Lawmakers discuss push for rare disease prevention, support

Lawmakers on Friday pushed for improving the production and access of lifesaving drugs for rare diseases at The Hill’s Improving Rare Disease Diagnostics & Care event.

Rep. Rodney Davis (R-Ill.), whose wife suffered from colon cancer and Lynch syndrome, said rare disease prevention is personal for many lawmakers.  

“There are so many people that I have met over my career in Congress whose families have been impacted by so many of those thousands of rare diseases and this is personal to all of us,” Davis said during the event Friday, which was sponsored by AstraZeneca Rare Disease and moderated by The Hill’s contributing editor Steve Scully.

Davis, a member of the Rare Disease Congressional Caucus, said in the short term COVID-19-related health protocols need to be adjusted to provide those suffering from rare diseases the care they need.

The suspension of elective surgeries in the early months of the pandemic prevented patients from receiving the early detection they needed, Davis said

“Short term, we’ve got to change the way that we’ve responded to COVID because I think we are going to have an increase in diseases that we know how to treat that are going to have a higher mortality rate,” Davis said.

In the long-term, Davis said there should be an emphasis on the relationships between doctors and patients.

“Long term we’ve got to keep the doctor-patient relationship paramount, and my fear is that many of my colleagues would rather see a government-patient relationship versus a doctor-patient relationship,” Davis said.

Davis — who supported the Right to Try Act, which provided patients access to some unapproved drugs — said there should be more ways for patients to access the unapproved drugs that can save lives. 

“When it comes to prescription drug access, and in my opinion the right to try any drug that could possibly help anyone who is fighting a rare disease right now, their access needs to be paramount in what policies we put in place,” Davis said.

“I was proud to support the Right to Try Act a few years ago that allows patients like my wife and others who may be in a more difficult situation in their fight to have access to those orphan drugs, have access to those pharmaceuticals and those therapeutics that might help that patient survive,” he added. 

Rep. Diana DeGette (D-Colo.) said approval of drugs for rare disease could model the fast-track production of COVID-19 vaccines.

“We always have to make sure that we’re not cutting corners on approval because we want to make sure that the drugs work and that they’re safe, but if you have a situation like this where you’re able to bring together teams from the agency itself, from the NIH and the FDA, but also from the pharmaceutical companies and the research universities to structure clinical tests to then be able to conduct those clinical tests on a widespread basis,” DeGette said at the event.

“And then to be able to approve on an emergency use basis, then that’s why we were able to get these vaccines as quickly as we could,” she added.

DeGette said lawmakers must also remove themselves from the research process for these drugs, especially when it comes to research involving stem cells.

“Policy makers need to give researchers the tools that they need to find cures and the methods that they need to find cures, the funding that we need to do the research, and then we need to step back,” DeGette said.

“We shouldn’t be micromanaging, for example, what types of cells researchers use as long as they’re used in an ethical way. We need to trust the scientists, and we need to work hand in hand,” she added.