Reversal of Fortune? Where Profits and Patients Collide

Posted Monday April 09, 2012 by Melissa

Collision of two chess figures, pawn and kingThe 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.

Shire Reversal on Rare Disease Drug Application Stumps Patients

I have several parting thoughts that come to mind:

  • Is this a power play on behalf of Shire against the FDA to back down on any suggested additional trials? (I have read no other suggestion of this; this is my musing.)
  • If it is a power play, as a strategist, is it one I would have recommended be played? I mean, the downside is patient disloyalty and distrust, not to mention that of the FDA, the upside potential being the elimination of the time and cost of additional trials. There are so many variables here, not the least of which are financial projections, profit margins, and market share versus Genzyme/Sanofi’s competing product Fabrazyme. Again, I have read no other suggestion of my hypothetical, but I ruminate on these things ever since my strategy brain somehow crossed wires with my personal, rare disease, mom brain.
  • Is this a wait and see approach since the drug is already approved in Europe and could they possibly, at a later time, navigate the process more quickly? The downside being that it gives Genzyme/Sanofi time to re-establish patient loyalty to their product.
  • Is there some information or data that Shire did not want to emerge from either these meetings or further trials other just the fact that they’d have to pay for additional trials and wait longer for approval?
  • At least Fabry patients have available another FDA-approved treatment. That is not to minimize this situation and the struggle that has occurred since Genzyme’s plant was infected with a virus and reduced or eliminated many patients’ dosing, but with so many rare diseases without treatment, I am at least happy for that. Without an alternative, I think the uproar from this decision would have been astounding.
  • I feel a little more tension and trepidation as rare disease pharma seems to become more big pharma-ish. Decisions at a more macro level cause palpitations in the mom-side of me, my child receiving a weekly drug and a clinical trial drug from Shire as well.
  • Drug clinical trials, FDA approvals, and market decisions are never in stone, until they’re in stone. While my child’s clinical trial drug is amazing, I have to remember that there are more factors involved in whether this is someday available to every child with Hunter Syndrome. Until then, however, you’ll hear my voice, loud and clear.

I look forward to your comments.

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2 Responses to “Reversal of Fortune? Where Profits and Patients Collide”

  1. Replagal was in competing clinical trials with Frabrazyme at the same time as Aldurazyme was in clinical trials and many patients including Fabry patients I know personally did feel Replagal worked better – but Fabrazyme won the clinical trial race and was FDA approved first here in the US. I think this is crap and patients should have a choice and if they chose Replagal then they should be able to do it; likewise if patients feel comfortable on Fabrazyme they should be able to use that. Seeing every other week (I am weekly, these patients are bi-weekly) how these patients feel and being very close with some of these patients and knowing the change they experienced from being in the Replagal clinical trial to then having to go on Fabrazyme I just think this other drug offers benefits to Fabry patients. Clearly it is working well in other countries where patients have the choice of which drug to use!

    Erica – MPS I Adult

  2. Interesting information!