As I said earlier this week, we’d be getting a Right-to-Try vote in the House, and well, we got that vote. Last night the House voted to approve the bill and will now send it to President Trump’s desk for his signature (it’s pretty much guaranteed conclusion that he will sign it).

As reported by STAT:

The legislation that passed the House would allow patients with life-threatening conditions ask drug makers to share treatments that haven’t yet been granted FDA approval, though the treatments must be in active clinical trials, and must have cleared initial preliminary testing. Patients who want to use the pathway must have exhausted all their other options, and must be unable to participate in a given clinical trial. The legislation doesn’t obligate drug makers to provide the treatments, and it doesn’t prevent them from charging patients for their associated costs if they do.

It certainly sounds nice, but I’m still not sure how it will be put to action. I am of the thought process that this could be bringing out some false hope to patients in dire need. Further it could also circumvent the FDA, whose responsibility is to keep Americans safe.

Several patient advocacy groups have expressed their displeasure, most notably (in our case, at least) the National Organization of Rare Disorders.

Here’s some of their statement published last night:

Today, the House of Representatives passed the Right to Try Act (S.204). We at the National Organization for Rare Disorders (NORD) are sorely disappointed that the House of Representatives has voted to send this legislation to the President’s desk…We have strongly opposed all Right to Try legislation, as we do not believe that it will safely and genuinely increase access to investigational therapies outside of clinical trials… S.204 does not set a standard for informed consent, has a much broader definition of eligibility, and contains vastly weaker reporting requirements. Thus, not only will this legislation be ineffective, as all Right to Try laws are, it will also present a danger to the many patients we represent…While we are disappointed that Congress has decided to ignore patient safety concerns, we will redouble our efforts to educate the rare disease patient population about clinical trial participation and enrollment as well as expanded access…The bill that passed the House today is dangerous, and we will do everything we can to protect our patients from unsafe and ineffective therapies.

I share most of NORD’s concerns, but I do believe that people with end-stage terminal illness should not be the endpoint of government legislation preventing access to potentially continuing life. This Right to Try Act seems to rollback some of the existing bureaucratic red-tape that currently exists for patients applying for the FDA’s compassionate use program, and that may help end-stage patients.

Ultimately it’ll be pretty interesting to see what will happen to patients in our community as we standby for this bill to be signed into law. As it currently stands we do have a number of very promising medications advancing through the drug development pipeline. Will this suddenly open access to those experimental medications for our friends who are in end-stage illness?

It remains to be seen.

Personally I’d like for people in end-stage CF (specifically) to be given as much opportunity as possible to continue to live without the need for moving towards transplant, or some cases, being given the opportunity to make oneself eligible for transplant. As for the rest of the CF population, those people who may be a bit healthier than others, I don’t think this means too much in the here and now.

What do you think?