Final Summary of the PPTA/FDA Liaison Meeting on September 14, 2022

The following is a final summary of the PPTA-FDA Liaison Meeting held virtually on September 14, 2022.

Welcome, Introductions & Meeting Guidelines

PPTA and Head of Global Plasma welcomed all participants and appreciated FDA’s participation in today’s meeting. PPTA’s Head of Global Regulatory Policy opened the meeting with introductory remarks. PPTA’s External Legal Counsel advised the participants on the meeting guidelines and the antitrust statement.

Brief Industry Update

PPTA provided FDA with a brief industry update. This included PPTA’s reorganization, status of 2022 and 2023 donor health studies, federal updates, including the CARES amendment to add plasma to the HHS awareness campaign, the successful exclusion of plasma-derived medicines from the “Inflation Reduction Act” and educational efforts in Congress. State updates were provided, including state harmonization with federal laws, communication and social media activities, challenges at the north and south US borders and continued difficulties of the industry with the FDA’s interpretation of 21 CFR 630.30.

PPTA also expressed the industry’s appreciation for the release of updated guidances issued by the FDA during the COVID-19 pandemic and pointed to difficulties for industry to implement FDA’s alternative provisions in other international jurisdictions, hence would welcome it, if the FDA could advocate for its science-based policies with international regulators.

PPTA also stressed to the FDA that they would welcome a closer collaboration on emerging infectious disease between the FDA and PPTA’s Global Pathogen Safety Working Group, especially given the impact of COVID-19 on plasma collections and the ongoing Monkeypox virus (MPXV) pandemic. Following the outbreak of the MPXV the PPTA pathogen safety experts issued a statement on the safety of plasma-derived therapies within a short matter of time. PPTA asked what FDA’s expectations were for the industry to continue collaboration under these circumstances, and what the FDA’s strategy will be in ending the Public Health Emergency (PHE). PPTA stressed that it is important that PPTA/Industry be notified sufficiently in advance to allow preparations, especially in relation to the 45/60-day inventory hold. FDA responded that it is no clear when the PHE will end as modelers expect a winter wave if people do not get vaccines and boosters, and the COVID-19 pandemic is not over for the FDA as the impacts continue to revibrate. FDA recognizes that industry will need extra time after the pandemic and will try to “overcommunicate” decisions by giving as much notice to industry as possible.

Some of FDA’s activities have re-commenced (such as in-person inspections) post-pandemic.

With regard to CFR 630.30 the FDA remarked that it has received PPTA’s comments and it will consider these as part of the guidance review. With regard to updated guidances for industry, FDA reported that a revision of the HIV donor deferral/ MSM guidance is expected following the results of the ADVANCE study. Industry will be given notice if guidances made during the pandemic will extend.

FDA responded that they collaborate as much as possible with international organizations, but do not know what a closer collaboration would entail for PPTA.

Finally, PPTA enquired  if there are opportunities for additional dialogue with FDA this year ('Annual Executive Liaison Meeting', as per CBER SOP), as the industry would highly value the opportunity to be able to meet with the FDA twice per year, with one meeting focused on general updates and another, more issues-driven meeting, such as CFR 630.30, CLIA – Total Protein Test, etc. The FDA responded that a second meeting would be dependent on resources.

FDA’s role in leading science-based revision and global convergence of ​regulatory policies for plasma

PPTA provided a presentation, requesting that the FDA, a dominant and well-respected regulatory body with decision making processes firmly science-based and engaged in many international regulatory fora, could assume an stronger educational role in the global regulatory circles to facilitate globally harmonized regulatory strategies and showcase its own regulatory strategies that would ultimately benefit the availability of plasma-derived medicinal products.

FDA responded that they are already involved with many international regulatory organizations, including WHO and ISBT and in their understanding this engagement is sufficient. FDA stated that their presence and opinions are valued at these organizations and most impact has been achieved by showing validated scientific documents and studies.

CBER Reorganization and Engagement

PPTA discussed industry’s challenges with FDA’s internal changes within OBRR. From an industry’s point of view this resulted in additional administrative burden due to  process changes such as modes of submission of documents, modes of contact (email submissions instead of direct exchanges with FDA staff, and others. PPTA further asked FDA for clarification on CBER’s organizational structure and provided examples of practical implications on industry.

FDA responded by sharing slides on CBER’s organizational charts; the main rationales for the change was the streamlining of processes within CBER and across the FDA overall for a harmonized process across OBRR, CBER and the entire agency as well as improving the turnaround times. It also remarked that a mailbox address (CBEROBRRBPBinquiries@fda.hhs.gov) is available to all stakeholders to answer any queries. The aim is to answer any queries ent to the mailbox within 3-5 business days. The FDA then explained that the centralized mailbox increased efficiency, resulting in improved turnaround times, and standardized and consistent advice and documentation. The turnaround times are also monitored by the FDA as a measure of performance.

FDA stated for Prior Approval Supplements (PAS) submissions, the new center process goal is 6 months. First, the submission is sent to OBRR in blood/plasma branch via the common email address. Then, there are plans for an on-site inspection (no desk review) and an email stating that FDA has received the PAS application. Next, it gets assigned to a Regulatory Program Manager (RPM). Teleconference may be set up with the RPM. Finally, when the review is complete, a signed letter will be sent. With this streamlined process, the time from PAS submission to approval is approximately 3.7 months. With this, the OBRR has completed 72 desk reviews of PAS in 2022. CBE has a similar process, but the timeline may be different.

