Tagged FDA

Trump’s Promise To Rein In Drug Prices Could Open Dam To Importation Laws

With prescription drug prices soaring and President Donald Trump vowing to take action, an old idea is gaining fresh traction: allowing Americans to buy medicines from foreign pharmacies at far lower prices. A new bill in Congress to allow the practice would modify previous safety standards and remove a barrier that proved insurmountable in past attempts to enable progress.

Congress came close to allowing importation through the Medicare Modernization Act in 2003, but added one firm precondition that has proved a nonstarter. The secretary of Health and Human Services had to guarantee that imported medications posed no additional risk to public safety and would save money.

“That is a fairly absolute standard and a high bar to cross,” said Elizabeth Jungman, director of public health at the Pew Charitable Trusts. Such an exacting standard — guaranteeing that no imported prescriptions posed a threat — has kept any secretary of HHS from condoning it.

In an open letter to Congress, four former commissioners of the Food and Drug Administration argue consumer drug importation remains too risky to permit. “It could lead to a host of unintended consequences and undesirable effects, including serious harm stemming from the use of adulterated, substandard, or counterfeit drugs,” they said in the letter distributed to media organizations. It was signed by Robert Califf, Margaret Hamburg, Mark McClellan and Andrew von Eschenbach, who headed the FDA at various times between 2002 through 2016.

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The recent proposal, from Sen. Bernie Sanders (I-Vt.) and such Democrats as Cory Booker from New Jersey and Bob Casey from Pennsylvania, drops that requirement. Instead, it sets up a regulatory system where Canadian pharmacies who purchase their supply from manufacturers inspected by the Food and Drug Administration would be licensed to sell to customers across the border. The bill allows not only individuals but drug wholesalers and pharmacies to buy from Canada.

After two years, HHS could allow importation from other countries that meet standards comparable to those of the U.S.

(Another bill in Congress, proposed in January by John McCain (R-Ariz.) and Amy Klobuchar (D-Minn.) focuses solely on allowing individuals to purchase from such pharmacies.)

Trump has promised that “pricing for the American people will come way down.” Last week, he had a high-profile meeting at the White House with Elijah Cummings, Peter Welch (D-Vt.) and the head of Johns Hopkins Hospital, Redonda Miller, to discuss allowing Medicare to negotiate prices on outpatient medicines. Cummings told reporters later that Trump said he supports Medicare price negotiation as well as the Sanders bill.

PhRMA, the drug industry’s trade group, has denounced Sanders’ proposal as it has others that enabled imports in the past.

“The bill lacks sufficient safety controls [and] would exacerbate threats to public health from counterfeit, adulterated or diverted medicines, and increase the burden on law enforcement to prevent unregulated medicines and other dangerous products from harming consumers,” said PhRMA spokeswoman Nicole Longo.

Surveys indicate that up to 8 percent of Americans have bought medicines outside the U.S. even though the practice is technically illegal and imported pills are subject to confiscation.

Around 45 million Americans — 18 percent of the adult population — said last year they did not fill a prescription due to cost, according to an analysis of data from the Commonwealth Fund by Gabe Levitt, president of PharmacyChecker.com, whose company helps Americans buy medications online by vetting overseas pharmacies and comparing prices for different drugs. Data compiled by the company comparing prices offered in Canada to those in New York, shows drugs are frequently three times or more as costly in the U.S. as over the border.

For example, a simple Proventil asthma inhaler costs $73.19 in the U.S. vs. $21.66 in Canada. Crestor, the cholesterol-lowering drug, is $6.82 per pill in the U.S. but $2.58 in Canada. Abilify, a psychiatric medicine, is $29.88 vs. $7.58, according to pharmacychecker.com.

Many previous bills to allow importation or to allow Medicare to negotiate prices for its beneficiaries have failed in the face of $1.9 billion in congressional lobbying by the pharmaceutical industry since 2003, according to Open Secrets. But Americans may be reaching a tipping point of intolerance. In polling just before the election by the Kaiser Family Foundation, 77 percent of Americans called drug prices “unreasonable” and well over half favored a variety of proposals to address them.

To address safety concerns, the Sanders bill institutes several new strategies. Canadian pharmacies that want to be registered to sell to Americans would have to pay a fee to pay for additional FDA monitoring. A General Accountability Office study would be required within 18 months of the final rule to address outcomes related to importation processes, drug safety, consumer savings and regulatory expenses.

Allowing people to legally import medications wouldn’t totally solve the problem of high prescription drugs, advocates say, but would be a step in the right direction. Said Levitt: “The best way for Americans to afford their meds is to enact polices here to bring the prices down here.”

