Investigator-Initiated Trial Management (PH212)

Expedite Approval Timelines and Establish Compliant Funding Practices
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Published 2015
253 Pages
500+ Metrics
213 Charts and Diagrams

Evaluate and Approve IIT Proposals Faster and Better While Staying Compliant

As the number of investigator-initiated trial (IIT) submissions increase, existing IIT management challenges magnify. An IIT’s final outcome is out of the drug manufacturer’s hands, so it is essential for teams to properly vet each proposal. Otherwise, companies risk putting their products — and the resulting data — in poorly designed studies with inexperienced or unqualified investigators. Successful teams will efficiently approve IIT proposals that demonstrate scientific merit and align with shifting corporate objectives. Finally, IITs promote KOL engagement, but proper documentation and FMV payments are a primary concern for teams navigating current compliance codes.

This report serves as a solution for IIT teams that struggle with these challenges. It is a detailed roadmap for improving IIT management at key steps, from proposal submission to follow-up with clinical investigators. Detailed benchmarks, diagrams and profiles show IIT management team structure, both stage-by-stage and cumulative timelines, as well as performance metrics for IIT teams across company sizes and regions. Top executives’ insights go beyond identifying problem areas to spotlight efficient processes and best practices for rigorous IIT evaluation.

As companies strive to accelerate IIT management processes, transparency must also be top of mind. A surefire way to boost efficiency while decreasing many compliance issues is to implement technology that allows investigators to submit proposals online. Internet portals have built-in systems that document submissions and approvals and act as a gateway for investigator communication and updates. IIT teams can also turn to third-party vendors for fair market value (FMV) assessments to safeguard against overpaying investigators.

Compliance is critical for IIT funding. This report contains extensive benchmarks and case studies to help build budgets that will adequately support investigator-initiated research. But it also provides strategies for how teams negotiate contracts and ensure FMV-compliant itemized trial budgets.

Industry executives working in investigator-initiated trials, investigator-sponsored trials, investigator-initiated research, and medical affairs will get the following benefits from this report:

  • Timelines for approving and beginning an IIT across 10 individual companies of various sizes and IIT teams with US, ex-US and global responsibility.
  • IIT volume benchmarks, including the number of proposals received, evaluated and approved and which channels are most prevalent for submissions.
  • Ratings and insights detailing 6 key criteria for assessing IIT outcomes — and red flags to watch for.
  • Infographics and data detailing IIT management structures across company size and region.
  • Metrics showing communication follow-up with investigators, including frequency of updates and medical science liaison (MSL) support.
  • Actual budget line items for IIT pass-through costs, direct costs and indirect costs.
  • Full budget case studies for 12 real-world IITs; each profile includes data on total company funding, available budget line items, therapeutic area, patient enrollment and trial duration.

 

Key Questions That This Study Answers

  1. Which performance metrics should companies measure to track IIT efficiency and program value?
  2. What are some best practices for companies to facilitate clinical trial agreement (CTA) negotiations?
  3. What IIT management structures and protocols can teams implement to accelerate the submission, evaluation and approval stages?
  4. What key criteria should teams consider when evaluating IITs? What red flags should they watch for?
  5. Once the IIT is approved, what are the best strategies for following up with investigators and ensuring that trials are progressing on schedule?
  6. What practices should teams employ to make sure that investigators’ itemized budgets are FMV-compliant? How do companies skillfully manage budget revisions?
  7. Which metrics are useful for setting annual IIT budgets?

 

Top Reasons to Buy This Report

Evaluate IIT submissions and negotiate contracts more efficiently: Delays reduce — or can even eliminate — IIT value, postpone or negate company research goals, expend resources and frustrate investigators. Learn proven strategies, such as pre-screening proposals, for expediting IIT evaluation, and consult six key criteria for selecting the right IITs. Performance metrics, including the number of proposals received, evaluated and approved, are also vital in accurately assessing IIT management efficiency. Examine timelines showing the number of days spent on nine IIT stages. Teams must track the time they are spending to know which stages can be shortened. These data will allow for more detailed planning — saving time and money down the road. Teams surveyed for this report also identify negotiating contracts as a top challenge. Benchmarking data indicate that establishing clear and consistent responsibilities, such as using legal teams to help with contracts, can significantly accelerate the process.

