Needs Assessment for the Creation of a Platform Trial Network in Metabolic-Dysfunction Associated Steatohepatitis

Endocrinology Hepatobiliary System Medicine
Metabolic Dysfunction Steatohepatitis Clinical Research Network Platform Trial Drug Development

Needs Assessment for Establishing a Platform Trial Network: A Case Study on Metabolic Dysfunction-Associated Steatohepatitis (MASH)

Academic Background

The public health issue posed by Metabolic Dysfunction-Associated Steatohepatitis (MASH) is becoming increasingly severe. MASH is a condition caused by fat accumulation, inflammation, and cell damage in the liver, often associated with obesity and diabetes. Globally, about 25-30% of the population is affected by Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD), with 5-8% suffering from MASH. This condition can lead to serious complications such as liver cirrhosis and liver cancer. However, there are currently no medications approved by regulatory authorities specifically for MASH, which significantly limits treatment options.

Research Origin

This study was co-authored by Elena Sena, Frank Tacke, Quentin M. Anstee, Nicholas Di Prospero, Mette Skalshøi-Kjær, Sergio Muñoz-Martínez, Jesús Rivera-Esteban, Alba Jiménez-Masip, Jesús M. Bañales, María Martínez-Gómez, Franz Koenig, Joan Genescà, Vlad Ratziu, and Juan M. Pericàs. The authors hail from multiple research institutions, including Vall d’Hebron University Hospital, Charité - Universitätsmedizin Berlin, Newcastle University, among others. The paper was published in the journal Communications Medicine.

Research Process

The study primarily employed a survey to understand the interest and capacity of European clinical research centers in joining a platform clinical trial (Platform Trial, PT) network for MASH. The study invited 141 European clinical research centers to participate, with a final response rate of 40%.

a) Research Process

  1. Preliminary Assessment and Survey Design:

    • Identify potential European clinical research centers and collect information on their interest and capacity for platform trials.
    • Design and distribute a questionnaire to assess these centers’ willingness to participate, existing capabilities, and understanding of platform trials (PT).

  2. Data Collection:

    • Data were collected and managed using the REDCap electronic data acquisition tool to ensure data integrity and accuracy.

  3. Analysis and Interpretation of Results:

    • Statistical analysis was conducted on the survey results to evaluate the centers’ willingness to participate and recognition of platform trials.

  4. Participant Feedback:

    • Gather feedback from participants on current MASH management models and their challenges, focusing specifically on non-invasive biomarkers.

b) Research Results

  1. Participant Background:

    • Respondents were mainly university hospital researchers, with 68% participating in national or international MASLD or MASH registries.

  2. Patient Needs and Participation Barriers:

    • 52% of respondents identified a significant treatment need in patients with advanced liver fibrosis (F3-F4).
    • The main barrier to participating in MASH trials was the lack of non-invasive testing methods.

  3. Awareness and Interest in Platform Trials:

    • 58% of respondents were familiar with adaptive trial design, but only 21% had participated in platform trials.
    • 94% of respondents believed platform trials could offer significant benefits to patients.

  4. Willingness to Participate:

    • All respondents expressed a willingness to participate in future platform trials.
    • 21% indicated that industry funding was necessary for their participation.

Conclusion and Value of the Research

Although this research is preliminary, its results have positive implications for establishing a MASH platform clinical trial network. Key conclusions include:

  • Patient Benefits: The study found that platform trials could offer more opportunities for patients to receive active treatments.
  • Need for Non-invasive Methods: It is crucial to explore and adopt non-invasive diagnostic and monitoring methods to reduce reliance on liver biopsies.
  • Strong Willingness to Collaborate: Most respondents showed a strong willingness to participate in platform trials if proper funding is provided, indicating a robust intention for collaborative efforts.

Research Highlights

  • MASH Patient Needs: The study highlights a significant unmet need for advanced liver fibrosis patients in MASH treatment.
  • Advantages of Platform Trials: Recognition of the potential of platform trials to improve clinical trial efficiency and therapeutic outcomes.
  • Multi-party Participation: Emphasis on the importance of multi-party collaboration among academic institutions, pharmaceutical companies, and patient organizations for successful implementation of platform trials.

Additional Information

Despite obtaining valuable data through the survey, the study has limitations such as a limited respondent composition and uneven geographical distribution. Future research should expand the survey scope to include more geographical regions and clinical research units for more comprehensive conclusions.

Conclusion

Establishing a PT in the field of MASH requires addressing multiple complex factors, including regulatory challenges, patient recruitment barriers, funding security, and multi-party collaboration. By tackling these key factors and leveraging existing clinical research networks and projects, advancements in MASH research and development of treatment options can be accelerated to address this growing public health issue.