Four Common Oversights of Investigator Initiated Trials

Within the past two years, investigator initiated trials (IITs) have seen a remarkable renewed interest. While research offices are doing their best to support these endeavors, the additional administrative responsibilities involved are often unfamiliar to research offices. Consequently, mistakes made during the initial development and start-up stages can cause compliance and financial ramifications. Here are four common mistakes to look out for when conducting an IIT study:

1.       Protocol Objective Development

Issues with IIT studies can arise as early as the protocol development stage. Determining whether the intent of a trial is interventional or observational and ensuring the protocol’s objective statement accurately portrays the PI’s intent is one of the most critical preliminary steps. This decision establishes a basis for billing strategy and leads to a more appropriate financial analysis. In essence, if the objective language implies that the study is interventional, and intends to assign patients to treatment groups, then claims should be submitted following the clinical trial billing policy. On the other hand, if the objective language implies that the trial will be conducted by simply collecting data from procedures and services performed as part of routine care, then the study can be deemed as observational and nothing should be billed under the clinical trial policy. These billing decisions should then influence how the institution files compliant insurance claims for enrollees.

2.       Achieving Medicare Qualifying Status

Achieving Medicare “qualifying status” allows institutions to bill for trial procedures under Medicare’s clinical trial policy, NCD 310.1, and potentially receive more comprehensive reimbursement. There are a few ways an IIT can achieve deemed qualifying status, one of which requires financial support by an approved governmental agency such as the NIH or the CDC. Alternatively, a study can be approved for an IND number or IND exemption. Often investigators are not aware that they need to apply for an IND number or an exemption and are unpleasantly surprised when insurance declines to pay for certain items or when their institution is reprimanded for failure to comply with the clinical trial billing policy. Educating PI’s often and early about IND submission procedures is a good way to avoid this error.

3.       Financial Impacts

Once preliminary status determinations have been made, it’s time to assess the financial impact of the study. Many institutions are unaware that large pharmaceutical companies like Merck, Sanofi, and Pfizer offer programs that allow them to partner with researchers on IIT studies. These Companies agree to offer financial support and/or drug provision in exchange for outside influence and perspective as they aim to supplement their own initiatives.

If an institution is unable to secure outside funding, the research team should anticipate and designate a payor for each study item. This can both minimize the potential for double billing and eliminate forgotten expenses. To better understand the finances of the trial, institutions can conduct a two-step analysis. First, an explanatory coverage analysis should be built to both predict the research fund expenses and provide a thorough explanation to the PI and staff as to why certain services can’t be billed. Step two of this financial analysis involves totaling these research expenses through an internal budget, which can then be used to determine the entire cost of the study and whether it is financially feasible.

4.       NCT Identifiers

The final oversight that often occurs in IIT studies arises in the final steps of study start-up, as institutions are responsible for registering the study with the federal government to obtain an NCT identifier number. Institutions may only become aware of their requirement to register once they receive claims denials or are confronted by CMS for failure to comply with the clinical trial billing policy. Skipping this step is a frequent oversight, as it is typically handled by the sponsor or an outside governmental agency. In order to bill trial services and procedures appropriately to Medicare, this identifier should be included on all patient claims. Consequently, failing to secure an identifier can cause trouble for the billing team as the trial proceeds. Once more, educating PI’s often and early about these procedures is the best strategy.


Emphasizing the common pitfalls of IIT studies is not meant to deter participation. In fact, there are numerous benefits to conducting IITs. All things considered, reward will only come about if there is a solid understanding of the administrative processes that are required to take on these studies.