{ "media_type": "text", "post_content": "Is there such a thing as durability of a small molecule? Like you take it once a day probably, but how do you measure the robustness of the therapy? I am thinking efficacy may be a slightly different metric. I understand durability of a gene therapy...like how long it should last. But can’t think of a similar metric for small molecule.", "post_id": "5fc3ffb00bb04e0024f9a333", "reply_count": 7, "vote_count": 3, "bowl_id": "5ae46a9f9f9aa00018cc41d5", "bowl_name": "Life Sciences Consulting", "feed_type": "bowl" }

Is there such a thing as durability of a small molecule? Like you take it once a day probably, but how do you measure the robustness of the therapy? I am thinking efficacy may be a slightly different metric. I understand durability of a gene therapy...like how long it should last. But can’t think of a similar metric for small molecule.

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Yes this is an efficacy endpoint known as DoR (duration of response) - most often used in oncology alongside ORR and CR/PR/SD/PD.

If you’re talking preclinical - you’re looking at PK T1/2, tumor inhibition % and duration of PD response.

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Listen to D1. He/she knows their shit.

OP I think you are looking to understand pk/pd

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Durability? So like half life?

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Pkpd would be a related way of measuring this, though it doesn’t necessarily translate to what you’re trying to get at I think.

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Robustness is an awkward word to use here I think. I think you're referring to stability which is related to shelf life or PK/PD which is related to frequency of administration. Both dependent on a lot of factors.

I saw a clinical trial use "durability of response" as a secondary endpoint a while ago. It was a biologic but I could see it making sense for small molecules

Durability is more of a long term efficacy question - sure we saw a 30% reduction in primary outcome for 18 months in clinical studies, but is that reduction maintained at the 3 years? 5 years? Etc. Real world evidence has entered the chat

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