FDA – R&R – Reachout and Rebuild

Tom Price Bought Drug Stocks. Then He Pushed Pharma’s Agenda in Australia.

David Posnett — Thu, 01 Jun 2017 21:26:12 +0000

Before he was named Trump’s health secretary, Price took a congressional trip to Australia and pressed officials to extend protections for drug companies in an international trade agreement.

by Robert Faturechi
ProPublica, June 1, 2017, 8 a.m.

In the spring before the 2016 presidential election, the Obama administration’s 12-nation trade agreement known as the Trans-Pacific Partnership, or TPP, was still alive. Negotiators worked on details as Congress considered whether to ratify the pact.

The Australian government was getting in the way of one change demanded by U.S. pharmaceutical companies. Makers of cutting-edge biological drugs wanted to have data from their clinical trials protected from competitors for 12 years, as they are under U.S. law — not the roughly five years permitted under the TPP. Australian officials insisted that an extension would deprive consumers of cheaper alternatives for too long.

On April 5, 2016, a bipartisan group of U.S. lawmakers arrived in Canberra, Australia’s capital, for meetings with government officials on a broad range of subjects. Among those on the routine congressional trip was Rep. Tom Price, a Georgia Republican who would go on to become President Trump’s secretary of health and human services. Three weeks before the trip, Price had purchased up to $90,000 worth of pharmaceutical stocks — trades that would come under scrutiny after his nomination to Trump’s cabinet.

In Canberra, Price and another Republican, Rep. John Kline of Minnesota, pressured senior Australian trade officials to modify their position on the 12-year extension, according to a congressional aide who was on the trip. The Australians explained that they had no intention of changing their laws or rules in ways that could increase drug prices. Price and Kline continued pushing, according to the aide, asking for a side letter or other written guidance that the period would be extended in Australia even if it weren’t spelled out in the TPP itself.

Price’s lobbying abroad, which has not previously been reported, is another example of how his work in Congress could have benefitted his investment portfolio. He traded hundreds of thousands of dollars’ worth of shares in health-related companies while taking action on legislation and regulations affecting the industry. ProPublica previously reported that Price’s stock trades are said to be under investigation by federal prosecutors.

Price, who did not respond to an interview request for this story, has said he did nothing wrong, that his broker generally chose stocks without his knowledge and that all of his trades were publicly disclosed.

Price’s financial disclosures submitted to the House Office of the Clerk show that on March 17, 2016, he purchased shares worth between $1,000 and $15,000 each in Eli Lilly, Amgen, Bristol-Meyers Squibb, McKesson, Pfizer and Biogen. All six companies had an interest in biological drugs, which are grown from live cells and are known for short as biologics. Eli Lilly, for example, is behind Portrazza, the first biologic approved to treat a common type of lung cancer. Amgen makes a top-seller for rheumatoid arthritis and psoriasis. Biogen developed a biologic for people suffering multiple sclerosis relapses.

Kline, who has since retired from Congress, said he could not recall if he or anyone else raised the biologics issue. His financial disclosures do not show direct holdings in pharmaceutical companies.

Australia has played another role in Price’s financial activities. In 2015 the congressman bought about $10,000 worth of shares in Innate Immunotherapeutics, a small biologics firm with an office in Sydney. After the congressional trip, which also made a stop in Sydney, Price purchased a larger stake in the company, about $84,000 worth, in two private placements, the first of which was announced in June. Price was invited to purchase the shares at a discounted rate.

It’s not known if Price had any contact with the firm while in Sydney. Price didn’t respond to questions about when and where he discussed the discounted offering with company officials. The company’s officials also did not respond.

Traveling congressional delegations typically meet with a variety of local officials, and at the time of the visit to Australia it wasn’t unusual for Republican lawmakers to side with the pharmaceutical industry on the trade deal’s protections for biologics. Price’s advocacy stands out because he pushed the cause directly with foreign officials, while at the same time owning stakes in companies that could have benefited.

