WASHINGTON, D.C. — The ORPHAN Cures Act is proving even more destructive than initially reported. Over the weekend, The Wall Street Journal revealed that the Congressional Budget Office (CBO) will re-score the bill to account for its impact on even more blockbuster drugs, meaning its already massive $5 billion price tag is set to grow.
When first scored, the ORPHAN Cures Act was projected to cost taxpayers nearly $5 billion over the next decade, allowing drugmakers to reap the rewards of a weakened Medicare Negotiation Program. But that initial estimate understated the damage: major high-cost drugs, including blockbusters like Keytruda, weren’t counted in the original analysis. With a new CBO score underway, the full scale of this pharma-backed carveout will soon be clear — and it’s worse than anyone thought.
This carveout is a blatant loophole designed for Big Pharma to protect its monopolies and keep prices artificially high. Pharma’s claim that ORPHAN is necessary to protect rare disease innovation doesn’t stand up to scrutiny either, since Medicare negotiation already preserves all the incentives for rare disease research under the 42-year-old Orphan Drug Act.
This should serve as a warning to Congress: the ORPHAN Cures Act is already funneling billions more to Big Pharma than lawmakers originally understood, doing even greater damage to Medicare negotiation. The last thing patients and taxpayers can afford is another pharma-backed giveaway like the EPIC Act that would hand the industry even more money and further undermine the program’s ability to lower drug price
The Wall Street Journal: Why Drug Prices for Some Big Medicines Will Remain High for a Longer Time
Joseph Walker | August 3, 2025
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Patients For Affordable Drugs Now is the only national, patient advocacy organization focused exclusively on policies to lower drug prices. We empower and mobilize patients and allies, hold accountable those in power, and fight to shape and achieve system-changing policies that make prescription drugs affordable for all people in the United States. P4ADNow is bipartisan and does not accept funding from organizations that profit from the development or distribution of prescription drugs. To learn more, visit: PatientsForAffordableDrugsNOW.org
Welcome to the Week in Review.
Pharma’s Mid-Year Money Grab
Despite one in three Americans being unable to afford their prescription drugs, pharma companies raised prices in July on over 150 brand-name medications as part of their mid-year price hikes. The majority of hikes were increases above the rate of inflation. A few lowlights: Novartis raised the price of its spinal muscular atrophy gene therapy, Zolgensma, by $119,585, the highest singular price increase on an already wildly expensive drug sold at over $2.5 million. And the largest percentage was for the osteoporosis drug Ibandronate Sodium, which was increased by 359% for three tablets. This is business as usual for Pharma: hiking prices and squeezing patients, regardless of the crushing financial burden they face.
Trump Administration Re-Ups MFN with New Letters to Pharma
On Thursday, President Trump sent letters to 17 major pharmaceutical companies ratcheting up his Most Favored Nation (MFN) drug pricing strategy. Americans shouldn’t be paying between four and eight times what other high-income nations pay for the very same brand-name drugs, and the president is recognizing that drug companies gaming the system are to blame. However, the letters still leave far too much room for the industry to protect its profits by raising prices for patients abroad — in line with their track record of exploiting even the most well-intentioned plans to maintain its monopolies at the expense of patients. It’s critical that any MFN plan contains safeguards to prevent pharmaceutical companies from simply raising prices in other countries instead of lowering them in the U.S., in addition to clearly defined ways for the administration to monitor and enforce compliance. — [White House, P4AD, NYT, POLITICO, Reuters, Inside Health Policy, Axios, The Hill, STAT News, CNBC, Bloomberg Law]
Tariffs Will Devastate Patients
Sunday’s EU trade deal will set a 15% tariff on imported pharmaceuticals from Europe, affecting a wide range of drugs, including blockbusters Ozempic and Keytruda. With Wall Street analysts predicting that the deal will cost the pharma industry $19 billion, P4AD is once again sounding the alarm that manufacturers will pass any new costs onto patients, raising prices and increasing drug shortages. Even if companies invest in reshoring, new price hikes will likely hit long before any potential benefits, harming the most vulnerable patients already struggling to afford their prescription drugs. — [White House, Reuters, Bloomberg, NYT, Axios, Endpoints, STAT News, Inside Health Policy]
Pharma Executives Push For Yet Another Handout
The Maintaining Investments in New Innovation Act, or MINI, is back in the news, with the CEO of Alnylam Pharmaceuticals penning an op-ed in support of this harmful bill. MINI would extend the exclusivity period for genetically targeted technologies (GTTs) to 11 years, further restricting them from negotiation and ensuring manufacturers can price-gouge patients for longer while increasing costs for taxpayers. If a drug company CEO is lobbying for a bill like MINI, you can bet it’s bad for patients. We urge Congress to put patients above industry interests — particularly after the industry was already handed a $5 billion handout with the ORPHAN Cures Act last month. — [Congress, STAT News, P4ADNow]
*Introducing a new weekly patient advocate spotlight. These advocates are the heart of our movement and courageously share their stories to drive change.
