Real World Evidence: Understand Trends & Seize Opportunities in the Changing Landscape of RWE Generation

Industries Profiled:
Health Care; Pharmaceutical; Diagnostic; Biotech; Manufacturing; Consumer Products; Medical Device; Biopharmaceutical; Clinical Research; Laboratories


Companies Profiled:
Abbott; Abbvie; Allergan; AstraZeneca; Bayer; Celldex Therapeutics; Dermira; EMD Serono; Flexion Therapeutics; GlaxoSmithKline ; Grifols; Janssen; Johnson & Johnson; Merck; Novartis; Sanofi; UCB Pharma; Vifor Pharma; Genzyme

Study Snapshot

Best Practices, LLC engaged 25 leaders with deep experience in managing Real World Evidence generation at 19 leading pharmaceutical companies in this benchmarking research.


Key topics covered in this report include:

• Real World Evidence Study Methods Utilized
• Methods of Data Collection
• Outsourcing
• Real World Evidence Budget
• Utilization of Vendors for Real World Evidence Generation

• Overview: Study methods that will be utilized the most in the future are Prospective Observational, Health Economic Modeling, Health Economic/HTA, Medical Record Abstraction, Drug Utilization Studies, and Disease Registries
• Economic/Reimbursement Studies: These RWE studies are the most utilized by respondents; future use to grow 26%
• Observational Studies: Three of the five studies in this category will be used by +70% of respondents in the future
• Extension Studies: This category of RWE studies used little but will Increase the most in use; future use to jump 123%

• Overview: A mix of primary & secondary data is a widely used data collection method for 9 of 16 RWE study types

• Overview: A majority of respondents use a mix of in-house and outsourcing for 12 (75%) of the 16 RWE studies

• Overview: Experience on Similar Studies, Scientific Expertise, & Prior Experience with Vendor are top 3 vendor traits

I.	Executive Summary: Methodology, Participants, Recommendations & Key Findings	pgs. 3-18
	Universe of Learning: Benchmark Class
	Study Structure & Design
	Study Recommendations and Findings
II.	Overview of RWE Study Methods Utilization, Data Collection Methods & Outsourcing	pgs. 19-39
	Economic / Reimbursement Studies Category
	Observational Studies Category
	Non-Safety Registries Category
	Intervention Studies Category
	Extension Studies Category
III.	RWE Budget and Rationale for Outsourcing	pgs. 40-46
	Average Spent on RWE Vendors
	Rationale for Using Outsourcing
IV.	Economic / Reimbursement Studies	pgs. 47-57
V.	Observational Studies	pgs. 58-83
VI.	Non-Safety Registries Studies	pgs. 84-104
VII.	Intervention Studies	pgs. 105-120
VIII.	Extension Studies	pgs. 121-131
IX.	Benchmark Class Profile	pgs. 132-137
X.	Appendix	pgs. 138-143

• Understanding future trends in the changing landscape of real world evidence generation
• Six RWE study methods that pharma will heavily utilize in the future
• RWE study methods with most growth potential
• Current and future use of RWE Studies
• Top vendor traits: Experience with similar studies and scientific expertise

• Economic / reimbursement studies – Current, past and future utilization; current and future growth potential; outsourcing
• Current and future use of economic / reimbursement studies
• Data collection methods utilized and future preference for the same for economic / reimbursement studies
• Top vendor traits for selecting a vendor for economic / reimbursement studies
• Observational studies category - Current, past and future utilization; current and future growth potential; outsourcing
• Current and future use of observational studies
• Data collection methods utilized and future preference for data collection method for observational studies
• Top vendor traits for selecting a vendor for observational studies
• Non-safety registries category - Current, past and future utilization; current and future growth potential; outsourcing
• Current and future use of non-safety registries
• Data collection methods utilized and future preference for data collection method for non-safety registries
• Top vendor traits for selecting a vendor for non-safety registries
• Intervention studies category - Current, past and future utilization; current and future growth potential; outsourcing
• Current and future use of intervention studies
• Data collection methods utilized and future preference for data collection method for intervention studies
• Top vendor traits for selecting a vendor for intervention studies
• Extension studies - Current, past and future utilization; current and future growth potential; outsourcing
• Current and future use of extension studies
• Data collection methods utilized and future preference for data collection method for extension studies
• Top vendor traits for selecting a vendor for extension studies

• Budget allocation for RWE generation and anticipated RWE generation budget in 2020
• Rationale for using in-house resources for RWE generation
• Rationale for using outsourcing approach
• Rationale for using mix resourcing approach
• The deciding factor for in-house or outsourcing RWE work

• Current and future utilization level for health economic modeling study method
• Number of studies conducted using health economic modeling method and anticipated use of health economic modeling study method
• Data collection methods utilized for health economic modeling studies and future preference
• Current and future utilization of outsourcing for health economic modeling studies
• Vendor selection criteria and vendor preference for health economic modeling studies
• Current and future utilization level for health economic / health technology assessment (HTA) dossier study method
• Number of studies conducted using health economic / health technology assessment (HTA) dossier method and anticipated use health economic / HTA dossier study method
• Data collection methods utilized for health economic / HTA dossier studies and future preference
• Current and future utilization of outsourcing for health economic / HTA dossier studies
• Vendor selection criteria and vendor preference for health economic / HTA dossier studies

