FDA approves first advanced cancer therapy - Axios

FDA approves first advanced cancer therapy

Elaine Thompson / AP

The FDA has approved the first advanced therapy to attack cancer. The brand-name drug, Kymriah, made by pharmaceutical giant Novartis, can be used for children and young adults who have a certain form of leukemia.

Why it matters: It's a major approval that has the potential to change how cancer is treated, although the eligible patient population appears small for now. "We're entering a new frontier in medical innovation with the ability to reprogram a patient's own cells to attack a deadly cancer," FDA Commissioner Scott Gottlieb said in a statement.

What the drug does: It's commonly known as a CAR-T therapy, and it's a hot research area across several drug companies (and is the prize of Gilead's $11.9 billion buyout of Kite Pharma from this week). CAR-T modifies a patient's immune cells, which are then infused back into the patient to kill cancer cells.

The price: $475,000 for a one-time treatment, according to a conference call with Novartis executives. The company also said it will only accept payments if patients respond to the drug within the first month. David Mitchell, president of advocacy group Patients For Affordable Drugs, met with Novartis yesterday to talk about "how to arrive at a fair price for its new CAR-T drug," but said the meeting was "disappointing."

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Some Republicans want to combine tax reform and health care

Sen. Lindsey Graham isn't ready to give up on health care. Photo: Andrew Harnik/AP

Graham-Cassidy is almost certainly dead, but some Republicans want to press on with health care — along with tax reform — through the 2018 budget, and momentum for the idea seems to be building. That way, Sept. 30 wouldn’t be their final deadline after all. "There's a pretty vocal do both tax reform and healthcare with FY18 reconciliation camp," one senior GOP Senate aide told me.

Why this matters: Some Senate Republicans don't want to give up on health care, but others worry combining the two difficult topics will sink the tax reform effort. "We don't have the political capital," said the aide, who is in the latter camp. But as we've seen over the last 10 days, it becomes politically difficult for the GOP to ignore a glimmer of hope when it comes to repealing the Affordable Care Act.

Who’s on board: Sens. Ron Johnson, Lindsey Graham and Rand Paul — so far.

Be smart: Writing budget resolutions and reconciliation instructions is complicated, but here's all you need to know: It would be possible to do both tax reform and health care through the same budget vehicle. So the argument isn't about whether this is possible — it's about whether the GOP should do it.

Johnson and Graham might be able to force the party's hand. "If we're not able to pass this this week, both Lindsey Graham and I have said — we're both on the Budget Committee — we'll insist on a budget resolution that'll give us the tools of reconciliation for health care and for tax reform," Johnson told reporters Monday.

  • Paul told reporters on Monday that "there's no reason you couldn't do health care and taxes at the same time."
  • Like health care, passing a budget — and then tax reform later — will only require 50 votes. However, that means Republicans can only lose two votes on a partisan bill to still pass it, with Vice President Mike Pence's tie-breaking vote.
  • "This will be the next problem [because] they won't have 51 votes for the budget," a GOP lobbyist said, adding that as far as tax reform goes, "I think this whole thing is going to get derailed by health care."
Yes, but: There are a lot of Republicans who are sick of dealing with health care. And very importantly, House GOP tax leaders have been clear that they do not want to mix health care with tax reform.

Zoom battles venture capital investor over cash, governance

Zoom, led by CEO Dave Sanders, is at odds with its venture capital investor. Photo: Zoom

Venture capital firm Endeavour Capital is asking a court in Oregon to appoint a receiver for Zoom, the health care startup that operates clinics and has been under investigation by the FBI for allegedly falsifying medical claims. Endeavour, which has poured $61 million into Zoom, says an independent third party overseeing Zoom would "clear the way for a significant, badly needed cash infusion."

Between the lines: The two sides say they are "deadlocked," but asking for a court-appointed receiver usually indicates a much deeper rift. And in this case, Zoom's finances appear to be on the line. Zoom did not immediately respond to questions.

The gritty details: Zoom has a four-person board: CEO and co-founder Dave Sanders, the other co-founder Albert DiPiero and two representatives from Endeavour. Since March, the two sides have been at odds over vague "company governance issues" as well as new money that needs to be injected into Zoom, which has laid off several people and had shut down its health insurance company.

Oregon law allows for a court-appointed receiver "in the event of a corporate deadlock or prospective insolvency," and Endeavour is asking for an expedited hearing.