Patient Advocate Spotlight: Steven Hadfield, 73, North Carolina
Condition: Rare Blood Cancer and Type 2 Diabetes
Drugs: Brukinsa ($15,744), Januvia ($527), Dupixent ($7,987), Metformin, Gabapentin, and Repaglinide, totalling roughly $24,221/month. Thankfully, Steven met the IRA’s $2,000 out-of-pocket cap this year.
This week, Steven participated in a press conference urging the repeal of the ORPHAN Cures Act. Here’s some of what Steven had to say:
“I used to take Imbruvica — a cancer drug priced at nearly $17,000 a month. I had to get grants to pay for it because it was simply unaffordable. Now, because it’s part of Medicare negotiation, in January that price will be cut by 40 percent — but if the ORPHAN Cures Act had been passed into law earlier, Imbruvica would not have been eligible for negotiation, leaving thousands of patients paying a higher price indefinitely… Patients like me fought hard to win Medicare negotiation. We cannot let Pharma gut that victory. It’s time to repeal the Orphan Cures Act and stand with patients — not Big Pharma’s profits.”
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WASHINGTON, D.C. — Patients For Affordable Drugs Now Executive Director Merith Basey issued the following statement in response to letters sent by President Trump to 17 major pharmaceutical companies regarding his Most Favored Nation drug pricing strategy:
“American patients deserve real relief from paying between four and eight times what other high-income nations pay for the very same brand-name drugs, and we welcome efforts to hold drug corporations accountable for their price-gouging and blame-shifting tactics. Yesterday’s letters make clear that the administration recognizes that drug companies are gaming the system to keep prices high here in the U.S.; however, the letters still leave far too much room for the industry to protect its profits while also raising prices for patients abroad.
Americans overwhelmingly understand that drug corporations are the root cause of high drug prices. And based on the industry’s track record, without proper enforcement and penalties, Big Pharma will exploit even well-intentioned plans to maintain its monopolies at the expense of patients.”
REMAINING QUESTIONS
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Patients For Affordable Drugs Now, is the only national, patient advocacy organization focused exclusively on policies to lower drug prices. We empower and mobilize patients and allies, hold accountable those in power, and fight to shape and achieve system-changing policies that make prescription drugs affordable for all people in the United States. P4ADNow is bipartisan and does not accept funding from organizations that profit from the development or distribution of prescription drugs. To learn more visit; PatientsForAffordableDrugsNOW.org.
Welcome to the Week in Review.