• Current and future utilization level for post-authorization safety studies
• Number of studies conducted using post-authorization safety method and anticipated use of post-authorization safety study method
• Data collection methods utilized for post-authorization safety studies and future preference
• Current and future utilization of outsourcing for post-authorization safety studies
• Vendor selection criteria and vendor preference for post-authorization safety studies
• Current and future utilization level for drug utilization studies
• Number of studies conducted using drug utilization study method and anticipated use of drug utilization study method
• Data collection methods utilized for drug utilization studies and future preference
• Current and future utilization of outsourcing for drug utilization studies
• Vendor selection criteria and vendor preference for drug utilization studies
• Current and future utilization level for prospective observational studies with HEOR objectives
• Number of studies conducted using the method of prospective observational studies with HEOR objectives and anticipated use of prospective observational studies with HEOR objectives
• Data collection methods utilized for prospective observational studies with HEOR objectives and future preference
• Current and future utilization of outsourcing for prospective observational studies with HEOR objectives
• Vendor selection criteria and vendor preference for prospective observational studies with HEOR objectives
• Current and future utilization level for post-authorization efficacy studies
• Number of post-authorization efficacy studies conducted and anticipated use of post-authorization efficacy studies
• Data collection methods utilized for post-authorization efficacy studies and future preference
• Current and future utilization of outsourcing for post-authorization efficacy studies
• Vendor selection criteria and vendor preference for post-authorization efficacy studies
• Current and future utilization level for medical record abstraction / retrospective chart reviews
• Number of studies conducted using the method of medical record abstraction / retrospective chart reviews and anticipated use of medical record abstraction / retrospective chart reviews
• Data collection methods utilized for medical record abstraction / retrospective chart reviews and future preference
• Current and future utilization of outsourcing for medical record abstraction / retrospective chart reviews
• Vendor selection criteria and vendor preference for medical record abstraction / retrospective chart reviews

• Current and future utilization level for disease registries
• Number of disease registry studies conducted and anticipated use of disease registries
• Data collection methods utilized for disease registries and future preference
• Current and future utilization of outsourcing for disease registries
• Vendor selection criteria and vendor preference for disease registries
• Current and future utilization level for product registries (not including vaccine registries)
• Number of product registry (not including vaccine registry) studies conducted and anticipated use of product registries (not including vaccine registries)
• Data collection methods utilized for product registries (not including vaccine registries) and future preference
• Current and future utilization of outsourcing for product registries (not including vaccine registries)
• Vendor selection criteria and vendor preference for product registries (not including vaccine registries)
• Current and future utilization level for pregnancy registries
• Number of pregnancy registry studies conducted and anticipated use of pregnancy registries
• Data collection methods utilized for pregnancy registries and future preference
• Current and future utilization of outsourcing for pregnancy registries
• Vendor selection criteria and vendor preference for pregnancy registries
• Current and future utilization level for vaccine registries
• Number of vaccine registry studies conducted and anticipated use of vaccine registries
• Data collection methods utilized for vaccine registries and future preference
• Current and future utilization of outsourcing for vaccine registries
• Vendor selection criteria for vaccine registries

• Current and future utilization level for expanded access programs
• Number of expanded access programs conducted and anticipated use of expanded access programs
• Data collection methods utilized for expanded access programs and future preference
• Current and future utilization of outsourcing for expanded access programs
• Vendor selection criteria and vendor preference for expanded access programs
• Current and future utilization level for randomized pragmatic trials
• Number of randomized pragmatic trials conducted and anticipated use of randomized pragmatic trials
• Data collection methods utilized for randomized pragmatic trial and future preference
• Current and future utilization of outsourcing for randomized pragmatic trial
• Vendor selection criteria and vendor preference for randomized pragmatic trial
• Current and future utilization level for other low-intervention clinical trials
• Number of other low-intervention clinical trials conducted and anticipated use of other low-intervention clinical trials
• Data collection methods utilized for other low-intervention clinical trial and future preference
• Current and future utilization of outsourcing for other low-intervention clinical trial
• Vendor selection criteria and vendor preference for other low-intervention clinical trial

• Current and future utilization level for concurrent outcomes studies
• Number of concurrent outcomes studies conducted and anticipated use of concurrent outcomes studies
• Data collection methods utilized for concurrent outcomes studies and future preference
• Current and future utilization of outsourcing for concurrent outcomes studies
• Vendor selection criteria and vendor preference for concurrent outcomes studies
• Current and future utilization level for extension studies
• Number of extension studies conducted and anticipated use of extension studies
• Data collection methods utilized for extension studies and future preference
• Current and future utilization of outsourcing for extension studies
• Vendor selection criteria and vendor preference for extension studies

• Benchmark partners’ geographic responsibility for RWE generation
• Therapeutic area responsibility of benchmark participants
• Benchmark partners’ organizational and product type responsibility
• Portfolio responsibility of benchmark participants
• Functional area responsibility of benchmark partners

• RWE study methods with significant growth opportunities in future
• Definitions of RWE study types