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Millions of Americans with rare diseases are caught up in an ongoing legal and political debate about how the U.S. supports drug makers and their research.
This article was published on Wednesday, February 22, 2023 in Kaiser Health News.
By Sarah Jane Tribble
A prescription drug that helps Lore Wilkinson walk and talk despite a rare muscle disease cost her so little for more than a decade that she didn't even use her insurance to pay for it. But now, her Medicare insurance is shelling out about $40,000 for a one-month supply of the drug, and she fears she'll be slammed with a $9,000 copayment.
"Who can afford that?" said the 91-year-old, who lives in Rochester, Minnesota. (Her first name is pronounced LOR-ee.)
Wilkinson, like millions of other people with rare diseases nationwide, is caught up in an ongoing legal and political debate about how the U.S. supports pharmaceutical companies and their research. The FDA made its latest move in the tug of war in late January by saying it would largely ignore a U.S. court ruling involving Firdapse, the drug Wilkinson needs.
Firdapse was approved in 2018 by the FDA as an "orphan drug," a designation that rewards drug companies for developing treatments for rare diseases. When a drugmaker wins approval for an orphan drug, the company is entitled to seven years of exclusive rights to the marketplace, which means the FDA won't approve another company's application for a competitive drug for the same use during that period.
But after the 11th U.S. Circuit Court of Appeals denied a motion in early 2022, the FDA stopped reviewing applications for certain drugs or handing out exclusivity, agency spokesperson April Grant said. The delay left drugmakers in limbo.
Often, drugs granted exclusivity are among the highest priced in the U.S. market. For example, Zolgensma, a one-time treatment for spinal muscular atrophy, carries a $2.25 million price tag. Mary Carmichael, a spokesperson for its manufacturer, Novartis, said Zolgensma has treated more than 3,000 patients globally and nearly all U.S. patients taking the drug as approved by the FDA are covered by commercial or government insurance.
The company also continues to invest in research and development as well as clinical studies for the drug to reach more patients, Carmichael said. Most drugs enter the U.S. market armed with a variety of patents and intellectual property protections that stave off competition and allow drugmakers to set prices as they see fit. For drugs that treat rare diseases, the seven years of market exclusivity is part of that armor.
A year's supply of Catalyst Pharmaceuticals' Firdapse, which Wilkinson takes to treat her Lambert-Eaton myasthenic syndrome, or LEMS, sells for about $375,000 after discounts, said Catalyst spokesperson David Schull. He said the company has financial assistance programs and donates to charitable foundations to help those in need. The goal, Schull said, "is that no LEMS patient is ever denied access to medication for financial reasons."
Catalyst was granted exclusive market rights for Firdapse in 2018, which meant that Wilkinson and other LEMS patients could no longer get a similar drug from another company free of charge.
In 2019, amid a patient uproar about the cost, which Sen. Bernie Sanders weighed in on, the FDA granted another company, Jacobus Pharmaceutical, the right to market a competitive product for a subset of pediatric patients.
Then Catalyst filed suit against the federal government, contending it had rights to be the exclusive provider for all LEMS patients, regardless of age. The case, Catalyst Pharmaceuticals Inc. v. Becerra, had potentially "far-reaching implications," wrote Grant, the FDA spokesperson, in an email to KHN. The court's decision also "raised several novel questions," she said.
The 11th Circuit sided with Catalyst in September 2021. But the FDA's recent move to effectively disregard the court's decision is "in the best interest of public health, rare disease patients and rare disease product development," Grant wrote.
Still, the multiyear saga highlights lingering questions about orphan drug exclusivity and how the FDA's policies may influence drug prices. At issue is the Orphan Drug Act, a 1980s-era law that incentivizes drug companies to research and develop rare-disease drugs. And it's not the first time the orphan drug program has raised concerns.
For decades, the FDA has overseen a two-step process: A drug is first granted an orphan designation because it shows promise to treat a rare disease or condition. Then, once the pharmaceutical company studies and develops the rare-disease drug, the FDA approves its use and awards seven-year market exclusivity, preventing competition.
