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Ask Me Anything: Regulatory Intelligence

  • 1.  Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 08:28

    Hi RegEx members,

    Recently, RAPS released it's quarterly Regulatory Focus Article Series: Regulatory Intelligence. We will be hosting an "Ask Me Anything" session today, Wednesday, 13 January, from 1:30-2:30 PM ET here on RegEx. Two regulatory experts, @Kirsten Messmer and @Richard Dennett will be available to discuss the articles and answer your questions in real time.

    If you cannot make the session, please feel free to submit any questions ahead of time by replying to this discussion or sending me an email at regex@raps.org. All of your questions will be answered on this thread at the time of the event.

    Looking forward to your questions!

    Best,
    Annie

    ​​

    ------------------------------
    Annie O'Brien
    Community Manager
    Regulatory Affairs Professional Society
    regex@raps.org
    ------------------------------


  • 2.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 13:29
    The AMA is starting - ask your questions below!

    ------------------------------
    Annie O'Brien
    Community Manager
    Regulatory Affairs Professional Society
    regex@raps.org
    ------------------------------

    Original Message


  • 3.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 13:32

    I would like to know if there is any certification we can get or online course for Regulatory Intelligence.

    I already started with this role within my company but would like to know more about tools and tricks that I can use to develop my expertise in Regulatory Intelligence.


    Thank you!



    ------------------------------
    Youmna Abboud
    Warsaw
    United States
    ------------------------------

    Original Message


  • 4.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 13:39
    Hello Youmna, thank you for your question. Although I am not aware of any specific certification for regulatory intelligence as such there are some options that might help: may be the RAC (Regulatory Affairs Certification) and also the book Regulatory Intelligence 101. Perhaps some local universities my have courses or offer remote options. I believe Temple University for example may offer some courses. Please note that the book on regulatory intelligence is being updated currently and you may want to wait until the 3rd Edition is released which is planned for March. Hope that help, Kirsten. ​

    ------------------------------
    Kirsten Messmer RAC
    Principal Regulatory Affairs Specialist
    Garner NC
    United States
    ------------------------------

    Original Message


  • 5.  RE: Ask Me Anything: Regulatory Intelligence

    This message was posted by a user wishing to remain anonymous
    Posted 13-Jan-2021 13:38
    This message was posted by a user wishing to remain anonymous

    Do you think a regulatory intelligence program can be a standalone function at an organization, or is it better off being combined with current reg affairs professionals responsibilities?
    Original Message


  • 6.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 13:46
    Hello, it depends on the size and the aims of the companies, e.g. the availability of personnel resources. Within large companies there might be specific departments that only work in regulatory intelligence. We have this within PPD and we have several professionals that work 95% in regulatory intelligence only. However, smaller companies my not have the human resources and regulatory intelligence becomes part of the regulatory function as such. Generally, it is very beneficially to have a specific regulatory intelligence function since the updates from regulatory agencies/ethics committees are coming out faster and faster globally. But as mentioned it might depend on the aims and resources that the company has. Please let me know if you have further questions on that, Kirsten.​

    ------------------------------
    Kirsten Messmer RAC
    Principal Regulatory Affairs Specialist
    Garner NC
    United States
    ------------------------------

    Original Message


  • 7.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 13:46

    I would appreciate thoughts on meaningful metrics around Regulatory Intelligence management. From discussion with peers in other CROs and pharma there's nothing particularly useful, tending to be along the lines of:

     

    • Number of change items versus previous YTD
    • Average delay between date of regulatory change/communication and notification to/identification by Regulatory Intelligence
    • Average duration of change management activity

     

    The variation seen with regards to whether something is a draft consultation/guidance or whether it's a full Regulation impacting multiple functions means some activities are not directly comparable with others, other than to gauge resource impact of assessing and implementing regulatory change or 'the cost of doing business'.



    ------------------------------
    Derek Johnston
    Maidenhead
    United Kingdom
    ------------------------------

    Original Message


  • 8.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 13:50
    Hello Derek, Thank you for your question. This isn't really my area expertise, but @Linda Bowen can probably advise on this question. Linda, do you mind jumping in on this question? Thanks, Kirsten.​​​

    ------------------------------
    Kirsten Messmer RAC
    Principal Regulatory Affairs Specialist
    Garner NC
    United States
    ------------------------------

    Original Message


  • 9.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 14:14
    I have just finished the Metrics Chapter for the 3rd edition of the RAPS RI Book. I'd be happy to share with you some of the ideas the DIA RI WG have discussed and we have presented on with respect to "Measuring Success" in policy and intelligence. 

