The Advocate

Volume 3, Issue 9

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September 2023

In This Issue:

Catch Up with FDA, CMMI, and ARPA-H On Demand Now 
House, Senate Unlikely to Reach Agreement By Sept. 30
Read New White Paper on Newborn Screening
October Hearing Set to Confirm NIH Director Appointee 
Policy News


ASGCT Advocacy

Catch Up with FDA, CMMI, and ARPA-H On Demand Now 

ASGCT works tirelessly to advocate for policies that support the work of our members and the field. This year’s Policy Summit welcomed nearly 300 in-person and virtual attendees to address the most important policy topics for the cell and gene therapy (CGT) field.

Policy Summit presenters discussed the pipeline of CGT approvals and associated challenges, highlighting the ways in which regulatory and legislative policy can help advance the field. The Summit featured several high-level fireside chats, kicking off with Dr. Nicole Verdun, new director of the Office of Therapeutic Products (OTP), focused on overcoming staffing and workload challenges to better accommodate the incoming CGT product applications. Dr. Verdun also emphasized ways to keep reviewers up to speed on this rapidly evolving field. Dr. Renee Wegrzyn, director of the Advanced Research Projects Agency for Health (ARPA-H), outlined ways her agency is working to fund high-risk, high-reward projects in the CGT space that might not be viable through other avenues. Meanwhile Laura McWright, director of the Seamless Care Models Group at the Center for Medicare & Medicaid Innovation (CMMI), unpacked the demonstration models announced by her Center earlier this year, specifically the model looking at standardizing value-based payments for CGTs. Other sessions tackled issues from patient-centered development, coverage and reimbursement, manufacturing challenges, the importance of public-private partnerships, and more.

All sessions at the Policy Summit were recorded and are available on the virtual platformfor registrants. You can still register and access all the on-demand content through Oct. 23!

House, Senate Unlikely to Reach Agreement By Sept. 30

The state of fiscal year 2024 (FY24) appropriations in the House and Senate is currently uncertain. To date, the Senate Appropriations Committee has successfully approved all 12 funding bills. The House Appropriations Committee has passed 10 out of the 12 bills. These funding packages will need to clear both the House and Senate, respectively, before moving towards final reconciliation and passage into law.

It is looking increasing likely that we will be facing a government shutdown come Oct. 1. A government shutdown would have severe consequences for researchers, disrupting critical funding, research initiatives, and regulatory processes. These scientists rely on federal grants and resources from agencies like the NIH and FDA to advance their research. A shutdown would also pause new initiatives, such as the CMMI demonstration models for CGT, the Cancer Moonshot Initiative, and ARPA-H.

With only a few days left before the Sept. 30 deadline, Congress may pass a short-term continuing resolution (CR) to fund the government while appropriations negotiations continue. The Senate and House must both pass the same CR before it can be signed into law by the President.

Read New White Paper on Newborn Screening

A new white paper, Pioneering the New Era of Newborn Screening: Collaborative Insights and Recommendations for Modernizing NBS Systems, has recently been released to help chart the future of the newborn screening (NBS) system in the United States. While NBS is widely considered one of the most successful public health programs in the U.S., the system also faces significant challenges. Arising from a series of newborn screening modernization roundtables in 2022, the white paper identifies a series of policy priorities that would collectively help drive positive change in the NBS system. ASGCT was a proud member of the white paper working group, which was led by the EveryLife Foundation.

October Hearing Set to Confirm NIH Director Appointee

In May, President Biden nominated Dr. Monica Bertagnolli, the current director of the National Cancer Institute (NCI), as the new director of the National Institutes of Health (NIH). Dr. Bertagnolli, a renowned surgical oncologist and cancer researcher, has made significant contributions to cancer prevention and treatment. During her tenure as NCI director, she advanced initiatives like the Cancer Moonshot, promoted cancer prevention, and expanded access to clinical trials.

Senator Bernie Sanders, Chairman of the Senate Health, Education, Labor and Pensions Committee in charge of confirming appointees, had initially opposed Dr. Bertagnolli's nomination due to concerns about drug pricing. After reassurances from the White House regarding efforts to lower prescription drug costs, Sanders plans to hold a confirmation hearing in October. The NIH director position has been vacant since December 2021 when Dr. Francis Collins left, with Dr. Lawrence Tabak serving as acting director. Sanders intends to discuss Dr. Bertagnolli's plans to address prescription drug prices during the confirmation process, citing recent actions by the Department of Health and Human Services regarding drug contracts as a positive step.


