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US FDA’s New Voucher Fee Reveals Standard, Priority Review Cost Spike
The number of standard and priority reviews also decreased significantly in FY 2023 compared to the previous year, which caused the fee for redeeming a voucher to rise.
Prior Authorization Reform: 2025 Could Be A Year Of Changes
Prior authorization reforms could emerge as an important under-the-radar area of drug policy next year. An upcoming Medicare proposed rule, the extraordinary uptake of weight loss therapies, and the launch of the overhauled Part D benefit design will all drive attention to the issue.
340B Rebate Dispute: HRSA Threat To Bar J&J From Medicare Part B, Medicaid ‘Unprecedented’
HRSA threatens nuclear option and Johnson & Johnson suspends its 340B rebate model. But the company also ‘reserves all of its legal rights with respect to this matter.’
The View From TOPRA
EMA’s AI Principles Intended To Be ‘Flexible & Long Lasting’
There is “a lot of flexibility” in the European Medicines Agency’s reflection paper on the use of artificial intelligence during drug development, which is principles-driven rather than setting rigid recommendations, says the agency’s Florian Lasch.
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Performance Trackers
US FDA’s June Approvals Forecast: Nearly 30 Goal Dates Suggest Hot Start To Summer
June goal dates include RSV and pneumococcal vaccines, two COPD therapies, Rocket and Sarepta gene therapies, and lots of oncology.
Global Pharma Guidance Tracker – August 2024
Stay up to date on regulatory guidelines from around the world with the Pink Sheet's Guidance Tracker. The complete Global Pharma Guidance Tracker, with sortable and searchable listings going back to 2014, is available online.
US FDA Performance Tracker
European Performance Tracker
Multimedia
Growth Spurt: Pediatric Labeling Is On The Rise, Taxing US FDA Safety Monitoring
The US FDA Pediatric Advisory Committee uses web-posted reviews of “low safety risk” products to keep up with postmarketing monitoring requirements amid rising interest in pediatric development.
US FDA's Slim October User Fee Calendar Brings Some Controversy
Advisory committee concerns cast clouds over Iterum’s oral antibiotic, Intercept’s Ocaliva, and perioperative immuno-oncology regimens, while CSL and Pfizer aim to take their hematology franchises in new directions.
BIOSECURE Act: Potential Supply Chain Hits And Pharma’s Path Ahead
With BIOSECURE's legislative progress on pause until after the election, a Pink Sheet infographic looks back on the Capitol Hill progress to date and looks ahead to the potential impact if it is enacted, using Evaluate Pharma data to highlight the likely holes in pharma’s supply chain.
Inflation Continues To Rear Head At US FDA User Fees
Inflation accounted for a larger portion of user fee revenue target increases for fiscal year 2025, compared to previous years, according to a Pink Sheet analysis.
Regional Comparisons
How The UK, EU, Canada, Australia And Japan Are Tackling Diversity In Clinical Trials
As efforts to improve diversity in clinical trials gain momentum globally, the Pink Sheet asked regulators in the UK, the EU, Canada, Australia and Japan about their efforts to support representative enrolment.
EU Revokes Intercept/Advanz’s Ocaliva Approval As Moment Of Reckoning Nears in US
The EU marketing authorization for the primary biliary cholangitis treatment has now been revoked. Meanwhile the drug's approval is in jeopardy in the US, where an advisory committee will opine on whether the accelerated approval drug has confirmed clinical benefit.
England Lags Behind Eight European Countries On Innovative Drug Availability
In this third article of a series on new drug reimbursement recommendations by the HTA body NICE, the Pink Sheet finds that fewer innovative medicines are reimbursed in England than in eight other European nations.
Decoding Regulatory Agency Decisions: The Case of Amylyx’s Relyvrio/Albrioza In The US And EU
Relyvrio/Albrioza, Amylyx’s ill-fated treatment for amyotrophic lateral sclerosis, was approved for marketing by the US Food and Drug Administration but rejected by the European Medicines Agency. The Pink Sheet explores how the two agencies applied the available regulatory flexibilities and the impact of other factors, such as regulatory precedence and patient influence, on decision-making.
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Recent And Upcoming FDA Advisory Committee Meetings
Recent and upcoming US FDA advisory committee meetings and a summary of the topics covered.
England Plans New Model For Use Of Lilly’s Mounjaro In Weight Loss
NHS England says it is proposing a “realistic but challenging uptake trajectory” that will make “this clinically and cost-effective drug” available to as many patients as possible without overwhelming health care providers.
EMA’s AI Principles Intended To Be ‘Flexible & Long Lasting’
There is “a lot of flexibility” in the European Medicines Agency’s reflection paper on the use of artificial intelligence during drug development, which is principles-driven rather than setting rigid recommendations, says the agency’s Florian Lasch.
US FDA Eager To INTERACT With Start-Ups, Not Check Boxes
A US FDA webinar for start-up sponsors showcased staff who sound enthusiastic about helping drug developers advance their projects, while also emphasizing the importance of early and continual engagement with the agency.
Adcomm Reform: Getting Needed Information More Important Than Vote Or Discussion
US FDA Office of New Drugs Director Peter Stein says review divisions have made the case that a discussion-only meeting would solicit the necessary input.
US Medicare Cost For Part D Redesign Could Far Exceed Earlier Projections, CBO Says
Instead of $5bn in 2025, the redesign could cost the federal government two to four times that amount, the Congressional Budget Office told Republican lawmakers. And the Part D premium stabilization demonstration will cost an additional $7bn, the office projected.
EU HTA Regulation: ‘We Don’t Want An Empty Dossier,’ Warns Coordination Group
Manufacturers that do not answer all questions they are asked during joint clinical assessments under the upcoming EU HTA Regulation could have their applications deemed incomplete, says a co-chair of the JCA subgroup.
Gene Therapy Hemgenix Gains Ground In Europe With Spanish Reimbursement Deal
Hemgenix has now secured reimbursement in several European markets, with more talks ongoing. While the path to reimbursement has not always been easy, innovative access deals have helped to smooth the way.
UK Expedites Cost Negotiations For Companies Conducting ATMP & Early Phase Trials
Companies dealing with commercial costings or contracts with the National Health Service for early phase clinical trials or trials of advanced therapy medicinal products should soon be able to conclude their negotiations more quickly.
Patient/CMS Roundtables For Medicare-Negotiated Drugs Will Be Interactive, But Not Public Next Time
The final Medicare price negotiation guidance for the next cycle includes improvements from last year’s patient listening sessions.
US FDA And AI: Who Is In Charge?
The FDA is developing several structures and a broad group of experts across disciplines to help craft artificial intelligence policy. But the proliferation of AI-related initiatives raises the question of who, ultimately, will make decisions about when novel applications of AI are acceptable.
Califf: Not Wise To Create Problems Related to Chinese Pharma Supply Chain Dependence
The US FDA Commissioner pushed for rebalancing the US’s pharmaceutical supply chains while also stressing that US-China commerce has a role that would be risky to compromise.
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