That final step, granting exclusivity, was in the spotlight in Catalyst's lawsuit against the FDA. Since the Orphan Drug Act was created, the FDA's staff routinely handed out exclusivity to companies for orphan drugs that treat a subset of patients, such as pediatrics. The goal was to make sure pharmaceutical companies didn't get total market control for a drug after doing studies on only the "smallest, easiest-to-study populations," the agency wrote on its website.
The Catalyst court decision could hurt children, agency officials wrote.
George O'Brien, a partner at Mayer Brown who represents companies regarding the FDA and regulatory practices, said he agreed with the FDA's decision and its long-term strategy of parceling out exclusivity because a drug's sales "should be limited to what you studied and got approved."
"Most people think the way the FDA has done it for years is a very sensible way to do it," O'Brien said. "Good for patients, good for pharma, and good for the FDA."
The FDA said that it will comply with the court's decision regarding Catalyst but that it doesn't apply to other companies or drugs. In response to the FDA's January announcement, Catalyst said it would not be affected. In July 2022, Catalyst bought the rights to Ruzurgi, the Jacobus drug.
Now, there is no competitive drug on the market that treats Wilkinson's disease.
Jacobus had provided Wilkinson with the active ingredient of its drug free of charge from 2004 to 2018: "The only thing I paid was shipping."
The FDA's move to largely rebuke the Catalyst case will likely mean another company will sue the agency again, O'Brien said: "They are in a really tough spot."
"My worry is there is just another lawsuit coming. And its uncertainty. Uncertainty is ultimately bad for patients," O'Brien said.
Drugmakers have taken the FDA to court before over how the agency administers the Orphan Drug Act. In 2014, Depomed won a suit against the agency demanding an exclusivity label on its drug Gralise, which treated nerve pain.
The FDA had given Gralise an orphan designation and approval but declined to give it exclusivity because it said it was not clinically superior to another drug already on the market. Then-federal district court judge Kentaji Brown Jackson, who was appointed to the U.S. Supreme Court last year, required the FDA to grant exclusivity, blocking a generic.
That case was focused on the clinical superiority of a drug, rather than the scope of exclusivity. After the Gralise decision, the FDA eventually persuaded Congress to amend the law, which may be needed now, O'Brien said. Rachel Sher, a former director of policy at the National Organization for Rare Disorders who is now at Manatt, Phelps, & Phillips, said companies that would benefit from a broader award of exclusivity will sue to force the agency for the same reading of the Orphan Drug Act.
"Congress will need to act at some point," said Sher, who also spent a decade on Capitol Hill as the FDA counsel for the House Energy and Commerce Committee.
Congress almost passed an amendment last year when it reauthorized the user fees that help fund the FDA. Then-Sen. Richard Burr (R-N.C.) argued to take the committee-added amendment out of the package, saying drugmakers would otherwise lack the incentives needed to develop drugs for rare diseases, according to Bloomberg Law.
Wilkinson, the patient advocate, has her own advice for Congress. The Orphan Drug Act itself — not just the exclusivity provision — needs to be fixed, she said.
"They have to change the law," she said. Pharmaceutical companies should only win orphan drug status and be given exclusivity when they develop "a really new medication, not just by changing one molecule."
Until then, Wilkinson said, she and others are still waiting: "I'm an old lady, and I don't know if it is going to get fixed."
This story was produced by KHN (Kaiser Health News), a national newsroom that produces in-depth journalism about health issues. Together with Policy Analysis and Polling, KHN is one of the three major operating programs at KFF (Kaiser Family Foundation). KFF is an endowed nonprofit organization providing information on health issues to the nation.
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This time it's Democrats accusing Republicans of wanting to maim the very popular federal health program.
This article was published on Tuesday, February 21, 2023 in Kaiser Health News.
By July Rovner, Kaiser Health News
The Medicare wars are back, and almost no one in Washington is surprised.
This time it's Democrats accusing Republicans of wanting to maim the very popular federal health program that covers 64 million seniors and people with disabilities. In the past, Republicans have successfully pinned Democrats as the threat to Medicare.
Why do politicians persistently wield Medicare, as well as Social Security, as weapons? Because history shows that works at the ballot box. Generally, the party accused of menacing the sacrosanct entitlements pays a price — although it's the millions of beneficiaries relying on feuding lawmakers to keep the programs funded who stand to lose the most.
