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.

FDA seeks comments on Increasing "Patient Access to At-Home Use Medical Technologies"

FDA's CDRH division is seeking public input on "advancing health equity and facilitating access to medical devices designed to be safe and effective when used outside of traditional clinical settings, for example, medical devices intended for use in the home".

From the website:

In particular, CDRH seeks comment from the public on the following questions:

• How can the FDA support the development of medical technologies, including digital health technologies and diagnostics, for use in non-clinical care settings, such as at home?
• What factors should be considered to effectively institute patient care that includes home-based care?
• What are ways that digital health technologies can (a) foster the conduct of clinical trials remotely and (b) support local or home-based healthcare models?
• How can the FDA facilitate individuals accessing medical technologies in remote locations when they are unable or unwilling to access care in clinical settings?
• What processes and medical procedures, including diagnostics, do you believe would be ideal for transitioning from a  hospital and/or healthcare setting to non-clinical care settings, for example, home use or school/work use?
  • What medical technologies could be ideal to transition to use in non-clinical settings? What aspects of those technologies could potentially benefit from modifications to optimize use in non-clinical settings?
• What design attributes and user needs would facilitate the use of medical technologies, including diagnostic and therapeutic devices, for use in a non-clinical setting, for example home use?
• For digital health technologies, what design attributes could better facilitate their use by diverse patient populations outside of a clinical setting? What other factors are important to consider which may improve use and acceptance of different digital health technologies by diverse patient populations (for example, older adults, non-English speakers, lower literacy)?
• What potential methods and strategies for evidence generation and data analysis could facilitate the regulatory review of medical technologies intended to be used in non-clinical settings, for example home use or school/work use?

Please submit all public comments to the docket (FDA-2023-N-1956), available at Regulations.gov. The public comment period will end on August 30, 2023.

Submit Comments by August 30