Establish IIT management structures that facilitate fast approval and submission tracking: Structure plays a key role in ensuring efficient IIT management. Involving local and global-level personnel during IIT approval processes, for example, helps to avoid duplication of efforts. Examine case study examples comparing different approaches to IIT team structures, as well as the IIT management support coming from other internal groups. As teams strive to accelerate IIT decision making, having efficient IIT proposal reviews is crucial. Creating standard operating procedures (SOPs) is one key step. Another important aspect involves IIT review committees. Data in the report explore IIT committee structures and which functions retain voting privileges during proposal evaluation.

Benchmark IIT spending and internal management costs to build the perfect budget: Examine benchmarks, best practices and case studies to build a solid IIT budget. Key metrics include the percentage of IIT budget dedicated at the beginning of each year, sources of IIT funding, percentage of budget spent on external spending and IIT internal management, average funding per IIT and IIT funding compared to the number of approvals. Along with providing overall team budgets, surveyed IIT managers were asked to elaborate on one recently completed study. Because life science companies can offer support for IITs in a variety of ways, this report examines which line items are company-funded and which items are left to the investigators. Benchmarks include budget line items for specific pass-through costs, direct costs and indirect costs. The report includes 12 full-budget profiles of recently completed trials.

Investigator-Initiated Trials Metrics

Chapter 1: Strengthen IIT Processes by Implementing Structure and Staffing Hierarchies

Major Takeaways

  • Develop standard operating procedures (SOPs) to facilitate consistent and transparent IIT program management.
  • Involve local and global-level personnel during IIT approval processes to prevent duplication of efforts.
  • Grant voting privileges to non-sales/non-marketing IIT committee members only to maintain separation between medical and commercial interests.
  • Incorporate milestones into existing payment structures to avoid challenging reconciliation procedures and to reinforce trial goals.
  • Implement protocols, milestone payments and timelines that ensure publications that increase IIT value.

Chapter Data

47 charts explore facets of IIT management: team structure; internal groups’ involvement at different IIT evaluation stages, including committee participation and voting privileges; and IIT oversight. This chapter also examines companies’ communications with investigators, including IIT teams’ preferences for updates and the role MSLs play in supporting IIT management.

  • Infographic showing the general process for managing IITs
  • Ways companies choose to structure IIT management (e.g., single dedicated group, multiple groups serving individual regions)
  • Percentage of companies with dedicated IIT departments in recent years (2006, 2008, 2011, and 2014)
  • Number of IIT groups, by company
  • Specific functions’ involvement in steps of the IIT process:
    • Proposal collection and initial screening
    • Proposal evaluation and approval
    • IIT oversight
    • IIT conclusion and use of data findings
  • Percentage of US and global teams with written SOPs for IIT processes
  • Ratings of challenges associated with IIT structure and management
  • Infographic showing a real-company approach to increasing efficiency in IIT reviews
  • IIT committee structure (charts show options for voting privileges and final decision-making power)
    • Total number of IIT committee members
    • Number of IIT committee voting members
    • Functions with voting privileges on committees that segment voting privileges
  • Milestone payments used for IITs
  • Teams’ preferences for receiving IIT updates
  • Frequency of receiving IIT updates from investigators
  • MSLs’ role in IIT management
  • Tactics to ensure publication strategies are met
  • Percentage of IITs published, by region

 

Chapter 2: Crafting IIT Approval Strategy and Performance Metrics

Major Takeaway

  • Pre-screen proposals so that the IIT committee only evaluates the highest-quality submissions.
  • Track time spent in each IIT approval stage to identify areas that can be expedited.
  • Entrust clinical trial agreement negotiations to legal personnel to save significant time and ensure trial progress.
  • Establish clear criteria and identify red flags when evaluating submissions.

Chapter Data

94 charts showing recommendations, timelines and benchmarks for improving IIT submission, valuation and approval processes. Data include the number of proposals received, evaluated and approved, as well as the number of days spent on various IIT stages to enable more accurate planning.

