How biomarkers can support rare disease pathways

BIO’s soon-to-be CEO John F. Crowley talks biomarkers and rare disease regulatory pathways, and U.S. Rep. Brett Guthrie explains the impact of policies to control drug prices. (557 words, 2 minutes, 47 seconds)

How biomarkers can support rare disease regulatory pathways

On Feb. 21, BIO’s soon-to-be CEO, John F. Crowley, joined a workshop about biomarkers and rare disease regulatory pathways, hosted by the Reagan-Udall Foundation for the Food and Drug Administration in Washington, D.C.—Bio.News has a quick recap.

The workshop:Qualifying Biomarkers to Support Rare Disease Regulatory Pathways explored primary disease activity biomarkers in rare genetic diseases. The workshop used heparan sulfate in neuronopathic mucopolysaccharidoses (MPS) as a case study for a biomarker to support accelerated approval.

Why biomarkers? They can be a tool for regulators and drug developers to use to determine safety and efficacy, explained Crowley. They can also help assess dose or biologic activity.

The challenge now: “How do we make sure that we’re achieving the gold standard of safety and efficacy grounded in key science?” he asked.

Why it matters: “We’ve come a long way since the Orphan Drug Act, but we’ve got a long, long way to go. And I think we’re finally at this inflection point where we now have the tools of science to offer hope tempered with reality of the challenges of drug development,” he said.

BIO’s take: “John and the panelists did a great job discussing the value of both the accelerated approval pathway and biomarkers in the development of treatments for rare diseases,” observes Dr. Cartier Esham, BIO’s Chief Scientific Officer and EVP of Emerging Companies. “It also highlighted how each treatment and each rare disease needs to be evaluated on the needs of both scientific understanding and the needs of that specific patient population.” 

Read the full recap on Bio.News.

More reading: What are biomarkers?

More listening: I am BIO Podcast- Accelerating Hope: The Pathway for Cures

Rep. Guthrie: ‘Biden’s coming for your cures’

That’s the message from Rep. Brett Guthrie (R-KY), who explains in a new op-ed the impact of Biden administration policies to control drug prices.

What’s happening: Although data shows branded drug net prices “have steadily declined” in recent years, “President Biden has resorted to price controls and removing incentives for innovation by taking the intellectual property of innovators,” writes Rep. Guthrie in The Hill.

First, the Inflation Reduction Act—“[d]espite flashing red lights that the bill would lead to significant decreases in research and development of critical drugs, including one such study indicating it would lead to as many as 342 fewer cures from 2021-2039,” says Rep. Guthrie.

Now, we’re talking Bayh-Dole—which the Biden administration is attempting to use to control drug prices, as we explained yesterday. (BIO opposes this.)  

There ARE good solutions to lower drug prices—like the bipartisan Lower Costs, More Transparency Act, which would require pharmacy benefit managers (PBMs) to disclose drug rebates and discounts, among other transparency measures. (BIO supports this.)

The bottom line: “I urge the president to work across the aisle alongside Congress to make prescription drugs more affordable without retreating on our shared visions of finding cures to previously incurable diseases,” concludes Rep. Guthrie.

Read and share the op-ed.

President Biden’s Friday: Welcoming the nation’s governors to the White House during the National Governors Association Winter Meeting.

What’s Happening on Capitol Hill: Recess.