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Our Take: FDA threatens legal action against Novartis over faulty data used in application for $2.1 million gene therapy

Aug 12, 2019

The FDA announced Tuesday that Novartis submitted the drug application for Zolgensma, the company’s novel gene therapy treatment for spinal muscular atrophy, with manipulated data. The FDA said it is assessing the matter and “remains confident” that the gene therapy will continue to be available to patients.

Echoing a statement from the agency’s press release, acting FDA commissioner Dr. Ned Sharpless tweeted that the agency “will use its full authorities to take action, if appropriate, which may include civil or criminal penalties.”

“It is the manufacturer’s responsibility to submit complete and accurate information in marketing applications for evaluation by the FDA,” the agency wrote in a statement. “If we become aware of a concern with data submitted to the agency as part of our review of a product application, it is in the best interest of patients, their caregivers, and the public that we disclose such information, to the extent permitted by law.”

Our Take: We’re covering this story as the lead because it grabbed a lot of headlines in the news cycle last week. What caught people’s attention was the acting FDA commissioner’s tweet that alleged potential criminal liability.

Best as we can tell, here’s what happened.

The FDA approved Zolgensma on May 24. (We wrote about its $2.1 million price tag on June 3.) A month later, AveXis Inc. — the product’s manufacturer, which Novartis owns — told the FDA about a data manipulation issue related to product testing in animals that was included in the company’s Biologics License Application (BLA).

The FDA says AveXis knew about the data manipulation before submitting the BLA, and apparently Novartis isn’t disputing that. FiercePharma reported that during a conference call on Wednesday, Novartis executives said a scientist at AveXis reported the manipulated data in mid-March and the company launched an internal investigation, which confirmed by early May that the scientist was correct. AveXis didn’t inform the FDA until June 28.

Vas Narasimhan, Novartis’ CEO, said on Wednesday’s call that the company had been conducting a “full technical investigation” in May to fully understand the issue. He added that Novartis would have shared the results of its investigation with the FDA before the drug was approved if the investigation had been completed sooner.

We don’t know what was behind Novartis and AveXis’ motivation to delay informing the FDA, nor do we have any idea how they manipulated the data. But for those who get their news by reading headlines, there are a few important facts to bear in mind.

First, the data issue, as the FDA acknowledges in its statement, occurred during animal testing — not during the crucial human trials that demonstrated the drug’s safety and efficacy.

Second, the FDA says it is “confident that Zolgensma should remain on the market.”

Bottom line: While the headlines have been sensational, there isn’t much to this story.

And let’s not forget that Zolgensma is truly a breakthrough therapy, offering hope for parents whose children were previously subjected to a horrible quality of life and certain death.

Novartis says it is taking steps to address the incident, such as “exiting the small number of AveXis scientists involved in these data inaccuracies” and hiring 1,000 new AveXis employees, and the FDA will determine the appropriate regulatory/legal action(s) to take. Fortunately, in this case, it looks like there hasn’t been and won’t be any harm done to patients.

You can read Novartis’ official statement on the matter here.

What else you need to know
Allscripts Healthcare Solutions reached a tentative $145 million settlement agreement with the Department of Justice in connection with investigations into “certain business practices” of Practice Fusion, an electronic health records vendor Allscripts acquired last year for $100 million (of note, Allscripts reportedly initially offered to pay $250 million). The investigations are centered on Practice Fusion’s compliance with the anti-kickback statute and HIPAA, as well as how the company obtained its software certification. In a recent earnings statement, Allscripts said it believes the agreement “will be sufficient to resolve all potential civil and criminal liability in connection with these investigations.” 

CMS haagreed to reimburse any FDA-approved (CAR) T-cell therapies, the agency announced Wednesday. CAR T-cell therapies are used to fight certain cancers by using a patient’s own genetically modified immune cells. CMS said it will cover CAR T-cell therapies when they are provided in health care facilities enrolled in the FDA’s Risk Evaluation and Mitigation Strategy (REMS) programs for FDA-approved indications and for off-label uses that are recommended by CMS-approved compendia. 

SSM Health is introducing an individual health plan in the St. Louis market this fall, for coverage to begin Jan. 1, 2020. The plan, called WellFirst Health, will utilize SSM Health’s network of physicians, hospitals, and clinics and will be offered on the ACA’s Health Insurance Marketplace. The plan is the health system’s first foray in the insurance market in St. Louis; it currently operates Dean Health Plan in Wisconsin, which provides coverage to about 400,000 members.

Provention Bio’s teplizumab received the FDA’s Breakthrough Therapy Designation, the company announced last Monday. Teplizumab, an investigational anti-CD3 monoclonal antibody, is being developed to prevent or delay clinical type 1 diabetes in at-risk individuals. The designation will expedite the drug’s development and regulatory review.

New York City launched NYC Care, a program that offers health care to uninsured residents who have lived in the city for at least six months and cannot afford or do not qualify for health insurance. For now, members can receive care at one of seven locations in the Bronx operated by NYC Health + Hospitals; the program will be implemented citywide by year-end. A statement by the governor’s office noted that about half of the city’s 600,000 uninsured residents are likely eligible for enrollment in NYC Care.

CVS has expanded its CarePass program to participating CVS pharmacies throughout the U.S., the company announced last Monday. For a membership fee ($5 monthly or $48 annually), members can get one- to two-day home delivery of qualifying prescription drug orders and other store items at no extra charge. Members also have access to a 24/7 pharmacist helpline and receive discounts on certain health-related CVS products. 

Walgreens customers can soon get select prescription Bausch Health dermatology products for a flat rate of $50 to $115 through Dermatology.com, a cash-pay prescription program Bausch Health launched in March. The two companies announced an expanded agreement on Aug. 1 that will give patients access to the prescription meds at more than 9,500 U.S. Walgreens pharmacy locations by the end of this month.

What we’re reading
Patient Assistance Programs and Anti-Kickback Laws. JAMA, 8.6.19 (subscription required) 

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