Generalized cost-effectiveness analysis (GCEA), a framework to comprehensively assess the societal value of innovative health technologies, will be officially debuted at the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) conference in Atlanta this week

This is the first acquisition of a fully-baked, “home-grown” company from RA Capital’s RAVen incubator, which builds companies that our venture team funds and helps govern, so we wanted to mark the occasion by sharing a bit more about how we got here. 

There is a real and urgent need for better flu prophylaxis and therapeutics. RA Managing Director Laura Tadvalkar writes about why RA is excited to lead a transformative transaction that returns a long-acting antiviral for flu to its original developer Cidara, and RA’s new initiative to help make outlicensing from pharma companies faster and more efficient.

Many people who dedicate their lives to discovering, developing, and making new, lifesaving drugs don’t understand the extent to which the drug industry has failed to communicate its value proposition to society and to inspire its customers to pay for that value. That is to say, when the public and even legislators representing our biotech innovation hubs focus on the price of a breakthrough drug without a sense of its value or how it came into being and insist that innovators should not only invent medicines but pay for them, too, that’s a self-inflicted wound. Vertex’s Trikafta, one of our industry’s greatest success stories, provides an example.

It’s fairly typical for companies to share confidential data with investors of their choosing before completing financing transactions, before publicly disclosing the data or the financing. How is this fair? Shouldn’t everyone get a chance to see the data so that no investor gets a better deal than anyone else? Peter Kolchinsky and Sarah Reed break down what’s allowed, what’s common, and what’s logical.

As a pioneering PDAB takes its first baby steps in Colorado, we’re getting the impression that we’re watching the rules being made up as it goes along. Fortunately, we’re also witnessing the power of organized and impassioned patient advocacy to slow, reverse, and maybe someday reshape misinformed public policy.

Congress has set its sights on China’s biotechnology industry and the US’s reliance on it. Legislators are worried about the Chinese Communist Party’s access to Americans’ genetic data and US taxpayer funds helping bolster CCP-affiliated companies and are proposing to sever ties between any federally funded work and Chinese “companies of concern,” which include BGI and Wuxi AppTech. 

We wanted to expand RA Capital’s Gateway library of teaching modules to cover compensation-related topics, so we peppered experienced board member Steve Hoffman with questions about everything from managing stock option grants to tying bonuses to stretch goals. His answers did not disappoint.

Over the course of just seven days, we saw two biopharmas do cannonballs into the collective cultural pool with marketing efforts that just might impact the trajectory of the national conversation about medicine, science, and progress.

A year ago we published the first “Semper Maior” piece, making the case that biotech was on firm ground and ready for a reboot. We put out the second piece last summer, when it felt like the rebound was underway. Had the year ended in October or even November… well, you know. But here we are after a general market and XBI surge feeling like biotech is now truly recovering from its prolonged downturn. So let’s mine the data, as we have before, to get a sense of what happened in 2023 and what lessons to take with us into 2024.

In what for ten months was shaping up as a brutal year for biotech, it should come as no surprise that all five of RApport’s most popular pieces of the year speak to the market downturn. So whether you’re a biotech employee looking to de-risk your next career move, a CEO wondering how and when to shut down a company, or an investor thinking about next year, grab a mug of peppermint mocha and dig in.

It’s time to start thinking about New Year’s Resolutions. We asked ten experienced biotech board members what they consider useful, not useful, and downright irrelevant in terms of corporate goals. How do you know if goals are effective? And how important is it that they can actually be measured?

In covering how Egypt managed to eradicate hepatitis C, the New York Times rewrites a key bit of history that betrays its misunderstanding of global drug pricing. Egypt’s access to the life-saving Harvoni was a gift from Gilead, not a result of a bare knuckle negotiation. It’s important to understand and explain the increasingly vulnerable system that underpins that gift before bad policy dismantles it.

European central planners should stop trying to talk the US out of incentivizing the development of better medicines that then also help Europeans and the rest of the world. It’s one thing to argue that any one country can’t afford to pay more for new drugs. It’s another thing entirely to put new drugs in jeopardy for everybody by exporting that thinking to the one country whose market-based system has underpinned the last several decades of pharmaceutical advances.

Medicare coverage of anti-obesity medicines would boost competition due to an expanded market, more investors in search of better drugs, and more competitors. This would result in a true weight-loss revolution that can save lives and dramatically drive down the cost of these drugs.

There’s a convention that public biotech companies should plan to raise money by the time they get down to one year of cash left on their balance sheets to avoid a dreaded “going concern” clause in their financial statements. But if you’ve got data on the way, it’s not always necessary to try to raise cash - particularly if the only terms you can get are draconian. Here’s a roadmap for deliberately navigating through that one-year threshold, owning the clause, and letting your data guide your financing strategy.

We asked three Gateway members who are or have been on Audit Committees – Kim Drapkin (CEO, Graphite Bio), Adam Mostafa (CFO, X4 Pharmaceuticals), and Sen Sundaram (CEO, Terns Pharmaceuticals) – to offer their best advice on Audit-related topics.

The technological renaissance in covalent inhibitor technology over the past decade is impressive. Relatively recent advances in structure-based drug design have unlocked the tantalizing opportunity to engineer covalency into small molecules. Now, RA Capital has incubated and seeded Hyku Biosciences to unlock histidines, tyrosines, and lysines for covalent modification to greatly expand the druggable proteome.

Silverback Therapeutics’s wind-down and reverse merger process can serve as a template for other companies when their clinical trials fail. The company’s response to its setback also teaches us about scenario planning, the importance of moving quickly when a key program doesn’t deliver hoped-for data, and why we should look ahead to a biotech ecosystem that anticipates the consequences of – and opportunities stemming from – its inevitable clinical setbacks.

Often, a drug’s initial approval only hints at its full therapeutic potential and the eventual number of patients who may benefit over time. Continued R&D investment can unlock much more utility before the drug ultimately goes generic, but those investments require adequate incentives.