REPORT KEY FINDINGS
- Adopt ePRO technology to maximize PRO efficiency.
- Focus on data quality for PRO endpoints to impact final product label.
- Combine standard PROs with therapeutic-area specific measurements.
- Leverage a dedicated PRO team to ensure strategic focus and regulatory compliance.
- Begin PRO planning and administration early in the product lifecycle.
WHY CLIENTS NEED THIS REPORT
- Add PRO endpoints and measurements into clinical trials to add value to drug dossiers/payer discussions.
- PRO measurement and analysis is still developing and unstandardized. Clients can use this report to learn:
- Which measurements to choose, how many to use and how to leverage them in regulatory and reimbursement discussions
- How to build and resource PRO teams
- Trends and emerging topics in PRO-driven initiatives
This report's five (5) chapters contain benchmarks and best practices exploring PRO usage, strategy, resources, timelines and efficiency.
- Benchmarks for PRO team structure, alignment, team size, budget management and average cost per patient.
- Detailed data for prevalence of specific PRO metrics measured, including signs and symptoms and health-related quality of life (HRQoL).
- Benchmarks for percentage of PRO endpoints on final product labels.
- Timelines for PRO planning, administration and data retrieval.
- Detailed benchmarks showing outsourcing levels and vendor selection practices at all PRO stages.
- Ratings for seven (7) PRO measurements in supporting quality of life and cost-effectiveness endpoints.
- Assessments showing PROs' effectiveness in achieving study goals in key therapeutic areas.
- Data and best practices analyzing prevalence of various PRO delivery mechanisms, spanning traditional pen-and-paper methods to tablets and other digital media.
- Leading executives outline the benefits and challenges associated with electronic PROs (ePRO) approaches.
- Timelines and costs to implement ePRO strategies.
- Average percentage of PRO endpoints on final product labels.
- Guidelines for optimizing PROs' impact on regulatory discussions and data effectiveness.
Data are split, where relevant:
- Company size:
- Top 10
- Top 50
- Small pharma
- Therapeutic areas:
- Infectious Diseases
KEY QUESTIONS THIS REPORT ANSWERS
Optimize PRO Strategy and Efficiency
- Which delivery mechanisms do companies commonly use to administer PROs?
- What are the benefits and problems posed by traditional methods?
- What advantages do companies gain from ePRO technology?
- What obstacles do companies face in implementing ePROs?
- How can teams win buy-in from senior management to adopt an ePRO approach?
- When during the product lifecycle should companies plan for PROs?
- When and how often should companies administer PROs and retrieve data to ensure maximum impact?
- How do teams balance in-house/outsourced resources and maximize vendor support throughout PRO projects?
Select the Right PRO Instrument to Impact the Final Product Label
- What PRO measurements are companies using to ensure the most accurate data?
- What criteria or processes do teams develop to find best-fit PRO tools?
- When are standard PRO measurements sufficient? When are customized PRO tools a better option?
- How successful are these PROs in supporting quality of life and/or cost-effectiveness arguments?
- How does PRO effectiveness vary across therapeutic area?
- How do companies overcome challenges in PRO endpoint approval?
- What strategies should teams employ to facilitate a smooth label claim approval process?
Ensure Strategic Focus and Regulatory Compliance via Dedicated PRO Teams
- How does having a dedicated group benefit PRO initiatives?
- Which functions oversee PRO teams?
- What advantages come from having clinical development manage PRO budgets?
- How large should a PRO team be to handle and balance increasing clinical- and market access-related activities?
- What is the average cost per patient for PROs in specific therapeutic areas?
CHAPTER BENEFITS AND METRICS BREAKDOWN
Chapter 1 examines patient-reported outcomes team structures and budgets. The chapter provides a brief background on PROs and their growing importance. It also discusses various ways to structure a PRO team, highlighting the benefits of having a dedicated group. Next, the chapter looks at PRO budget management and how this affects the PRO groups' overall operations. Benchmarks and executives' input stress the advantages of giving the clinical operations team budgetary oversight of PRO teams.
- Develop a cohesive PRO strategy to meet regulatory and payer demands in both US and EU markets.
- Create a dedicated PRO team and allot ample staff to develop customized PRO measurements.
- Align PRO teams by therapeutic area to create targeted PRO strategies.
- Keep budget within clinical operations for easier trial integration.
11 charts detailing patient-reported outcomes team structure and resources. Throughout the chapter, data are often broken down by company size (top 10, top 50 and small pharma).
- Regions involved in PRO strategy development
- Percentage of companies with a dedicated PRO team
- Function providing oversight of PRO teams (by company)
- PRO team alignment, by company size
- PRO team size (number of FTEs)
- Number of PRO team FTEs, by level of executive
- Function responsible for PROs in absence of dedicated team
- Function managing PRO team budget (by company)
- Average cost per patient for PROs (by company and therapeutic area)
The prevalence of PRO measures in clinical trials has grown as the pharmaceutical industry shifted toward increased patient-centricity. This chapter examines pharmaceutical manufacturers' PRO usage in clinical testing as well as their outsourcing practices. Data show the percentage of clinical trials leveraging PRO endpoints and when companies begin planning for these patient-reported outcomes initiatives. Further research highlights industry outsourcing levels in addition to vendor selection best practices.
