- In early cancer drug development, we are amid of an important transition: From the pursuit of the maximum tolerated dose (MTD) that dominated the development of cytotoxic chemotherapy to the attempt to define the biological optimal dose for targeted anti-cancer agents and immune oncology treatments .
- This transition – accentuated by the FDA’s Project Optimus and the agency’s draft guidance on dose-optimization – will require significant changes to the early development of anti-cancer agents. With additional dose-optimization studies development timelines and costs will increase but it is also clear that non-optimized dosing is likely to reduce the efficacy and increase the toxicity of an agent.
- New study designs, pre-clinical research, updated mathematical modelling of PK/PD relationships and potentially new early endpoints (e.g. ctDNA) are among the sophisticated tools needed to achieve a new balance in early drug development.
- A very interesting aspect is the exploration of the use of patient-reported outcomes early in drug development for dose-optimization based on short- and longer tolerability. These data could provide important additional information for dose selection and could lead to an earlier understanding of the patient experience.
- The workshop will offer a neutral and collaborative platform for evaluation and discussion of innovation in these areas with active participants from all relevant stakeholders. Informative lectures and panel discussions open to all attendees will alternate.
- The main objective of the workshop will be to formulate how dose optimization in the future could be implemented in early drug development based on new methodologies, but also considering the views from all relevant stakeholders. CDDF intends to publish the outcome .
Learning outcomes / Meeting objective
- Explore challenges to the implementation of dose-finding studies in oncology
- Discuss opportunities to improve dosing strategies given ongoing challenges
- Identify key considerations for selecting appropriate dose optimization strategies in oncology in light of FDA’s project OPTIMUS
- Potential implications of project OPTIMUS for dose-finding strategies in oncology early development in Europe and beyond
The target is a multidisciplinary audience of academia representatives, EU and US regulatory bodies (EMA, FDA, National Agencies), the pharmaceutical industry, HTAs, and patient advocates.