Target Product Profiles for Antimicrobials
Cystic Fibrosis antimicrobial development is challenging, here are some reasons why:
- It can be difficult for drug developers to access insights from people with CF and therefore understand their needs for future treatments.
- In CF, different clinical needs need to be targeted: eradicating infection, pulmonary exacerbations, and suppression of chronic infection. In addition, there are many different bacteria, viruses and fungi that infect people with CF. This makes it difficult for drug developers to navigate this complex area.
- The preclinical landscape for CF antimicrobials is rapidly evolving, with a lack of guidance on which preclinical models and assays should be used.
- There is a lack of consensus on how to set up clinical studies to test the efficacy of CF antimicrobials, with concerns about existing commonly used endpoints (e.g. microbiology, FEV.). However, there is not enough evidence about alternative endpoints such as patient-reported outcomes.
To guide and catalyse the discovery or development of CF antimicrobials that meet the needs and priorities of people with CF, the CF AMR Syndicate has created Patient-Focused Target Product Profiles (TPPs) for CF-Related Infections.
TPP Development
TPPs are tools used by drug developers, which define the criteria a new drug needs to meet in order to fulfil specific patient and clinical needs. To accelerate the development and translation of novel CF antimicrobials to the clinic, the CF AMR Syndicate has developed a suite of patient-focused TPPs to define patient priorities and clinical needs for new CF antimicrobials.
The TPP development process began with a focus group involving people with CF and their relatives; this enabled identifying their unmet needs and priorities. The insights of people with CF were reinforced with clinical and industry expertise gained through focused engagement activities.
Process
Focus group of people with CF
Clinical focus group
Industry insights and expertise
Delphi Survey
Virtual Symposium
The burden of care faced by people with CF has been a key insight raised throughout the TPP development process. This is a key theme within the TPPs.
To enable consensus on the TPPs, an international Delphi survey was conducted across a range of respondent types; this identified the strong consensus on the TPPs key recommendations, and some areas for further discussion, including:
- The treatment durations suggested for new therapeutics
- The use of CF animal models and traditional infection rodent models in preclinical studies
- The use of patient-reported outcomes (PROs) as primary clinical trial endpoints in clinical studies
These areas were discussed in detail at
a TPP Virtual Symposium in April 2021.
Download a copy of the TPP Virtual Symposium Meeting Report.
Access the TPPs
To request a free copy of the TPPs, please complete the form below. The TPPs will be sent to you by email; please contact CF AMR Syndicate if you do not receive the TPP email within seven working days.
