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Phase IV Clinical Trials (PH157)

Best Practices in Post-Marketing Study Management
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Published 2011
197 Pages
500+ Metrics
173+ Charts and Diagrams

Boost Your Post-Marketing Study Program

Whether building a better product profile, establishing comparative effectiveness or accomplishing any number of other objectives, post-marketing research grants companies a range of clinical and commercial benefits. Amid rigorous payer demands and competitive pressures, Phase IV studies are a growing necessity — but they often remain deprioritized and under-resourced as teams struggle to marshal needed support for both strategy and execution.  

Top Reasons to Buy this Report

Use this study to reinforce Phase IV research and perfect study management. Examine benchmarks for US and EU data across major therapeutic areas and five trial types: Integrate study planning and execution: Explore real-company structures as you work to maximize expertise and dissolve communication blocks. Clarify roles so that Phase IV objectives receive adequate attention. Win post-marketing study resource support: Match your needs to trial size and scope — compare planned versus actual spending and peruse staffing tables for 51 real-world Phase IV trials. Track per patient budgets and headcounts by trial type, geography and company size. Along the way, learn strategies for balancing outsourcing with in-house capabilities to ensure consistent trial support and top performance. Accelerate the trial timeline: Track timing using data broken down by study types, geography and nine key trial activities. Measure performance: Discover the metrics top companies use to track performance and drive improvement in their Phase IV operations.

Phase IV Study Management Metrics

Chapter 1: Study Management Structure, Oversight and Strategy

Chapter Benefits
  • Ensure adequate infrastructure and support for Phase IV trials.
  • Understand and designate clear roles for Phase IV strategy, management and execution.
  • Benchmark patient enrollment numbers for specific therapeutic areas.
Chapter Data 54 charts reveal Phase IV structure and strategy data in the US and EU:
  • Examples of real-world structures for Phase IV program management
  • Percentage of Phase IV work handled in-house
  • Number of ongoing Phase IV studies, with breakdowns by trial type
  • Departments included in Phase IV strategic development and execution
  • Patient enrollment and average patients per investigator site across all therapeutic areas (comparative effectiveness trials, registries and observational trials, investigator initiated studies (IIS), large simple safety trials)
  • Average patients per investigator site data for specific therapeutic areas (oncology, cardiovascular diseases, cardiology & thrombosis, CNS/neurology, vaccines, type 2 diabetes, musculoskeletal)
 

Chapter 2: Phase IV Trial Timelines, Activities and Performance Metrics

Chapter Benefits
  • Target areas/activities that present opportunities to accelerate trials.
  • Understand which trial types are being conducted where, and why.
  • Learn which metrics other companies use to track Phase 4 trial performance.
  • Understand the unique challenges patient enrollment presents in post-marketing research.
Chapter Data 63 charts focused on trial execution and measurement:
  • Percentage of drugs for which groups conduct Phase IV trials (in US and EU)
  • Point at which US and EU groups initiate studies
  • Average and percentage difference between planned versus actual trial duration
  • Time consumed by key trial activities
  • Planned versus actual enrollment
  • Opportunities for accelerating trial timelines (in US and EU)
  • Performance measurement self-ratings for study design & concept development, protocol development/writing, CRO/vendor selection, site selection/enrollment/contracting, patient enrollment, data collection and cleaning, and statistical analysis
  • Percentage of companies tracking performance metrics (in US and EU), including efficiency/operations metrics, resource management metrics, time milestone metrics
  • Weighted rankings of performance metrics and KPIs
 

Chapter 3: Phase IV Study Budgets and Outsourcing

Chapter Benefits
  • Use real-company data to justify and win resources for your Phase IV programs.
  • Benchmark costs for Phase IV trials in four major therapeutic areas.
  • Examine real-company staffing data for 51 trials.
  • Balance the benefits and disadvantages of Phase IV outsourcing trends.
Chapter Data 49 charts and tables focusing on resource benchmarks for the five examined post-marketing trials:
  • Average cost per patient for oncology, cardiovascular diseases, CNS/neurology, cardiology & thrombosis
  • Staffing tables with in-house and outsourcing data for 12 clinical positions for 51 trials
  • Average numbers of patients and investigator sites
  • Average number of patients per CRA and patients per site ratios
  • Average percentage of trial budgets outsourced
  • Planned versus actual cost for IIS

