Featured Stories
Decentralized Manufacturing Sponsors Must ‘Do Their Homework’ To Impress US & EU Regulators
Decentralized manufacturing methods for cell and gene therapies will be critical for improving patient access to treatments, but sponsors must prepare to demonstrate “comparability” with centralized manufacturing.
Has Competition Reassurance Helped Counter Combination Woes In The UK?
The rate of non-submissions for combination therapies in England has been on a downward trend in the last two years.
EU Preparing ‘Urgently Needed’ Guidance On Therapeutic Radiopharmaceuticals
The EMA’s proposed new guidance will focus on the regulatory and methodological aspects of clinical trial design and marketing authorization for tRPs, according to a concept paper that has just been issued for public consultation.
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 – September 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
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.
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.
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.
Recent Stories
Death of a PBM Reform Bill: Why Big Employers Helped Scuttle California’s SB 966
Big corporations fought to scuttle a California PBM reform bill, despite favoring federal efforts, in part from a desire to be held to one national standard.
PBM Reform Legislation: PhRMA ‘Laser Focused’ On Passage This Year
But the prospects for attaching the legislation to a federal spending package in December will be dependent on the outcome of the upcoming elections.
Teva Inks $450m Deals Over US Kickback Claims, Including Alleged Copay Violations
Teva resolved two US Department of Justice civil suits accusing the firm of violating the US Anti-Kickback Statute and the False Claims Act by allegedly conspiring to fix the price of three generic drugs and paying Medicare patients’ copays for its multiple sclerosis brand product Copaxone.
Stealth’s Elamipretide: Is A New Preapproval Trial Feasible?
Members of the Cardiovascular and Renal Drugs Advisory Committee repeatedly challenged Stealth’s assertion that a new randomized trial in Barth syndrome was not possible, but also said potential functional unblinding and other challenges could compromise a new study.
Animal Testing Alternatives Need Central Office At US FDA, Science Board
Slow adoption of alternatives to animal testing in the current decentralized regulatory framework
Pink Sheet Podcast: Inside Medicare Price Negotiations, New US FDA Vaccine Pathway
Pink Sheet reporter and editors discuss experts’ experience helping drug sponsors negotiate prices with CMS and the FDA’s proposal for a new pathway to update vaccines before a pandemic is declared.
ICH Guidelines: Regulatory Members & Observers Show Strong Compliance
An examination of the global implementation of guidelines from the International Council for Harmonisation by regulators has yielded positive outcomes.
Philippines Seeks Feedback On Plan To Recognize BE Studies Under ASEAN Deal
The Philippines medicines regulator explains how it intends to implement the ASEAN mutual recognition agreement under which member states have committed to accept bioequivalence study reports for generics issued by approved BE centers.
For Generic Manufactures, New Medicare Drug Pricing Demo Is a Dud
The proposed CMS innovation center's $2 drug list model will not address the barriers to newer generics getting on Part D formularies and plans may have little incentive to participate in the demo, an industry group said.
US FDA Advisors Urge Faster Movement, mRNA Consideration, In Updating Pandemic Flu Vaccines
The FDA’s vaccine advisory committee endorsed a new plan to update pandemic influenza vaccines “inter-pandemic,” but encouraged the agency to consider how to make the approach feasible for novel vaccine technologies.
Stealth’s Elamipretide Gets US FDA Panel Nod, But Not A Glowing Endorsement, For Barth Syndrome
The Cardiovascular and Renal Drugs Advisory Committee voted 10-6 that efficacy had been shown for the ultra-rare disease, but even panelists in the majority questioned whether the product satisfied the threshold requirement for an adequate and well-controlled study.
EU Authority Mulls Paying For Late-Stage Research As A Pull Incentive For Neglected Disease Drugs
It is time for a much greater focus on pull incentives to ensure viable market conditions for medicines for neglected diseases that are traditionally regarded as unprofitable.
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