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Set Alert for Interviews

A selection of in-depth interviews and profiles of policymakers and industry executives.

NGGT Progresses First US Gene Therapy Trials For Rare Conditions

Next Generation Gene Therapeutics is fast-tracking clinical trials in the US for its gene therapy candidates for Bietti's crystalline dystrophy and classic phenylketonuria, supported by China data, the Chinese venture's CEO says in an interview.

China Clinical Trials

‘Antiquated’ Regulations Slowing Adoption of Innovative Clinical Trial Approaches

The current regulations were written when clinical trials involved far fewer entities, and a certain amount of ‘contortion’ is necessary when sponsors are trying to introduce decentralized, community-based and other modern elements, the FDA’s Kevin Bugin says in an interview with the Pink Sheet.

Clinical Trials Diversity & Inclusion

Relyvrio’s Failure Sparks NeuroSense ALS Development Changes

NeuroSense plans to adjust its upcoming clinical trial to avoid the fate of Amylyx’s ALS candidate, which was recently withdrawn from the market due to a lack of efficacy.

Neurology Clinical Trials

‘Do It Or We’re Not Going To Approve Your Drug:’ Industry Reps Ask FDA For Trial Diversity Sticks

FDA leadership has generally seemed apprehensive of going too hard on companies that don’t meet clinical trial diversity goals, but some industry insiders say a stronger FDA is needed to spark real change. Delayed guidance could indicate if the agency will change course.

Clinical Trials Diversity & Inclusion

BIO Seeks ‘Substantial Runway’ To Shift Away From China As Momentum For Manfacturing Bill Grows

Association CEO John Crowley tells the Pink Sheet that a survey which found decoupling from China could be disruptive underscores the need to do so because having the supply chain ‘tied up with a global adversary is profoundly worrying to all of us.’

Manufacturing Lobbying

What’s The Key To Successful R&D With Artificial Intelligence? ‘Bilingual’ Scientists

Before J&J’s former chief data scientist Najat Khan moved to Recursion, she talked to the Pink Sheet about how AI is transforming trials and driving collaboration between industry and regulators.

Artificial Intelligence Clinical Trials

ICER Debuts Clinical Trial Diversity Assessment Framework

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.

Diversity & Inclusion Clinical Trials

EMA Pilots Partnering With Companies To Gain Efficiencies In Preparing Drug Assessment Reports

The European Medicines Agency is looking to increase the review efficiency and consistency of its assessors by experimenting with a US FDA-style approach in which companies can voluntarily submit certain information in advance to facilitate the product’s review.

Europe Regulation

Woodcock Takes On Rare Disease Challenges In Retirement, Keeps FDA, Industry At Arm’s Length

Recently retired US FDA Principal Deputy Commissioner Janet Woodcock will be advising the Haystack Project, with the goal of helping rare disease organizations encourage creativity in drug development programs without jeopardizing regulatory success, Woodcock told the Pink Sheet in an interview.

Rare Diseases Research & Development

EU Needs Market Access Improvements Beyond New HTA Regulation

While the EU Health Technology Assessment Regulation could reduce divergence in reimbursement decisions made across member states, many national-level HTA hurdles and challenges will remain, market access experts from EFPIA say.

Europe EU

‘Unbalanced’ EU HTA Timelines Exacerbated By Rare Disease & Cancer Drugs

As it stands, drugmakers will have just 90 days to prepare their dossiers for EU-wide joint clinical assessments under the new Health Technology Assessment Regulation. Market access experts from EFPIA tell the Pink Sheet that this short deadline could delay patient access to complex medicines, such as innovative cancer drugs.

BioPharmaceutical Health Technology Assessment

Estonian Biobank’s Personalized Medicine Project To Unlock New Insights For Drug R&D

A new center for personalized medicine in Estonia will see PacBio’s long-read whole genome sequencing technology used to unlock new information about how genetics impact patients’ drug responses, experts from the project tell the Pink Sheet.

Medical Device BioPharmaceutical

Breaking Barriers: Japan's PMDA To Open First Global Office, In D.C.

Through a new full-time office in the US capital, Japan's drug regulator aims to provide essential regulatory information to help US bioventures enter the Japanese market at an early stage.

Japan United States

'Don't Compromise The Pharma Innovation System,' Says Outgoing IFPMA Head Thomas Cueni

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. 

Intellectual Property Innovation

When Using Artificial Intelligence In Pharma R&D, Start With Identifying Problem To Solve

VeriSIM uses generative AI for questions such as changing a drug molecule’s chemistry and machine learning to better predict potential biological implications, says CEO Jo Varshney.

Artificial Intelligence Research & Development

How The EU & UK’s Contrasting Approaches To AI Regulation Could Impact Pharma

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.

Artificial Intelligence Digital Health