Cystic Fibrosis antimicrobial development is challenging
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.
The challenges we currently face
01
It can be difficult for drug developers to access insights from people with CF and therefore understand their needs for future treatments.
02
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.
03
The preclinical landscape for CF antimicrobials is rapidly evolving, with a lack of guidance on which preclinical models and assays should be used.
04
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.
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.
Our Process
Focus group of people with CF
Delphi Survey
Clinical focus group
Virtual Symposium
Industry insights and expertise
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.
A copy of the TPP Virtual Symposium Meeting Report is available to download.
Access the TPPs for Antimicrobials
Please complete the form to request a free copy of the TPPs and a digital copy will be sent to you.