Table 3.
Case examples of RWE for drug development strategy and clinical trial design
| Use | Citation | Study Objective | Data Source(s) | Insight |
|---|---|---|---|---|
| Understanding patient populations | Broder et al. (2018) 17 | Estimate prevalence and incidence of neuroendocrine tumors | IBM MarketScan and IQVIA PharMetrics claims databases | Prevalence and incidence increasing over time. |
| Dellon et al. (2014) 66 | Estimate prevalence of EE | IQVIA PharMetrics claims | Updated estimates for number of patients with EE in the United States following the introduction of a new ICD‐9 diagnosis code specific to EE. | |
| Wallin et al. (2019) 16 | Estimate national prevalence for MS by analyzing multiple US databases, covering different population segments. | Optum, IBM, Kaiser Permanente, Department of Veterans Affairs, and the Centers for Medicare and Medicaid claims databases | The 3‐year prevalence of MS was 309.2 per 100,000, with an estimated 727,344 cases in the United States, higher than previous studies. | |
| Halpern et al. (2019) 67 | Estimate prevalence of agitation among patients with AD | Optum EHR database | Prevalence of agitation over a 2‐year period was 44.6%. NLP was used to analyze unstructured data for keywords related to agitation. | |
| Chehade et al. (2021) 68 | Describe patient journey for individuals with EG/EoD | Symphony Health Patient Source claims database | Many EG/EoD patients initially diagnosed with irritable bowel syndrome or dyspepsia, highlighting the need for improved diagnosis. | |
| Morgan et al. (2021) 69 | Describe diagnostic journey of patients with PSP | Patient interviews and physician chart reviews in France, Germany, Italy, Spain, the United Kingdom, and the United States | Diagnostic delays may be related to patients first presenting to primary care providers before being evaluated by movement disorder specialists. | |
| Understanding treatment patterns | Zhu et al. (2019) 70 | Characterize current treatment patterns for AA in China | Disease Registry in China | Only 1 in 5 AA patients were receiving first‐line care concordant with evidence‐based guidelines |
| Stewart et al. (2021) 71 | COVID‐19: understand medication use, hospital‐based mechanical therapies, disease progression, and re‐infection | HealthVerity used tokenization to link multiple data sources | Use of hydroxychloroquine with or without azithromycin among hospitalized patients with COVID‐19 was described. | |
| Murage et al. (2019) 54 | Examine treatment patterns for patients with psoriasis receiving biologic therapies. | IQVIA PharMetrics database linked to the Modernizing Medicine EHR database | Results on combination therapy, switching, adherence, and discontinuation are valuable for biopharmaceutical companies developing therapies targeting specific patient subgroups (i.e., treatment failures) | |
| Shah et al. (2017) 65 | Applied eligibility criteria from phase III clinical trials for MM to assess the proportion of patients being excluded from trials. | Disease Registry | Estimated that 40% of MM patients – 52.7% of African American patients – would not qualify for any clinical trials | |
| Foerster et al. (2021) 72 | Describe the diagnostic journey for women with breast cancer in Sub‐Saharan Africa | Prospective Cohort Study | White patients in Nigeria had a median diagnostic journey of only 2.4 months, compared with 11.3 months for patients in Uganda. | |
| Bakouny et al. (2021) 73 | Effect of COVID‐19 pandemic on cancer screening and diagnosis | EHRs from one integrated delivery network | Cancer screening procedures decreased 60%‐82% from 2019 to 2020. New cancer diagnoses decreased 19%–78%. | |
| Understanding diseases | Bali et al. (2017) 53 | Natural history study of ALS with A4V SOD1 genotype | EHRS from 15 North American medical centers | Genotype is adequately defined and understood to study in clinical trials. Data on disease course used to inform future trial sample size calculations. |
| Scher et al. (2015) 59 | Build a dynamic progression model for prostate cancer | NCI‐SEER | Findings could be used to design clinical trials targeting a patien t subgroup with the greatest unmet need. | |
| Tabrizi et al. (2012) 60 | Understand disease progression in HD | Disease Registries | Endpoint selection for future trials should use serial brain imaging rather than measures related to quality of life. Former was more sensitive to changes in clinical presentation. | |
| Ataga et al. (2020) 58 | Understand how hemoglobin concentration is related to stroke, cerebrovascular disease, kidney disease, pulmonary vasculopathy, and mortality in patients with SCD | Meta‐Analysis including disease registries | Changes in hemoglobin concentration is a validated intermediary measure of disease progression in patients with SCD. |
AA, Aplastic Anemia; AD, Alzheimer’s disease; ALS, amyotrophic lateral sclerosis; COVID‐19, coronavirus disease 2019; EE, eosinophilic esophagitis; EG/EoD, eosinophilic gastritis or duodenitis; EHR, electronic health record; HD, Huntington’s disease; ICD‐9, International Classification of Disease 9th revision; MM, multiple myeloma; MS, multiple sclerosis; NLP, natural language processing; PSP, progressive supranuclear palsy; RWE, real‐world evidence; SCD, sickle cell disease; SEER, Surveillance, Epidemiology and End Results.