RE:RE:RE:RE:FDA ready to review novel cancer vaccine Platform TechnologyNew medicines, particularly biologics in the treatment of cancer, are increasingly being identified as efficacious across multiple cancer indications and can be viewed from a commercial and reimbursement perspective as drug platforms. Increasingly manufacturers are prioritising the development and launch of multi-indication medicines and in terms of how medicines become available in a given health care setting. First, manufacturers show a tendency to prioritise development of niche indications, with high disease severity and unmet need for the first indication of multi-indication medicines.
To a considerable degree this strategy seems to resonate with the objectives of health systems prioritising treatments that address significant unmet need and disease severity.
Relative to subsequent indications, first indications were more likely to be based on conditional approval or priority review, indicative of a prioritisation of patient populations with high disease severity and unmet need for the first indication.Further, a higher proportion of first indications received an orphan designation, as ONCY has been granted for pelareorep. These findings remain consistent when excluding EMA from analysis (where conditional approval is only granted to new drug submissions, rather than indication extensions).
Second, the evidence base of subsequent indications tends to be based on more robust study designs. Subsequent indications are more likely to be approved on the basis of phase III head-to-head trial designs, while first indications are more dependent on phase II, single arm trials. These findings are aligned with a higher proportion of conditional approvals and lower MCBS scores in first indications and a tendency to develop indications with high disease severity and unmet need in the first indication.
Consequently the development and marketing of multi-indication (platform) oncology medicines requires balancing a variety of factors that must be adjusted to the specific characteristics of a clinical setting.
As a result Big Pharma manufacturers of biologics are showing a tendency to launch first in niche indications with high disease severity and unmet need, a strategy that seems to be compatible with what health systems demand, and then expand on the drug platforms capabilites into earlier conditions and more common disease states.
https://delta4.ai/drug-indication-expansion/