Set Phase IV Study Strategy Within the Medical Affairs Function

The following excerpt is taken from Chapter 1, “Study Management Structure, Oversight and Strategy.” It discusses departmental roles for setting Phase IV strategy among companies in the US and the EU using examples from surveyed companies. The full chapter explores the spectrum of post-marketing study management structures in place across the industry, citing benefits and disadvantages for each. It also explores oversight for Phase IV strategy and execution. Finally, it contains patient enrollment benchmarks for five different Phase IV trial types across seven different therapeutic areas. Today, medical affairs teams remain the dominant players who set the strategy for Phase IV studies, but decisions are not made in a vacuum. Several other corporate functions impact strategy. According to Figure 1.17, 72% of surveyed companies indicate that their medical affairs teams play a role in the development of Phase IV clinical strategy. But clinical and marketing teams widely maintain influence on Phase IV strategy development. Clinical teams impact strategy development at half of all surveyed companies, while marketing teams are involved at 44% of all surveyed companies. When survey responses are broken out into US and European data, patterns emerge. US pharmaceutical companies ask fewer functional groups to take part in the development of a corporate Phase 4 strategy than do European companies. Half of the US companies surveyed (Figure 1.18 [not shown]) use only one department to set Phase IV strategy; two companies use medical affairs and two companies use clinical development. In contrast, European companies (Figure 1.19 [not shown]) employ a committee approach to strategy development. Of the 10 surveyed European companies, only 2 rely on a single department to set strategy — both use medical affairs. In fact, 30% of European companies ask four or more departments to assist in Phase IV strategy development. One reason for such heavy Phase IV responsibility within medical affairs is due to the use of IIS as Phase IV studies. In addition, once pharmaceutical brand teams launch their products, companies often transition responsibilities away from the initial launch team. For example, many companies employ a lifecycle management team to oversee product activities after launch. This shift in responsibility allows the LCM team to prepare for the drug’s inevitable patent expiration. Phase IV studies play an important role in lifecycle management, especially when companies use study results to expand into new markets. In much the same way, medical affairs teams take over responsibility for Phase IV studies after product launch. Company B, for example, shifts its post-marketing research operations away from its commercial teams to medical affairs. The company’s Phase IV study management organization is housed within medical affairs; however, the responsibility for setting strategy and executing the trials falls to each therapeutic area leader. Each therapeutic area also has medical directors who provide input on study strategy and execution, but the two responsibilities are not separate. Company A’s medical affairs department sets the strategy for all Phase IV studies. For IIS, the medical affairs department works closely with each investigator to develop protocols that fit best with company strategy. The company’s medical affairs team is also responsible for approving the IIS and processing payments to investigators. For company-sponsored studies, Company A’s vice president of medical affairs designed the main post-marketing research strategy with input from the company’s medical director and MSL director. However, the marketing or commercial operations group, in collaboration with medical affairs, often develops the strategy for a Phase IV study’s direction. According to one interviewed executive at Company A, Phase IV studies are designed to support the brand franchises in each therapeutic area. In addition, the technical aspects of the study design, protocol, endpoints and analyses are all medical affairs’ responsibilities.   The following excerpt is taken from Chapter 2, “Phase IV Trial Timelines, Activities and Performance Metrics.” The chapter examines trial timelines and performance measurement, identifying areas that cause delays and providing solutions for trial acceleration. Benchmarking data for the EU and US are broken out by therapeutic area and by trial type. This excerpt discusses one of the greatest challenges — and areas for improvement — in Phase IV research: patient enrollment.

Phase IV Patient Enrollment

Companies of all sizes struggle with patient enrollment. Surveyed companies offered a variety of reasons why patient recruitment and enrollment occupies such a significant amount of time. Companies face two major problems in recruiting patients. First, they have difficulty finding patients on the whole, whether the companies are looking for general practice patients or treatment-naïve patients. Second, even if there is a potential patient population to recruit, the competition for available patients is fierce. Figures 2.31 to 2.38 show planned versus actual patient enrollment for IIS, comparative effectiveness, and registries and observational trials. IIS present the largest percentage deficit between planned and actual patient numbers, largely because IIS investigators tend not to have the experience and resources of the traditional investigators and centers used in Phase III trials. Registries and observational trials have the largest average difference between planned and actual patient enrollment numbers, but this is due to Company 20’s outlier data, in which it reported a 61% difference. Lack of Patients In Phase IV trials, many companies look outside the typical patient centers that would be used for Phase II and III studies. This is mainly to engage the more specialized patient populations sometimes required by post-marketing studies. And for companies that are accessing investigators and patients though the typical early-phase centers, many find it hard to locate qualified investigators and even harder to enroll the general practice patients in studies without enlisting the physician as an investigator. Companies will often provide additional training to investigators to encourage them to enroll, and in return, the company receives the benefit of enrolling that investigator’s patients in the post-marketing study.

