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  • 1.  TDI for process impurities, ICH Q3A/M3

    This message was posted by a user wishing to remain anonymous
    Posted 08-Dec-2021 10:19
    This message was posted by a user wishing to remain anonymous

    Hi RAPS community,

        I work on biologics, and small molecule CMC is certainly not my area of expertise, but I have a question on some apparent inconsistencies (to me as a layman) among ICH guidelines.
       Say hypothetically, I am trying to develop a generic version of a hypertension drug that the patient needs to take for life.   The RLD has a dose of 800 mg/day.  I have a process impurity at 0.07% that is not present in the RLD.  There is no obligation of identifying this impurity as it is below the identification threshold, but I performed the identification out of an abundance of caution.  The impurity is predicted to be negative for bacterial mutagenicity.  I propose an acceptance limit of 0.1%.  If this is agreeable to the agency, it would mean that from a regulatory standpoint, 0.8  mg TDI for chronic dosing is acceptable.  
       1) If 0.8 mg TDI lifetime exposure is an acceptable risk for an unqualified impurity, why does ICH M3 require a 14-day tox study for microdose trials (single dose of 0.1 mg or lower) ?
       2) Applying the approach of Less-than-Lifetime exposure, a single dose of 50 mg of this impurity (neat) should be considered safe in humans (1.5 ug of lifetime exposure is similar to 120 ug of short-term exposure less than a month, as most toxicities depend on both dose and duration, ICH M7) ?

      Thank you very much for insights

    Tobias
       



  • 2.  RE: TDI for process impurities, ICH Q3A/M3

    Posted 09-Dec-2021 10:10
    Hello Tobias,
    I'll try to address your two questions based on my experience with small molecules. For full disclosure, I am not a toxicology expert; therefore, my understanding of ICH M3 is not much different from yours.

    1) ICH M3 is about the drug (as finished product and API), not the impurities. Drugs intended to be administered at low dose are not exempted from nonclinical studies to ascertain their safety and tolerability before they are given to humans. As you can see from the details of Table 3, a variety of approaches are possible-the extent of the possible studies therein listed is more limited than what would be required for drugs at higher doses.
    2) the LTL principle typically only applies within the context of ICH M7. With the possible exception of drugs introduced in Phase 1, for which  a  justification for thresholds higher than those indicated in ICH Q3A and Q3B is being accepted by regulators worldwide nowadays (see the 2017 Harvey paper).

    As a general principle, please keep in mind that an impurity by its own definition does not bring any benefit, only risk. That is why there is an expectations that impurities are controlled at limits that are considered generally safe, in the absence of qualifying toxicological studies.

    I have a question for you: if you intend to include this now identified impurity in the specification of the drug substance, in the absence of toxicological qualification the highest limit you could claim is not more than 0.15%. If you specify it at 0.1%, do you mean that at 0.14% a batch would still pass?
    Have you monitored this impurity on stability? Does the limit takes into account the possibility that it may grow on storage? In other words, is it a drug substance degradation product?
    Finally, what about the drug product? Does it grow on storage, could it be considered a degradation product?

    Hope this hleps,
    Maurizio

    ------------------------------
    Maurizio Franzini
    Regulatory Affairs CMC, Associate Director
    San Francisco CA
    United States
    ------------------------------



  • 3.  RE: TDI for process impurities, ICH Q3A/M3

    This message was posted by a user wishing to remain anonymous
    Posted 09-Dec-2021 23:41
    This message was posted by a user wishing to remain anonymous

    Thank you so much Maurizio for the comments
         1) I am not a toxicologist either, but I think one of the basic tenets of toxicology is that it depends on both dose and duration.  If nothing else, there is typically a significant difference in plasma concentration between single and repeated dose of a chemical at steady state.  The exact factor for conversion may be up to debate, but I think most people would agree that the risk to patient safety is quite different weather a chemical is administered as a single dose or a 20-year chronic dose.  I am interested to know if the agency allows higher qualification thresholds for drugs that are intended as a single dose/very short duration.
        2) The genesis of my question actually was from my reading of the Harvey paper.  They stated that 95% of in vivo tox studies on a variety of chemicals have NOEL greater than 0.22 mpk (no allometric scaling) or 0.038 mpk (with allometric scaling), therefore 1 mg TDI is scientifically sound.  Harvey et al were key opinion leaders, but it seems to me that they did not account for human to human variation (F2=10) and the fact that most of these studies were less than 3 months (F3=10).  Using these uncertainty factors, it would seem that 10 ug TDI lifetime exposure is more reasonable
        3) 10 ug TDI chronic exposure as a general safe level seems to be more in line with the 100 ug single dose in microdosing studies
        4) As you correctly pointed out, impurities do not typically provide benefits to the patients.  Therefore, our risk tolerance should be lower than a drug in microdosing which might eventually benefit the patient.  I think such a consideration is the reason why WHO's TTC is 10x lower than that in ICH M7.
        5) The example in my original post was hypothetical to illustrate what I see as significant difference in qualification thresholds between Q3A and M3.  A 0.14% impurity level would have exceeded the 1 mg limit.