  • Infographic outlining three key steps for increasing IIT efficiency
  • Ratings of challenges associated with IIT approvals
  • Criteria ranked most important when evaluating an IIT proposal
  • Reasons for automatically rejecting an IIT proposal
  • Average number of proposals received, evaluated and approved in 2014, by region
  • Average percentage of:
    • Submitted proposals that were evaluated in 2014, by region
    • Evaluated proposals that were approved in 2014, by region
    • Number of proposals submitted, evaluated and approved in 2013, 2014 and 2015
  • Percentage of proposals received, evaluated and approved, by year (2013–15)
  • Percentage of IIT proposals received, by channel (e.g., Internet, MSLs, meetings or other)
  • Percentage of IIT submissions first received as concept and protocol proposals
  • Frequency of IIT proposal evaluation meetings, by region
  • Frequency of meetings compared to number of evaluated IIT proposals
  • Number of days spent on the following nine (9) stages:
    • IIT concept submission to concept review
    • Concept review to concept decision
    • Concept decision to investigator notification
    • Investigator notification to protocol submission
    • Protocol submission to protocol review
    • Protocol review to protocol decision
    • Protocol decision to contract negotiation start
    • Contract negotiation start to contract execution
    • Contract execution to first patient in study
  • Ten (10) individual company timelines — across company sizes and team regions — showing number of days spent for each of these IIT stages.
  • Average percentage of IITs, by target patient enrollment ranges
  • Number of approved IITs in 2014 by target patient enrollment
  • Average percentage of IITs with different number of sites, by region
  • Average percentage of approved IITs that:
    • Are retrospective studies
    • Are interventional studies
    • Gather data about a secondary indication
    • Gather data about a new patient population/subgroup
    • Gather patient-reported outcomes (PROs)
    • Gather data about new dosing

 

Chapter 3: Build the Perfect IIT Budget

Major Takeaways

  • Calculate average funding per IIT to build annual IIT budgets.
  • Evaluate proposed IIT itemized budgets for FMV — and only approve those that are FMV-compliant.
  • Be wary of overhead staffing costs that exceed 25% of the total direct and pass-through costs.

Chapter Data

55 charts showing IIT budgets, costs and spending. This chapter also includes case studies highlighting budgets for 12 IITs, which will help teams set accurate annual IIT budgets.

  • Ratings of challenges associated with IIT budgets
  • Percentage of IIT budget dedicated at beginning of year
  • Average percentage of IIT spending from medical affairs, clinical/R&D, and marketing
  • Percentage of budget spent on IIT funding and internal management
  • Internal IIT management costs, by year (2013–15)
    • IIT funding compared to the number of approvals for 2014
    • IIT budget changes from 2015 to 2016 (increase, decrease, or stay the same)
    • IIT funding for investigators, by year (2013–15)
    • IIT teams’ responses to unacceptable proposed budgets (Chart shows different options for coordinating how budget revisions are handled.)
    • Number of allowed IIT budget revisions
    • Total company funding for an IIT with more/fewer than 100 patients enrolled
    • Total company funding for an IIT: oncology trials
  • Budget line items for specific pass-through costs:
    • IRB (initial review, continued review, and amendments)
    • IND and other regulatory requirements
    • Publication
  • Budget line items for direct costs:
    • Study and laboratory supplies
    • Diagnostic fees and services
    • Pharmacy start-up fees
    • Data management
    • Document storage fees
    • Patient recruitment
  • Budget line items for indirect costs:
    • Staffing overhead
    • Initiation fees
    • Cost of office supplies

In addition to analyzing each budget line item individually, this chapter contains case studies for 12 recently completed IITs. Each trial case study contains the following information:

  • Total company funding
  • Available budget line items
  • Therapeutic area
  • Patient enrollment
  • Number of sites
  • Trial staff per site
  • Trial duration

 

Chapter 4: IIT Team Profiles

Major Takeaways

  • Quickly compare your IIT program against others with similar characteristics and resources.
  • Use these benchmarks to improve your IIT team and as evidence for increasing resources or adjusting processes.