An itinerary for the trip reviewed by ProPublica mentions TPP in relation to one of the meetings, but does not list the biologics provision. A former Australian trade official, who asked not to be named and attended one of the meetings, confirmed that the 12-year lockup was addressed, but said he could not recall which Congress members were pushing it.

Others on the trip, organized by the House’s Education and the Workforce Committee, were Robert Scott, D-Va., Ruben Hinojosa, D-Texas, Erik Paulsen, R-Minn. and Dan Benishek, R-Mich. Those members who responded to requests for comment said they could not recall whether the provision was discussed.

The data collected during clinical trials of drugs can save competitors time in developing the cheaper alternatives to biologics known as biosimilars. Keeping the data proprietary longer extends the original drugmaker’s monopoly. While some big brand-name pharmaceutical companies also make biosimilars, they and their trade association — the Pharmaceutical Research and Manufacturers of America — advocated strongly for longer exclusivity.

In the end, the debate over the provision became moot. Trump scrapped the TPP days after taking office. Price divested his drug stocks upon taking the cabinet post. His investment in Innate Immunotherapeutics yielded a profit of at least $150,000.

Another Trump attack on the FDA

David Posnett — Sun, 05 Mar 2017 06:06:45 +0000

I have previously warned about defanging the FDA. The FDA (Federal Drug Administration) has saved this country from major catastrophies like the malformations (see title image) caused by thalidomide taken during pregnancy. This reached worldwide epidemic numbers in the 1950s but the US was largely spared because the FDA did not approve the drug.

In last week’s address to Congress Trump did it again. He wants to do away with FDA regulations. It is a terrible idea. Here is a great article on the subject by David Hilzenrath. It appeared on March 3rd on pogo.org.

In his address to Congress this week, President Trump argued that slashing regulatory restraints at the Food and Drug Administration (FDA) could unleash medical miracles. To make his case, he pointed to 20-year-old Megan Crowley, who was expected to die years ago until her father helped develop a drug for her rare disease.

“[O]ur slow and burdensome approval process at the Food and Drug Administration keeps too many advances, like the one that saved Megan’s life, from reaching those in need,” Trump said.

Crowley’s story is a moving one, but a closer look at that story and at the FDA more generally could point to very different conclusions.

The FDA approved Myozyme, the drug that saved Crowley from Pompe disease, in April 2006, nine months after drug-maker Genzyme submitted its application to market the drug. That illustrated a sharp decline over recent decades in the amount of time the FDA takes to review new drugs.

In 1993, the approval time was typically 19.1 months for drugs and biologic products involving a new medicine, according to FDA data. As of 2016, the median time to approval was 7.8 months, the FDA reported.

The FDA approved Myozyme despite finding that the drug itself could hurt people. The label the FDA negotiated in 2006 prominently warns of potentially “life-threatening” side-effects, adding: “BECAUSE OF THE POTENTIAL FOR SEVERE INFUSION REACTIONS, APPROPRIATE MEDICAL SUPPORT MEASURES SHOULD BE READILY AVAILABLE WHEN MYOZYME IS ADMINISTERED.” The agency’s approval seemed to reflect the philosophy that, for patients suffering from a terrible and otherwise incurable disease, a chance at life—or a chance at an improved quality of life—can be worth potentially deadly risks.

An internal FDA memo dated April 27, 2006—the day before the FDA approved Myozyme—shows why it is important for the FDA to scrutinize research on experimental drugs before approving them.

Under the heading “Outstanding Safety Issues,” the memo says an FDA review team “has come to have concerns that the applicant applied an overly narrow interpretation as to what safety information should be presented to the FDA.”

“The review team also has serious concerns that significant safety information that should have been clearly identified, analyzed, and discussed” in a safety summary “was effectively obscured in the way it was presented in the application.” For example, according to the memo, the FDA learned from “a third party” that a patient chose to stop treatment after a reaction to the drug led to a stay in an intensive care unit. The application to the FDA apparently did not explain that that patient discontinued use of the drug due to an adverse event, the memo says.

