🚨 FDA'S NEW PLAUSIBLE MECHANISM PATHWAY
@DrMakaryFDA @VPrasadMDMPH just published the @US_FDA most important position piece on biotech regulation in America.
It's short.
but it's 🔥
What you need to know about where the US rare/incurable-disease biotech is headed 🧵
@DrMakaryFDA @VPrasadMDMPH just published the @US_FDA most important position piece on biotech regulation in America.
It's short.
but it's 🔥
What you need to know about where the US rare/incurable-disease biotech is headed 🧵
"...a path to market entry for products where a randomized trial is not feasible."
While RCTs are the "gold-standard" for assessing efficacy, it's not always feasible to run or power.
FDA typically requires two "successful" RCTs for approval.
It's now going to be flexible.
While RCTs are the "gold-standard" for assessing efficacy, it's not always feasible to run or power.
FDA typically requires two "successful" RCTs for approval.
It's now going to be flexible.
"The Food and Drug Administration (FDA) is committed to providing regulatory guidance and encouragement..."
It's easy to think of regulators as the enemy.
But they are your friend.
They have seen thousands of trials.
Listen to them. There is wisdom in resistance.
It's easy to think of regulators as the enemy.
But they are your friend.
They have seen thousands of trials.
Listen to them. There is wisdom in resistance.
The FDA’s plausible mechanism pathway
What does this require?
» Specific biological/cellular/molecular cause is "known"
» Treatment targets most proximal mechanism
» Disease has predictable natural history
» Confirmation that mechanism was targeted
» Clinical course improves
What does this require?
» Specific biological/cellular/molecular cause is "known"
» Treatment targets most proximal mechanism
» Disease has predictable natural history
» Confirmation that mechanism was targeted
» Clinical course improves
"The FDA will consider previous clinical course and, in some cases, will view patients as their own control."
AND
"Clinical data must be strong enough to exclude regression to the mean."
Translation: no control arm required, but stats must hold.
AND
"Clinical data must be strong enough to exclude regression to the mean."
Translation: no control arm required, but stats must hold.
IMPORTANTLY:
"Once a manufacturer has demonstrated success with several consecutive patients with different bespoke therapies, the FDA will move toward granting marketing authorization for the product."
You heard it.
SEVERAL PATIENTS → FDA APPROVAL
"Once a manufacturer has demonstrated success with several consecutive patients with different bespoke therapies, the FDA will move toward granting marketing authorization for the product."
You heard it.
SEVERAL PATIENTS → FDA APPROVAL
MOST IMPORTANTLY:
"Manufacturers will then be able to leverage platform data from such personalized products to gain marketing approval for similar products in additional conditions"
Translation: You can use data from previous trials to accelerate new trials.
"Manufacturers will then be able to leverage platform data from such personalized products to gain marketing approval for similar products in additional conditions"
Translation: You can use data from previous trials to accelerate new trials.
"For instance, a single disease with 150 different genetic mutations with the same functional implication may require 150 different therapies, and the plausible mechanism pathway would be ideally suited to such therapies"
Translation: Severe Autism Spectrum Disorder
Translation: Severe Autism Spectrum Disorder
Source: nejm.org/doi/full/10.10…
• • •
Missing some Tweet in this thread? You can try to
force a refresh
