The public has taken a renewed interest in discussing vaccines, with many people asking to be convinced that vaccines are safe and effective enough to be used as widely as modern medicine recommends. In the hope of reaching some more people seeking answers to common questions, we’ve prepared this Q&A document. Medicine is complicated, and the current discourse is noisy, but it’s possible to bridge the gap between experts and patients by breaking down the jargon. We’ll try to do that here. We wrote this with our own friends and families in mind because we believe that everyone should be able to get clear answers to their questions. We hope it is useful to you.

Big pharma once again showed up for the Superbowl, or their ads did at least. But those pricey spots, while didn’t connect the dots to inspire the American public with their role in getting important therapies to the patients that need them. Were they moving? Sure. But will they move the needle where it really counts? Probably not.

Since the original version of this article was published in 2023, the market has continued to be tight for biotech investment. New companies continue to form and get funding, but overall, investors are reducing risk with an increasing preference for companies with clinical data – or at a minimum validation in animal studies – before follow-on investments are attractive. 

One of our guiding principals here at RApport and RA Capital is that we learn collaboratively; not just internally, but with the rest of our community as well. RApport is an important part of that (and as always, we encourage you to reach out with questions, comments, and op-ed ideas: rapport@racap.com), but RA University – in which many of our readers are already enrolled – is another. Recently, we’ve been rolling out RAU’s newest, on-demand course: Biotech Unveiled: Understanding the U.S. Biomedical Innovation Marketplace and its Global Role.

Wouldn’t it be better if everyone in our ecosystem were candid with one another?

After all, within companies it’s common to encourage everyone to speak their minds. If you see a problem, call it out; give honest feedback; feedback is a gift; people have a duty to dissent if they disagree with something. So, what stops this corporate if-you-see-something-say-something from going beyond managers and employees? If we normalized candor, we could learn more from one another, cross-pollinate best practices, and correct misunderstandings.

As we break for the holidays and begin to look forward to 2025, we're also taking a beat to look back on what most drew our readers' attention in 2024. 

On the occasion of a documentary about living with cystic fibrosis and the drug that turned his life around, Gunnar Esiason reflects on his personal struggle, the roles of patients and their caregivers in drug development, and the scientific struggle we so rarely see publicly depicted.

On RA Capital's Gateway, Peter Kolchinsky, Managing Partner at RA Capital Management, and Travis Wilson, Partner at Gurnet Point Capital, delve into how biotech companies can achieve more with lean, strategically staffed teams. Small, agile teams of versatile professionals who leverage external expertise when needed lets companies maintain operational efficiency and financial discipline.

Unlike our healthcare spending that goes to services and should be considered an expense, the mere 8% we spend on novel medicines doesn’t just pay for the same things year over year – it’s an investment in progress. Surprised it’s such a small fraction? Most people are.

Hear from our cast of biotech executives and board members on Gateway as they discuss how they utilize the investors on their boards.

Last week’s publication in the Forum for Health Economics and Policy of Valuing the Societal Impact of Medicines and Other Health Technologies: A User Guide to Current Best Practices is a watershed moment for the field of health economics and outcomes research. 

Thanks to the relentless efforts of scientists at Vertex Pharmaceuticals, there will likely soon be a novel non-opioid pain medicine. How insurance plans, including Medicare, decide to cover this drug will make a world of difference to whether we get even better non-opioid options down the road — and whether this progress makes a dent in the ongoing opioid misuse crisis.

Today, AbbVie announced it is acquiring Aliada for $1.4 billion, motivated by the potential for ALIA-1758, Aliada’s lead program, to be the best-in-class for the treatment of Alzheimer’s disease, and by the potential of the BBB-crossing platform to enhance its discovery and development efforts for other difficult-to-treat neurological diseases. As we congratulate the Aliada team, we wanted to share a bit more of the story behind the second significant acquisition from our Raven incubator this year and the first full-circle acquisition of a company we built with technology spun out from a larger company.

In the high-stakes world of corporate governance, conflicts among board members and executives are almost inevitable. But how can these tensions be turned into constructive conversations that drive organizations forward?

Policies favored by both parties’ leadership – who find it politically easier to battle made-up bogeymen than tackle real issues – threaten to undermine our national health security and eviscerate America’s world-leading innovative drug development industry.

Discover the keys to outstanding board leadership in this Gateway video featuring top industry experts. This engaging discussion explores what truly defines a great board chair, bringing together insights from:

Starting with a blank slate is exciting – the ability to create a platform, a pipeline, a team from scratch is a big reason why one comes to biotech – but it requires a fearless and innovative team and bold investors and partners. The story of how PKU became the first killer app for Jnana’s SLC discovery platform -- a key pivot point in demonstrating the platform's value -- is emblematic of the calculated risk taking that we embrace in biotech.

A LinkedIn discussion about “crossover” financing rounds deepened my sense that we’re not all saying the same thing when we’re saying the same thing. And that’s a challenge, if not necessarily always a problem, in an industry that’s only becoming more scientifically and financially complex as it matures. Because to understand one another and avoid unnecessary hiccups it helps to have a common language.

Our usual top-down biotech sector metrics showed that 1H24 was… pretty boring (though for us bottom-up folks, individual companies offer plenty of excitement). After the last few years, boring ain’t bad. With expectations of Fed easing interest rates due to waning inflation, the excitement level for the second half of the year has already picked up, but that’s not reflected in the metrics here.

The bottom line is that when it comes to global revenues for innovative medicines, US revenues for “winners” (the top selling drugs) matter most, since these come at the highest profit margin and launch more quickly, and serve as the primary incentive for biomedical innovation.

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.