“It is the opinion of the review team that in order to evaluate the safety of this product adequately it will be necessary to obtain additional information from the applicant, and additional time will be needed to assess the significance of the new information,” the memo says.

As noted, the drug was approved the next day.

Though many drugs undergo clinical testing in thousands of patients before the FDA approves them, the testing of Myozyme reflected the fact that Pompe is a rare disease. Margaret Hamburg, a former FDA commissioner, cited the agency’s approval of Myozyme as evidence of the agency’s “considerable regulatory flexibility.”

“[I]n 2006 we approved a treatment for Pompe Disease—a rare genetic disorder that causes debilitating muscle weakness—on the basis of a single pivotal study of 18 patients,” Hamburg said in a 2011 speech.

Does Genzyme think the FDA’s handling of Myozyme was “slow and burdensome?” We asked a company spokeswoman that question by email Wednesday. In a reply on Thursday, the spokeswoman, Lisa Clemence, declined to comment. Efforts to reach biotech executive John F. Crowley, the father of Megan Crowley, were unsuccessful.

Years after Myozyme won FDA approval, the FDA issued a public warning that Myozyme and other Genzyme products could be contaminated. “The foreign particles, believed to be found in less than 1% of products based on product lots assessed to date, include stainless steel fragments, non-latex rubber from the vial stopper, and fiber-like material from the manufacturing process and could potentially cause serious adverse events in patients,” the FDA warned. Again, the FDA showed forbearance, allowing the potentially compromised drugs to remain on the market.

“The Agency is acutely aware of the critical need for patients to have continued access to these important products,” the FDA said.

In an article about Trump’s FDA critique, Julia Belluz of Vox wrote this week that the major impediment isn’t getting the FDA to approve new medicines; it’s the scientific difficulty inherent in discovering or devising those medicines.

“It’s not the FDA that gets in the way of innovation—it’s the limits of our knowledge,” the article said.

As recent history and reporting by the Project On Government Oversight show, the public interest may be endangered by an FDA that places too little emphasis on the safety and efficacy of the drugs it is reviewing, by haste and lack of thoroughness, and by the pharmaceutical industry’s drive for potentially looser drug approval standards.

Last year, the FDA approved a $300,000-a-year drug for another devastating disease—Duchenne Muscular Dystrophy—despite having concluded that there was no good evidence the drug actually worked. As the head of the FDA at the time wrote, the drug’s “clinical benefit has not yet been established.”

A top FDA scientist who argued against the drug’s approval expressed himself more bluntly.

“By allowing the marketing of an ineffective drug, essentially a scientifically elegant placebo, thousands of patients and their families would be given false hope in exchange for hardship and risk,” Ellis F. Unger, director of the FDA’s “Office of Drug Evaluation – I,” wrote in a July memo.

“I argue that this would be unethical and counterproductive,” Unger wrote. “There could also be significant and unjustified financial costs—if not to patients, to society.”

It’s understandable that desperate patients and their families might seize upon any chance of a cure or treatment, no matter how slim. But approving drugs on that basis entails tradeoffs. As POGO has reported, it could not only harm patients but also spur wasteful spending by consumers, private health insurers, and government programs.

Approving unproven drugs, or approving drugs on the basis of unscientific evidence, could be tantamount to writing a blank check to drug companies, contributing to unsustainable health-care costs.

It could divert spending from more promising treatments, and it could dilute economic incentives for researchers and pharmaceutical firms to come up with cures that actually work and work safely instead putting patients at greater risk.

By: David S. Hilzenrath
Chief Investigative Reporter, POGO

David Hilzenrath is the Chief Investigative Reporter for the Project On Government Oversight.

FDA – R&R – Reachout and Rebuild

Tom Price Bought Drug Stocks. Then He Pushed Pharma’s Agenda in Australia.

The Trump Administration

Another Trump attack on the FDA