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

Categories: Cost and Quality, Pharmaceuticals

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$89,000 Orphan Drug Gets A New Owner — And Likely A New Price

Marathon Pharmaceuticals’ controversial $89,000-a-year drug that has drawn outrage from patients and intense questioning from Congress is getting a new owner.

After striking a deal Wednesday evening, PTC Therapeutics announced plans early Thursday to buy the Duchenne muscular dystrophy drug Emflaza from Marathon for $140 million in cash and stock. The drug’s new price was not announced.

While declining to answer questions about price, PTC Chief Executive Stuart Peltz built up the value of Emflaza, which is a steroid, in an investor conference call. Emflaza, he said, is an “important new drug” and “disease-modifying.”

On price, PTC believes “a change needs to be made, however it’s premature to speculate exactly what that will be,” Peltz said.

Emflaza was approved as an orphan drug with the Food and Drug Administration last month, and PTC executives said they expected it to treat 9,000 Duchenne patients in the U.S. The drug has been available outside the U.S. for decades under the generic name deflazacort.

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It is a steroid used to lessen the symptoms of Duchenne, a fatal muscle-wasting disease that affects mostly boys. For years, many American patients have imported the generic version at a cost averaging from $1,000 to $1,600 annually. The cost typically was not covered by insurers.

But the controversial price difference between the older generic and the newly approved drug has outraged patients and lawmakers.

“It’s just all insane,” said Dana Edwards, a New Jersey mother whose 12-year-old boy uses deflazacort, from overseas, to treat his Duchenne muscular dystrophy. Edwards said the generic costs her about $1,000 a year, and she worries that the PTC purchase will not affect Emflaza’s price.

“Honest to God, I think it’s going to have to remain pretty high,” Edwards said. “I pray to God that they do right by our community.”

Shortly after Marathon announced the price of Emflaza, Sen. Bernie Sanders (I-Vt.) and Rep. Elijah Cummings (D-Md.) sent a letter to the company demanding answers about pricing, saying the drug isn’t new. Marathon executives responded by delaying the drug’s market launch.

In early March, seven Democratic U.S. senators and one independent also demanded answers from the company in a letter that said they’re concerned that the price “exploits” patients. Marathon had responded by saying it was committed to ensuring that all patients had access to the drug.

Sanders and Cummings sent a letter to the FDA this week, asking about the approval process for Emflaza and saying that the price was “especially troubling” in light of the financial incentives Marathon received under the orphan drug approval process.

Those incentives include a seven-year market exclusivity for the drug as well as tax breaks on development and a waive in fees. Marathon also won a pediatric review voucher with its Emflaza approval. And the lawmakers’ letter zeroed in on the voucher program, which Congress approved in 2012 to encourage the development of drugs for rare pediatric disorders. Companies can keep the voucher to expedite the approval process for their next drug or sell it to another company. The vouchers have fetched prices of $350 million.

On Thursday, a Sanders aide confirmed that the lawmakers will continue examining the pricing and approval process for Emflaza.

Marathon executives declined interview requests. But the company sent a letter to the patient community stating that the deal will “create the opportunities needed” to make sure the greatest number of Duchenne patients could get the drug.

PTC’s Peltz, just as Marathon’s executives said last month, told investors that the company would reach out to the Duchenne community, physicians and payers. The company plans to disclose its price after the acquisition is finalized, which is expected by the end of June.

As part of the deal, Marathon will be entitled to payments based on Emflaza’s sales plus a one-time $50 million payment when the drug reaches an undisclosed sales-based milestone. In addition, Marathon executives will remain on the transition team to aid in the drug’s U.S. rollout.

PTC sees Emflaza as a first step into the U.S. Duchenne market, Peltz said. The company talked of pursuing a pediatric approval for the drug’s use.

PTC has another drug, Translarna, which received conditional approval for use in the European market to treat people with a specific mutation of Duchenne. The drug targets a different set of patients than those treated by Sarepta Therapuetics’ Exondys drug. It is currently being reviewed for use in the U.S.

Parent Project Muscular Dystrophy, a patient advocacy group, released a statement saying that PTC has a “long history” with the community. PPMD’s statement urged PTC to be “transparent about the methodology they will use when establishing their pricing.”

“The players may have changed,” PPMD said, but that doesn’t mean concerns about pricing have lessened.

KHN’s coverage of prescription drug development, costs and pricing is supported in part by the Laura and John Arnold Foundation.

Categories: Cost and Quality, Health Industry, Pharmaceuticals

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By The Numbers: Trump’s Choice For FDA Chief Is Versatile, Entrenched In Pharma

President Donald Trump’s pick to lead the Food and Drug Administration has deep ties to the pharmaceutical industry as a consultant, investor and board member. Scott Gottlieb, 44, also has worn many hats in a career that included two previous stints at the FDA, practicing as a physician, and writer/editor roles at prestigious medical journals.

