Phase I Clinical Trials (PH173)

Optimizing Cost Drivers for Clinical Pharmacology Programs
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  • Manage Phase I Clinical Trial Cost Drivers to Move Compounds through Early-Stage Development

    Phase I is a pivotal step in a compound’s journey toward regulatory approval, as clinical teams shepherd each new wave of investigational medicines toward large-scale trials.

    The challenges are increasing. Cost pressures stretch limited budgets along with issues involving timelines, recruitment, sites and more — which rise and fall according to shifting sets of variables. Phase I protocol, once predictable, now must account for rapid expansion into more complicated areas, such as oncology and orphan diseases.

    Use this report to address the major factors behind uncertainty in Phase I trial management. Its benchmarks and best practices explore Phase I clinical team resources, trial timelines, challenges and vendor management: 

     

    Control critical cost drivers

    Total trial costs increase with protocol complexity. Manage key cost-drivers, including number of patients, patient visits, trial duration and specialty trial requirements. Examine benchmarks detailing total trial costs, per-patient costs and per-patient visit costs across 10 key therapeutic areas, including oncology and central nervous system.

     

    Boost Phase I planning to prevent delays — and propel teams toward trial completion

    Set defined milestones and explore trial duration data for each stage of the process, and draw on executives’ strategies for keeping ahead of schedule.

     

    Right-size your clinical team and improve vendor management

    Balance in-house and vendor capabilities to increase patient recruitment, and analyze staffing metrics, including sponsor headcounts, for 25 Phase I trials across therapeutic areas. Lastly, follow strategic recommendations for vendor selection and management.

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  • Clinical Pharmacology and Phase I Metrics

     

    Chapter 1: Early-Stage Clinical Structure and Trial Staffing

    Chapter Benefits

    • Determine Phase I management group structures — stand-alone or combined with later-stage trial management — based on company preferences and resource needs.
    • Staff Phase I teams to account for the complexity of trial protocol and vendor usage.
    • Leverage internal boards of experts to determine if compounds are able to advance into and out of Phase I clinical trials.
    • Save time and money by implementing a gating system to ensure that a compound meet set criteria before advancing into Phase I.
    • Leverage early marketing groups to analyze compounds’ commercial endpoints and cost-effectiveness during early-stage clinical trials.

     

    Chapter Data

    17 charts focused on Phase I team structure, staffing and processes:

    • See Phase I clinical development team structure for four unique companies.
    • Study two examples of processes and pathways for advancing products into Phase I clinical testing.

     

    Average sponsor headcount for all Phase I trials and broken out by therapeutic area:

    • Oncology
    • Cardiovascular
    • Central Nervous System/Neurology
    • Women’s Health
    • Gastroenterology
    • Diabetes (Type 2)
    • Endocrinology
    • Infectious Disease
    • Respiratory
    • Rheumatic Diseases

     

    Chapter 2:  Phase I Trial Budgets and Cost Drivers

    Chapter Benefits

    • See how executives at top-performing companies begin Phase I trial planning and budget creation.
    • Control critical Phase I cost-drivers, including patient enrollment, patient visits and trial duration.
    • Consider the increasing costs of first-in-patient testing versus first- in-healthy volunteer trials.
    • Project budgets according to per-patient costs and therapeutic area needs.
    • Examine CNS trials’ high total costs and per-patient costs.
    • Evaluate oncology trial costs and cost-drivers for 12 trials.
    • Explore the advantages and disadvantages of clinical trial simulation to help fine-tune Phase I trial protocol design.

     

    Chapter Data

    29 charts focused on Phase I trial costs, including total costs and per-patient costs, and patient enrollment and completion.

    Total costs for Phase I clinical trials broken out by therapeutic area:

    • Oncology
    • Cardiology
    • Central nervous system
    • Gastroenterology
    • Women’s health

     

    Data broken out by therapeutic area (typically oncology, cardiology, central nervous system, gastroenterology):

    • Average trial costs for all Phase I clinical trials by therapeutic area
    • Therapeutic areas with only one sample (Diabetes Type 2, Endocrinology, Infectious Diseases, Respiratory, Rheumatology)
    • Average patient enrollment in Phase I clinical trials (by therapeutic area)
    • Average patients completing Phase I clinical trials (by therapeutic area)
    • Average patient dropout rate in Phase I clinical trials (by therapeutic area)
    • Average per patient costs in Phase I clinical trials (by therapeutic area)
    • Per patient costs in Phase I clinical trials (by therapeutic area)
    • Average number of patient visits in Phase I protocol (by therapeutic area)
    • Average cost per patient in Phase I clinical trials by number of patient visits in protocol
    • Average cost per patient per visit in Phase I clinical trials (by therapeutic area)
    • Cost per patient per visit in Phase I clinical trials (by therapeutic area)
    • Average Phase I cost per patient (by trial duration)
    • Average cost per patient per month in Phase I clinical trials (by therapeutic area)
    • Cost per patient per month in Phase I clinical trials (by therapeutic area)

