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It was an alluring pitch. Patidegib was chemically similar to Erivedge but supposedly safer: By rubbing the stuff on to skin, the thinking went, you could avoid the taste loss, hair loss, and muscle cramps that came when you swallowed it — reactions that kept some from taking the pills. ...To scientists, it’s still an exuberant story of discovery against the odds. To patients like [Kaylene] Sheran, it’s a story tempered with worry: Every side effect, it seems, has been prepared for except financial toxicity. (Boodman, 10/23)

It all began with a hypothesis: That the biology behind Gorlin might hold the secret to other cancers. After tracing this rare disorder to a mutation in one of the hedgehog genes, which help shape bodies during development, researchers began to look for the same clue elsewhere. They found it in the tumors of patients who didn’t have the syndrome but who grew the same sorts of cancers: A molecular lock was missing, allowing hedgehog proteins to proliferate unchecked. In many other cancers, the signs they found were subtler. There were traces of hedgehog proteins but no telltale mutations, a whiff of smoke but no gun. Still, smoke was smoke, and the news wafted from conference to conference, lab to lab. (Boodman, 10/24)

As the Marine Band warmed up and White House aides scurried to set up chairs in a sun-soaked Rose Garden for perhaps the most consequential health care address of the Trump presidency, a brazen mid-level budget aide was battling to jettison the entire event. President Trump was set to take the stage in barely an hour to unveil a “blueprint” to lower prescription drug prices. But on that afternoon in May 2018, Joe Grogan refused to sign off on either the remarks or the policy rollout. (Facher, 10/22)

The House Ways and Means Committee late Tuesday approved Speaker Nancy Pelosi’s drug pricing bill, sending the legislation to the full House floor for a vote expected before the end of this month. The legislation, which passed three committees along partisan lines, has a high chance of approval in the House, where Democrats control the chamber with 234 seats versus 197 held by Republicans. The Education and Labor Committee and the Energy and Commerce Committee made few changes to the bill last week, despite Republican members expressing concerns that the legislation was rushed and dead on arrival in the Senate. (Lovelace, 10/22)

Prescription drug companies and trade groups shelled out millions of dollars to lobby Congress as it considered legislation aimed at reining in skyrocketing drug prices, according to new lobbying disclosure reports. The Pharmaceutical Research and Manufacturers of America (PhRMA) — the trade group representing branded drug companies — spent $6.2 million on lobbying in the third quarter of 2019, which ran from July through the end of September. (Hellmann, 10/22)

The Association for Accessible Medicines wants generic medicines excluded from new Medicare rebates the bill mandates if companies raise their prices above the rate of inflation, the trade group's leader Chip Davis said during a press briefing today. The Senate Finance Committee's drug pricing package, S. 2543 (116), excluded generics from a similar provision. (Karlin-Smith, 10/23)

As debate grows over the role that taxpayer dollars play in drug discovery, an analysis finds one in four new medicines approved by regulators over the past decade benefited from publicly supported late-stage research or spinoff companies that were created by public sector research institutions. Moreover, drugs that were approved following major public financing were more likely to have won speedier approvals by the Food and Drug Administration. These medicines were also more likely to be so-called first-in-class treatments, suggesting they offered novel approaches to therapeutic care and therefore were potentially more clinically important. (Silverman, 10/23)

Eli Lilly Co. posted lower-than-expected revenue as the pharmaceutical company provided more funding for Medicare participants according to a change in federal requirements this year. The Indianapolis-based drugmaker on Wednesday reported third-quarter revenue of $5.48 billion, up 3.2% from a year earlier but below the $5.5 billion analysts polled by FactSet had expected. Lilly said an 8% volume increase drove revenue. (Sebastian and Loftus, 10/23)

Eli Lilly and Co third-quarter sales missed Wall Street estimates on Wednesday as rebates limited revenue from its top-selling diabetes drug Trulicity, and its shares fell about 4%. The Indianapolis-based drugmaker has been banking on newer drugs such as Trulicity and psoriasis therapy Taltz as it faces competition from cheap generic versions of erectile dysfunction treatment Cialis and other older medicines. (10/23)

Humana is suing a slew of pharmaceutical companies for allegedly fixing the prices of widely used generic drugs to boost their profits at the expense of insurers. The Louisville, Ky.-based health insurer alleged in the lawsuit that the drug companies violated antitrust laws by conspiring to obstruct competition among generic manufacturers that would normally lead to reduced prices for those drugs. (Livingston, 10/21)

Novartis reported a strong launch for its new gene therapy Zolgensma—the world’s most expensive drug—overcoming concerns about whether insurers would cover the treatment and a data-manipulation scandal at the unit that makes it. Zolgensma, which costs $2.1 million per patient, brought in $160 million in the three months to Sept. 30, its first full quarter of sales. That was well above analyst expectations of around $98 million. (Roland and Martuscelli, 10/22)

Novartis sales data on Tuesday suggested the Swiss drugmaker is reaping less than the $2.1 million U.S. list price for its gene therapy Zolgensma, as insurers may be getting breaks on the world's most-expensive one-time treatment. The spinal muscular atrophy (SMA) treatment, approved by the U.S. Food and Drug Administration in May, has reaped $175 million in revenue this year, including $160 million in the third quarter. (10/22)

On Monday, Vertex (VRTX) Pharmaceuticals secured approval of a new cystic fibrosis drug. Margot Cleveland bought steaks. She was in the mood to celebrate. Cleveland’s son, 10, was born with cystic fibrosis. He recently started taking an older Vertex medicine, but hopes to switch soon to the newly approved drug, called Trikafta. Life with cystic fibrosis was about to get a lot less stressful. (Feuerstein and Herper, 10/23)

Silicon Valley billionaire Sean Parker, founder of Napster and an early investor in Facebook (FB), has more recently recast himself as a scientifically minded philanthropist, spending much of his vast fortune to fund academic scientists working on new treatments that harness the immune system to attack cancer. Now, Parker is taking on a new role: creating biotech companies. His Parker Institute for Cancer Immunotherapy played a key role in the creation of ArsenalBio, a new biotech startup debuting Thursday that aims to use CRISPR genome editing tools, synthetic biology, and other technologies to engineer immune cell therapies that can better find and kill cancer cells. (Feuerstein, 10/17)

The National Institutes of Health and the Bill and Melinda Gates Foundation will together invest at least $200 million over the next four years to develop gene-based cures for sickle cell disease and HIV with an attribute even rarer in the world of genetic medicine than efficacy, the groups announced on Wednesday: The cures, they vowed, will be affordable and available in the resource-poor countries hit hardest by the two diseases, particularly in Africa. The effort reflects growing concerns that scientific advances in genetic medicine, both traditional gene therapies and genome-editing approaches such as CRISPR, are and will continue to be prohibitively expensive and therefore beyond the reach of the vast majority of patients. (Begley, 10/23)

Do you have expired or unused prescription medicine at home? Get rid of it on Saturday during National Prescription Drug Take Back Day. The nationwide event, sponsored by the Drug Enforcement Administration and law enforcement agencies nationwide, helps people dispose of drugs safely. (Blakemore, 10/22)

Canada's Liberal government is more likely to pass a universal prescription drug plan after losing its majority in Monday's election, setting the stage for what would be the biggest shakeup of the country's public healthcare system since it was created in the 1960s. The Liberals won the most seats in the election but fell short of a majority, which means Prime Minister Justin Trudeau will need the support of rivals like the left-leaning New Democratic Party (NDP) to govern. Both the Liberals and NDP have promised a new national drug plan. (10/22)