CEOs to Congress: Pass ORPHAN Cures Act

BIO members and CEOs sent a letter to congressional leaders urging them to pass the ORPHAN Cures Act and support rare disease R&D. Plus, more highlights from the BIO CEO and Investor Conference, including what executives are saying about the policy outlook and AI. (676 words, 3 minutes, 22 seconds)

BIO member companies, CEOs urge Congress to pass ORPHAN Cures Act

At the start of Rare Disease Week, BIO leaders and more than two dozen member companies and CEOs sent a letter urging congressional leaders to pass the ORPHAN Cures Act and support rare disease patients and innovation.

Catch up: The Inflation Reduction Act (IRA) exempts orphan drugs from price controls— “provided they are approved for one rare disease only,” says the letter.

Why it matters: “In effect, the IRA creates a strong financial disincentive against companies like ours investigating whether an orphan drug might have multiple uses—as they often do. If this provision remains in place, investigation into the efficacy of new treatments for a wide range of rare diseases will decline, leaving potentially life-saving applications unexplored. Quite simply, the financial ‘reward’ for investment in such investigations, if successful, could actually be negative,” they explain.

The ORPHAN Cures Act would fix this—“clearing the way for much-needed progress in treating a wide range of rare diseases,” which affect more than 30 million Americans.

But you don’t have to take our word for it…BIO Board Chair Dr. Ted Love, Travere Therapeutics CEO Eric Dube, and the Council of State Biosciences Associations (CSBA) have all joined BIO in explaining why it needs to pass.

American voters agree—with 4 in 5 saying incentives for rare disease treatments should be a critical policy priority, and two-thirds wanting their lawmakers to vote for the bill, according to a Morning Consult poll. 

Download and share BIO’s ORPHAN Cures Act one-pager.

CEOs talk policy outlook and AI

The panel: Day 2 began with a panel about the policy outlook for the year, with a focus on what it means for pricing and access to medicines.

The key takeaway: “Increasingly, we are seeing a far more punitive regulatory environment,” said BIO’s Chief Policy Officer, John Murphy—and biotech companies need to keep an eye on price controls and challenges to patents. The industry needs to communicate about pricing “in more layman’s terms,” said Bristol Myers Squibb’s Chris Mancill—and talk about the value the industry brings.

What they’re saying: “In the election, drug pricing is going to be a hot topic on both sides of the aisle,” said Murphy in an interview. “The industry’s really going to have to look for signals as to how both sides are going to address drug pricing while also continuing to push a narrative about how important it is to provide innovation for patients and how expensive and uncertain that environment is.”

The panel:Executives discussed the growing importance of artificial intelligence (AI) and machine learning in drug development, emphasizing the need for applications that deliver tangible value to patients and investors alike.

The key takeaway: The true value of AI lies in its ability to drive the development of safer and more effective therapies for patients, noted Liz Schwarzbach, Chief Business Officer of BigHat Biosciences. While AI presents immense potential, firms must focus on validating innovations and identifying specific benchmarks for success to harness its full potential in drug discovery.

What they’re saying: “I’m very excited about accessibility of medication—just having people understand exactly how they can leverage this technology towards achieving the end goal, which is getting drugs to patients faster or getting better drugs to patients,” said Petrina Kamya, Ph.D., VP and Global Head of AI Platforms and President of Insilico Medicine Canada.

How to catch up on the BIO CEO & Investor Conference

Did you miss the BIO CEO & Investor Conference—or want to catch up on all the news and insights? Here's how you can read our coverage:

• Follow Good Day BIO and Bio.News for panel recaps, exclusive Q&As, and much more.
• Join the conversation on X with @IamBiotech and #BIOCEO24.

President Biden’s Wednesday: Quiet day after yesterday’s Michigan primary.

What’s Happening on Capitol Hill: Congress has until Friday to pass funding measures (including for the FDA) to avoid a partial government shutdown. According to reports, they’re making progress. Agriculture Secretary Tom Vilsack will testify before the Senate Agriculture, Forestry, and Nutrition Committee today. Meanwhile, Rare Disease Week continues – read more at Bio.News.