Use of RWD for frontline MF approval

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Hoosier Investor
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Joined: Thu Jun 18, 2020 5:48 pm

Use of RWD for frontline MF approval

Post by Hoosier Investor » Mon Jun 22, 2020 3:56 am

Recent presentations have made it clear the FDA will not consider Real World Data (RWD) in support of a drug's initial approval. This explains why we weren't allowed A) an early approval based on our P2 IMbark results, or B) an open-label P3 Registration trial using RWD as a synthetic control arm (as CKTC indicated may be a possibility). However, it appears the FDA will consider RWD for approvals involving additional indications.

Based on this information, I hope we can use RWD to expedite our frontline MF approval....after we gain regulatory approval in MDS or R/R MF.

ashah
Posts: 20
Joined: Wed Jan 09, 2019 1:32 pm

Re: Use of RWD for frontline MF approval

Post by ashah » Mon Jun 22, 2020 5:14 am

Question for the regulatory experts.
The limited use of the RWD by the US FDA seems to not be as scientific. Either allow the use or don't, but to say it cannot be used for 1st induction makes it more arbitrary than scientific.
Or have constraints that the use of RWD for approval will be tied to safety data... that makes it more manageable or science based.

Anyway, US FDA not withstanding, what is the policy for approval in the EU? Does EMA have similar constraints as the US FDA or are the Europeans more driven by data and unmet medical needs?

biopearl123
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Joined: Fri Jul 20, 2018 5:13 pm

Re: Use of RWD for frontline MF approval

Post by biopearl123 » Mon Jul 06, 2020 9:38 pm

Ashah, I think your question re EU becomes even more relevant and prescient given recent events. Even if the EU does not value RWD in the same way we had hoped the FDA would (and as they seemed to indicate via various guideline papers they published), it does appear that after orphan status is granted in the EU, (already for MF and pending in MDS) that a conditional EU marketing approval might be possible. I have no information on this other than provided by other board members regarding the EU approval process. Certainly the ramp up in regulatory specialists and the apparent push to enhance production and meet regulatory production guidelines by the company speak for something of this nature. bp

ashah
Posts: 20
Joined: Wed Jan 09, 2019 1:32 pm

Re: Use of RWD for frontline MF approval

Post by ashah » Tue Jul 07, 2020 12:26 pm

BP,
Thank you as always for thoughtful response.
One more follow up. What is the requirement for a +ve MDS Orphan Status in EU?
A question arises, why now, versus earlier or later?

biopearl123
Posts: 409
Joined: Fri Jul 20, 2018 5:13 pm

Re: Use of RWD for frontline MF approval

Post by biopearl123 » Tue Jul 07, 2020 4:34 pm

Hi Ashah, I think the answer lies in Geron needing data to differentiate it from Luspatercept which they now clearly have. bp

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