- Use PROs to target signs and symptoms measurements and HRQoL data.
- Balance PRO endpoints with traditional clinical endpoints.
- Start planning and administering PROs early in timelines, often Phase 1 or Phase 2.
- Outsource PRO activities to complement internal capabilities.
- Maintain a list of innovative, preferred vendors when outsourcing specific study tasks.
27 charts detailing prevalence of PRO measurements, planning stages, vendor selection for PRO initiatives and percentage of budget outsourced. In some cases, data are broken down by company size (top 10, top 50 and small pharma).
- Average percentage of company trials implementing PRO measurements
- Percentage of companies measuring specific PRO metrics
- Signs and symptoms
- Health-related Quality of Life
- Physical functioning
- Quality of Life
- Patient satisfaction
- Health status
- Psychological functioning
- Treatment adherence
- Reports and ratings of healthcare
PRO Planning and Administration Timelines
- Of trials with PROs, percentage of endpoints that are PROs (by therapeutic area)
- Phase at which PRO planning begins
- Phase and frequency at which a PRO is administered to patient
- Phase and frequency of PRO data retrieval
PRO Initiative Budget Outsourcing and Vendor Selection
- Average percentage of PRO initiative budget outsourced for nine (9) therapeutic areas
- Average percentage of PRO initiative budget outsourced, by company for these therapeutic areas:
- Vendor selection by PRO initiative stage (by company size)
Chapter - 3
This chapter examines different PRO instruments, including broad overall health functioning measures as well as therapeutic area-specific instruments. PRO measurements are assessed on their ability to support quality-of-life endpoints and cost-effectiveness endpoints. In addition, this chapter examines PROs' ability to achieve key study goals in four therapeutic areas: CNS, respiratory, immunology and oncology. Chapter 3 discusses the use of disease-specific PRO measurements in these therapeutic areas.
- Start with standard PRO measurements (SF-36, EQ-5D) to capture basic HRQoL or cost-effectiveness data.
- Use PROs to support diverse goals, including marketing initiatives and medical affairs/publications strategies.
- Select or develop therapeutic area-specific measurements to complement broader, standard measurements.
20 charts detailing PRO effectiveness in supporting trial endpoints and the prevalence of specific PRO measurements in trials. In some cases, data are broken down by company size (top 10, top 50 and small pharma).
- Ratings of PRO effectiveness in supporting quality of life endpoints and cost-effectiveness endpoints (by company size)
- Percentage of companies implementing PROs, by therapeutic area
- Ratings of PRO effectiveness in achieving study goals, by therapeutic area
- Establish relationships with patients
- Bolster reimbursement decisions
- Support marketing initiatives
- Support medical affairs/publications strategies
- Support clinical trial endpoints
- Percentage of trials (CNS, respiratory, immunology and oncology) using specific PRO measurements, including:
Chapter - 4
This chapter discusses PRO delivery mechanisms and modalities, a key aspect of PRO selection and implementation. Modalities, both electronic and paper-and-pen, are examined across surveyed companies. Next, this chapter offers alternatives to purely patient-reported questionnaires, such as clinician- and proxy-reported formats. Finally, this chapter examines the benefits of implementing an ePRO strategy and the key metrics of companies already using this technology.
- Transition from paper format to ePRO via mobile devices to improve data quality.
- Include ClinRO and ObsRO in relevant trials.
- Budget significant proportion of ePRO budget to project costs and hardware expenses.
7 charts detailing companies' use of electronic PROs and timelines for implementing ePRO systems.
- Percentage of companies using PRO modalities, by company size and modality type:
- Interactive Voice Response System (IVR)
- Personal journal/paper questionnaire
- Tablet/mobile device
- Percentage of companies using specific additional PRO delivery mechanisms (caregiver, clinician, or proxy)
- Percentage breakdown of ePRO initiative costs
- Percentage of companies using ePRO technology
- Diagram showing benefits of adopting ePRO technology
- Number of years since ePRO implementation
- Timeline of planned ePRO implementation
Chapter - 5
The patient experience is increasingly valuable to those charged with making regulatory and reimbursement decisions, making PROs particularly important. This chapter examines how regulatory bodies tend to perceive PROs and how likely PROs are to end up on the final product label. It references regulatory guidance, industry consortia and guidelines to maximizing PROs' impact on regulatory discussions and data effectiveness.
- Target favorable therapeutic areas to land PRO data on product labels.
- Leverage PROs in submissions in EU and US.
- Look to consortia for development of standardized PRO measurements in specific therapeutic areas.
- Look to FDA PRO Guidances when developing PRO strategy.
10 charts detailing the presence of PRO endpoint on the final product label.
- Ratings of PRO effectiveness in discussions with regulatory bodies (by region)
- Average percentage of PRO endpoints on final product labels (by company), for the following therapeutic areas
- Infectious Disease