The System Lost Me

A few weeks ago I hit a rare mom bottom. The last one. I was told my daughter’s Expanded Access could be at risk, and I sat up all night nauseated, heartbroken, staring into the dark like my body already knew something my mind was still trying to outrun. This was the drug I had fought for relentlessly. This was the fight I had given six years to. Six years I will never get back, and for what? So she could still lose access?

A regulatory setback, a Complete Response Letter, Warning Letters, a missed endpoint in a tiny and heterogeneous population does not exist in isolation. It triggers a cascade. Stock collapses, funding evaporates, and confidence disappears. A company that was advancing a therapy becomes vulnerable overnight not just scientifically, but financially and structurally. This is the moment no one accounts for. I remember being in a meeting and asking after an error in docmunetation had been disclosed that compromised the study my daughtr wa in… I said “so we did all this for nothing” and the room fell quite.

We need to start with something uncomfortable. In rare disease, collapse has quietly become part of the model—not because the science fails, not because patients disappear, but because the system cannot carry innovation through uncertainty. And when that collapse happens, something predictable follows. Companies like Cycle Pharmaceuticals step in and acquire distressed rare disease assets at a fraction of their prior value. These moves are often framed as rescue—stepping in where others could not continue. On paper, that sounds like stability. In reality, for many patients and families, it feels like something very different, because by the time an acquisition happens, the damage has already been done…. long before even aquisition was a thought. There is a whole market in buying up rare diseasse companies post FDA CRLs.

I had already done everything families are told to do. Learn the science. Speak carefully. Be credible. Advocate harder. Stay grateful. Keep going. I did all of it because I thought if I pushed long enough, if I said it clearly enough, if I kept showing up with facts and force and discipline, someone inside industry or at the FDA would eventually meet this moment like a child’s life actually sat in the middle of it.

But that is not what this system was built to do.

It was built to protect institutions first. Shareholders. Optics. Timelines. Regulatory theater. Corporate survivability. Everybody loves the word innovation until a real child depends on it. Then suddenly access becomes conditional, urgency gets buried under process, and the people with the least skin in the game still get the most control. Families carry the fear. Children carry the risk. Everybody else gets to call it complexity.

I am done with that arrangement.

What broke that night was not my love for my daughter. Not my willingness to fight. What broke was the last piece of belief I had in this game. The industry version. The FDA version. The whole polished game where power moves slowly, speaks softly, and still expects families to bow to it while our children absorb the consequences. I do not have that kind of reverence left in me.

I have seen too much.

There is something clarifying about hitting a bottom so hard it burns the fantasy out of you. You stop wanting a seat at the table. You stop begging broken systems to become moral. You stop spending your best energy trying to be legible to people who will never understand what it costs to live inside this kind of uncertainty. And a different question starts to take shape: what would we build if we stopped building for them?

That is where I am now.

I want to put my middle finger up and ride straight into the sunset of new dreams of innovation and design that belong to patients. Not shareholders. Not regulators. Not the people who can debate risk because it is never their child paying for the outcome. I want to build things that work for my little girl and for other families like mine. Things shaped by real life. Things that do not make access feel like ransom. Things that begin where the burden actually lands.

Because mothers like me know things this system keeps refusing to value. We know where care breaks. We know where the language turns fake. We know which parts are theater and which parts help a child live. We know what it looks like to sit through the polished versions by day and come apart alone at night. We know how many promising things die in rooms where nobody has to take that grief home.

And once that knowledge settles into your body, it changes what ambition looks like.

Maybe the next real innovation will not come from the people protecting the gates. Maybe it will come from the mothers they exhausted. The families they underestimated. The patients they kept reducing to endpoints, case counts, and strategy decks. Maybe the future gets built by people who have finally lost respect for a system that keeps asking their children to carry uncertainty as the price of someone else’s caution.

There is a quiet defiance, and calming peace in that realization. I will build this better because it begins with my daughter. And nothing built for shareholders can ever outmatch something built from a mother’s love.