Good Day BIO: The economic case for investment in rare diseases

On Rare Disease Day, we look at a new report that shows the economic burden of rare disease—and the value of investing in new treatments. Plus, we recap what one BIO Board member said about animal genomics during last week’s USDA Agricultural Outlook Forum. (659 words, 3 minutes, 17 seconds)

The economic case for investment in rare diseases

On Rare Disease Day, a new report finds rare diseases impose “a substantial economic burden”—which can be reduced with innovative new treatments.

By the numbers: There are more than 7,000 known rare or “orphan” diseases, which combined affect 25-30 million Americans. Sadly, 95% have no treatment or cure.

“A lack of treatment for a rare disease is associated with a 21.2% increase in total costs per patient per year,”says a just-released report from Chiesi Global Rare Diseases, which studied direct, indirect, and mortality-related costs for a sample of 24 rare diseases across five therapeutic areas. “The cost for 8.4 million patients in the U.S. impacted by 373 rare diseases considered in this analysis is estimated to be $2.2 trillion per year.” 

For all known rare diseases, “the societal responsibility…may be in the range of $7.2 trillion to $8.6 trillion per year.” 

Why it matters: “These results highlight the need for policymakers to nurture and sustain innovation based on the positive economic return from rare disease therapies and justify an increased governmental investment in diagnosis and newborn screening to ensure wider patient access to therapies,” concludes the study. “Incentives for drug development, particularly restoring the Orphan Drug Tax Credit to 50% and maintaining its current applicability to multi-indications, encourage investment and have led to progress in rare disease drug approvals.”

Watch: “Equity is a word that's used a lot right now, and when it comes to rare disease, it means ensuring research and development gives these conditions the attention they deserve,” BIO President and CEO Dr. Michelle McMurry-Heath told the RareKC Symposium last week. "I call it just science"—watch her brief remarks.



More Health News:

Modern Healthcare: Will we maintain the momentum on new therapy breakthroughs?
Scientists had an astoundingly productive 2021. The Food and Drug Administration fully approved more than 50 new drugs—one of the best years on record—and granted emergency use authorization to numerous other therapies, including antiviral pills that reduce the risk of death from COVID-19 by up to 89%. Those COVID therapeutics and vaccines generated the biggest headlines. But other breakthroughs were just as scientifically impressive—and could be just as transformative for patients.

When it comes to animals, we need ‘more science, not less’

Animal genomics can drive animal health and welfare as well as the sustainability of our food supply, said a BIO Board member during USDA’s 98th Agricultural Outlook Forum last week.

A key theme: productivity and sustainability “don’t have to be at opposite ends of the spectrum,”said USDA Chief Economist Seth Meyer. “You don’t have to be either sustainable or increase productivity—there are innovative ways to accomplish both.” 

A key solution: innovations in animal breeding, which can “help protect our food supply and create a more healthy, sustainable food system,” said BIO Board member Elena Rice, Chief Scientific Officer of Genus PLC during the opening plenary on market opportunities. 

Animal genomics are important but “often overlooked,” said Rice, whose company is using gene editing to improve the health, welfare, and sustainability of food animals with a focus on disease resistance.

Their most advanced project: pigs “completely resistant” to porcine reproductive and respiratory syndrome (PRRS). They’ve developed hundreds of pigs that look, develop, grow, and breed like non-gene-edited pigs, “except they do not get sick from PRRS.”

They’re also working on feed additives to reduce enteric methane emissions from beef and dairy cows,which account for about 30% of methane emissions. Scientists recently said atmospheric methane emissions are growing “dangerously fast.”

The bottom line: “We need more science and not less” to improve animal health and welfare and meet “global protein demand in a sustainable manner,” concluded Rice—and we need policy that supports the development and deployment of thesetechnologies.

Did you miss it?You can register for free and access the full event on-demand.

President Biden’s Monday: Ahead of tomorrow’s state of the union address, he’s accelerating “the return of the federal workforce this week, as the White House seeks to show the country that in-person work can be safe,” reports Axios. This morning, meeting with Allies and partners to discuss Russia’s attack on Ukraine. This afternoon, holding a Black History Month celebration with Black Cabinet secretaries, lawmakers, and civil rights leaders.

What’s Happening on Capitol Hill: Back from recess. Meanwhile, the Supreme Court will hear what The New York Times calls the “biggest climate change case in a decade,” which could “restrict or even eliminate the Environmental Protection Agency’s authority to control the pollution that is heating the planet.”