FDA also answered the following questions posed by PPTA:

  1. Will CBER/OBRR perform the PAIs or will they be shared with ORA? ​

OBRR PAI will be done by blood and plasma branch.

  1. Will CBER/OBRR still provide firms with advance notification of the PAI dates? Previously firms were notified approximately 30 days in advance of the inspection. ​

Yes, OBRR will notify in 30 days in advance.

  1. Does CBER/OBRR have sufficient resources for timely approval of the supplement? (PAS record review process appeared to be successful with supplement approvals occurring on average within 8 months of center openings)​

Yes, timelines have been met when three months data has been received. The time starts when the complete package is received, not when the center opens.

  1. Approximately how soon after the onsite inspection will firms receive the Establishment Inspection Report (EIR)? ​

After the onsite inspection, the EIR will be issued by ECBQ, preceded by the approval letter.

  1. What steps are taken post-CBER onsite inspection and what is the expected turnaround time to obtain supplement approval?

The steps are the same as the required submission and expect to receive approval within six months. Currently authorized officials (i.e., corporate contacts) are not notified as a cc via email and a process adjustment would need be necessary to include these contacts. FDA suggested contacting OCBQ to have this process changed.

PPTA asked FDA if these processes could be sent in writing to share with the membership as there seems to be a need for clarification. FDA stated that the processes have been sent to individual member companies following individual requests and have been communicated in the 2020 and 2021 PPTA-FDA Liaison Meetings. PPTA responded that it would address this internally and put together the combined information to be shared amongst members.

Total Protein Test: Assessment of Risk/Simplicity

PPTA provided a presentation asking FDA’s direction and assistance to facilitate, along with other government agencies, to add the total protein (TP) test, when using a digital refractometer as part of pre-donation screening for Source Plasma, to the list of waived tests within 42 CFR 493.15 or Guidance for Industry. PPTA presented it’s rationale that due to the simplicity and accuracy of performing the test. 42 CFR 493.15(b) allows for tests to be waived under CLIA if it poses no reasonable risk of harm to the patient (donor in our case) if the test is performed incorrectly or if the methodology is simple and it renders the likelihood of erroneous results negligible.

FDA responded that CLIA waivers are handled by Center for Devices and Radiological Health (CDRH) and device manufacturers should contact CDRH as CBER does not issue guidance on device waivers.

Closing Remarks

PPTA thanked the FDA for their time and willingness to continue to have this liaison meeting and thankful for their international leadership. FDA stated they were appreciative of the engagement.

 

Participants

PPTA Members

Roger Brinser, Head of Regulatory Affairs, Takeda/BioLife Plasma Services
Nelli Cherny, Director of Regulatory Affairs, KedPlasma
Chaaya Ganorkar, Executive Director, US Regulatory Affairs, BPL
Barbara Glantschnig Global Head of Quality, Plasma Operating Unit, Takeda Pharmaceuticals
Kaitlin Kestenberg, Vice President, Compliance & Project Management, ADMA Biologics
James Knowles, Senior Director, Global Regulatory Affairs, Grifols Plasma
Thomas R. Kreil, Associate Professor of Virology, Global Pathogen Safety, Takeda
Michael Messick, Regulatory Authorized Official, ImmunoTek Bio Centers
Eva Quinley, Senior Director, Regulatory Affairs and Laboratory Operations, CSL Plasma
Cynthia Tolman, Senior Vice President, ADMA BioCenters Georgia, Inc.
Gina Truscott, Director and Team Lead, Global Regulatory Affairs CMC Plasma-Derived Therapies, Takeda Pharmaceuticals

U.S. Food and Drug Administration (FDA)

Office of the Center Director (OD)

Peter Marks, MD, PhD, Center Director
Celia Witten, MD, PhD, CBER Deputy Director     
Diane Maloney, JD, Associate Director for Policy, OD 
Sherry Lard, PhD, Associate Director for Quality Assurance

Office of Blood Research and Review (OBRR) 

Nicole Verdun, MD, Director
Anne Eder, MD, PhD, Acting Deputy Director 
Jennifer Scharpf, MPH, Associate Director for Policy and Communications 
Orieji Illoh, MD, Director, Division of Blood Components and Devices (DBCD)
Wendy Paul, MD, Deputy Director, DBCD
Rick McBride, Chief, Blood DBCD, Plasma Branch (BPB)
Miriam Montes, Lead, Consumer Safety Officer (CSO)

Office of Tissues and Advanced Therapies (OTAT)

Scott Brubaker, Director, Division of Human Tissues (DHT) 

Office of Compliance and Biologics Quality (OCBQ) 

Melissa Mendoza, Director, OCBQ 
Beth Rogerson, Branch Chief, Program Surveillance Branch      

Office of Communications Outreach and Development (OCOD)  

Pauline Cottrell, CSO  

PPTA Staff

Dominika Misztela, Head of Global Regulatory Policy
Joshua Penrod, Head of Global Plasma
Bill Speir, Lead for U.S. Regulatory Policy
Evelina Kozubovska, Lead for European Regulatory Policy
Michelle Fransen, Lead for Project and Study Management
Michelle Mason, Coordinator, Regulatory & Quality Support
David Evans, External Legal Counsel, Kelley Drye & Warren LLP

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