Unintended consequences of the IRA

We recap Day 1 of the BIO Patient & Health Advocacy Summit in D.C. with a look at the unintended consequences of the Inflation Reduction Act—and why patients must be centered. And this World Polio Day, we see an opportunity to eradicate polio worldwide—if we continue vaccination efforts. (643 words, 3 minutes, 12 seconds)

The unintended consequences of the Inflation Reduction Act

The Inflation Reduction Act (IRA) was a hot topic on Day 1 of the BIO Patient and Health Advocacy Summit, with patient advocates discussing the impact on drug development for orphan and rare disease.

Catch up: The IRA exempts orphan drugs for rare diseases from price negotiations—but only if the drugs are approved for a single indication. Drug manufacturers are disincentivized to test whether a new drug can treat other indications.

There’s a potential solution:The bipartisan Optimizing Research Progress Hope and New Cures (ORPHAN Cures) Act, which changes the incentive structure to encourage follow-on investment into orphan drug development.”

Why it matters: “Decisions are being made now based on the legislation that passed over a year ago,” explained Amy Rick, Chair of the IRA Orphan Task Force. “I fear problems have already arisen, and we will be struggling with those decisions for 10 years.”

Specifically: “Once these orphan drugs apply for a second designation, even though there is no revenue associated with it, they will become negotiable,” said Karin Hoelzer of the National Organization of Rare Disorders (NORD). “We think that it has a tremendous chilling effect on the pipeline, in particular for the small biotech where we get a lot of really big innovation in the orphan drug space.”

The bottom line: “My main concern about the IRA is those 95% of patients that have no treatment right now will have to wait longer,” said Jamie Sullivan of the EveryLife Foundation for Rare Diseases. “And our mission is to get people the right treatment at the earliest possible moment.” 

Read more at Bio.News.

Good Day BIO Live at PHAS: ‘It’s all about the patient’

Speaking on Day 1 of the BIO Patient & Health Advocacy Summit in Washington, D.C., BIO Chair Dr. Ted W. Love spoke about how things have changed since he began his training as a physician and why the industry focuses on patients—read more at Bio.News.

We can eradicate polio ­– with vaccination

This World Polio Day, global eradication of polio seems possible—if we continue vaccination efforts.

Why it matters: The polio vaccine has been so effective that there are only a handful of cases globally, where vaccine coverage is inadequate.

The situation today: The World Health Organization (WHO) Polio Committee reported declining cases at their Aug. 25 meeting. Wild polio virus is nearly gone, endemic only in Pakistan and Afghanistan, where cases are at record lows; isolated cases have been found in Malawi, Mozambique, Tanzania, Zambia, and Zimbabwe.

But risk remains: A few recent cases stem from an older oral vaccine containing weakened live virus. This vaccine is no longer used in the U.S., but “shedding” can pose a risk to under-vaccinated communities. (Luckily, infections of this kind are declining, with just 14 cases reported globally last week.)

The bottom line:Vaccines work. Once a terrifying, potentially deadly disease for children, polio is preventable—and the only case in the U.S. since 1979 was in an unvaccinated person last year.  

What’s next: The Global Polio Eradication Initiative started in 1980 helped bring the number of cases from 365,000 to just a handful. The effort continues to get that number to zero.

More Health News: 

STAT News: Maternal COVID-19 vaccination offers infants immunity for up to 6 months
“The risks of severe neonatal morbidity, neonatal death, and admission to the neonatal intensive care unit were all significantly lower during the first month of birth in infants whose mothers were vaccinated against COVID-19, and protection against the virus continued for up to six months after birth, according to a new study published Monday in JAMA Pediatrics.”

Good Day BIO Live at PHAS: ‘Patient advocacy, for us, has always been a choice’

Speaking on Day 1 of the BIO Patient & Health Advocacy Summit in Washington, D.C., BIO Vice Chair John Crowley spoke about his family’s journey into biotech and why the “mission moment” is key to keeping patients front and center—read more at Bio.News.

President Biden’s Tuesday: Awarding the National Medal of Science and National Medal of Technology and Innovation in a ceremony at the White House.

What’s Happening on Capitol Hill: The House continues the process of choosing a Speaker.