Chapter Data

16 charts showing profiles of eight (8) IIT teams. These profiles include global, US and ex-US teams of various sizes. They present a range of IIT team structures, activities and budgets against which companies can benchmark their IIT programs. Data are conceptualized in infographics and charts to facilitate comparison across profiles.

Each team profile has the following three sections:

  • Structure
    • Team region and company size
    • IIT program structure for the entire company
    • Group the surveyed IIT team represents
    • IIT committee structure — by function and voting privileges
    • Person or team that makes the final decision about IIT proposals
  • Budgets and Spending
    • Internal management costs for 2013, 2014 and 2015
    • IIT funding for 2013, 2014 and 2015
    • Percentage of budget dedicated at beginning of the year
    • Percentage of IIT budget from medical affairs, clinical/R&D and marketing departments
  • Number of IIT Proposals: the number of IIT proposals the team received, evaluated and approved in 2013, 2014 and 2015

Sample Content from Investigator-Initiated Trial Management

The following excerpt is from Chapter 1, “Strengthen IIT Processes by Implementing Structure and Staffing Hierarchies.”

A Tale of Two Top 25 Companies: Comparing IIT Management Techniques Across Large Pharma Organizations

In general, companies may opt to align their IIT structures based on whether supporting functions fulfill IIT approval or trial execution responsibilities. For many teams, clinical trial managers take on the bulk of IIT management responsibility, both in terms of trial approval and contract execution. When approving IITs, medical staff may help companies determine whether or not a proposed IIT is a good fit for their existing pipeline. On the IIT execution side, global and local-level teams may incorporate the expertise of legal teams to help them develop and negotiate clinical trial contracts. However, a look at two teams from Top 25 pharmaceutical organizations — one group based in the US and another based abroad — further emphasizes that companies’ IIT structures are not necessarily uniform.

A single structure — which houses both the IIT review committees and the FTEs supporting the operational components of approved trials — supports IIT management at Company B. On the approval side, clinical trial managers at Company B prepare committee agendas, track meeting minutes and record committee votes into a database. On the trial execution side, these staff members conduct financial forecasting, assist with managing trial budgets, and track clinical trial payments.

Like Company B, clinical trial managers at US Company C handle most IIT activities. However, Company C prefers to divide these responsibilities into two separate structures.

The company’s 15-person scientific coordination group handles the IIT approval process. This group—comprised of full- and part-time staff, each with medical backgrounds, including registered nurses — makes up Company C’s IIT committee.

The organization’s 10-person operations group is more focused on the logistics behind companies’ clinical trials. Business-oriented individuals in this group handle trial payments and financial forecasting responsibilities.

Recently, Company C reduced the size of its IIT staff. The company’s operations group lost five personnel and its scientific group is now an outsourced operation. Yet despite its structural adjustments, Company C maintains its use of these two, distinct groups.

Investigator Initiated Trials Report Sample

 

The following excerpt is taken from Chapter 2, “IIT Submission Review and Systems.” The full report contains an in-depth discussion on the benefits and drawbacks of adopting digital systems to expedite the IIT proposal submissions process.

Overcoming Digital Submission Hurdles to Reap Benefits

Figure 2.1 [data shown in full report] breaks down how each company receives its IIT submissions, showing the percentage of submissions obtained via the Internet and digital submission technologies, MSLs, sales, meetings and congresses and other methods of delivery.

Of the six companies offering the option of a fully digital, online submission portal, two are Top 25 global pharmaceutical companies that receive hundreds of annual IIT submissions per business unit. The sheer volume of proposals makes digital submissions attractive to large companies. The efficiency gained by automating the submission system allows IIT teams to look at uniform, complete proposals; in other words, those teams can be sure that the proposals they review meet the company’s standards for IIT submissions. At other companies, those submissions can be incomplete or noncompliant, thereby wasting precious review time.

