Outlook 2024
Annual Industry Ranking And Forecast
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Thomas Cueni is stepping down after seven years at the helm of the International Federation of Pharmaceutical Manufacturers and Associations. In an interview with the Pink Sheet ahead of his retirement, he reflects on the proposed pandemic treaty, what still needs to be done to ensure equitable access to medicines, the importance of tackling AMR, moves towards regulatory reliance – and what his post-IFPMA life might look like.
Significant cost sharing reductions under the redesign will enhance uptake of the pulmonary arterial hypertension drug. The trade-off is a 20% mandatory price discount in the catastrophic phase.
Global regulators have yet to develop hard and fast rules for the use of artificial intelligence in drug development. Until they do, pharma companies should be guided by transparency and best practice principles when preparing submissions, a consultant says.
As FDA is readying to go back to in-person advisory committee meetings this fall, the centers have different ideas about how to use their expert panels.
Read all the Pink Sheet stories about FDA advisory committees...
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.
New poll from advocates pushing for courts and the states to stay out of FDA’s scientific decisions indicates majority of US voters support FDA drug safety determinations across party lines, though Republican support is more tentative.
US FDA’s drug center reported a net increase of more than 300 employees in FY 2023, while the biologics center saw an overall increase of more than 30.
Infographic details who gave what to whom as all segments of the US healthcare industry gear up for the presidential election cycle.
As Congress pressures FDA to increase foreign inspections, the agency unveils plans to strengthen its overseas presence, including new offices and more staff in the New Delhi, India, post. Deputy Commissioner Kimberlee Trzeciak notes the moves are resource dependent.
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.
While approaching the third year of its launch, the parallel advice program has seen a handful of applicants, but regulators on both sides of the Atlantic remain optimistic.
US FDA biologics center officials spoke about their efforts to increase collaboration and harmonization with the drugs center, and to internally involve more review disciplines in evaluating biomarker evidence, during a Reagan-Udall Foundation meeting that weighed potential use of accelerated approval for neuronopathic mucopolysaccharidoses disorders.
While more significant reforms are less likely under a second Biden term unless Democrats control both chambers of Congress, former President Trump could look to put his stamp on drug pricing reform by resurrecting one of his former policies involving international reference pricing and Medicare Part B drugs.
GSK, Stealth BioTherapeutics and Blueprint Medicines discuss the difficulties getting participation of physicians, the excessive cost of expanded access, and whether physicians should report research data.
In Germany Novo Nordisk’s obesity drug Wegovy will remain excluded from reimbursement for obesity, unlike Rhythm’s Imcivree.
Leaders from Novartis, Bayer, Sun Pharma, the Indian Pharmaceutical Alliance, Médecins Sans Frontières and Anand and Anand discuss India’s evolving intellectual property rights landscape, including pre-grant oppositions, enforcement action and other realities. Concerns around evergreening, restrictions on patent-eligible subject matter and compulsory licensing were also key talking points at a recent conference in Hyderabad.
The agency's and the pharma industry’s worst fears – that a potential ruling in the mifepristone case might usurp the FDA’s scientific authority and throw the stability of the US drug approval process into question – seem unlikely to materialize as the majority of the Supreme Court justices signal the Alliance for Hippocratic Medicine lacks standing to sue.
Anticipating a ‘tsunami of therapies’ for rare diseases, commissioner says the agency will have to think of creative approaches and employ regulatory flexibility for them. FDA considers copying the oncology center’s Project Facilitate for expanded access to other diseases.
Ensuring inspectors understand the innovative trial designs in use before an inspection will be important to increasing industry confidence in their use, CDER Director Patrizia Cavazzoni said.
Medicare is making clear that Part D ‘can’ cover GLP-1 inhibitors that are approved for cardiovascular indications – but in language that seems to leave plans considerable discretion for now. The formulary review process for 2025 may be a more important milestone for changes.
New guidelines that cast doubt over use of single-arm and non-randomized studies are “discouraging” for rare disease patients and the advanced therapy sector, says ARM, the Alliance for Regenerative Medicines.
The EU’s pharmacovigilance committee says that a review of the available evidence showed that in several cases, blood levels of immunosuppressants increased rapidly to toxic levels in patients who were taking the Pfizer drug.
The company received two complete response letters from the US FDA for the CD20xCD3 bispecific antibody odronextamab due to clinical trial requirements for accelerated approval.
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