US Health and Human Service Department questions recent federal court decision striking down the government’s policy of allowing plans to broadly exclude manufacturer copay assistance from counting toward patient deductibles or out-of-pocket maximums.
The European Medicines Agency has been on a steep learning curve with its qualification of a digitally-measured outcome for use in Duchenne muscular dystrophy trials, smoothing the way for other developers entering the digital biomarker space.
When Janet Woodcock retires early next year, Namandjé Bumpus will take over as No. 2 in command at the FDA. Since joining the agency in 2022 from Johns Hopkins, her profolio has included health disparities, cosmetics, and drug review disputes.
Rare Disease Policy Changes
The National Academy of Medicine is launching a Congressionally mandated study of the US FDA’s regulatory approach to rare disease treatments. The first question is whether there is anything more that FDA can do.
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AstraZeneca’s Truqap and Evive’s Ryzneuta bring FDA center for drugs’ 2023 novel agent total to 51, while biologics center already sits on a record-breaking 14 novel approvals.
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
While a decline in complete response letters in FY 2023 is good, the continued drop in ANDA submissions could be a bad sign for the industry’s long-term health, a former FDA official worries.
In 17 suits against FDA in the last two years, firm seeks to block generics of its sleep-wake disorder drug and gain a jet lag indication. CEO Mihael Polymeropoulos tells the Pink Sheet that FDA has retaliated against Vanda for its first suit challenging a partial clinical hold on another drug.
Pink Sheet infographic breaks down the breakthrough therapy designations made by the US FDA, with statistics on what’s been approved, rescinded, and everything in between.
The vast majority of meeting requests eligible for the in-person/virtual format were not requested as such, according to Center for Drug Evaluation and Research data.
European Union reform proposals could reduce ‘clock stop’ time in drug reviews, which a new study identified as the primary driver of longer review times at the EMA compared with the US FDA.
Regulators from Canada, the UK and Australia discuss their experiences of working together under both the ACCESS Consortium, an international regulatory worksharing coalition, and Project Orbis, a parallel review process designed to speed up access to oncology medicines.
The UK joined Project Orbis in 2021 with the aim of speeding up the regulatory process for promising cancer drugs, but fewer than half of the drugs licensed under this pathway are available on the NHS in England.
The move expands access to the Pharmacopoeial Discussion Group’s quality standards to the Indian pharmaceutical industry.
MSD and Kyowa Kirin have separately criticized England’s HTA body, NICE, for assessing their respective rare disease medicines Welireg and Crysvita using its standard appraisal pathway. Both medicines were provisionally rejected for reimbursement.
The developers of survodutide and elsunersen will be able to benefit from the early and enhanced support that the European Medicines Agency offers through its priority medicines scheme.
Overall survival is the only viable endpoint for pragmatic trials in oncology, the US FDA and a panel of stakeholders agree, although other novel endpoints could be validated over time. In the meantime, FDA says, sponsors should focus on other aspects of trials that can be streamlined.
A policy report by a major South Korean pharma industry association puts forward suggestions on what it will take for a new pan-government control tower to function properly.
A single-arm trial using a biomarker and animal model could be enough for an accelerated approval, especially in a pediatric rare disease where randomization and placebo controls may not be feasible, the CBER director said.
On the back of a successful pilot of early consultations with patients, the European Medicines Agency is extending the initiative so that patient and health care professional groups can give valuable input in areas such as unmet needs, clinical trial endpoints and expectations for future treatments.
The upcoming retirement of Janet Woodcock will mean the loss of one of the US FDA’s most experienced and influential drug regulators. However, the drug center still has plenty of long-tenured staff peppered across all of its key functions.
Aldeyra’s bid to rely on a single Phase III trial after an initial one failed is rejected by FDA, but firm already has a special protocol assessment under review for a trial that could provide the symptom data it says agency needs to approve the RASP modulator in dry eye.
US FDA has received a total of 19 reports of T-cell malignancy involving BCMA- or CD19-directed autologous CAR-T cell immunotherapies and is evaluating the need for regulatory action. Focus on lentiviral and retroviral vectors could extend to safety of other types of gene therapies.
Legislation to delink PBM payments from drug prices advances in House Energy and Commerce health subcommittee, putting a key pharma priority closer to the finish line. Whether patients will see any benefit remains a worry.
Since October, reported cases of infectious respiratory diseases have been rising in China, prompting an information request from the WHO. However, no new types of pathogen or unusual clinical symptoms have been detected so far.
People have challenged patent listings more than 60 times in the past six years but it is up to new drug application holders to decide whether to revise or delist their submissions.
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