How to collect and submit RWD to win approval for a new drug indication: FDA spells it out in a long-awaited guidance

October 21, 2021 11:09 AM EDTUpdated October 22, 04:34 PM

How to collect and submit RWD to win approval for a new drug indication: FDA spells it out in a long-awaited guidance

Zachary Brennan

Senior Editor

Real-world data are messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data are exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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Biotech investors and CEOs see two paths to growth, but are they equally viable?

Noël Brown

Head of US Biotechnology Investment Banking, RBC Capital Markets

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

October 4, 2022 06:00 AM EDTUpdated 11:21 AM

People

Pharma

In Focus

Father starts lab after intellectual property issues stymie rare disease drug development

Jared Whitlock

Features Editor

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Vlad Coric, Biohaven CEO

October 4, 2022 03:06 PM EDTUpdated 03:37 PM

Pharma

Vlad Coric charts course for new Biohaven with neuroscience push and Big Pharma vets on board

Nicole DeFeudis

Editor

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

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Houston, TX, USA

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In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

October 4, 2022 02:54 PM EDT

Pharma

Marketing

AstraZeneca debuts annoying purple ‘Phil’ creatures, personified asthma eosinophils ‘behaving badly’

Beth Snyder Bulik

Senior Editor

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

October 4, 2022 12:55 PM EDT

Pharma

Marketing

Q&A: Novo Nordisk digital innovation chief Amy West discusses pharma pain points and a healthcare 'easy button’

Beth Snyder Bulik

Senior Editor

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Christophe Bourdon, Leo Pharma CEO

October 4, 2022 03:39 PM EDT

Pharma

Marketing

Leo Pharma looks 'beyond the skin' in atopic dermatitis awareness campaign

Nicole DeFeudis

Editor

As Leo Pharma aims to take on heavyweight champ Dupixent in atopic dermatitis, the company is launching “AD Days Around the World,” an awareness campaign documenting real patient stories across Europe.

The project, unveiled on Monday, spotlights four patients: Marjolaine, Laura, Julia and África from France, Italy, Germany and Spain, respectively, in short video clips on the challenges of living with AD, the most common form of eczema.

Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

October 3, 2022 07:31 AM EDTUpdated 02:42 PM

Deals

Updated: AstraZeneca nabs a small rare disease gene therapy player for 667% premium

Max Gelman

Senior Editor

Kyle LaHucik

Associate Editor

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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Head of Quality Systems

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Dave Marek, Myovant CEO

October 3, 2022 07:27 AM EDT

Deals

R&D

Myovant board balks as majority owner Sumitomo swoops in with a $2.5B deal to buy them out

John Carroll

Editor & Founder

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Ying Huang, Legend CEO

October 4, 2022 02:52 PM EDT

Pharma

Manufacturing

Lentiviral vector ramp-up: J&J and Legend to invest $500M in New Jersey manufacturing to support Carvykti

Zachary Brennan

Senior Editor

In response to a question on manufacturing scale at Legend Biotech’s R&D day yesterday, the company’s top exec said its partnership with Johnson & Johnson will be doubling its investment in its New Jersey manufacturing center and will be investing a total of $500 million.

With an eye on their BCMA-directed CAR-T therapy Carvykti (cilta-cel), approved in February as a fifth-line treatment for multiple myeloma, Legend CEO Ying Huang said that the ramp-up in production and the decision to manufacture its own lentiviral vectors — currently in shortage worldwide — means they won’t have to deal with that shortage.