     

    Chapter 3:  Phase I Trial Duration

    Chapter Benefits

    • Compare planned Phase I trial durations and actual trial durations across 5 key therapeutic areas.
    • Examine the average duration of each trial phase (Final protocol to first-patient-in, FPI to 50% enrollment, 50% to last-patient-in, LPI to last-patient-out, LPO to data lock) for 5 key therapeutic areas.
    • Engage in pre-planning to save time and prevent unnecessary delays.
    • Leverage existing CRO relationships and set tight, realistic deadlines.
    • Combine multiple Phase I studies to limit the number of IRB and regulatory submissions and lower trial costs.
    • Overcome patient enrollment delays by working with vendors and research sites to understand potential patient populations.
    • Plan extra time in trials for therapeutic areas with specialty patient populations such as oncology or pediatrics.

     

    Chapter Data

    21 charts focused on the planned and actual duration of Phase I clinical trials. Data are broken out by therapeutic area (cardiology, central nervous system, gastroenterology, women’s health, oncology):

    • Planned versus actual Phase I trial milestones for all clinical trials
    • Planned Phase I clinical trial duration (by therapeutic area)
    • Actual Phase I clinical trial duration (by therapeutic area)
    • Average time dedicated to specific activities in Phase I clinical trials (by therapeutic area)
    • Average time spent in each clinical operations stage for all Phase I trials (by therapeutic area)

     

    Chapter 4: Outsourcing and Vendor Management

    Chapter Benefit

    • Explore vendor management best practices from top executives.
    • Leverage CROs and other vendors to save time and money — but be mindful of potential downfalls.
    • Develop criteria for site and vendor selection when setting outsourcing strategy:
    • Preferred provider versus bidding process
    • Number of trial sites
    • Effect of number of sites on length of time before all sites are active
    • Scale outsourcing strategies to in-house capabilities.
    • For biotechnology companies, find the most capable vendor within the trial’s budget.
    • Match vendor size to company size, but be wary of being passed over in favor of larger company contracts.
    • Increase patient recruitment and CRO motivation by balancing capabilities of the lab and sponsor.
    • Consider the advantages and disadvantages of preferred vendors.
    • Employ detailed, consistent interaction with vendors to increase patient recruitment and CRO motivation.
    • Define CRO responsibilities and establish clear processes for managing vendor oversight.

     

    Chapter Data

    7 charts focused on Phase I trial outsourcing and trial site management:

    • Average percentage of Phase I trial costs outsourced for all trials and by therapeutic area
    • Percentage of surveyed trials using number of sites (1 trial site, 2–5 trial sites, 6+ trial sites)
    • Average number of sites per trial (by therapeutic area)
    • Percentage of sites unable to recruit patients for trials with multiple sites
    • Average lengths of time (in months) until all sites are active by number of sites
    • Average lengths of time (in months) until all sites are active by therapeutic area

     

     

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  • Phase I Clinical Trials Excerpt

     

    The following excerpt is a key finding from the full report’s Executive Summary:

    Incorporate Defined Milestones and Diverse Expertise into the Decision to Move a Compound to Phase I

     At several companies surveyed, the decision to advance a compound from pre-clinical testing to a Phase I trial is made by a collaboration of experts from across pre-clinical development. An interviewed executive from Company B describes how toxicologists and physicians contribute to the approval board’s decision to move it into Phase I. “All of the specialties come together to figure out if it’s safe to move into human testing,” he explains. At Company C, experts from many stages of Discovery together make the final decision to move the drug to Phase I. At Company F, even more collaboration makes up the process —experts from both pre-clinical and Phase I clinical groups work together to examine the testing data and move the compound forward.

    In addition to this multidepartment collaboration, companies find it useful to establish a set list of criteria a drug must meet before being considered for Phase I trials. Whether established by the pre-clinical group, clinical group, or partner company, these milestones ensure that a company does not waste resources on a compound that is unsafe or financially impractical. As shown in Figure E.3 [figure appears in full report], a project review committee at Company A will ensure that criteria are met at five gates. Only after these gates have been passed will the company invest in large-scale toxicology studies and manufacturing campaigns. Approval of at this final milestone will allow the compound to move into Phase I.

    Blending expertise and list of criteria in the pre-clinical phase allows companies to best judge whether a drug will be safe in humans and valuable to the company. Establishing a set of milestones within this early phase simplifies the final approval process because the compound has already fulfilled important benchmarks. Rather than a pre-clinical executive simply pushing the compound to the next step, a collaboration of experts from the present and future stages of the process can all assess the compound’s safety as well as other possible indications and the drug’s overall financial viability.

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