Our Chance to Make a Difference
Last week’s blog talked about the BIO convention, and the most important message I brought home is that policy must never hinder innovation. Greater access to care depends on continued innovation—one cannot exist without the other. It is up to us to ensure lawmakers do not jeopardize America’s position as the global leader in pharmaceutical innovation. When people blame “big Pharma” for limiting access to medicines, it is always focused on the price. We need to remember that the most limiting factor to access is when a drug is never discovered. This week, I want to detail some legislation that needs to be passed, either in the One Big Beautiful Bill (OBBB) that is working its way through the legislative process, or in the near future to support the goals outlined above. It seems these days all legislation is complicated, but I will do my best to demystify and explain the legislation and regulations that are important to you and me.
Let’s start with the Optimizing Research Progress Hope And New Cures Act (ORPHAN Cures Act). This proposed piece of bipartisan (that’s right, bipartisan) legislation was introduced to fix the restrictions that were included in the Inflation Reduction Act (IRA) which said that the exemption granted to drugs for rare diseases only applied to the first indication and not to follow-on drugs that were discovered after the first approval. This restriction has already severely reduced the investment in follow-up research for rare diseases. Since the IRA became law, the number of drugs that received a second orphan drug designation has dropped by 48%. We’ve cut the number of secondary drugs available for those afflicted by rare diseases in half! This is certainly a policy that is already restricting innovation. This legislation, which members of both parties have recognized as a problem, has been pulled out of the Senate’s version of the OBBB. If it is not included in the bill during the hectic vote-a-rama happening today (Monday), then the only chance we have to influence our lawmakers is when the Senate and the House work to reconcile the two versions of the OBBB. It is very important that you tell your lawmakers how you feel about this legislation. At the end of the blog are ways to contact your lawmakers and have your voice heard.
I’ve talked quite a bit about the Most Favored Nation (MFN) Executive Order (EO). You can read more about it here. It is another way the government is fixing the price on drugs and another way that limits innovation. Letting other nations free load off of our research investments is not right but using the government’s “sledgehammer” approach, especially through the use of EOs, is never a good idea. There are ways to deal with this inequity and other solutions that need to be considered. This approach will threaten our ability to maintain our position as the center for health innovation. The Supreme Court just ruled that a nationwide injunction to EOs is not permissible, which gives the President even more power to circumnavigate Congress. It means that stopping these types of restrictions on price fixing and other “sledgehammer” solutions will be even harder. We might have a little more time to talk with our lawmakers about the MFN EO, but we need to do it before the August recess.
The Strengthening Medicare and Reducing Taxpayer Prices Act (SMART Act) was reintroduced in May. This piece of legislation wasn’t good when it was first introduced, and it still isn’t any good. Contrary to its labored acronym, this is dumb legislation, as I pointed out in an earlier blog. It doubles down on the price fixing scheme that the IRA introduced without even considering that the impact of the IRA has not yet been determined. It doubles or triples the impact on innovation. We need to tell our lawmakers that this legislation will help crush our hopes for new medicines.
Lastly, I want to discuss a not so well-known law that is now sunsetting, ceasing to be law, if it is not renewed. This law, the Rare Pediatric Disease Priority Review Voucher (PRV) was created to incentivize drug development for serious, life-threatening rare diseases that primarily affect children. I know that this blog deals with healthcare issues affecting older Americans. But as far as I’m concerned, this law is the perfect way that our government should interact and promote healthcare innovation, and we really do care about our grandkids and, gasp, our great grandchildren. This law worked by first having the drug developer apply for and receive a Rare Pediatric Disease Designation from the FDA. Then, upon FDA approval for the drug, they received a Priority Review Voucher which allowed the developer to receive priority review (6 months instead of the standard 10) for a different drug application. This voucher could be used by the developer or sold to other developers, some going for over 100 million dollars. There was no cost to the government, except for supporting an accelerated review, and gave the developer an asset that could be used to entice investment or support their own future research. This was a no cost method for spurring innovation for rare drugs for children. What a great public/private partnership between our government and drug developers. We need to work to get this approach reinstated.
Well, we’ve got our work cut out for us. We need to tell our lawmakers to:
Put the ORPHAN Cares Act back in the OBBB,
Find a better way to get other countries to pay their fair share, and rescind the MFN EO
Make sure that the dumb SMART Act never sees the light of day, and
If you still have some time left, tell them to reintroduce the Rare Pediatric Disease Priority Review Voucher law
You can do all this by clicking here to contact your Senators or your House Representative. For those who are social media savvy, click here for ways to use social media to speak out. You can make a difference.
Best, Thair