@JB1975- FDA guidelines require mesoblast to submit validated potency assays. So , I would suggest that an independent lab validated the work and , as they often remind us, they have constant dialogue with the FDA. Highlighted in purple is where the FDA says- hey , send us yo data and we will check it.
Reg
3. Biologics License
To obtain a biologics license, a validated potency assay or assay matrix with defined
acceptance criteria must be described and justified in the BLA (21 CFR 601.2(a) and
211.165(e), see also Section II.B). The acceptance criteria should be based on
knowledge gained through manufacturing experience and data collected from assays
performed during all phases of product development and clinical investigation (Ref.
5). As you evaluate product conformance lots or lots manufactured explicitly for use
in your pivotal clinical studies, acceptance criteria should be refined to reflect these
data.
The potency assay acceptance criteria defined in your BLA, which are intended for
subsequent lot release testing, should reflect the potency limits established for product
lots used in the pivotal clinical studies demonstrating clinical effectiveness (see FDC
Act, Section 505(d), 21 U.S.C. 351).
C. What Should be Considered for a Potency Assay Validation Plan?
1. Regulations
To obtain a biologics license, you must submit data in your BLA demonstrating,
among other things, that your product meets prescribed requirements of potency
(21 CFR 601.2),
which requires that you validate your potency assay (see
21 CFR 211.165(e)). The validation process identifies potential sources of error and
quantifies them within the assay method. During assay development, you should
evaluate assay performance and suitability for use. Numerous resources are available
for analytical methods validation (Refs. 9 through 11). You should perform analysis
and validation of all relevant assay parameters (Refs. 9 through 11), including:
Accuracy;
Precision (Repeatability, Intermediate Precision);
Specificity;
Linearity and Range;
System Suitability; and
Robustness.
You may provide data collected from potency assay validation studies in
electronic format to facilitate statistical evaluations by the CBER review committee,As discussed in Section III.B.3, qualitative assays may be used as part of an assay
matrix to assess potency, provided that you conduct suitable correlation studies.
You
should validate all parameters relevant to your qualitative assay and provide a
rationale for those parameters that you determine are not relevant.
As this guidance indicates, a considerable amount of data might be necessary to
develop a suitable measurement of potency for your product (see also Ref. 15). In
addition, your assay(s) might change over time in response to new information
obtained as you develop your product. Therefore, w
e recommend that you have
timely discussions with your CBER review team as you design, evaluate and validate
your potency measurement.
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