“It seems like the main question is, ‘Which Gottlieb are we going to get?’” said Dr. Robert Califf, who stepped down from his position as the commissioner of the Food and Drug Administration in January.

Here’s a look at Gottlieb’s career, by the numbers:

188 Companies

New Enterprise Associates, the venture capital firm where Gottlieb is a partner, is currently or has been invested in 188 health care companies.

8 Boards Of Directors

Gottlieb serves or served on eight boards of directors, according to his LinkedIn profile. The firms include pharmaceutical companies Gradalis and Tolero Pharmaceuticals, which are developing cancer treatments, among other things; and Glytec, which offers glycemic management tools for patients with diabetes.

8 Drug And Device Companies

Eight pharmaceutical companies disclosed payments to Gottlieb in 2015, according to the open payments database: Vertex Pharmaceuticals, GlaxoSmithKline, Daiichi, Valeant, Pfizer, Millennium, SI-BONE and E.R. Squibb & Sons. Payments included travel to Philadelphia, San Francisco and London.

9 Recusals

In a memo, Gottlieb wrote that he had a consulting relationship with nine health care companies before his stint as the FDA’s deputy commissioner for medical and scientific affairs during the George W. Bush administration. Those ties disqualified him from dealing with matters concerning those companies for at least a year. The firms included Eli Lilly, Roche and Sanofi-Aventis.

The recusals generated some headlines during the avian flu scare because Gottlieb had to recuse himself from some of the planning efforts around vaccines.

7 Years

Starting in 1997, Gottlieb spent seven years as a staff writer for BMJ, the British Medical Journal. BMJ is one of the top medical journals in the world. And he was an editor at the Pulse section of JAMA, the Journal of the American Medical Association, from 1996 to 2001.

Gottlieb also spent the past seven years as an adviser to drugmaker GlaxoSmithKline’s product investment board, according to his LinkedIn page.

33 Years Old

Gottlieb was 33 when he got the No. 2 job at the FDA in 2005. He had done a short stint at the agency a few years earlier, but was considered a controversial pick because of his ties to Wall Street. He stayed until 2007.

“He has done a lot of thinking about how FDA should be managed — his operational sophistication is going to be a great asset,” said former FDA attorney Coleen Klasmeier, who worked with Gottlieb and co-authored articles with him. “He also has very strong relationships among career FDA personnel and will be able to hit the ground running on a range of important initiatives.”

18 Times Before Congress

Gottlieb testified before Congress as an expert witness 18 times. He has spoken about drug prices, revamping the FDA approval process and the vaccine supply.

“Most people watching at the FDA would breathe a sigh of relief,” Dr. Joshua Sharfstein, who served as the FDA’s principal deputy commissioner until 2011, said of Gottlieb’s impending nomination. He added that Gottlieb is “not someone who has expressed antagonism to the core principles of the agency.”

36,000 Twitter Followers

More than 36,000 people follow Gottlieb on Twitter, though he follows only 845 people, as of Tuesday. He has tweeted at least 10,400 times, lately about different strains of the flu.

72 Percent

Almost three-fourths of the 53 drug companies surveyed by Mizuho said they’d prefer Gottlieb to head the FDA.

$414,000

Pharmaceutical companies paid Gottlieb nearly $414,000 from 2013 through 2015, according to federal open payments data, for speeches, consulting, travel and meals.

That included $65,780 from a pharmaceutical company to promote a controversial cystic fibrosis drug called Kalydeco. Only one other doctor received more money toward promoting the drug.

The drug’s price tag was controversial because the nonprofit Cystic Fibrosis Foundation kicked in $150 million toward finding a cure for the fatal disease and got a rich $3.3 billion payday for selling its rights to royalties for the drug. Vertex Pharmaceuticals priced Kalydeco at more than $300,000 a year.

Nearly $30,000

Gottlieb has contributed nearly $30,000 toward Republican political campaigns and joint fundraising committees from 2005 to 2014. He has donated toward the presidential runs of Mitt Romney and Sen. John McCain. He also donated more than once to Speaker Paul Ryan.

Gottlieb contributed the most money toward Republican Sen. Ben Sasse of Nebraska, spending $2,600 in the primary and $2,600 again in the general election in 2013. Sasse was a vocal anti-Trump supporter, penning an open letter in February 2016 about how he could not support Trump, who was “dividing” the nation, in his view.

“But have you noticed how Mr. Trump uses the word ‘Reign’ — like he thinks he’s running for King?” Sasse asked in the post.

Categories: Health Industry, Pharmaceuticals, Syndicate

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