Lilly's Principles on Accelerated Approval
June 20, 2023 Posted by: Eli Lilly and Company
By Carl Garner, Senior Vice President, Global Regulatory Affairs at Eli Lilly and Company
We know the positive impact that the Accelerated Approval Program has had for millions of people, and at Lilly we are committed to always doing what’s right on behalf of patients. The Accelerated Approval Program advances innovation and provides early access to safe and effective therapies that save lives. We also know that concerns have been raised about the program.
We take our responsibilities in the accelerated approval process seriously. As part of our commitment, we’ve outlined our own set of guiding principles on Accelerated Approval.
We are committed to providing timely access to FDA-approved, safe and effective new medicines for patients with unmet need.
We are committed to transparency.
Lilly has a responsibility to be transparent about its pursuit of accelerated approval.
Lilly will be clear about why accelerated approval is appropriate for a given drug and patient population, including the rationale behind the surrogate endpoint, and will ensure that research regarding the use of the endpoint is published and/or publicly available.
We are committed to expeditiously produce confirmatory trial data.
Alignment with the FDA on confirmatory study design should occur prior to accelerated approval.
As a general rule, enrollment in confirmatory studies should be underway prior to accelerated approval.
If confirmatory studies fail to verify the benefit, Lilly will promptly initiate discussions with FDA to determine appropriate next steps.
If Lilly and FDA agree that withdrawal is appropriate, Lilly will act quickly to discontinue the product while working to ensure patients currently on therapy have the option to complete treatment.
There may be legitimate scientific reasons to continue to pursue confirmatory studies, even in the face of unclear data. Such circumstances would require full transparency and significant consultation with FDA and external stakeholders about the rationale and timeline for ongoing research and any continued availability of the product.
These principles aren’t just hypothetical for us. As a recent example of our commitment to expeditiously produce confirmatory trial data, the FDA recently converted our accelerated approval of Retevmo® (selpercatinib) for non-small cell lung cancer to a traditional approval. This happened a little over two years after the accelerated approval was granted in May 2020. While not every conversion will happen this quickly, such timely production of data happens when pharmaceutical companies align on study design early, and work to enroll those studies in advance of accelerated approval.
On a different note, FDA has stated that the process to withdraw a drug’s approval can be cumbersome. We are supportive of efforts to streamline this process, but also believe it’s important for pharmaceutical companies to be proactive when a treatment with an accelerated approval has confirmatory data that fails to verify a clinically meaningful benefit. When the confirmatory study for Lartruvo® (olaratumab) failed to verify a clinical benefit in 2019, we worked with the FDA to discontinue the product while helping to ensure patients on therapy had the option to complete treatment.
We are committed to continue employing an appropriate, proactive approach for our accelerated approval products across our portfolio.