Article | August 23, 2023

What To Include In (And What To Leave Out Of) Your Clinical Trial Protocol

Source: Clinical Leader

By Clinical Leader Editorial Staff

GettyImages-1274428125 data quality

Designing an effective clinical trial protocol boosts a sponsor’s likelihood of FDA approval and ensures that researchers are asking the appropriate questions to benefit the patient. In the early stages of your trial, it may be tempting to include extraneous details in the protocol, i.e., too many different objectives.1 However, this could potentially distract your FDA reviewer, lead to unclear results and an unrealistic amount of work for the study period, and, depending on their condition, increase discomfort for the patient. To create a protocol that adequately captures your data requirements in a simple and streamlined fashion, consult early and often with patients and regulators to leverage their perspective.

Prioritize The Patient Experience

As you consider what elements to include in your protocol, think critically about how each component will impact the patient’s experience. Some examples include:

  • Will the trial be conducted in a decentralized fashion with at-home components, or will it be fully conducted at the clinic?
  • If the patient is required to come into the clinic, what time of day will they arrive and how long will they need to stay?
  • Will they need to fast in preparation for the treatment and, if so, is their arrival time a feasible time of day to be fasting until?
  • Are they taking other medications that could impact a fasted state?

Thinking through a patient’s routine requires you to consider these details and more. Map out each step and create a corresponding schedule that accommodates patients as best as possible. Account for the specificities of a patient’s indication. For example, requiring a four- to six-hour clinic visit would be incredibly taxing for an Alzheimer’s patient; similarly, conducting an MRI for a Parkinson’s patient would be difficult. Each step in a protocol should be carefully considered in terms of whether it will produce clean data and how it will be executed efficiently with an emphasis on a positive patient experience.

Collaborate With Regulators Early And Often

Building a strong, collaborative relationship with regulators from the outset of your drug program will help significantly when it comes time to design a protocol. Ask FDA regulators to provide guidance and recommendations for what should be included in a protocol to help you gather the necessary data. Some sponsors opt to work with former FDA regulators who now offer independent consulting services. An experienced consultant will help you interrogate the advantages of additional endpoints and observational studies as well as the potential obstacles of including too much in the protocol. Gathering these expert perspectives will help your team determine whether assessments are warranted and if they have any accompanying consequences.

Achieve A Balance Between Complexity And Simplicity

Ultimately, your clinical trial will benefit from a protocol that is precise and written with patients in mind. It is vital to have well-outlined, clearly written procedures so that a protocol can be conducted at different sites and still produce consistent data sets. Sponsors in this phase will benefit from gathering insight and feedback from patients, regulators, and peers in the industry. Consider how other sponsors have written protocols to inform what works well and what might be unnecessary. Furthermore, ensure that your approach is truly patient-centric. This can be done in a number of ways, including conducting physiologically-based pharmacokinetic (PBPK) modeling to gain a better understanding of how the therapy will interact with the patient’s biological functions. Sponsors can also team up with patients and patient advocacy groups to determine what outcomes are most critical and impactful for their quality of life. By prioritizing clarity and succinctness in your protocol, you better serve the trial, FDA regulators, and, most importantly, the patients in need of your therapy.

For more information about designing clinical trial protocols, watch the recent Clinical Leader Live event Trial Protocol Design Do’s and Don’ts.

Reference

  1. Al-Jundi, A., & Sakka, S. (2016). Protocol Writing in Clinical Research. Journal of clinical and diagnostic research : JCDR, 10(11), ZE10–ZE13. https://doi.org/10.7860/JCDR/2016/21426.8865