         Thank you again for the insights.

    Tobias


  • 4.  RE: TDI for process impurities, ICH Q3A/M3

    This message was posted by a user wishing to remain anonymous
    Posted 15-Dec-2021 13:43
    This message was posted by a user wishing to remain anonymous

    I am wondering if there are participants on this forum who strongly disagree with my statement in the original post that there appear to be some inconsistencies on the qualification thresholds between ICH Q3 and M3, and the 1 mg TDI might not be reasonable.  I certainly appreciate that Q3 is intended for impurities and M3, typically API.  That said, a chemical is a chemical, and it will do harm to the patient if taken above a certain level and for longer than a certain duration.  The body can not really tell, nor does it care, the difference between an impurity and an API.  Our risk assessments, therefore, should be consistent across ICH guidelines.

    Regards,

    Tobias




  • 5.  RE: TDI for process impurities, ICH Q3A/M3

    This message was posted by a user wishing to remain anonymous
    Posted 09-Dec-2021 11:15
    This message was posted by a user wishing to remain anonymous

    Are you talking about clinical trials for a new molecular or new biological entity, since you refer to "microdose trials" and ICH M3?

    Yours is an approved RLD product versus developing ANDA, correct? Make sure you are applying the right guidelines!



  • 6.  RE: TDI for process impurities, ICH Q3A/M3

    This message was posted by a user wishing to remain anonymous
    Posted 09-Dec-2021 23:41
    This message was posted by a user wishing to remain anonymous

    Hi Anon

        Thank you for the comments.  Almost all chemicals, be they environmental toxins, food additives (intentional or unintentional), or pharmaceutical impurities, have the potential to harm human beings above a certain level and/or taken for a longer than a certain duration.  The example in my original post was hypothetical, but I wanted to make the point that our risk assessments should be consistent among ICH guidelines, be it Q3A or M3 7.1.  I have zero expertise in small molecule CMC, and would certainly welcome your thoughts on this

    Tobias


  • 7.  RE: TDI for process impurities, ICH Q3A/M3

    This message was posted by a user wishing to remain anonymous
    Posted 15-Dec-2021 16:45
    This message was posted by a user wishing to remain anonymous

    Your blanket assessment that all chemicals (whether approved, NCE, ) are same in risk is a big issue from the regulatory perspective especially in the drug development world! You gave hypothetical scenario using ANDA drug development which is hugely different to a NCE development in risk assessment! Your question does not appear to be anything to do with biological or small molecule CMC experience. Sorry, you need to be more specific in your question! Good luck!


  • 8.  RE: TDI for process impurities, ICH Q3A/M3

    This message was posted by a user wishing to remain anonymous
    Posted 20-Dec-2021 10:40
    This message was posted by a user wishing to remain anonymous

    Hi Anon,

    Thank you for the comments, and I can not agree with you more that NCE and generic developments are different.  I did not say all chemicals have the same risks, and I am sorry if my choice of words led to such a confusion.  The chemical at issue is an unspecified impurity with unknown tox profile.

    For an NCE, impurities below 0.15% can also be considered qualified by virtue of IND enabling tox, as impurity levels in the tox batch are often at least as high as the clinical batches.  In addition, the API with defined impurity specs is characterized extensively in further tox studies such as the 2-year genotox in late development.  For a generic, however, a sponsor has no obligations, nor will it likely to volunteer, to do a tox evaluation for a new impurity below the qualification threshold.  Therefore, I think the risks to patient safety are not negligible for generics with new impurities.

    NCE or generic, hypothetical example or not, my puzzlement remains the same: why is it logical to require tox of an 80 ug single dose in a phase 0 trial (where the drug may have some eventual benefits to the patients), when the same sponsor can have an impurity of 800 ug for chronic dosing without qualification if it has a different development program for a generic (where the impurity is not expected to have any benefits)?  If we agree that M3 is reasonable, we will likely have to conclude that 1 mg TDI is not.

    Furthermore, if 1 mg TDI is sound, we also have to conclude that an 1 mg single dose of a chemical of unknown tox profile (let's say the sponsor has reasons to believe that it is a life-saving medication) should be considered safe without tox evaluations.  I know of no regulatory agencies in the world that would allow such a human study to proceed.

    I would love to hear your arguments against the above contractions.

    Best, 

    Tobias