What we're hearing: Sources familiar with Zoom's operations have said the company's finances have been in trouble since last year, when Sanders realized an Affordable Care Act program called risk adjustment would cost Zoom a lot of money. Risk adjustment is at the heart of the FBI's investigation into Zoom.


CBO: “Millions” fewer covered under Senate health care bill

Sen. Bill Cassidy's health care bill would leave millions uninsured, CBO said (AP Photo/Andrew Harnik)

Senate Republicans' latest health care would save the federal government at least $133 billion over the next decade, the Congressional Budget Office said today. That means the bill is eligible for the special rules that would allow Republicans to pass it with a simple majority.

Yes, but: The budget office didn't have enough time to evaluate the bill's effect on premiums or the number of Americans who have health insurance. It said only that "millions" fewer people would have insurance under the GOP bill than under the status quo. But that was enough for Sen. Susan Collins, who announced her opposition to the bill immediately after CBO released its analysis.


Collins will oppose Senate health care bill

Sen. Susan Collins said she opposes the Senate's health care plan. (AP Photo/Robert F. Bukaty, File)

Sen. Susan Collins officially said she will oppose the Senate's latest bill to repeal parts of the Affordable Care Act — yet another nail in the coffin for a bill that's moving further away from the 50 votes it would need to pass.

Why it matters: It would only take three "no" votes to kill the bill. And Collins' opposition makes it a total of four Republicans who say they won't vote for the bill — two moderates (Collins and Sen. John McCain) and two conservatives (Sens. Rand Paul and Ted Cruz).


Cassidy-Graham hearing: A dramatic political showdown over a dying bill

Capitol police remove protestors from the Finance hearing on Cassidy-Graham. Photo: Caitlin Owens/Axios

The Senate Finance Committee hearing on Republicans' latest health care bill is about as chaotic as congressional hearings get, with senators talking over one another, testifying about bills they wrote (which is unusual), and protestors in wheelchairs being dragged out of the committee room.

Be smart: This dramatic political showdown is all happening for a bill that's widely assumed to be dead. Not only are Republicans highly unlikely to pass this bill, but now they'll also have to deal with photos of disabled people in wheelchairs being removed from a hearing about a bill they fear could take away their health care coverage.

What's happened:

  • Protestors attending today's hearing on Republicans' health care proposals were immediately removed by Capitol police after they began chanting, "No cuts to Medicaid, save our liberty" as soon as Chairman Orrin Hatch gaveled the hearing into session. The hearing couldn't begin for roughly 20 minutes — until the protestors, many in wheelchairs, had been removed. "If you can't be in order, then get the heck out of here," Hatch said once the protestors were removed.
  • Sen. Bill Cassidy, one of the bill's sponsors and one of the witnesses testifying at the hearing, said the most recent version of the bill is also the final one, aside from corrections to drafting errors.
    • Ranking Member Ron Wyden asked if the most recent version of the bill is the one the Senate will vote on. "Yeah, I believe so…I hope a correction of a drafting error doesn't constitute a whole 'nother version," Cassidy replied.
    • Why this matters: Sens. Rand Paul and Ted Cruz are withholding their votes for the bill unless they get policy changes they've asked for. Cassidy made it sound like those changes aren't coming. Sen. John McCain is the third public "no" vote, meaning the bill is looking pretty dead.

Cruz still a "no" on Graham-Cassidy

Sen. Ted Cruz currently wouldn't vote for the Graham-Cassidy bill. Photo: Jose Luis Magana / AP

Sen. Ted Cruz still is not a "yes" vote on the Graham-Cassidy health care bill, according to a Cruz aide, despite changes made in a new version of the legislation released publicly Monday morning. These changes include more conservative regulatory provisions. It's not clear why Cruz doesn't support the bill at this time.

Why this matters: Cruz is the third Republican senator to say he opposes the bill. It only takes three to kill it.


McCaskill calls out ER company for surprise medical bills

Sen. Claire McCaskill wants answers from Envision Healthcare. Photo: Susan Walsh / AP

Sen. Claire McCaskill has sent a letter to Envision Healthcare CEO Chris Holden, demanding answers about the company's practices of billing emergency room patients for out-of-network charges. McCaskill heavily cited a New York Times report and a study that found an Envision subsidiary called EmCare routinely manages hospital ERs and avoids contracting with insurers so it can bill higher rates to patients.

