The rare disease world, and especially the non-precisely-defined ultra rare disease world, is a small one. You know the players. You meet over and again. Your pharma company knows your name and you’ve probably met many of their people in person.
We play on the delicately balanced chess board of profits and life, business and family, shareholders and children. Notice that I could have said profits versus life, business versus family, and shareholders versus children. But that would not have been entirely appropriate. In many circumstances, the business objectives of a rare disease pharma company align with those who would benefit from their treatment.
Develop a treatment? Patients are helped, company makes profits…. Good for all.
Develop a patient support program? Patients are encouraged and helped, patients are more likely to continue treatment, payors more likely to understand and pay…. Good for all.
Raise awareness? Families are more supported, payors are more aware, children are diagnosed earlier…. Good for all.
Diagnose more children and diagnose them earlier? Treatment started sooner…. Good for all.
But when the interests of profits and patients collide, who is the winner? Is it ever the patient? Should it be? It is difficult to dissect a solution.
Shouldn’t a pharma company, any company, make wise business decisions? Decisions that are in the best interest of their shareholders? Also, their employees, who also have families and children and need a paycheck each week?
But then again, aren’t there obligations to a patient population that depends on that company? Or was led to believe that they could, at present and in the future, depend on that company?
Again, rare disease is a small world. In many cases, the patient population may significantly overlap with the investor population. I read investor call transcripts, 10Ks, 8Ks, annual reports. And I would venture to say that I’m not alone.
So when I read about a situation such as this, I have great pause, because I can feel the pull of my securities lawyer/strategic consultant brain against my parent-of-a-child-with-a-similar-rare-disease-who-gets-his-current-and-clinical-trial-drugs-from-the-same-company brain. Please read this article.
I have several parting thoughts that come to mind:
I look forward to your comments.