Global health regulators collaborate: International Coalition of Medicines Regulatory Authorities

Founded in 2019, the International Coalition of Medicines Regulatory Authorities (ICMRA) collaborates to review changes to manufacturing or add manufacturing sites to “enable better industry quality management to reliably supply critical medicines for patients in need”.  An article posted by the FDA announces recent progress and future plans (here).

ICMRA has just completed a pilot.  The article notes that the “FDA and EMA regulators completed the first collaborative assessment of a proposed post-approval change for a critical oncology biologic with the Japanese Pharmaceuticals and Medical Devices Agency (PMDA) serving as an observer. The work, in which FDA and EMA reviewed and approved a proposal to add new manufacturing and quality control sites, can help assure the supply of the medicine.”

 

Other activities and future plans make an interesting read, and if successful will simplify and speed future approvals of new therapies and subsequent modifications of their manufacturing to ensure global therapy supply.

FDA new FINAL Guidance: CDER’s Program for the Recognition of Voluntary Consensus Standards Related to Pharmaceutical Quality

For my CMC colleagues, this will be of interest. I didn't know what a "voluntary consensus standard" meant, and fortunately the Guidance addresses that as a foot note to the Introduction:

"a standard that is developed or adopted by domestic and international voluntary consensus standards bodies . . . . These bodies often have . . . policies that include provisions requiring that owners of relevant patented technology incorporated into a standard make that intellectual property available to implementers of the standard on non-discriminatory and royalty-free or reasonable royalty terms."

My initial thinking in reading this was "OK - NIST, USP" but another footnote indicates that other bodies can submit to have other standards recognized: "Although regulated industry would be the primary user of standards that are within the scope of this program, any interested party may request recognition of a standard. "

It's also noted that CDER’s program is different than the standards recognition program in the CDRH.

To read the new final guidance, access it here: https://www.fda.gov/media/121305/download

Updates to the FDA’s Clinical Laboratory Improvement Amendments (CLIA) Web pages Make Processes Clearer, Easier to Understand

FDA just released information on the improvements to their website for users and FDA's role in establishing assay complexity standards:

--The Clinical Laboratory Improvement Amendments (CLIA) and the associated regulations provide the authority for oversight of clinical laboratories and laboratory testing. While the Centers for Medicare and Medicaid Services (CMS) is responsible for many aspects of the CLIA program, including the certification of clinical laboratories, the U.S. Food and Drug Administration (FDA) is responsible for categorizing the complexity of tests, and the categorization is then used to determine which laboratories can perform which tests.

The website: https://www.fda.gov/medical-devices/ivd-regulatory-assistance/clinical-laboratory-improvement-amendments-clia?utm_medium=email&utm_source=govdelivery

 

Publication of Interest: Neutralizing Antibody Validation Testing and Reporting Harmonization

Experts from the AAPS's Therapeutic Product Immunogenicity Community and the Food and Drug Administration (FDA) recently published an open access article on "Neutralizing Antibody Validation Testing and Reporting Harmonization" in the AAPS Journal. This industry-Agency collaboration helps align the science and data needed for regulatory review of immunogenicity data.

 

The Abstract notes the areas required to be tested during validation and included in the validation reports:

(1) format selection;

(2) cut point;

(3) assay acceptance criteria;

(4) control precision;

(5) sensitivity including positive control selection and performance tracking;

(6) negative control selection;

(7) selectivity/specificity including matrix interference, hemolysis, lipemia, bilirubin, concomitant medications, and structurally similar analytes;

(8) drug tolerance;

(9) target tolerance;

(10) sample stability; and

(11) assay robustness

 

Kudos to Heather Myler and the team for the in-depth consideration of the issues related to NAb assays and in developing recommendations for standardized reporting formats.

Quanterix Launches LucentAD Biomarker Blood Test To Aid Physician Diagnosis Of Alzheimer’s Disease In Patients

With Eisai and Biogen’s Leqembi treatment for Alzheimer’s disease recently receiving full FDA approval, Quanterix has launched a prescription test, LucentAD, for the p-Tau 181 protein.  The test will be used to support physicians in diagnosing patients with early symptoms and assessing if drug therapy is warranted.  The test has also recently been shown to correlate with reduction in amyloid load in the brains of amyloid patients on anti-amyloid drug therapies. Performed on its SIMOA platform, LucentAD is a lab developed test (LDT), i.e., not approved by FDA’s CDRH division.  Quanterix is offering the assay through its just launched Lucent Diagnostics lab, which operates under CLIA regulations in the US. 

 

Link to the announcement.

FDA CDER updates internal policy for Risk-Based Manufacturing Site Selection

 For those in the CMC space, the latest from the FDA's CDER on how they do risk-based manufacturing site selection:

FDA recently published a revision to Manual of Policies and Procedures (MAPP) 5014.1, Understanding CDER’s Risk-Based Site Selection Model (https://www.fda.gov/media/116004/download?utm_medium=email&utm_source=govdelivery). This MAPP outlines how the Office of Pharmaceutical Quality (OPQ) will manage the Site Selection Model (SSM) to prioritize manufacturing sites for routine quality-related (i.e., current good manufacturing practice) surveillance inspections. The MAPP revisions include:

--Updating the model by adding a risk factor about the compliance history of establishments in the country or region in which an establishment is located.

--Providing clarity on the risk factors associated with patient exposure and inherent product risk.

--Adding language about quality system effectiveness.

FDA Requesting Comments on the draft Pharmaceutical Quality/Chemistry Manufacturing and Controls (PQ/CMC) Data Elements and Terminologies

Of interest to those working in the PQ OR CMC spaces and wanting to keep abreast of the latest in FDA thinking, the FDA is requesting comments on the draft "Pharmaceutical Quality/Chemistry Manufacturing and Controls (PQ/CMC) Data Elements and Terminologies" document.  These data elements and terminologies are used in electronic submissions of PQ/CMC data within filings. "... the Agency is continuing to seek comment on the accuracy, suitability, and appropriateness of revised and/or new data elements and terminologies for submission of PQ/CMC data."

 Extensive details on the Background and the Establishment of a Docket are available at the provided link.