Outlook 2024
Annual Industry Ranking And Forecast
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Bolstering regulatory expertise in low- and middle-income countries could help prevent a safety-related disaster that chills enthusiasm for the field, while greater regulatory convergence could help tamp down the proliferation of stem cell clinics, the FDA’s Peter Marks says.
New research is gauging how much savings CMS may see on the prices of the first 10 drugs selected for negotiation.
The head of orphan medicines at the European Medicines Agency has encouraged sponsors to engage with regulators early and take “a little bit longer” to develop their product, rather than cut corners, to boost their chances of marketing authorization success.
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.
Keep up with latest biopharma regulatory and policy developments with Pink Sheet podcasts.
More PodcastsIn a year full of regulatory milestones for the firm, PTC Therapeutics hopes to set some approval precedents – and basically hopes its candidates have an easier time of it than they have had before.
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.
After creating a list of anticipated product-specific guidances to be published for generic sponsors and increasing PSG production, public requests for the agency to write a PSG declined.
The schedule for the second round of negotiations is different from the first cycle, in part because the Inflation Reduction Act allowed for extra time initially as the program launched. The first- and second-year timelines also overlap: The second negotiation cycle will begin before the first cycle prices are implemented.
The new tool provides a way of quantifying the degree of diversity by race/ethnicity, sex and age in clinical trials, but the results will not factor into the US health technology assessment body’s cost effectiveness determinations for new drugs.
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.
EMA charges participants to receive scientific advice through the fledgling program, unlike the US FDA, which may not fit the budgets of some complex generic sponsors. At the same time, sponsors also may simply not be aware the program exists yet.
Acknowledging that it may be a barrier to some extent, EMA’s senior scientific specialist of clinical pharmacology Kevin Blake notes that EU reference medicinal products are a legal requirement for bioequivalence studies and cannot be circumvented.
While EU preparations are underway to introduce strict legal requirements for all AI systems, the UK has doubled down on its flexible, non-regulatory framework. In this second of a two-part article, a lawyer explains the pros and cons of each for pharma and medtech firms.
Flexible thinking and rigorous standards will both be needed to develop psychedelics as drug therapies in order to surmount the many complicating factors, from unique ‘set and setting’ aspects to functional unblinding, speakers at Reagan-Udall Foundation meeting agree.
Brazil has listed 42 international regulatory bodies as equivalent authorities, including US FDA and the EU’s EMA.
EU-level action on market access could include a “solidarity fund” for ultra-rare disease treatments and a new framework for managed entry agreements.
Congratulations to the winners of the 2024 Citeline Awards, held 8 May in Boston.
The FDA commissioner said an H5N1 vaccine can be developed from the same mRNA platform used to create the COVID-19 vaccines, but funds are needed to ensure production can begin quickly if a human outbreak occurs.
As sponsors explore development in autoimmune and infectious diseases, plus a range of hematologic and solid tumors, manufacturing processes can be centralized or decentralized, but Marks says the FDA is ‘neutral’ on that decision.
The FDA would only withdraw a rare disease gene therapy for “pretty clear” reasons, such as when there is minimal benefit with extensive side effects. The one-and-done nature of administration also makes withdrawal easier, CBER Director Peter Marks says.
The Netherlands’ Central Committee on Research Involving Human Subjects is gauging sponsor demand for a new scientific advice service for clinical trials which is for now free of charge.
Psychedelic drug developers are focusing on the US market, and there are no approvals in sight in the EU. The European Medicines Agency wants to change that.
The EMA received more than 1,000 responses to its draft reflection paper on AI in drug development, some of which will be incorporated into the final document, an agency representative told this week’s RAPS Euro Convergence 2024.
After creating a list of anticipated product-specific guidances to be published for generic sponsors and increasing PSG production, public requests for the agency to write a PSG declined.
A public-private partnership or other solution may be needed for the FDA to handle the costs of reviewing the growing number of drug applications with AI and machine learning components, CDER Director Patrizia Cavazzoni said.
Industry has been slow to embrace the idea of pragmatic clinical trials with streamlined data collection, so the Oncology Center of Excellence now is turning to the broader cancer community for research proposals.
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