Republicans have repeatedly warned they would hold raising the federal debt ceiling hostage unless Democrats negotiated changes to Medicare, Medicaid, and Social Security. The three programs together, along with funding for the Affordable Care Act and Children's Health Insurance Program, account for nearly half of the federal budget.
The political bomb that went off during President Joe Biden's State of the Union speech on Feb. 7 had been ticking for weeks. In his speech, Biden threatened to veto any Republican efforts to cut Social Security or Medicare. It was one of only three veto threats he made that night. During a trip to Florida after the speech, he said it more forcefully: "I know a lot of Republicans, their dream is to cut Social Security and Medicare. Well, let me say this: If that's your dream, I'm your nightmare."
Senior Republicans have distanced themselves from the proposals Biden was referencing, notably ideas from the House Republican Study Committee and Sen. Rick Scott (R-Fla.) to make cuts or even let Medicare expire unless Congress votes to keep it going.
"That's not the Republican plan; that's the Rick Scott plan," Senate Minority Leader Mitch McConnell said on a Kentucky radio show Feb. 9, echoing his opposition to the plan last year.
"Cuts to Social Security and Medicare are off the table," House Speaker Kevin McCarthy declared the day before Biden's veto threat.
McConnell and McCarthy know something that Rick Scott apparently does not: Politicians threaten big, popular entitlement programs at their peril. And, usually, it's been Republicans who suffer the electoral consequences.
This dates at least to 1982, when Democrats used threats of Republican cuts to Social Security to pick up more than two dozen House seats in President Ronald Reagan's first midterm elections. In 1996, President Bill Clinton won reelection in part by convincing voters that Republicans led by House Speaker Newt Gingrich wanted to privatize Medicare and Social Security.
At the beginning of his second term, in 2005, President George W. Bush made it his top priority to "partially privatize" Social Security. That proved singularly unpopular. In the following midterm elections, Democrats won back the House for the first time since losing it in 1994.
In 2010, Republicans turned the tables, using what they described as "Medicare cuts" in the Affordable Care Act to sweep back to power in the House. (Those "cuts" were mostly reductions in payments to providers; beneficiaries actually got extra benefits through the ACA.)
The use of the Medicare cudgel likely reached its zenith in 2012, when Democrats took aim at Medicare privatization proposals offered by Paul Ryan, the House Budget Committee chair and Republican vice presidential candidate. That debate produced the infamous "pushing Granny off the cliff" ad.
The reality is that Medicare's value as a political weapon also sabotages any effort to come together to solve the program's financing problems. The last two times the Medicare Hospital Insurance Trust Fund was this close to insolvency — in the early 1980s and late 1990s — Congress passed bipartisan bills to keep the program afloat.
Even the word "cut" can be political. One stakeholder's Medicare "cut" is another's benefit. Reducing payments to medical providers (or, more often, reducing the size of payment increases to doctors and hospitals) may reduce premiums for beneficiaries, whose payments are based on total program costs. Raising premiums or cost sharing for beneficiaries is a benefit to taxpayers, who help fund Medicare. Increasing available benefits helps providers and beneficiaries, but costs more for taxpayers. And on, and on.
There are fundamental differences between the parties that can't be papered over. Many Republicans want Medicare to shift from a "defined benefit" program — in which beneficiaries are guaranteed a certain set of services and the government pays whatever they cost — to a "defined contribution" program, in which beneficiaries would get a certain amount of money to finance as much as they can — and would be on the hook for the rest of their medical expenses.
This would shift the risk of health inflation from the government to the beneficiary. And while it clearly would benefit the taxpayer, it would disadvantage both providers and beneficiaries of the program.
But there are many, many intermediate steps Congress could take to at least delay insolvency for both Medicare and Social Security. Some are more controversial than others (raising the payroll tax that funds Medicare, for example), but none are beyond the steps previous Congresses have taken every time the programs have neared insolvency.
Republicans are correct about this: Medicare and Social Security can't be "fixed" until both sides lay down their weapons and start talking. But every time a granny in a wheelchair gets pushed off a cliff, that truce seems less and less possible.
HealthBent, a regular feature of Kaiser Health News, offers insight and analysis of policies and politics from KHN's chief Washington correspondent, Julie Rovner, who has covered health care for more than 30 years.
