On Novartis' Data Manipulation

by Greg Dombal, Chief Operating Officer

The news this past week that scientists at Novartis manipulated data in comparability studies for their gene therapy (Zolgensma) was surprising and disheartening. You can read one of the original articles here. 

Our industry has been plagued by bad actors on the data side and news like this about Novartis is just one example. It is difficult to understand exactly what happened within the walls of Novartis, but the timeline is curious – FDA was notified after approval of Zolgensma. To further complicate matters, FDA says that the comparability data (material used in Phase 1 compared to that of Phase 3) from animals does not change the view that the human results are persuasive. Sounds like the FDA team has recently embraced their inner Machiavelli! Like many others in the industry, this news has me thinking about what may have driven Novartis’ decision making, and what drives mine.  

Gene therapy, and for that matter, any therapeutic destined to go inside the human body, is typically the result of years and years of research; it is said that tens of thousands of ideas fail for each one that becomes a drug. We live in an industry that is eternally optimistic – the next idea will be the biggest and best yet. The idea that a successful treatment is only one experiment away draws many to the field who are passionate about improving the lives of patients and willing to surmount the necessary obstacles. Our push for success, on a patient-centric level, is noble and motivating. Yet, the riches that await a scientist whose idea becomes reality can be intoxicating.  Successes are celebrated and those behind them are idolized. Finance has Buffet and Science has Langer. The podium and the board room await. Understanding whether a person or a company is motivated by the patients or the paycheck is not always clear.

Given what we know of the case of Zolgensma, we could assume the worst. As of now – only a few scientists are going to “be exited” (Novartis might patent that phrase). Sacrificing a few scientists but keeping the spoils does not seem like a complete outcome. I am curious about the Project Leads, the Regulatory people, the lawyers, the Sales people and the Executives who knew about this along the way – do they have culpability?  Or is this just part of the show for Novartis to get to market? Perhaps as the Novartis CEO said in the last investor call, the road to transparency is bumpy, and more changes will come out of this at Novartis.

Or maybe one small issue grew into something of much greater significance without deliberate dishonest intent. It’s easy to see how adjusting a study protocol after a pilot run fails could turn into adjusting a piece of data that doesn’t fit that analysis could turn into data manipulation on a bigger scale. Perhaps no one in the lab thought they were doing anything wrong.  Maybe no one in Regulatory caught on, and maybe no one in Quality audited the studies in question. It is not difficult to imagine being the executive who did not want to rush to judgement before an internal investigation was complete. For if it was a false alarm, why ruin careers, why impact stock price, and why risk taking the drug away from patients–infants born with spinal muscular atrophy, which often leads to death within months? Where is the ethical line in this case?  It’s not clear. 

It will not be long before we hear from a company, with greater confidence than ever, that the animal data doesn’t really matter, or that FDA will have to approve their new drug. And they will can point to Novartis as proof. Our job as expert resources for our clients will continue to be complex – challenging us to constantly evaluate and revisit what the data is, what the data tells us and what our responsibility to patients and our clients demands of us. Doing the right thing is an easy value statement to make, and it seems we all enthusiastically are on board with that value. Defining the right thing and acting appropriately when the right thing is not easy or even clear is our challenge. 

I believe we are a company that truly prioritizes doing the right thing. Without passing judgement on Novartis, it is important to reiterate that Halloran will always strive to do the right thing, as urgently and specifically as we can. Arthur Ashe said the “Success is a journey, not a destination.” Our journey as a company will continue to be a success because we hire people who are great at their jobs AND are willing to relentlessly do the right thing. Halloran will always support us in that effort.