BIO tells Congress price controls hurt R&D

Price controls are already hurting drug R&D, especially for rare disease, BIO’s Vice Chair John Crowley told Congress yesterday. Meanwhile, in New York, BIO’s SVP Phyllis Arthur spoke on a panel during the UN General Assembly about what we need to do to get ready for the next pandemic. (653 words, 3 minutes, 15 seconds)

BIO’s Vice Chair to Congress: ‘Ill-conceived’ price controls limit cures

Drug price controls in the Inflation Reduction Act (IRA) harm drug R&D, John Crowley, CEO of Amicus Therapeutics and BIO Board Vice Chair, told Congress yesterday.

What happened: Crowley outlined several problems with the price controls at the House Energy & Commerce Oversight and Investigations Subcommittee hearing, “How the IRA’s Price Setting Scheme Means Fewer Cures for Patients.”

The law halts R&D for rare diseases, said Crowley, who works to develop treatments for rare diseases like Pompe and Batten. Orphan drugs are exempt from price controls—but only if they’re approved for a single indication, thereby disincentivizing manufacturers to test for other indications. (Learn more about Amicus' work.)

Why it matters: With 30 million Americans impacted by rare diseases and 95% lacking treatment, “this is a public health crisis of epic proportions,” said Crowley.

And the price controls kill investment overall—including with provisions disincentivizing investment in small molecule drugs, which account for 90% of approved medicines, he said.

Watch highlights:

CMS makes things worse: Calling for congressional oversight, Crowley said implementation of the law by the Centers for Medicare and Medicaid Services (CMS) discourages incremental drug improvements and is overall unpredictable, which chills investment.

Members of Congress concur: Subcommittee Chair Morgan Griffith (R-VA) said the drug price provisions appear unconstitutional. Energy & Commerce Chair Cathy McMorris Rodgers (R-WA) agreed IRA crushes innovation, while Rep. Debbie Lesko (R-AZ) said 24 companies have already curtailed drug development.

BIO’s view: “We need to break down barriers, encourage massive private capital flows to fund biotech entrepreneurs (and) incentivize our largest biopharmaceuticals companies to invest in rare disease programs,” Crowley testified. “We are instead now facing a massive headwind brought about by an ill-conceived drug control pricing law.”

Read in-depth coverage in Bio.News.

Get ready now, BIO’s Phyllis Arthur tells UN

We need to make progress now against the next pandemic, BIO’s Phyllis Arthur said during a panel on pandemic prevention, preparedness, and response at the UN General Assembly yesterday.

What’s been happening: The World Health Organization (WHO) has been working on a pandemic preparedness treaty to ensure better global preparation for the next pandemic. Yesterday, UN member states took a small step forward with a political declaration to cooperate on pandemic preparedness.

Why it matters: “The best outcome during an emergency comes from partnerships during the interpandemic period when there is time to make thoughtful investments,” said Phyllis Arthur, BIO’s SVP of Infectious Disease and Emerging Science Policy, during the UN panel.

What we need: Governments and stakeholders must collaborate to expand clinical trial networks, strengthen regulatory capacity and harmonization, build stronger supply chains, and incentivize manufacturing across regions, she said.

The role of governments: “Governments, in partnership with manufacturers, should invest in geographically diverse regional manufacturing expansion during the period between pandemics. This will aid in the development of new technologies that could be rapidly deployed around the world,” she added.

Beyond R&D, we must prepare for procurement and distribution, as well, she continued.

The big picture: “Government and industry should invest in new platform technologies and mechanisms for the development of vaccines and antivirals against the most important pandemic pathogens,” Arthur said. “That must be done in a universe where there’s a strong regimen of intellectual property, and voluntary and strongly incentivized collaboration between companies, governments around the world.”

Read in-depth coverage in Bio.News.

More News:

Colorado Politics (Opinion): Powerful state panel poses threat to patients with rare diseases
“The Prescription Drug Advisory Board (PDAB) is one of the most powerful panels ever created in Colorado. It has been tasked with cutting the cost of medicines through setting mandatory prices. What could be wrong with using the power of state government to simply cut drug costs? As it turns out, there are numerous short-term, and long-term, red flags for patients,” writes Amber Freed, Founder and CEO of SLC6A1 Connect.

President Biden’s Thursday: Meeting Ukrainian President Zelensky. CNN and Washington Post have more.

What’s Happening on Capitol Hill: A relatively quiet day in committees as Congress works toward a Sept. 30 spending deadline.