Phase IV Clinical Trials Report Sample

The following excerpt is taken from Chapter 1, “Study Management Structure, Oversight and Strategy.” It discusses departmental roles for setting Phase IV strategy among companies in the US and the EU using examples from surveyed companies. The full chapter explores the spectrum of post-marketing study management structures in place across the industry, citing benefits and disadvantages for each. It also explores oversight for Phase IV strategy and execution. Finally, it contains patient enrollment benchmarks for five different Phase IV trial types across seven different therapeutic areas.  

Set Phase IV Study Strategy Within the Medical Affairs Function

  Today, medical affairs teams remain the dominant players who set the strategy for Phase IV studies, but decisions are not made in a vacuum. Several other corporate functions impact strategy. According to Figure 1.17, 72% of surveyed companies indicate that their medical affairs teams play a role in the development of Phase IV clinical strategy. But clinical and marketing teams widely maintain influence on Phase IV strategy development. Clinical teams impact strategy development at half of all surveyed companies, while marketing teams are involved at 44% of all surveyed companies. When survey responses are broken out into US and European data, patterns emerge. US pharmaceutical companies ask fewer functional groups to take part in the development of a corporate Phase 4 strategy than do European companies. Half of the US companies surveyed (Figure 1.18 [not shown]) use only one department to set Phase IV strategy; two companies use medical affairs and two companies use clinical development. In contrast, European companies (Figure 1.19 [not shown]) employ a committee approach to strategy development. Of the 10 surveyed European companies, only 2 rely on a single department to set strategy — both use medical affairs. In fact, 30% of European companies ask four or more departments to assist in Phase IV strategy development. One reason for such heavy Phase IV responsibility within medical affairs is due to the use of IIS as Phase IV studies. In addition, once pharmaceutical brand teams launch their products, companies often transition responsibilities away from the initial launch team. For example, many companies employ a lifecycle management team to oversee product activities after launch. This shift in responsibility allows the LCM team to prepare for the drug’s inevitable patent expiration. Phase IV studies play an important role in lifecycle management, especially when companies use study results to expand into new markets. In much the same way, medical affairs teams take over responsibility for Phase IV studies after product launch. Company B, for example, shifts its post-marketing research operations away from its commercial teams to medical affairs. The company’s Phase IV study management organization is housed within medical affairs; however, the responsibility for setting strategy and executing the trials falls to each therapeutic area leader. Each therapeutic area also has medical directors who provide input on study strategy and execution, but the two responsibilities are not separate. Company A’s medical affairs department sets the strategy for all Phase IV studies. For IIS, the medical affairs department works closely with each investigator to develop protocols that fit best with company strategy. The company’s medical affairs team is also responsible for approving the IIS and processing payments to investigators. For company-sponsored studies, Company A’s vice president of medical affairs designed the main post-marketing research strategy with input from the company’s medical director and MSL director. However, the marketing or commercial operations group, in collaboration with medical affairs, often develops the strategy for a Phase IV study’s direction. According to one interviewed executive at Company A, Phase IV studies are designed to support the brand franchises in each therapeutic area. In addition, the technical aspects of the study design, protocol, endpoints and analyses are all medical affairs’ responsibilities. The following excerpt is taken from Chapter 2, “Phase IV Trial Timelines, Activities and Performance Metrics.” The chapter examines trial timelines and performance measurement, identifying areas that cause delays and providing solutions for trial acceleration. Benchmarking data for the EU and US are broken out by therapeutic area and by trial type. This excerpt discusses one of the greatest challenges — and areas for improvement — in Phase IV research: patient enrollment.  

Patient Enrollment

Companies of all sizes struggle with patient enrollment. Surveyed companies offered a variety of reasons why patient recruitment and enrollment occupies such a significant amount of time. Companies face two major problems in recruiting patients. First, they have difficulty finding patients on the whole, whether the companies are looking for general practice patients or treatment-naïve patients. Second, even if there is a potential patient population to recruit, the competition for available patients is fierce. Figures 2.31 to 2.38 show planned versus actual patient enrollment for IIS, comparative effectiveness, and registries and observational trials. IIS present the largest percentage deficit between planned and actual patient numbers, largely because IIS investigators tend not to have the experience and resources of the traditional investigators and centers used in Phase III trials. Registries and observational trials have the largest average difference between planned and actual patient enrollment numbers, but this is due to Company 20’s outlier data, in which it reported a 61% difference. Lack of Patients In Phase IV trials, many companies look outside the typical patient centers that would be used for Phase II and III studies. This is mainly to engage the more specialized patient populations sometimes required by post-marketing studies. And for companies that are accessing investigators and patients though the typical early-phase centers, many find it hard to locate qualified investigators and even harder to enroll the general practice patients in studies without enlisting the physician as an investigator. Companies will often provide additional training to investigators to encourage them to enroll, and in return, the company receives the benefit of enrolling that investigator’s patients in the post-marketing study.