BIO Notebook: Policy Uncertainty Constraining Dealmaking

 

Highlights from Day Four of the BIO International Convention include policy concerns helping constrain dealmaking, Novartis discussing its approach to partnering, and Generate looking for funding to move into Phase III.

Woodcock: Do Not Do The Wrong Study, Even If Against US FDA Advice

 

The former CDER director said she tells sponsors not to conduct an FDA-recommended study design or randomized trial if it will not work.

AI Will Bolster, Not Squelch, Patient Voice In Clinical Research, US FDA Says

 
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Artificial intelligence tools used for clinical research should be developed with input from patients and focus on their needs and abilities, FDA experts said.

UK Looks To Auto-Match NHS Patients With Clinical Trials To Boost Recruitment

 
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The UK government is making it easier for millions of people to participate in clinical trials and is boosting transparency around how studies are delivered across the National Health Service.


UK’s Decentralized Manufacturing Rules Designed To Allow For ‘Future Developments’

 

The UK drug regulator acknowledges that technology will “move on” and, as such, has designed its decentralized manufacturing regulation to be as “enduring” as possible. Experts from the MHRA explain what products are covered by the new framework and how it has been future-proofed.

Global Guideline Signals ‘Paradigm Shift’ For Pregnant And Breastfeeding Women, Says EMA

 

The European Medicines Agency expects the International Council for Harmonization’s new guideline will help address the critical lack of data on the use of medicinal products in pregnant and breastfeeding populations.

Profit-Sharing AI Model Proposed To Boost Korean Drug R&D

 
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A differentiated profit-sharing model to accelerate the use of AI in drug development using clinical and patient data is being proposed in South Korea, but the idea faces multiple practical challenges

US FDA’s Top Cell and Gene Therapy Regulators Forced Out

 

Office of Therapeutic Products Director Nicole Verdun and her deputy Rachael Anatol were escorted out of FDA headquarters on June 18. Disagreements over CAR-T regulation and Capricor’s DMD treatment may be to blame.


First Among Equals: US FDA CBER Director Prasad Gains Agency-Wide Authority

 

As Chief Medical and Scientific Officer, Vinay Prasad will advise the FDA Commissioner on cross-cutting issues, provide strategic leadership and be a public face of the agency.

Not Over Yet: Eisai And Lilly To Appeal England’s ‘Flawed’ Refusal To Fund Leqembi And Kisunla

 

Eisai and Lilly explain why they disagree with today’s conclusion by the health technology assessment institute, NICE, that the benefits of their respective drugs for Alzheimer’s disease are too small to justify the additional cost to the National Health Service.

BIO Notebook: MFN Pricing, Obesity R&D, US FDA’s Rare Disease Hub And Reaction To Review Program

Highlights from Day Three of the BIO International Convention include the realities of MFN pricing, AstraZeneca's R&D plans for obesity, the need for resources for the FDA's rare disease hub and reactions to the Commissioner's National Priority Review Voucher program.

Brazil Could Look To Germany, UK And Australia For ATMP Drug Pricing Rules

 

There is a place for using external reference pricing to set prices of advanced therapies, but any such mechanism must take into account the specificities of the Brazilian market and health system.


Lerodalcibep And First Generic Palbociclib Among 10 New EMA Filings

 

Lerodalcibep and palbociclib are among the latest new drugs that the European Medicines Agency has started to review for potential EU marketing authorization.

Welireg Among Japan Recommendations; Economic Policy Raises Hopes Of Higher Prices

 
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Country recommends eights new drugs for approval, including six for rare diseases, and also announces macroeconomic policy the pharma industry says would enable some reimbursement prices to be raised.

BIO Notebook: Policy Problems, ACIP’s Future, BI Is Popular, Makary On FDA-Industry Ties

 

Highlights from Day Two of the BIO International Convention include BIO officials raising concerns about Trump Administration policies, the future of ACIP, an interview with BI's head of global business development, and FDA Commissioner Martin Makary's view of the FDA-industry relationship.

National Priority Voucher Gives US FDA Commissioner Tool For Radical Regulatory Surgery

 

US FDA Commissioner Martin Makary will establish "national priorities" used to select sponsors for the pilot of “tumor board-style” drug reviews that would start before Phase III is completed.


US FDA’s CoGenT Pilot At Risk? Project Orbis Success May Not Save Cell/Gene Therapy Initiative

 

Despite following the path of the popular Project Orbis, the cell and gene therapy international collaborative review pilot is being reconsidered by the FDA's new management.

HTA Expert Warns Of Escalating Measures If Pharma Fails To Tame Prices

 

Too many “uninformative” drug trials fail to justify the excessively high prices of many medicines, while there is too much evidentiary uncertainty in European pricing and reimbursement systems, warned Anja Schiel from Norway’s NOMA.

Kisunla Back In The Spotlight As EMA Reconsiders Alzheimer’s Drug Rejection

 

It appears that the European Medicines Agency is making progress on its re-examination of Eli Lilly’s Alzheimer’s disease drug Kisunla, following an initial rejection in March based on safety concerns.

Canada Joins Global Push To Drop Costly Clinical Trials For Biosimilars

 

Health Canada’s proposal to no longer require biosimilar manufacturers to prove the safety and efficacy of their product through Phase III clinical trials marks a pivotal change in Canada’s regulatory approach.