A group of senators wants U.S. regulators to use their full authorities to hold Novartis AG and its subsidiary AveXis Inc. accountable for manipulating preclinical data to obtain approval of Zolgensma, a gene therapy that sells for $2.1 million — the most expensive medicine in American history.
Last week, the U.S. Food and Drug Administration said it had been informed that data in Zolgensma's U.S. marketing application had been manipulated. Novartis had known about the data manipulation since March — two months before the drug won approval — but waited until late June to tell the FDA.
Acting FDA Commissioner Ned Sharpless
"This scandal smacks of the pharmaceutical industry's privilege and greed, and Americans are sick of it," Sen. Dick Durbin, D-Ill., the Senate's Democratic whip, and four other senators told Acting FDA Commissioner Ned Sharpless in an Aug. 9 letter.
"Anything short of a forceful response would signal a green light to future pharmaceutical misbehavior," the senators said.
The lawmakers also wanted to know why the Trump administration withdrew a proposed rule in October 2018 that would have required sponsors of certain clinical trials to promptly report suspected data manipulation to the FDA and asked if the agency would reconsider reissuing the regulation in light of the Novartis situation.
"We have received the letter and will respond directly to the senators," FDA spokeswoman Sarah Peddicord told S&P Global Market Intelligence.
Trump importation ideas criticized
Meanwhile, critics and policy experts continued to raise doubts about the feasibility of two pathways the Trump administration outlined on July 31 as potential courses of action for importing prescription medicines into the U.S. as a way to lower Americans' drug costs.
Even the U.S. Department of Health and Human Services does not appear eager to implement the ideas, which face a number of roadblocks and could take years to put into place, said Rachel Sachs, an associate professor of law at Washington University in St. Louis.
HHS Secretary Alex Azar had previously called drug importation a "gimmick," though in recent months he said the administration was searching for a safe way to allow it.
HHS' two ideas, however, rely on other parties — states, wholesalers, pharmacies and manufacturers — to do the federal government's work for it on demonstrating whether drug importation can be done safely and effectively, Sachs wrote in the policy journal Health Affairs.
HHS has not provided a timeline for officially proposing the pathways, which involve pilot projects run by states, wholesalers and pharmacists for importing medicines from Canada and a complicated process that would let manufacturers sell products intended for foreign markets in the U.S.
The FDA's Sharpless acknowledged there were "many operational challenges" ahead that must first be addressed.
In an Aug. 8 blog, the Pharmaceutical Research and Manufacturers of America, which lobbies for brand-name drugmakers, noted it is not alone in opposing the Trump administration's importation ideas and identified a number of organizations that have stated they were against the two pathways.
Officials in Canada also criticized the Trump administration's plans, saying they were concerned the proposals, if implemented, could lead to drug shortages in their country.
In an Aug. 6 tweet, Canada's Minister of Health Ginette Petitpas Taylor said she plans to convene a meeting on Aug. 12 of pharmacists, patients, industry and experts to discuss HHS' importation ideas and the next steps to safeguard her nation's prescription drug supply.
Sen. Chuck Grassley, R-Iowa, who has partnered with Democrats on drug importation legislation, said if the Trump administration moves forward with its two pathways, it should at least reinstate surprise inspections of foreign manufacturing facilities.
In his Aug. 6 letter to Azar and Sharpless, Grassley said he was also concerned that it was not clear how drugs intended for foreign markets imported into the U.S. by manufacturers would be tracked and traced.
Trump targets September
White House Counselor Kellyanne Conway last week confirmed reports that President Donald Trump plans to unveil his healthcare plan in September, though she said it would not be a replacement for the Affordable Care Act.
President Donald Trump
"We're not here to repeal and replace Obamacare," Conway told reporters Aug. 7.
She said the core provision in Trump's health plan would ensure that Americans with preexisting medical conditions could not be discriminated against by insurers — protections already granted by the ACA.
Conway said the White House was "waiting with bated breath" on whether the U.S. Court of Appeals for the 5th Circuit would uphold a district judge's ruling that the ACA is unconstitutional.
If the courts do not dismantle the ACA, Sen. Lindsey Graham, R-S.C., last week said Republicans on Capitol Hill would try again to repeal it.
Conway also said drug pricing was a piece of Trump's healthcare plan, though the White House would not confirm if the president would include HHS' pending proposal to test an international pricing index model as part of his September package.