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Mismatch Between Growing Vaccine Pipeline and CDC’s ACIP Capacity

The Centers for Disease Control and Prevention’s ACIP is not able to take on all vaccine-like preventative medicines and still has to grapple with how to handle therapeutic vaccines. 

Advisory Committees Vaccines

Keeping Track: Two Breakthrough Oncologics, Another Antibiotic Clear US FDA Along With Pfizer’s Hemophilia B Gene Therapy

The US FDA approved Day One’s pediatric brain cancer drug Ojemda, ImmunityBio’s bladder cancer immunotherapy Anktiva, an uncomplicated UTI claim for Utility Therapeutics’ Pivya, which has a long history in Europe, and Pfizer’s hemophilia B gene therapy Beqvez.

US FDA Performance Tracker Approvals

Priority Review Vouchers: Tropical Disease Candidates Wait Years For FDA Action On Qualifying List

There are pending requests to add 13 candidates to the tropical diseases that qualify for a priority review voucher, according to a Pink Sheet analysis, as FDA Commissioner Robert Califf faces congressional pressure on the years since the list's last update.

Review Pathway Infectious Diseases

Pink Sheet Podcast: US FDA Biosimilar Strategy, Gene Therapy Accelerated Approvals, FDA v. Partisan Politics

Pink Sheet reporter and editors discuss the impact of FDA biosimilar promotion guidance on the future of the interchangeability designation, upcoming guidance on accelerated approval for gene therapies, and partisan attacks on the agency from Capitol Hill.

Biosimilars Gene Therapy

Cancer Trials: FDA Wants Lower Performance Status Eligibility But Primary Analysis Exclusion OK

New draft guidance pushes industry to broaden clinical trial eligibility criteria while offering some protection from fears it could hurt efficacy results.

Clinical Trials Guidance Documents

US FDA's Califf Defends Agency Work On Marijuana Rescheduling, Animal Testing

House appropriations subcommittee chair Andy Harris, R-MD, says the agency ignored several important factors in recommending that marijuana be reclassified as a Schedule III drug; Califf tells ranking member Sanford Bishop, D-GA, that although work is progressing on animal testing alternatives, 'we're a long way right now' from eliminating animal studies before first-in-human trials.

Cannabidiol CBD Research & Development

Overall Survival Is Not A Required Primary Endpoint In All Cancer Trials, US FDA Says

Seeking to clear up a ‘misconception,’ Oncology Center of Excellence officials say earlier endpoints, such as progression-free survival and overall response, continue to be useful in getting therapies to market quicker, but sponsors still should plan to systematically collect OS data to ensure there is no detriment to survival.

Clinical Trials Drug Approval Standards

Pharma Can Pursue Claims Against Providers For 340B Duplicate Discounts In Medicaid Managed Care, HRSA Says

The statement, which is part of a final rule on the 340B administrative dispute resolution process, could facilitate manufacturer efforts to seek repayments from hospitals in such cases.

Reimbursement Pricing Strategies

The Aspirin Test For AI?

US FDA Commissioner Robert Califf tells Congress that regulators don’t have to know how artificial intelligence works in medicine, but must make very sure it actually does work.

Artificial Intelligence Politics

Mary Beth Clarke, CDER Exec Programs Office Director And Generic User Fee Shepherd, Will Retire

Clarke helped launch GDUFA I and negotiate GDUFA II, chaired the CDER Executive Committee and oversaw many other programs in the US FDA’s drugs center.

FDA Leadership

Rx Advertising: Interchangeability Doesn’t Mean Clinical Superiority To Other Biosimilars, FDA Says

Updated draft guidance on promotion of biologic reference products, biosimilars and interchangeable biosimilars offers another indication of the US’s push to do away with the interchangeability designation.

Advertising, Marketing & Sales Biosimilars

Generic Or Innovator? Sandoz Sues CMS Over Potential Change In Rebate Classification

Sandoz argued against paying higher rebates for two of its drugs via the Centers for Medicare and Medicaid Services’ Medicaid drug rebate program in a US court six years after the suggestion was first made.

Generic Drugs Legal Issues

US FDA Gene Therapy Accelerated Approval Guidance Will Describe ‘Buckets’ Of Use Scenarios

Forthcoming guidance is expected to describe areas of “low-hanging fruit” and those that are more challenging for use of the expedited pathway, Center for Biologics Evaluation and Research Director Peter Marks said.

Gene Therapy Rare Diseases

FTC Early Findings In PBM Investigation Coming By Mid-Summer, Chair Khan Says

The agency is also poised to announce another development in its scrutiny of the drug patents listed in the FDA’s Orange Book.

Reimbursement Patents

Gene Therapy: Pediatric Development Could Start Sooner Than Sponsors Think – FDA OTP Director

Nicole Verdun said children could participate in gene therapy clinical trials earlier if the necessary controls are in place.

Rare Diseases Gene Therapy

Puberty Blockers: FDA's Califf Asked If REMS, Boxed Warning Against Off-Label Use In The Works

FDA Commissioner Robert Califf tells House appropriations subcommittee chair the agency will 'consider any information that may be available' on off-label use of puberty blockers for gender dysphoria before making a regulatory decision.

Hormonal Drug Safety
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