Seems like we JUST had a presidential election, right?
But we’re in the dead of summer 2019, inching closer to a presidential election that will once again take over our TV, Internet, and every day conversations.
And while there’s a lot of bluster and high-falutin words being kicked back and forth, you’ll be surprised to know that the 2020 contenders for the Democratic Party’s presidential nominee have been having a nuanced debate about healthcare policy regarding rare diseases!
Ozy.com published a great article, titled “DEMS ARE TORN ON HOW TO TREAT RARE DISEASES” — revolving around the candidates’ positions on the Obama-era policy “21st Century Cures Act” and unsigned legislation “Orphan Products Extension Now.”
It’s worth a read, but we’ll break down the most essential bits below!
What is the 21st Century Cures Act?
Signed into law in 2016 by President Obama (and passed by a Republican Senate), The 21st Century Cures Act promotes and funds the acceleration of research into preventing and curing serious illnesses; accelerates drug and medical device development; attempts to address the opioid abuse crisis; and tries to improve mental health service delivery.
It included many provisions directly impacting and promoting the discovery, development, and delivery of orphan therapies for rare disease patients, including:
- An extension of the Rare Pediatric Disease Priority Review Voucher Program, which incentivizes the development of new therapies to help the more than 15 million children with rare diseases;
- Streamlining of U.S. Food and Drug Administration (FDA) review of genetically targeted and protein variant therapies for rare diseases;
- Creation of funds in the amount of $4.8 billion over 10 years for the National Institutes of Health (NIH) to fund the Precision Medicine Initiative, BRAIN Initiative, and the Cancer Moonshot;
- Further expansion of the Patient-Focused Drug Development Initiative and requirements for the FDA to report on how patient experience data was used in regulatory review.
The law also amends FDA regulations to allow for companies to — in some cases — seek expedited drug approvals without clinical trials, simply by providing other data that points to success.
However, the Trump administration has withheld funds meant for the NIH to expand research on rare diseases.
Among presidential hopefuls, Vice President Joe Biden, Sen. Kirsten Gillibrand, Sen. Amy Klobuchar, Sen. Cory Booker, former Rep. Beto O’Rourke and Rep. Eric Swalwell support the law.
What is the OPEN Act?
Also known as the Orphan Products Extension Now Accelerating Cures and Treatments Act — the OPEN Act is a bill (not law) that would extend the exclusivity period for rare disease drugs — currently five years — by an additional six months. During this period, no other company can sell that drug, irrespective of whether it’s patented.
The OPEN Act’s chief cheerleaders are Klobuchar and Swalwell, who are on the rare diseases caucus in Congress.
What is the Advancing Access to Precision Medicine Act?
Sponsored by Swalwell, the Advancing Access to Precision Medicine Act would facilitate customized genomics-based treatment for rare diseases.
Which Dems Oppose these Bills and Why?
Senators Elizabeth Warren and Bernie Sanders are opposed to the aforementioned bills, because they argue they would allow pharmaceutical companies to profit by short-circuiting scientifically established procedures and rushing drugs to the market.
Warren even voted against the 21st Century Cures Act.
The Bottom Line (For Now)
So the debate falls on these two markers:
One side (Biden, Klobuchar, Swalell) is keen on the need to incentivize research and development into potential cures for rare diseases, in the hope that this will lead to much-needed drugs.
The other side (Warren, Sanders) is more focused on curbing Big Pharma’s influence. By introducing drugs that haven’t been through the most rigorous of clinical trials, these argue that these pharma companies risk patient health. And with longer periods of exclusivity, pharma can also monopolize the market of a key drug patients need — enabling them to set higher prices than would be the case in a competitive market.
We don’t have the answers, but we are thrilled that candidates for the highest office in the land are asking the questions.
We’ll keep an eye on these issues as the campaign season heats up.