I Like Rare Disease

“I like rare disease.” On the face of it, this statement seems innocuous enough. But as with everything, context here is critical. Who said this?

Was it an internist expressing intellectual curiosity?


Was it a scientist musing over the challenge of research?


Was it a pediatrician wanting to connect her patient with a new therapy?


It was an investment expert I met today. She was explaining what it takes to get funding from her institution. She was asked how she selects the young companies in which she invests. She said it comes down to “liking the story.” When pressed to share her definition of “the right story,” she said “I like rare disease.”

Again, context is everything.

If she had said “there is an unmet need in the rare disease community and by developing drugs to help them we can also build a business,”

I might have felt better.

If she had said “You know there is a HUGE business opportunity in orphan drugs, and how great that people get helped along the way,”

I might have felt better.

If she had said “We can make a ton of money on these drugs, and if patients get helped in the process then that’s okay too,”

I might have felt better.

But she didn’t. And I don’t.

It was just “I like rare disease.” It was just “that’s a successful investment.”

What happened to the patient?

The one who has been sick for a decade?

The one who can’t figure out what’s wrong?

The one who doesn’t know anyone else with the same symptoms?

I fully recognize that this sounds “holier than thou.” (Oh there goes that bleeding heart doctor blathering on about patients again…)

And yes, you could say I am a “pot calling the kettle black.” (Well, Dr. Grabowsky, you are the one who left clinical practice for industry…)

So here’s the deal. I have no problem with people making all of the money in the world – if they don’t hurt anyone along the way. I do have a problem with exploitation, and that’s what this felt like.

There are plenty of people in the investment community taking big risks by funding small companies developing orphan drugs. Without that money, we wouldn’t be slowly working down the number of incurable rare diseases. And for that, I am profoundly grateful. But no one – NO ONE – should “like rare disease” if they are only talking about money.

Yes, orphan drugs are profitable.

Maybe, there are some contexts in which one can like rare disease.

But let’s try to remember:

Living with rare disease is awful.


Tara Grabowsky, MD
Chief Medical Officer, Vencore