    And I teach a 36 hour course on Regulatory Intelligence in the Master's in RAQA program at Temple University.


  • 10.  RE: Ask Me Anything: Regulatory Intelligence

    This message was posted by a user wishing to remain anonymous
    Posted 13-Jan-2021 13:49
    This message was posted by a user wishing to remain anonymous

    We are an early stage CT (MSC) company that has just demonstrated proof of principle & are in the process of seeking investment funding for the development of our product to GMP clinical manufacture / FIH clinical trial - any pointers of an optimal approach?
    Original Message


  • 11.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 13:58

    Thank you, quite a loaded question!

    • A first step is to understand and establish your Target Product Profile (TPP) and quality attributes of the product; including - active elements / mode of action etc.
    • Adopt a 'begin with the end in mind' approach – what is the target indication?; what will the commercial product look like?, how much product will be needed?, process basis?, formulation etc.
    • Next, line up a CMO to get a timeframe & costings; also, for contracting out non-clinical development / toxicology studies -  this will be important to build a model for the investors
    • You will need to develop a potency assay or surrogate model
    • The non-clinical process needs be representative of the clinical process; therefore, need to demonstrate product comparability i.e. non-clinical/clinical characterization of principle CQAs of the product
    • The bench process might get you into clinical  / first into human but will need proper development to make it suitably robust and commercially orientated - invest time to do this as a) a characterized and consistent product and a robust and reproducible process are key -  not expected at phase 1 of course , but it is good to adopt this general philosophy as early as possible
    • Make sure all necessary procurement is in place re: donor eligibility, consent, testing and make sure tissue establishment licenses are in place

    Lots more to think about but they're some of the  basics to hopefully get you going.

    ------------------------------
    Richard Dennett
    Paris
    France
    ------------------------------

    Original Message


  • 12.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 13:57
    Hi experts - Another question for you:

    What is the biggest challenge as you see it for CGTs ?

    ------------------------------
    Annie O'Brien
    Community Manager
    Regulatory Affairs Professional Society
    regex@raps.org
    ------------------------------

    Original Message


  • 13.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 14:08
    Thank you. 


    Probably many!   Across Cell, Gene and Tissue therapies, Product Diversity and product specific/nuances are a main one as i see it  - no one size fits all and therefore whilst we have good backbone guidelines in place, there is still the  need to base CGT development on a 'risk based approach'  - in the demonstration of project quality and safety.



    ------------------------------
    Richard Dennett
    Paris
    France
    ------------------------------

    Original Message


  • 14.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 14:14
    ​As Richard says, there are many challenges - I think a particular one is also comparability of the CGT products throughout the development program. Is the product being developed for commercialization still comparable to the product that was used initially to investigate safety and efficacy. The FDA has noted in a recent webinar that this comparability and demonstrating it is key in approval evaluations. The FDA suggests to develop critical quality attributes as early as possible and also validate assays as early in the development process as possible.

    ------------------------------
    Kirsten Messmer RAC
    Principal Regulatory Affairs Specialist
    Garner NC
    United States
    ------------------------------

    Original Message


  • 15.  RE: Ask Me Anything: Regulatory Intelligence

    This message was posted by a user wishing to remain anonymous
    Posted 13-Jan-2021 14:09
    This message was posted by a user wishing to remain anonymous

    We have seen reports of occasional CGT products receiving Clinical Hold re CMC – do you know of the principle reasons?
    Original Message


  • 16.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 14:25
    'Clinical hold' is principally applied for Quality & Safety reasons:  For CMC 'news headline' -  clinical holds -  the reasons aren't always stated but mostly this is down to: not actually having the clinical material produced in time (I have seen this happen a few times) - i.e. 'a case of the 'the clinical cart moving before the CMC horse' ; certain characterization not having been performed; including correct coverage of process and product impurities; having a suitable potency assay in place -  these are some reasons I am aware of for cell and gene therapies

    ------------------------------
    Richard Dennett
    Paris
    France
    ------------------------------

    Original Message


  • 17.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 14:13
    Another question for our experts: What benefits does a regulatory intelligence program provide a regulatory affairs professional?

