DCAT Value Chain Insights’ “5 MINUTES WITH,” part of the DCAT Member Company Community section, features interviews with business and industry leaders on issues impacting the bio/pharmaceutical manufacturing value chain.

This 5 MINUTES WITH features Gil Roth, President of the Pharma & Biopharma Outsourcing Association (PBOA), a non-profit trade group that advocates for the regulatory, legislative, and general business interests of the CMO/CDMO sector, to discuss the latest on the Generic Drug User Fee Amendments (GDUFA III) and other policy issues of importance to CDMOs/CMOs.

Q. PBOA was a participant in the recent negotiations between the US Food and Drug Administration (FDA) and industry on the third iteration of the Generic Drug User Fee Amendments (GDUFA III), which sets user fees for the industry and performance goals for the FDA as it relates to generic-drug review times. GDUFA III applies for fiscal years 2023 through 2027. Overall, what are some key highlights from GDUFA III? What is new in GDUFA III?

Roth (PBOA).The Generic Drug User Fee Amendments (GDUFA) program is still a work in progress, just finishing its second five-year authorization in 2022. For GDUFA III, industry and FDA came together to refine aspects of the program that could help speed up review and approval of abbreviated new drug applications (ANDAs). The next iteration, if authorized by Congress and signed into law, will include the following: more communications and meetings between applicants and FDA; new timelines for drug-master-file assessments; commitments to (finally) tackle Suitability Petitions; expansion of Controlled Correspondence to include post-approval changes; increased commitments for Product-Specific Guidances, especially for complex drugs that require more work on the part of both FDA and ANDA filers; post-Warning Letter (WL) meetings; and a reinspection pathway to clear sites that are Official Action Indicated (OAI) and have received a WL. The program will also shift—as requirements under the Prescription Drug User Fee Act (the user-fee program for innovator drugs) already has—from a “front-load cost” model to one that adjusts its full-time equivalent (FTE) needs from year to year, utilizing the FDA’s Resource Capacity Planning and Capacity Planning Adjuster methodology.

Q. From a CDMO/CMO perspective, what elements of GDUFA III are of particular importance?

Roth (PBOA): One of the key aspects from a CDMO perspective was simply trying to attain cost control for facility fees, given the new funding model that GDUFA III will adopt. We think we’ve achieved that by further reducing the amount CMO finished-dosage-form (FDF) facilities pay, compared to non-CMO FDF facilities. In GDUFA II, that was one-third of a full facility fee, and under GDUFA III, that will go to 24% of a full fee. This should shield CMO sites from a significant cost increase, even if the overall program costs go up as we project.

The reinspection process for OAI + WL facilities may be of some benefit for CDMOs, although of course, those timelines also apply for in-house ANDA facilities, which account for a larger portion of that pool than CDMO sites.

And overall, the philosophy is that if high-quality ANDA submissions get reviewed and approved more swiftly—especially in the complex ANDA territory—that will be a benefit for the CDMOs who help manufacture them. Of course, market realities sometimes interfere with that notion.

Q.  Process-wise, what are the next steps for GDUFA III to take effect, and when would it take effect?

After industry and FDA wrapped up negotiations and ratified the agreement and the Commitment Letter that outlines FDA’s deliverables, we—industry and FDA—took part in a public meeting on GDUFA III, FDA received comments and docket submissions, and we all held several briefings with Congressional committees to detail what’s in the agreement. Then FDA, via the US Department of Health and Human Services and the Office of Management and Budget, submitted the “legislative package”—along with those for PDUFA and the Biosimilar User Fee Amendment (BsUFA)— to Congress. From there, Congress will work on an overall FDA Reauthorization bill covering these User Fee Amendments—PDUFA, GDUFA, and BsUFA—and other FDA-related priorities. The bill will go through committees in the House and Senate, amendments and riders will be considered—these generally do not seek to change the User Fee Amendments themselves—and the bill will move from committees to the floor, and if both houses of Congress approve it, any major differences will go through reconciliation, and the legislation will go to the White House for the President’s signature to become law. Five years ago, there were some delays, and it was signed into law in mid-August, but I’m hoping for a shorter timeframe this time. It’ll go into effect the following fiscal year, which begins on October 1, so the earlier it’s enacted, the sooner FDA can officially issue that year’s fees, get to hiring, etc.

Q. For 2022, what other policy issues will PBOA be addressing and/or what policy issues are of interest to CDMOs/CMOs?

Now that the GDUFA heavy lifting is over, we’re focused on a number of pandemic-related issues. Of course, supply-chain concerns are important, especially with various countries’ ideas about “onshoring” and the impact of export bans, and this Administration’s use of the Defense Production Act. We’re interested in working more with the Biomedical Advanced Research and Development Authority (BARDA), an office of the US Department of Health and Human Services, to better understand some of its aspirations for preparing for the next pandemic. I think we have a lot to share with them about how CDMOs work and what “surge capacity” would really mean. We’re also working on Workforce Development legislation to try to boost the bio/pharmaceutical manufacturing base in the US and will continue working with Congress and FDA to explore ways that CDMOs can help accelerate patient access to critical medicines.

As a matter of regulatory policy (as opposed to legislative and executive issues), PBOA members are concerned about the Reporting Amounts draft guidance issued by the FDA in response to the CARES Act of March 2020. Concerns relate to the technical aspects of the reporting requirements and format, the potential for duplicative or contradictory information coming from CDMOs and their license-holder customers, and the incredibly short timelines to submit 2020 and 2021 data, respectively February 15 and May 15 of this year (2022). All these issues, particularly when the guidance hasn’t yet been finalized, make this a potentially disastrous rollout and one that could lead to incorrect assumptions about the supply chain.

We’re also putting together a new working group on Environmental, Social and Governance (ESG) issues to help our members learn from each other and outside experts about how to negotiate that terrain. We held a member panel on it at our recent annual meeting (virtual, of course), and the response was terrific. It’s an area where all our members have questions, no matter how large or small the company is. Between that and other new and existing special interest groups, such as our Quality Technical Group, Supply Chain Task Force, Serialization Working Group, and others, we’ve managed to create forums for CDMOs to work together, learn from each other, and build consensus on key topics.

Gil Roth is the Founder and President of the Pharma & Biopharma Outsourcing Association (PBOA), a non-profit trade group that advocates for the regulatory, legislative, and general business interests of the CMO/CDMO sector. PBOA has grown from 15 members at its inception in 2014 to more than 40 today. He previously served as the Founding Editor of Contract Pharma magazine. He can be reached at gil.roth@pharma-bio.org.