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. 

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.

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.

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.

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.

All else being equal, people care more about outcomes that happen in the near future than about outcomes that happen later. The discount rate represents how much timing matters. We sit down with Josh Cohen, Deputy Director of the Center for the Evaluation of Value and Risk in Health (CEVR) at the Institute for Clinical Research and Health Policy Studies at Tufts Medical Center, and Research Professor of Medicine at Tufts University School of Medicine, to learn about how changes to the discount rate can alter how we value medicines.

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

Whether you enjoy air travel or not, it’s increasingly common and persistently bad for the environment. Almost three million passengers fly in and out of US airports each day and global demand for air travel is expected to double by 2050. In addition to emitting pollutants that lower air quality, aviation is recognized as a hard-to-decarbonize sector and accounts for ~3% of global CO2 emissions. Airlines have recently announced commitments to switch to "green" fuels, but as of today 95% of jet fuel is produced from fossil fuels. Why is that? 

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.

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.

Let’s dig into some of the most recycled materials in the world – metals – and how we might make metals recycling even better. We are obsessed with metals because they are critical materials to human civilization and well-being, which is why improved scrap metal recovery is good for the economy, good for the environment, and good for people – exactly the kind of win-win-wins we live for at Planetary Health.

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. 

Let’s talk about something everyone produces but nobody really likes – trash. People produce billions of tons of waste annually. While there are many ways to reduce the amount of waste we produce, it is not possible to stop it entirely. Waste is, quite literally, inevitable. The disorder (entropy) in the universe increases with time, which just means that things will always break and there will always be some amount of waste. Once our things become our trash, we have two options: we can dispose of them or we can try to reclaim them as a resource. From a planetary health perspective, there are a lot of reasons to want to reclaim wastes as resources. Unfortunately, it is not easy to do so economically.

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.

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.

It may surprise you to find out just how badly we’re failing to take a comprehensive approach to quantifying societal value from new innovations in medicine. The IRA’s investment in our climate future should be lauded; its treatment of medical innovation is unfortunately hobbled by its goal of lowering Medicare spending today at the expense of tomorrow’s patients – that is to say, all of us.

It’s long been assumed that as long as NICE-like cost-effectiveness analyses stay on the other side of the ocean, our US biotech ecosystem will be fine. But in recent years, toxic math has begun to make its way to US soil. Peter Kolchinsky sat down with Jayson Dallas to get his take on NICE’s tactics, their increasingly global reach, and how biopharma can fight back.

The treatment of NDA-path drugs in the Inflation Reduction Act materially degrades the investment case for these treatments in age-related diseases and thus jeopardizes future biotechnology progress and the health of America’s seniors.

The federal government must address the failure of Silicon Valley Bank (SVB) with speed and clarity before it metastasizes into a national crisis, so the ecosystem of institutions in which SVB has operated, including other banks and investors, can help bridge companies so that they can continue to operate while they wait for their deposits.

Our year-end wrap of the site’s most popular articles of 2022 reflects a challenging year for the biotech sector, featuring tumultuous markets and the shifting drug pricing policy landscape. But those pieces also reflect the ingenuity, hopefulness, and opportunity that is inherent in drug discovery and development.

Traditional cost-effectiveness analyses done by organizations like ICER and NICE overlook much of the value of new drugs, including factors with crucial societal impact like genericization, risk reduction, and community spillover. Peer-reviewed research has made this clear again and again, but these organizations continue to insist on using outdated formulas to determine the value of drugs. With the passage of the IRA and imminent drug pricing “negotiations” (read: price controls), it’s more important than ever to get the math that values our medicines right.

The recent announcement by Alnylam Pharmaceuticals that it was holding off on a planned Phase 3 pivotal study for vutrisiran (marketed as Amvuttra in its sole approved indication so far, hATTR amyloidosis with polyneuropathy) in Stargardt disease illustrates how the Inflation Reduction Act is already having a negative impact on small molecule and orphan drug R&D prioritization. Alnylam’s announcement also brought out skeptics of the biotech industry, who argue the new law is being scapegoated by innovators who want to overturn its Medicare drug-negotiation provisions.

As the founder of two biotech companies in Arizona that are currently fundraising to support drug development, it’s clear to me that the Inflation Reduction Act will steer me away from developing cancer drugs for older Americans. This is a problem that can be fixed.

Over the past four decades, David Beier has observed and participated in the evolution of the US biotechnology industry for several key vantage points. The industry’s success and freedom to innovate, he says, has been underpinned by – and helped create – a marketplace that was open enough to fully reward risk-taking investors devoting capital to cutting edge science. Today, that success faces new threats.

A conversation with former Pfizer research head John LaMattina about the persistence of the misperception that NIH invents the drugs that biopharma companies sell, the chance encounters that can lead to breakthroughs years or even decades later, and the tough math facing investors and heads of research across the industry when deciding whether to invest in a project that’ll only enjoy nine years of market exclusivity.

We talk a lot about access to care and the hurdles that might keep people from getting the medicine they need. Usually, we’re talking about the price of healthcare or the inanity of insurance requirements. But other hurdles hide in plain sight, as is the case with naloxone (Narcan), birth control, and other drugs that could (and should) be available over-the-counter (OTC). 

If the FTC begins to look askance at M&A between commercial-stage biopharmaceutical companies and the innovative smaller biotechs that so often are the sources of new medicines, that would be bad news for future R&D and the patients who depend on our industry for life-saving medicines. That they might do so in the name of preserving or protecting innovation in the first place would add insult to injury.