Baby Pia Denied Compassionate Use: Something ‘Ain’t’ Right in Basel

Novartis just can’t keep out of bad news. They have denied a request from the Belgian federal government to make the most expensive drug in the world (Zolgensma at $2 million) available to a Belgian toddler named Pia who suffers from spinal muscular atrophy, despite crowdfunding success from her parents.

The Family Raises the Funds

Apparently, Pia’s family worked frantically and furiously to save their 9 month old daughter and organized a crowd-funding campaign, raising € 1.9 million needed to afford Zolgensma, a drug yet to receive approval in Europe.

Novartis Refuses to Entertain the Option

Despite the fundraising, the Swiss drug makers denied the 9-month old access to the drug. Belgian Federal Minister Maggie De Block made a plea to the company to “remain human.” The company denied anyway.

The Drug

The world’s most expensive drug, it has been hailed as a breakthrough for the treatment of SMA, a progressive genetic disease leading to muscular failure. Most children diagnosed with the disease die in infancy, reports Ms. Gabriela Galindo in the Brussels Times.

Compassionate Use Access

As the therapy is not approved on the European market, the Belgian minister noted the “largely public-funded” company should make the drug available under the European Medicines Agency compassionate use program or CUP. CUP enables national governments to request to drug makers to offer the drugs to a suffering patient pending market approval, reports the Brussels Times.

Novartis Rejects: A Generic Letter

De Block’s spokesperson, Audrey Dorigo, reports that Novartis refused a compassionate use request. She continued, “We find that their refusal is not justified,” noting other cases where drug companies offer help in such circumstances.

Novartis refused to commend but did issue a statement, noting “We are in constant dialogue (…) with government bodies to find solutions for early access for all of our innovative treatments for patients who can benefit from them.”

Family Response

Apparently Pia’s parents have tried to contact Novartis multiple times, so it is not a surprise that Novartis is denying their daughter access under compassionate use. The did note that “What is news is the public pressure,” noting they we would be open to speak to Novartis “as soon as possible,” reports Ms. Galindo.

Novartis Mission & Finances

TrialSite News notes Novartis’ mission “is to discover new ways to improve and extend people’s lives. We issue science-based innovation to address some of the society’s most challenging healthcare issues. We discovery breakthrough treatments and find news ways to deliver them to as many people as possible.”

With a current market capitalization of nearly $200 billion, they expect about $54 billion in revenue with $16.9 billion in profit—very healthy. The company sits on over $10 billion in cash.

Integrity & Values Ultimately Impact Brand & Market Cap

Novartis is a publicly traded company and does owe a duty to shareholders to ensure that it generates not only a return but also growing value. It is a business. It also operates in a sector that has significant public supports, including government-created monopolies via the patent process. This, of course, is because of the tremendous expense and risk involved with creation of drug development intellectual property. This is understandable and the way the world works.

Novartis didn’t develop this drug themselves—they obtained it in April 2018 as part of the AveXis acquisition. They paid a handsome $8.7 billion, so they are absolutely justified for seeking a return on their investment.

But along the way something just doesn’t seem right in Basel and it starts with their CEO. We wrote about their shift of business model from volume to value and how pricey acquisitions were part of a fundamental shift for the Swiss biopharma company. We wondered about the model—depending on drugs that cost consumers $2+ million. Of course, there is a rational argument that the costs are far greater for supporting those patients with such conditions as SMA, and we get that. But the reality of the actual market place versus theory can, in all actuality, be fundamentally different worlds. Novartis has made some big bets and they may, unfortunately, be caught in the middle of a deep chasm.

Then we covered the news that Novartis, under the order of the CEO undoubtedly, withheld important information about the AveXis drug and awaited FDA approval before releasing that certain information was manipulated. Frankly, “our gut flipped” from hearing about this story. We have worked with big pharma and understand they are massive organizations with complicated labyrinth of departments and divisions—and stuff does happen— but this kind of call has to come with the blessing of the top.

Patient Engagement

Novartis is investing heavily in patient engagement. Examples are many, but we offer one in the form of the Novartis-supported patient research exchange, which seeks directly to communicate with patients and their advocates on number of core issues involving research—direct patient engagement evidences their intent to work closely, directly with patients.

A very different message materializes in their formal response to Pia’s plea where they note: “only physicians can comment on patients and the potential use of any treatment option.” On the Novartis website, they declare “Novartis works with the patient community around the world to discover new ways to improve and extend people’s lives.” They emphasize “working together” and “respecting and understanding” the patient by “actively seeking out and listening to insights from the patient community to inform decision-making” and the like. They remind the reader in “expanding access to our medicines” that they are committed to offer “tailored and scalable access solutions” but associate it with generics. The company website reminds the reader of their commitment “to exploring options to provide patients with access to our investigational medicines.”

Basel needs to understand that it is moments like this where they can break the mold, take a different approach and show leadership—it might carry some risk of liability but certainly compassionate use programs offer coverage—a new path towards Pia offers something much deeper and more profound for enduring brand and value.