Why it matters: Envision has faced heat from patients and shareholders, and now an influential politician is pushing to know sensitive matters, like how many complaints its ERs have fielded and what percentage of ER visits involve out-of-network billing. Envision has until Oct. 11 to respond to McCaskill's office.


Paul still opposes GOP health bill

Sen. Rand Paul opposes the latest health care bill. Photo: Manuel Balce Ceneta / AP

Sen. Rand Paul still opposes the Senate's latest bill to repeal parts of the Affordable Care Act, despite changes that pulled parts of the bill to the right.

Why it matters: This bill almost certainly cannot pass without Paul's support, yet his opposition is not a surprise. He wants a much more conservative bill. Winning his support would lose the support of even more moderate Republicans than the bill has already lost. That quandary sunk previous repeal efforts this summer, and seems likely to sink this one, too.


Pfizer spins out shelved experimental drugs

Pfizer is taking Johnson & Johnson to court. Photo: Mark Lennihan / AP

SpringWorks Therapeutics, a rare disease drug development startup, has raised $103 million in Series A funding from Pfizer, Bain Capital Life Sciences, Bain Capital Double Impact, Orbimed and LifeArc. The company is launching with four compounds licensed from Pfizer, including a pair – one in desmoid tumor and another for neurofibromatosis – that are ready for Phase 3 clinical trials. Pfizer also has some royalty rights via the transaction, although financial details were not disclosed.

Why it matters: Because big pharma rarely pays much attention to its shelved experimental compounds, let alone create a spin-out company to further develop them.

But that's what Pfizer did here, spearheaded by a strategy exec named Lara Sullivan who now leads SpringWorks. It could become a model for Pfizer's peers, many of which have just out-licensed compounds on a one-off basis to inbound third parties. As for SpringWorks, it intends to acquire other candidates from non-Pfizer sources (both corporate and academic).

Bottom line from Xconomy's Bed Fidler: "Pharma companies scrap development of experimental drugs all the time. Sometimes those drugs don't work or are unsafe. Other times they no longer fit a company's strategic goals, or they might be best suited for a different disease. Maybe they even lose an internal fight for interest and resources, and the cash to run larger, more expensive trials is put elsewhere."


Graham-Cassidy: New policies, same politics

Sens. Lindsey Graham and Bill Cassidy will introduce a new health care bill. (AP Photo/Alex Brandon)

The substance of the Senate's latest health care bill is different from its predecessor, but the politics are not, in part because only a few Republican senators care about the substance of the bill.

The bottom line: The revised health care bill Sens. Lindsey Graham and Bill Cassidy are set to release today follows a familiar model: more money for the moderates (Sens. Lisa Murkowski and Susan Collins) and more regulatory rollbacks for the conservatives (Sens. Rand Paul and, perhaps, Ted Cruz). We've seen this before, but the path to 50 votes might be even narrower this time.

Déjà vu: As much as the Graham-Cassidy process might have felt like a new thing playing out, with new developments, it's largely a repeat of July:

  • The latest effort — throw more money at moderates' states and beef up the regulatory flexibility to win over conservatives — is pretty much the same calculation GOP leaders made with the last-minute changes to the Better Care Reconciliation Act, the first bill to fail over the summer.
  • It's still very hard to win over conservatives without at least edging up to the ACA's pre-existing conditions protections — and that still alienates moderates.
  • McCain — and to a lesser but non-trivial degree, Murkowski and Collins — are upset not just about the contents of these bills, but about the rushed, frantic and incomplete process. Another last-minute rewrite, days before the final deadline, won't help alleviate any of those concerns.

What's changed:

  • The latest revisions offer new funding streams or recalculated formulas that would benefit Alaska, Arizona, Kentucky and Maine. Those states would still see cuts, overall — but those cuts appear to be smaller this time around.
  • States would have even more flexibility to roll back some of the Affordable Care Act's insurance regulations — including the guarantees it provides for people with pre-existing conditions.

Why it matters: The deadline to pass a bill with just a majority vote is Saturday. If this strategy is going to succeed now where it has failed before, it'll need to happen fast — but still in a climate of frantic deadline pressure, little independent analysis and, now, a fight with Jimmy Kimmel.