Figure 2.2 [data shown in full report] shows a breakdown by company size of the percentage of IIT submissions received online. As the graphic indicates, large companies receive the largest portion of IIT submissions online at 75%. Early adoption by large companies of online IIT submission is likely due to two primary factors: the financial burden of such programs and the regulatory compliance pressure to do so. Large companies are more apt to invest in the technical infrastructure and IT support necessary to implement digital submission capabilities into their existing online presences. Moreover, those same companies are at the forefront of regulatory scrutiny into FMV practices and therefore are motivated to establish data tracking systems that help them remain compliant and provide evidence of compensation when necessary.

Company 12, also a Top 25 pharma company, does not permit investigators to complete submissions online. As a result, this company is over-reliant on its sales and MSL teams to solicit and shepherd IIT proposals from investigators in the field. This inefficient strategy puts more pressure on individuals working in the field and detracts from their ability to address other needs and job duties. Driving IIT submissions does not contribute to a sales rep’s prescription rates, her commission or her company’s bottom line. The same is true for MSLs, although to a lesser degree. They are tasked with fielding physicians’ clinical questions and can thereby help physicians identify areas of potential exploratory research. But if they were less responsible for supplying their company’s IIT strategy with proposals, they could spend more time developing physician relationships and brand loyalty, which contribute to the commercial team’s success.

 

The following is excerpted from Chapter 3, “IIT Spending and Budgets.” The full chapter analyzes budget and spending data from companies surveyed and breaks down sources of IIT funding and the way that IIT teams structure budget requests.

Budget Structures: Ongoing Nature of IITs Leads to Ad Hoc Preference

Most surveyed companies approve IIT budgets throughout the year, as opposed to one lump sum at the beginning of the year (Figure 3.1, data shown in full report).

Just over a third of surveyed companies receive IIT funding as part of a dedicated budget line item, and 64% do not have a dedicated budget for IITs. A dedicated budget does not necessarily mean a company’s entire IIT expenditure is accounted for at the beginning of the year. Of the five companies with dedicated budgets, two report that while they receive a dedicated budget, it amounts to 60% of the companies’ total IIT expenditure (Figure 3.2). The remaining money is granted for projects and studies on an as-needed basis, requiring the IIT department head to send requests for additional funds up the command chain, typically to the head of the medical affairs department. Furthermore, of the five surveyed companies that receive an IIT budget allocation at the beginning of the fiscal year, four have decentralized structures. This finding would seem to indicate that for those companies yet to consolidate IIT operations into a centralized structure, the solution to funding is to allocate money at the beginning of the year and when the funding runs out, so do IIT approvals. At companies with centralized structures, IIT teams are more developed and more readily able to source funding on an ongoing, ad hoc basis. Hierarchical proximity and integration within medical affairs departments offer centralized IIT teams streamlined access to the funding process.

The ad hoc nature of budgetary allocation for IITs presents challenges to teams. The head of an IIT team has to look at several factors when planning the year’s spending. The first, decided at the beginning of the year, is how much — and whether — money will be allocated to IIT spending. The next consideration is the number and cost of ongoing IITs; most IITs will draw from several years’ worth of budget, and the individual in charge of a company’s IITs must be aware of how much money is already committed to ongoing IITs before determining how many new studies can be undertaken. An executive at Company C described the process of determining future budget this way:

The way we determine our budget is we look at the studies we have funded — the way we do our bookkeeping, we accrue them, so if a study is going to take 12 months and it’s going to cost $120,000, we put $10,000 into it every month over the life of it, and that’s how we do our accounting. So if we have a study that bleeds into next year, we know how much money to have available. It’s not balancing our checkbook; we have to project what we will need in years to come for studies. We look at historical data, what I call new spend, which are new projects, and how much they end up accruing for a given year. We come up with our budget based on what our fixed costs are and what we anticipate we need to spend for new studies, and that’s what we ask for.

An executive from Company B described a process in which IITs sit within a broader, Phase 4 group, leading to decisions on how to divide the Phase 4 funding between in-house studies and IITs. “The Phase 4 group that sits within medical affairs is given their dollars like anyone else,” he said. “So they have to put up a plan at the beginning of the year and look at doing [IITs] based on gaps that we see in the literature — to answer those questions. Part of that money needs to go to company-initiated studies and part of that money needs to go to external investigators submitting their ideas.”