    ------------------------------
    Annie O'Brien
    Community Manager
    Regulatory Affairs Professional Society
    regex@raps.org
    ------------------------------

    Original Message


  • 18.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 14:22
    Thanks for the question, Annie. In general, regulatory affairs is moving fast and the new guidances, regulations, trends etc are coming out faster and faster. A dedicated regulatory intelligence professional can keep on top of the new developments and alert stakeholders inside and outside of the company of the new developments that might have a business impact. Regulatory intelligence is not only reading and passing on ​information, but also analyzing what it all means and how it might influence regulatory strategy. We see a lot of regulatory intelligence utilized to form regulatory product development and commercialization strategies as well as scoping out the competition in the space of specific products and/or for various indications. The utility of regulatory intelligence is limitless and if possible, a company should consider investing in having a team of dedicated professionals. Please let me know if you'd like me to elaborate on a specific aspect, Kirsten.

    ------------------------------
    Kirsten Messmer RAC
    Principal Regulatory Affairs Specialist
    Garner NC
    United States
    ------------------------------

    Original Message


  • 19.  RE: Ask Me Anything: Regulatory Intelligence

    This message was posted by a user wishing to remain anonymous
    Posted 13-Jan-2021 14:18
    This message was posted by a user wishing to remain anonymous

    What are the GMO assessment situation for China ?
    Original Message


  • 20.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 14:37
    The assessment of genetically modified organisms (GMO) varies by country. This is normally a requisite before the clinical trial is able to proceed.  In the EU this by separate health and safety committees /environmental review board or in the US, similarly - Institutional Review Boards and Institutional Biosafety Committees (IBC) -  In China there is currently no separate review of GMOs for their technical  construction, attenuation etc. and environmental risk assessment but this is rather reviewed at the point of a pre-IND  meeting and during assessment of the IND dossier itself for GMO / gene therapy products

    ------------------------------
    Richard Dennett
    Paris
    France
    ------------------------------

    Original Message


  • 21.  RE: Ask Me Anything: Regulatory Intelligence

    This message was posted by a user wishing to remain anonymous
    Posted 13-Jan-2021 14:23
    This message was posted by a user wishing to remain anonymous

    We are developing a cell based allogenic product that has a short shelf life -  what are the logistics considerations for the clinical trial ?
    Original Message


  • 22.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 14:45
    For an allogeneic cell based product, this will depend upon if the cells can be cryogenically frozen or not (linked to viability at recovery).  Cryogenic transit can therefore be conducted by an certified bio-courier or the cells may have to be shipped usually at a controlled ambient lowered temperature. Coordination of the patient to be at the clinical site (that may not necessarily be near by also needs to be factored to receive the administration -  so there are a few parts that need alignment and this can be challenging.


    Consider also 'in use' stability as viability still needs to be maintained and within specification during administration



    ------------------------------
    Richard Dennett
    Paris
    France
    ------------------------------

    Original Message


  • 23.  RE: Ask Me Anything: Regulatory Intelligence

    Posted 13-Jan-2021 21:29
    Another huge consideration - particularly for biological materials such as cells - is shipping internationally. Logistics can become complicated when needing to follow several different import/export requirements and obtaining licenses in addition to cell stability maintenance and respecting the respective subject privacy laws. As you can probably see there are many logistical implications and building a strong strategy taking into account the various regulatory and practical requirements to sustain product integrity and ensure patient safety will be imperative. ​

    ------------------------------
    Kirsten Messmer RAC
    Principal Regulatory Affairs Specialist
    Garner NC
    United States
    ------------------------------

    Original Message


  • 24.  RE: Ask Me Anything: Regulatory Intelligence

    This message was posted by a user wishing to remain anonymous
    Posted 13-Jan-2021 17:06
    Edited by Eileen Quinn 13-Jan-2021 17:05
    This message was posted by a user wishing to remain anonymous


    I'm working on a Phase 1 Drug Product and we're thinking about increasing the packaging size for our DP being manufactured soon.  The DP formulation will remain the same, the components of the packaging will remain the same and all grading of the packaging will remain the same.  We want to increase the volume of product, so the vial size will increase; however, the head space will remain the same.  If we use the new packaging, which is equivalent to the last packaging, can we still base our expiry on the last DP formulation?  We also file globally.

    Thank you
    Original Message


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