The FDA intends to make the development and review of cancer drugs more efficient under the Real Time Oncology Review (RTOR) pilot program. Under this supplemental applications pilot, launched last year, evaluation of clinical data begins as soon as clinical data becomes available and results are ‘locked’. This means that the FDA is in a position to approve a formal application much sooner. In July Kisqali (ribociclib combination treatment) was approved less than one month after the June submission date and many months ahead of the scheduled approval date.
A lag time between approval and commercial launch could cost businesses that are not prepared to launch millions of dollars in lost revenue when factors such as intellectual property protection, licensing and potential sales revenue are considered.
Even more consequential is the time that patients may lose when new, potentially life-saving therapies are not available. From a competitive perspective, being first to market or first to launch is a favorable outcome for manufacturers, but only if the manufacturer is launch ready and prepared for a potentially expedited approval.
The Diaceutics end to end strategic and tactical planning services can help organizations be ready to launch. We support any launch that includes biomarkers for patient stratification, safety and efficacy, including market access initiatives.
This week, the FDA granted approval for pembrolizumab in combination with chemotherapy for first-line treatment of metastatic non-squamous NSCLC, making it the second FDA approval using RTOR. Previous submissions from Merck have included the use of a PD-L1 companion diagnostic.
The process is certainly faster and more efficient, nonetheless there are implications for the manufacturer. Smarter launch strategies need to be designed to accommodate the shorter review times to prevent any delay to launch and commercialization. Complex submissions that include companion diagnostics may be excluded in the pilot program - the FDA are more interested in drugs likely to demonstrate substantial improvements over available therapy (‘Breakthrough Therapy’ designation) with simple study designs and easy to interpret clinical endpoints such as overall survival. The FDA claim that the new process is ‘good for patients, good for health care providers, good for product developers, and good for the FDA’ despite stipulating that studies conducted exclusively outside the United States are excluded from the RTOR program. No justification is provided for this ‘US clinical trials’ rule and there is a risk that this could lead to higher costs for clinical trials as manufacturers may need US, Chinese and Japanese specific trials for global launch. There are further implications for treatments for rare diseases because clinical trial recruitment could be delayed if insufficient patients with a particular disorder can be recruited in a timely way from the US patient population.
The new review process is reported to be piloted in a number of applications for expanded use of already approved cancer drugs and if the process succeeds (no timeline is given), it will be expanded to applications for new cancer treatments. If recent trends continue, we are likely to see a number of additional therapies and indications gaining rapid approval, highlighting the need for the manufacturer to be launch ready sooner in order to achieve commercial launch excellence.