Good Day BIO: Orphan drugs now at risk

September 17, 2021
Another important thing in the budget reconciliation this week: Democrats included a provision that would change the Orphan Drug Tax Credit and disincentivize R&D. We have details, plus more on what needs to happen at the UN Food Systems Summit next week. (783 words, 3…
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Another important thing in the budget reconciliation this week: Democrats included a provision that would change the Orphan Drug Tax Credit and disincentivize R&D. We have details, plus more on what needs to happen at the UN Food Systems Summit next week. (783 words, 3 minutes, 54 seconds)

And before you head out for the weekend be sure to listen to the latest I am BIO podcast.

 

What happened with the Orphan Drug Tax Credit

 
 

In this week’s reconciliation markup, Democrats included a change to the Orphan Drug Tax Credit, which would negatively impact research on rare diseases affecting millions of Americans. 

ICYMI: It was a busy week in the House with several committees marking up the Senate’s $3.5 trillion budget reconciliation package. (Here’s our readout, including news on drug price controls and the sustainable aviation fuel tax credit.)

But there was an unwelcome surprise: Section 138141 (page 609), which would make drug manufacturers ineligible to collect the Orphan Drug Tax Credit “if the drug had previously been approved by the Food and Drug Administration for a separate indication,” explains STAT News

“The ODTC can help to offset the cost of developing and testing orphan therapies as they move through the clinical trial process,” explains the National Organization for Rare Disorders (NORD). “This longstanding incentive is particularly important for the many smaller companies focused exclusively on rare diseases.”

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—and if the provision is passed, R&D will be disincentivized further. 

Before the Credit, there were only a handful of treatments for rare disease patients. Now there are hundreds being developed. Like for cystic fibrosis. The first treatment helped less than 1,000 patients. Today, 90% of CF patients have a life-changing therapy. If this provision were in place, thousands of those patients may not have a treatment option.

Masters’ Message: The idea of taking away the Orphan Drug Tax Credit is certainly no surprise. What is a complete surprise is that Congress would consider a move to destroy research into cures for vulnerable populations afflicted by rare diseases such as sickle cell and cystic fibrosis. We will be aggressively fighting back on behalf of patients in the rare disease community—just because their numbers are not huge doesn’t mean their suffering is any less real. It is shocking some Members of Congress would put those patients’ cures at risk. – Rich Masters, BIO’s Chief Public Affairs and Advocacy Officer

Read: Marking Rare Disease Day 2021

 

More Health Care News: 

The San Diego Union-Tribune (Opinion): Antibiotic-resistant superbugs are a ticking time bomb in global healthcare
The PASTEUR Act and the DISARM Act could help, write BIO President & CEO Dr. Michelle McMurry-Heath and Dr. Andrew Tomaras, Chief Scientific Officer at Forge Therapeutics.

RealClearHealth (Opinion): White House plan on healthcare reform: mostly brickbats, some bouquets
“That healthcare package released by the White House has some good ideas and some dangerous ones,” including federal price negotiations, drug importation, and a federal board to determine “value,” says former FDA Associate Commissioner Peter J. Pitts, President of the Center for Medicine in the Public Interest and Visiting Professor at the University of Paris School of Medicine.

 
 
 
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Why gene editing should be on the menu

 
 

Next week’s UN Food Systems Summit will provide a global platform to discuss solutions for securing our food supply—and gene editing must be on the menu.

Rapid increases in climate instability and population are major threats to our food supply—some of the biggest challenges agriculture has seen in its 10,000-year history

Gene editing is one of the best tools available to respond, Innovature explains—IF governments remove obstacles to using it. 

The U.K. took a step in the right direction by approving the first field trials of CRISPR-edited wheat in Europe. “The wheat has been edited to reduce levels of the naturally occurring amino acid, asparagine, which is converted to the carcinogenic processing contaminant, acrylamide, when bread is baked or toasted,” says U.K. Research & Innovation

Read: BIO encourages new rules for gene editing technology in the U.K. 

CRISPR works “by removing or replacing genetic sequences in an organism’s DNA. Rather than inserting foreign genetic material, it simply accelerates beneficial mutations that would occur naturally,” Innovature explains.  

Read: Gene Editing 101 

This can make crops more resilient—helping them endure harsher environments by requiring less watertolerating drought, and being more productive. Recent innovations include:

Now, world leaders need to ensure equitable access to these advances—by “eliminating barriers to the benefits of science,” wrote experts in the International Business Times

Read: How We Can Stave Off the Hunger Pandemic

 

More Agriculture and Environment News: 

Axios: CRISPR companies aim to bring gene editing to the market—and beyond
“Mammoth Biosciences—whose co-founder Jennifer Doudna shared a Nobel Prize for chemistry last year for her role in discovering the gene-editing tool—announced $195 million in funding late last week, valuing it at more than $1 billion.” 

Grist: A deadly fungal disease on the rise in the West has experts worried
“Researchers haven’t pinned down exactly what’s behind the rise in Valley fever cases or how to stop it. One thing is nearly certain, though: climate change plays a role.”

 
 
 
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After seeing firsthand how few Hispanic nurses there were to serve Hispanic communities in San Antonio, Texas, Dr. Ildaura Murillo-Rohde dedicated her career to changing this dynamic. She became the first Hispanic dean of nursing at New York University and founded  the National Association of Hispanic Nurses to advocate for Latinx nurses. The American Academy of Nursing named her a " living legend” in 1994.

Meet more Hispanic and Latinx scientists and innovators you should know.

 
 
 
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President Biden’s Friday: Early this morning, he reconvened the Major Economies Forum on Energy and Climate to press for climate action. The White House COVID-19 Response Team will hold a briefing at 11:45 AM ET. President Biden will head to Delaware this afternoon. 

What’s Happening on Capitol Hill: Enjoy the weekend.

 
 
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