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Investigator-Initiated Clinical Trials

July 27, 2023
 - Tim Hardman

Investigator-Initiated Clinical Trials (IITs) are clinical studies led by academic researchers or clinicians rather than pharmaceutical companies or sponsors. While IITs can offer numerous benefits, they also come with challenges. Regulatory authorities have levied substantial fines on pharmaceutical companies for irregularities around the conduct of IITs. Investigators face challenges around regulatory requirements and following Good Clinical Practice (GCP), often due to lack of experience or training. In our recent Insider's Insight we explore the pros and cons of IITs [1] which are summarised below:

PROS:

Scientific Independence

  • Freedom in Research: Investigators have greater autonomy in study design and execution, allowing them to explore innovative hypotheses that might not align with the commercial interests of pharmaceutical companies.
  • Novel Therapeutic Approaches: IITs often focus on unmet clinical needs or unique patient populations that may not be priorities for industry-led trials. This can lead to new therapeutic avenues and deeper understanding of diseases.

Filling Knowledge Gaps

  • Supplementing Existing Data: IITs can fill important gaps in medical knowledge, particularly for off-label uses of drugs, rare diseases, or understanding the long-term safety and effectiveness of therapies that have already been approved.
  • Focus on Real-World Evidence: Many IITs address practical, real-world clinical questions such as optimising treatment protocols, which may not be the primary focus of industry-sponsored trials.

Reduced Bias

  • Minimised Commercial Influence: Since the trials are led by academic investigators, there may be less bias towards producing commercially favourable results, leading to more objective and transparent findings.

Collaboration and Innovation

  • Fosters Academic Collaboration: IITs promote collaboration between clinicians, researchers, and academic institutions, leading to cross-disciplinary innovation and knowledge-sharing.

Patient-Centric Focus

•Improved Patient Care: IITs are often designed to improve specific aspects of patient care, such as exploring different dosing regimens, evaluating quality of life, or identifying biomarkers for better treatment response.

 

CONS:

Limited Funding

  • Restricted Resources: IITs often rely on limited funding, such as government grants or institutional support, which may not be sufficient to conduct large-scale, multi-centre trials. This can result in smaller studies with less robust data.
  • Challenges with Drug Supply: Without industry backing, investigators may struggle to secure drug supplies for the study, particularly for expensive or experimental therapies.

Regulatory and Administrative Burden

  • Complex Regulatory Requirements: Investigators leading IITs must navigate a complex regulatory landscape, often without the support structure provided by pharmaceutical companies. This includes preparing clinical study reports, obtaining ethical approvals, and complying with GCP (Good Clinical Practice) standards.
  • Time-Consuming Administration: IITs require significant administrative oversight, such as managing trial documentation, data collection, and reporting, which can divert focus from scientific research to bureaucratic tasks.

Limited Infrastructure

  • Lack of Operational Support: Unlike industry-sponsored trials, which have established infrastructures for monitoring, auditing, and managing trials, investigators may lack the necessary infrastructure for large-scale studies, potentially affecting trial quality and consistency.
  • Data Management Challenges: Efficient data management and analysis can be difficult without the advanced tools and support systems typically provided by pharmaceutical companies.

Challenges in Trial Design and Execution

  • Design Limitations: IITs may face limitations in trial design, including smaller sample sizes, single-centre studies, or insufficient statistical power to draw definitive conclusions.
  • Less Rigour in Reporting: Due to resource constraints, IITs might sometimes lack the same rigour in terms of reporting adverse events, monitoring, and documentation compared to industry-sponsored trials.

Limited Impact and Generalisation

  • Less Generalisable Results: IITs tend to have smaller patient populations or focus on specific centres, which can limit the generalisability of the findings to larger, more diverse populations.
  • Publication Challenges: It can be harder to publish results from IITs, especially if the study lacks sufficient statistical power, or if findings contradict industry-sponsored research.

Investigator-Initiated Clinical Trials offer significant advantages in terms of scientific freedom, innovation, and filling unmet medical needs. However, they are often limited by funding, infrastructure, and administrative challenges, which can impact the scale and generalizability of the research. Successful IITs require careful planning, access to resources, and collaboration with multiple stakeholders, including regulatory bodies and industry, to overcome these hurdles and ensure meaningful impact on clinical practice. There is no doubting the potential benefits that can be delivered by IITs when they are conducted correctly. However, despite the pressure on clinicians to get involved in research, teaching of the skills and understanding to conduct clinical trials is still largely neglected. Inexperienced physicians and teams are often left struggling to build understanding from the relatively poor materials available in the scientific literature and more commercially orientated publications [2]. Many published reports and books that claim to instruct readers about IIT conduct are expensive and/or locked behind paywalls [3]. Equally, valued content about IITs is often padded out with the more general requirements for any normal clinical trial.

Here we have focused on the challenges at hand, providing a summary from our own experience of the key considerations for those thinking of conducting an IIT and a ready source of further reading. Don’t just take our word for it – check out some of the successful projects we have completed, such as the MRC funded RASP-UK industry partnership (www.RASP-UK.org) and the Horizon 2020 MID-Frail initiative (www.midfrail-study.org). Please contact us if you would like further support bringing your planned project to fruition.

References

  1. The Insider’s Insights on Investigator Initiated Trials. Niche Science & Technology Ltd., 2023
  2. Nebie EI, et al. Operational Differences between Product Development Partnership, Pharmaceutical Industry, and Investigator Initiated Clinical Trials. Trop. Med. Infect. Dis. 2024, 9, 56. https://doi.org/10.3390/tropicalmed9030056
  3. Review of “Guide for Investigator Initiated Trial Management.” J Clin Res Best Prac 2012; 8: 1-4

 

About the author

Tim Hardman
Managing Director
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Dr Tim Hardman is Managing Director of Niche Science & Technology Ltd., a bespoke services CRO based in the UK. He is also Chairman of the Association of Human Pharmacology in the Pharmaceutical Industry, the representative industry body for early for early phase clinical studies in the UK, and President of the sister organisation the European Federation for Exploratory Medicines Development. Dr Hardman is a keen scientist and an occasional commentator on all aspects of medicine, business and the process of drug development.

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