“I believe in common sense, and I believe in clinical trial diversity both. So I believe if you’re going to make results extrapolated with the general population, you should have results in those populations that you are making recommendations.” – US Food and Drug Administration’s Commissioner nominee Martin Makary on clinical trial diversity. Find out more: Makary Signals Some Interest In US FDA Staff Cuts, Fighting Patent Thickets
“There is nothing on EU stockpiling, nothing on the public medicines infrastructure, nothing to ensure transparency for the public, nothing innovative in terms of financing, nothing that would ensure that public support actually translates into the availability of medicines” – European Parliament’s Tilly Metz on the proposed EU Critical Medicine Act. Find out more: EU’s Critical Medicines Act: A Major Milestone Or Too Weak?
“I think that societies in modern countries like the EU and the US ought to also bear or take responsibility for the financial risk and the health risks that are implicated in getting a drug to market with limited data.” – Bart Leroy of Belgium’s Ghent University Hospital on why reimbursement authorities and payers need to share financial risk with gene therapy developers. Find out more: Pharma Firms Will ‘Walk Away’ From Gene Therapies Unless Payers Share Risk
“Right after the benefit’s revision was explained at the annual budget committee at the Lower House, we have been receiving numerous messages from the patients concerned about the financial challenges this revision may cause,” – the Japan Federation of Cancer Patient Group’s Shinsuke Amano on the prime minister’s decision to freeze plans to revise the country’s High-Cost Healthcare Benefit scheme. Find out more: Japan PM delays Revision of High-Cost Healthcare Benefit After Patient Protests.
“If the Medicaid funds get cut, [states] are going to look for savings everywhere and one place they could look is medications. They could try to dial up additional manufacturer supplemental rebates, or they could try to do more aggressive prior authorization, things like that.” – Former US Centers for Medicare and Medicaid Services official John Coster on how states could increase cost management for prescription drugs to offset the loss if Congress decides to impose major Medicaid cuts. Find out more: Medicaid Cuts Could Prompt States To Tighten Drug Cost Controls, Former CMS Official Says
Stripping patients of their voting rights within the EMA is not just a step backwards – it is an outright dismissal of the very people these decisions impact most.” – EURORDIS-Rare Diseases Europe’s Valentine Bottarelli on fears that plans to include patients on European Medicines Agency committees might be scrapped. Find out more: EU Pharma Reform: Negotiations Could See Patient Voice ‘Discarded,’ Warn Patient Groups
“Forcing out Hilary Perkins as FDA chief council days after her appointment was announced raises serious questions about the integrity of the FDA and whether Martin Makary will be able to protect the agency from political interference,” – US Public Citizen’s Health Research Group’s Robert Steinbrook on Perkin’s resignation. Find out more: Makary US FDA Nomination Advances, Last Minute Hiccup May Offer Clues On Mifepristone
“Life sciences companies are rapidly losing confidence in the UK as an attractive and predictable place to do business,” – Gilead Sciences’ Peter Wickersham on the need for the government to fix the Voluntary Scheme for Branded Medicines, Pricing, Access and Growth. Find out more: Branded Pharma And Generics Industries At Odds Over UK VPAG Framework
“I feel like there’s nothing fundamentally going to change in the support for moving rare disease drug development forward. I feel that that support is very clear from the administration that’s come in.” – US FDA’s Peter Marks on efforts to spur rare disease therapeutic development, and the expectation for continued support under the Trump administration. Find out more: CBER’s Marks Says US FDA’s Rare Disease Work Expected To Win Trump Administration Support
“Although the Clinical Trials Regulation (CTR) in Europe is supposed to harmonize everything, my personal experience is that it really doesn’t. You do end up still with the national requirements. You still end up with documents outside of the mandatory document listing.” – Fondazione Telethon’s Sean Russell on how the CTR could instead see sponsors drawn to nations with faster trial approval timelines. Find out more: EU Clinical Trials Regulation Failing To Harmonize Requirements, But UK has ‘Great Opportunity’
“I’ve already heard from industry concerns…They’re not getting things on time. They feel like there’s a lot of delay.” – Former US FDA acting commissioner Janet Woodcock on concerns that the Trump Administration’s changes at the agency are beginning to negatively impact industry. Find out more: US FDA’s Speed Of Work Under Trump Quietly Raising Industry Concerns
“Our interest is in having this guidance not only be an in depth assessment of all of these potential elements and methods and their applicability to HTA, but to have that guidance be specific enough so that if there is something we need to operationalize, we have a sense of how to do that,”– Dan Ollendorf, of the US Institute for Clinical and Economic Review, on how a transatlantic guidance being developed could eventually inform pricing and bring changes to the way health technology assessments are conducted. Find out more: ‘Hope And Scientific Spillover’- Cross-Atlantic HTA Alliance To Focus On Novel Benefits
