Orthop Relat Res 2010;468(11):3070-6.

Kurtz SM, Ong KL, Lau E, Bozic KJ, Berry D, Parvizi J. Prosthetic joint infection risk after TKA in the Medicare population. Clin Orthop Relat Res 2010;468(1):52-6.

Kurtz SM, Ong KL, Schmier J, Zhao K, Mowat F, Lau E. Primary and revision arthroplasty surgery caseloads in the United States from 1990 to 2004. J Arthroplasty 2009; 24(2):195–203.

Ong KL, Kurtz SM, Lau E, Bozic KJ, Berry DJ, Parvizi J. Prosthetic joint infection risk after total hip arthroplasty in the Medicare population. J Arthroplasty 2009;24(6 Suppl):105-9.

Schmier JK, Covert DW, Lau EC, Matthews GP. Medicare expenditures associated with diabetes and diabetic retinopathy. Retina 2009; 29(2):199–206.

Lau E, Ong K, Kurtz S, Schmier J, Edidin A. Mortality following the diagnosis of a vertebral compression fracture in the Medicare population. J Bone Joint Surg Am 2008; 90:1479–86.

Schmier JK, Covert DW, Lau EC, Robin AL. Trends in annual Medicare expenditures for glaucoma surgical procedures from 1997–2006. Arch Ophthalmol 2009; 127(7):900–905.

Kurtz SM, Ong KL, Schmier J, Mowat F, Saleh K, Dybvik E, Kärrholm H, Garellilck G, Havelin LI, Furnes O, Malchau H, Lau E. Future clinical and economic impact of revision total hip and knee arthroplasty. J Bone Joint Surg 2007; 89 Suppl 3:144–51.

Schmier JK, Halpern MT, Covert DW, Lau EC, Robin AL. Evaluation of Medicare costs of endophthalmitis among patients following cataract surgery. Ophthalmology 2007; 114(6):1094–1099.

Schmier JK, Halpern MT, Covert DW, Matthews GP. Evaluation of costs for cystoid macular edema among patients following cataract surgery. Retina 2007; 27(5):621–628.

Halpern MT, Schmier JK, Covert DW, Venkataraman K. Resource utilization and costs of agerelated macular degeneration by disease type. Health Care Financ Rev 2006; 27(3):37–47.

Halpern MT, Zilberberg MD, Schmier JK, Lau EC, Schorr AF. Anemia, costs and mortality in chronic obstructive pulmonary disease. Cost Effect Resource Alloc 2006; 4:17.

Commercial Claims Databases:

Schmier JK, Hulme-Lowe CK, Covert DW. Adjunctive therapy patterns in glaucoma patients using prostaglandin analogs. Clin Ophthalmol 2014; 8:1097–1104.

Schmier JK, Covert DW, Lau EC. Medical service encounters and payments associated with topical adjunctive therapy use of timolol for glaucoma. Clinical Drug Investigation 2012; 32(12):835–42.

Schmier JK, Covert DW. First-year treatment costs among new initiators of topical prostaglandin analogs: Pooled results. Clinical Ophthalmology 2010; 4:437–45.

Schmier JK, Covert DW, Robin AL. First-year treatment costs among new initiators of topical prostaglandin analog identified from November 2007 through April 2008. Current Medical Research & Opinion 2010; 26(12):2769–77.

Schmier JK, Lau EC, Covert DW. Two-year treatment patterns and costs in glaucoma patients initiating treatment with prostaglandin analogs. Clinical Ophthalmology 2010; 4:1137–43.

Schmier JK, Covert DW, Robin AL. Estimated first-year costs of prostaglandin analogs with/without adjunctive therapy for glaucoma management: a United States perspective. Curr Med Res Opin 2007; 23(11):2867–75.

Schmier JK, Covert DW, Robin AL. First-year treatment costs among new initiators of topical prostaglandin analogs. Clin Ophthalmol 2009; 3:637–44.

Schmier JK, Covert DW, Robin AL. First-year treatment patterns among newly-diagnosed glaucoma patients starting treatment with a prostaglandin analog. Curr Med Res Opin 2009; 25(4):851–8.

Professionals

Knowledge