Table of Contents
Earlier this summer, I had a front-row seat for the amazing progress biopharmaceutical companies are making in the battle against cancer.
Stephen Ubl
The deluge of data at this year’s American Society for Clinical Oncology (ASCO) meeting provided many reasons to cheer. There were blows struck against some of the biggest cancer killers and impressive progress against some of the toughest-to-treat tumors. Cancer-killing approaches once thought impossible — “undrugable targets,” in industry terms — are now a reality.
But there are reasons to be concerned this winning streak might be at risk. There is no debate the price-setting provisions in the Inflation Reduction Act (IRA) will lead to fewer new medicines, and the ones that are likely to be hit first — and hit hardest — are the ones developing the next generation of breakthrough cancer medicines.
Cancer drug development usually starts with testing a medicine in the sickest patients, where patients with more advanced stages of cancer have exhausted treatment options and the need is greatest. These treatment populations are also where a new therapy may be most impactful. After the Food and Drug Administration approves a medicine, biopharmaceutical companies often conduct additional research on patients with earlier stage cancers, or test it on different types of cancers to see if it can help more patients beyond the initial indication. This critical post-approval R&D often requires lengthy and complex clinical trials which can take up to four years to complete.
A new study from the Partnership for Health Analytic Research shows that nearly half the time, approvals for additional indications for medicines take place seven or more years after the first approval. It’s a system that works well: companies have an incentive to continue to look for new ways to leverage their innovation and an ever-larger group of patients benefits.
This year’s ASCO meeting showed how that works in practice. Two of the most important studies presented this year involved evidence that cancer-fighting pills — Kisqali plus endocrine therapy for breast cancer, Xtandi plus leuprolide for prostate cancer — were highly effective in far broader patient populations than had previously been shown. Because of continued investment in research, both of them have the potential to improve the lives of tens of thousands more Americans.
Yet these are the very type of post-approval R&D treatment advances that may be jeopardized under the IRA.
That’s because the law selects small molecule medicines, generally pills, capsules or tablets, for government price setting as early as seven years after they are first approved. And the government’s mandated price takes effect after just nine years.
This policy — the “pill penalty” — will further discourage companies from moving forward with the types of advances highlighted at ASCO and removes incentives for companies to continue investing in research as products mature. That’s exactly the opposite of what patients want and need.
Kisqali has been on the market for more than six years, and Xtandi’s initial approval came in 2012. Indeed, the Xtandi Phase 3 study that garnered attention at this year’s ASCO meeting took nine years to read out. That raises the question: Would these breakthrough results have happened if the IRA’s punitive approach had been in place a decade ago? Would the studies have even been attempted?
The IRA’s attack on ongoing R&D is forcing a full-scale rethinking of how companies develop oncology medicines. Since there is only a small window when a medicine is not subject to price setting, it may make more sense to try to win approval for larger groups of patients first, even if it means it takes longer to get a medicine on the market.
Companies are already struggling with that choice. At a press briefing at ASCO, Genentech’s chief executive officer, Alexander Hardy, said that the company feels the weight of this huge disincentive for getting medicines approved as quickly as possible for a small group of sicker patients, rather than in the broadest possible group.
A year ago, Genentech could have pushed for both earlier access for the neediest patients and expanded use into larger populations. Because of the pill penalty, they’re now being forced to make a much more difficult choice about choosing early indications with the greatest economic value rather than the greatest unmet need. As a result, in many cases, small treatment patient populations and those with rare types of cancers may have to wait for new treatment. And there’s the possibility that it’s no longer feasible to pursue those indications at all.
It’s a terrible position to put researchers and patients in. Regardless of which decision is made, patients and the treatments they need will be harmed. That’s a hard reality to accept, particularly when we know — and as the ASCO meeting proved — that the future of cancer treatment is otherwise so bright.
Stephen Ubl is president and chief executive officer PhRMA.