Register for CBER Workshop on Advocacy for Policy Development

The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) is hosting a public virtual webinar on Thursday, October 5from 11:00 a.m. – 12:15 p.m. ET. The webinar, Warrior Families: Advancing Regenerative Medicine Through Science, will feature a panel of families sharing their experiences with advocacy and contributing to therapeutic product development for loved ones living with rare diseases. Registration is now open.  

Public Workshop Will Focus on Enhancing Clinical Trial Diversity

Nov. 29 and 30, 10:00 a.m. – 2:00 p.m. ET both days

FDA, in collaboration with the Clinical Trials Transformation Initiative (CTTI), is convening a public workshop to solicit input on increasing the enrollment of historically underrepresented populations in clinical studies and encouraging clinical study participation that reflects the prevalence of the disease or condition among demographic subgroups. The meeting fulfills a requirement under section 3603 of the Food and Drug Omnibus Reform Act of 2022 (FDORA).

Attend the 2023 NORD Breakthrough Summit in Washington, D.C.

Register now for the National Organization for Rare Disorders (NORD) Rare Disease and Orphan Products Breakthrough Summit being held in person in Washington, D.C. Oct. 16-17. This event brings together rare disease experts, patient advocates, and more from around the world to tackle the most pressing issues facing the rare disease community. Learn more on NORD's website.

Discuss Challenges and Opportunities in CGT Manufacturing

The National Academies will be hosting a public workshop Oct. 17 to explore challenges and opportunities in manufacturing regenerative medicine products. Presentations and discussions will consider how new developments in areas such as closed systems, decentralized manufacturing, and automation can affect manufacturing cell- or gene-based therapies. 

Nov. 16-17: Join a Standardization Workshop at USP Headquarters

Join a free hybrid workshop Nov. 16-17 to learn about the current global status of gene therapy, gene modification, and T-cell therapy with a specific emphasis on current standardization and collaborative efforts, as well as the current challenges and gaps. The workshop will be held at the USP headquarters in Rockville, MD, and talks will also be available virtually.

Policy News

  • After receiving feedback from sponsors, FDA has extended the comment period for the Manufacturing Changes and Comparability for Human Cellular and Gene Therapy Products draft guidance to Nov. 12. ASGCT plans to submit comments ahead of the November deadline. 

  • CBER has published a request for information (RFI) from stakeholders regarding scientific challenges and opportunities to advance individualized cellular and gene therapies. FDA intends to use the comments to inform potential planning of future town halls, workshops, or discussion papers which could ultimately facilitate the development of additional regulatory science tools, standards, or guidance. ASGCT plans to respond. 

  • The National Academies of Science, Engineering, and Medicine have published a summary on the Third International Summit on Human Genome Editing: Expanding Capabilities, Participation, and Access. The Summit explored science, equity, access, and public and patient engagement around genome editing.  

  • The Centers for Biologics Evaluation and Research has announced a new grant funding opportunity through FDA’s Office of Orphan Products Development. This opportunity will support clinical studies of orphan products addressing unmet needs of rare diseases. CBER also recently updated their rare disease webpage to include additional opportunities for sponsors.  

  • FDA has released a new guidance document for sponsors titled Formal Meetings Between the FDA and Sponsors or Applicants of PDUFA Products. ASGCT’s CMC and Regulatory Affairs Committees will plan to comment. The docket will be open for public comment through Dec. 21.  

  • FDA has released details on their new CMC readiness pilot aimed at reducing manufacturing bottlenecks for critical drugs. This pilot was included in the PDUFA VII commitments and will begin accepting applications Oct. 3. For sponsors participating in the pilot, FDA will provide product-specific CMC advice during product development, to include two additional CMC-focused Type B meetings, as well as a limited number of additional CMC-focused discussions, based on readiness and defined CMC milestones. 

Want more policy news? Register to watch the 2023 Policy Summit on demand through Oct. 23.

View the agenda + register


ASGCT Policy Summit

Watch on demand through Oct. 23!

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