When I come across items like this in Rare Disease Report,  I feel very ambivalent.  While much of the comments I can’t argue with,  I don’t entirely agree with this stance on compassionate use & right-to-try laws.

I understand the pressing need to not give false hope & protect those in fragile health.  However, the other side of the coin applies as well. If you are facing a shortened life expectancy with no good treatment options & a uphill battle in maintaining your quality of life because of debilitating symptoms & constant (potentially invasive) interventions needed to alleviate them (in some cases/diseases) isn’t the gamble worth it? Especially if you have done your homework with your doctor?

While I understand there are unforeseen risks with drugs that are new & have not had long-term testing, there are a few drugs that have been on the market for some time that have been tested & are worth the risk. Especially if the side effects are manageable & quality of life improves.  Why block compassionate use in those cases? Why such slow approvals to change an indication?

Yet it happens all the time, even when other off-label uses have been approved. It seems to take forever with rare diseases & terminal illness when most people in that populace don’t have the luxury of extra time.

If one is facing a grim prognosis & there’s something that could buy them some more quality time, if they’ve weighed out the risks & benefits for themselves, why block access?

I understand the professional reluctance & points raised in this analysis, but with all due respect, most of these bureaucrats making these decisions are not living with the day-to-day devastation of these illnesses.   They’re not stuck weighing the toll that constant interventions are taking on their body or fighting to preserve the quality of life that is slipping through their fingers.

Most people are realistic & pragmatic enough to know that some of these agents won’t cure them or solve all their problems. In some cases, it may only buy them a short period of time.

At a certain point, what do these people have to lose?  Especially if they’ve had discussions with their doctors & have weighed the pros & cons.

While I agree that nonprofits need to protect & come up with their own unique stance & advocacy bent, I feel this article is pretty much anti-compassionate use & right-to-try.  This surprises me because in rare diseases (more often than not) options are limited & most of us in need of a lifeline must take some calculated risks.

My organizations (the LAM Foundation & TS Alliance) I think do take a balanced approach with their advocacy.  Some of the life-threatening experiences those of us have in the LAM & TSC communities has led to honest & frank discussions & finding our own ways to safely fast track research & options in tandem with researchers. Many of these researchers are clinicians who treat & see us at our best & worst.  They see the devastation because they are in the trenches & in the fight with us.

Neither the organizations or researchers believe in taking unnecessary risks that could harm our community & leave those they advocate for in a more vulnerable state.  I realize that not organizations & research operates this way, but to say that there aren’t examples where it does is a glaring oversight.

There are organizations out there who do both test & try & compassionate use pushes the right way.  They aren’t even given a line in this article.

For balance to the community it addresses, I would have liked to see the author point out some instances of success stories & possible case studies where improvement is needed.

Yes, it would have made it longer, but it certainly would have been a more balanced & well-rounded perspective on the subject.

Documentary nut that I am, I have one I’d like to recommend to people are who are interesting in learning more about this subject.

I recently watched an older, but still relevant & well-done documentary that addresses these issues called  Fight To Live. This is a great one to watch to learn more on the subject. Especially if general news & articles like this aren’t your cup of tea.  (A few of the streaming channels on Roku have it for free).

Though the documentary is from 2012, it’s still evergreen.  The documentary shows the devastating ramifications on people’s lives when opportunities for compassionate use are completely missed or purposefully blocked.