Details Emerge in FDA Voucher Program
The FDA is continuing to build out its new priority review voucher program, now formally adding “affordability” as one of the priority criteria when considering vouchers. According to new guidance, companies could potentially be eligible for a voucher if they lower U.S. brand-name drug prices to match those in comparable high-income nations – a nod to President Donald Trump’s ‘Most Favored Nation’ pricing proposal. While we are encouraged to see affordability being raised, without congressional authorization and with only five vouchers available in the pilot year, it raises serious questions about the program’s viability. As always, we know pharma will find ways to abuse even the best-intentioned programs. P4AD will continue to monitor this program as it develops. — [CNBC, Endpoints]
Pharma’s Silence Gives The Game Away
Pharma loves to claim that the popular Medicare Negotiation Program will devastate innovation – but when a CBO report found that a 10% cut to the NIH budget would result in more than four times the harm to new drug development, the industry has largely remained quiet. Why? Because pharma’s fearmongering about the IRA isn’t really about innovation – it’s about protecting their monopoly pricing power. According to the CBO, NIH cuts would result in 53 fewer drugs coming to market over the next 30 years, compared to just 13 from Medicare negotiation. — [CBO, KFF]
AstraZeneca Begins Reshoring But Patients Still Get Squeezed
With 200% tariffs on pharmaceutical products still being considered by President Trump, AstraZeneca announced a $50 billion investment this week in U.S. manufacturing. But let’s be clear; reshoring won’t happen overnight, and drugmakers will pass any increased import costs onto patients long before domestic supply chains can catch up. And while AstraZeneca is using this announcement to cozy up to the administration, its CEO is also using the spotlight for attacking potentially transformative drug pricing reforms like Most Favored Nation – a reminder that Big Pharma’s top priority remains protecting profits, not patients. — [Bloomberg, STAT News]
In Case You Missed It
A new report commissioned by the Association for Accessible Medicines claims that Medicare negotiation will harm generic competition and drive up drug prices. But a closer look reveals that their entire argument is based on faulty assumptions and ignores key facts, including inaccurately suggesting that Medicare negotiation is a substitute for market-based competition, conveniently overlooking that generic or biosimilar competition is already ineligible for negotiation.
*Introducing a new weekly patient advocate spotlight. These advocates are the heart of our movement and courageously share their stories to drive change.
Patient Advocate Spotlight
Elaine Kniepfel, 83, Kansas
Condition: Multiple Sclerosis
Drugs: Copaxone ($100,000/year), Provigil ($8,000/month)
Background: Retired educator with 47 years of service as a teacher and administrator
In her words: “Even with Medicare, the $2,000 cap is too much for retired people on fixed incomes. It’s abhorrent that drug companies charge so much to the people who need medicine most.”
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Welcome to the Week in Review.
CMS Model Could Expand Access to Sickle Cell Therapy
This week, CMS announced that 33 states, DC, and Puerto Rico will join an initiative intended to improve access and lower costs for gene therapies for sickle cell disease (SCD), a condition that disproportionately impacts Black and Latino communities. A continuation of the Biden administration’s model, the Cell & Gene Therapy Access model ties payment to clinical outcomes, aiming to expand access while holding drug companies accountable. With a massive price tag upwards of $2.8 million for SCD treatments, we’re encouraged to see CMS working to make these life-changing therapies more affordable and more widely accessible for patients. — [CMS, NIH, CMS, BioPharma Dive, Fierce Healthcare, BioPharma Dive, Pink Sheet, Healthcare Finance]
Big Pharma’s Discount Won’t Cut It
Bristol-Myers Squibb (BMS) and Pfizer announced a new direct-to-patient program this week, offering a “discounted” price on their blockbuster blood thinner Eliquis. While marketed as a win for patients, the new discounted price is still higher than what patients on Medicare will pay under the new Medicare Negotiation Program and will still be too high for many patients. In the wake of growing public and political pressure, this announcement comes just weeks after the Trump administration issued an executive order reviving the idea of ‘Most Favored Nation’ pricing including calling for direct-to-consumer sales at a lower price, and at a time when patients and the majority of Americans demand more action to lower drug prices. At the same time, BMS is actively trying to weaken Medicare negotiation, asking for the “maximum level of flexibility”. Make no mistake, pharma is looking for ways to stay ahead of reforms that threaten their pricing power. — [WSJ, Reuters, Endpoints]
FDA Proposes Tying Priority Approval Vouchers to Drug Affordability
In an interview with CNBC late last week, FDA Commissioner Makary said the agency would consider drug affordability as a factor in its new priority review voucher program. This announcement comes after the department introduced the new program last month, stating that its goal is to “reduce inefficiencies” and expedite decision-making. Though some experts have claimed the new voucher program doesn’t hold up to legal scrutiny, Makary and the FDA have stated they’re aiming to use the program as an incentive for companies that commit to lowering their drug prices — though we’re always on the lookout for pharma to try and game the system here too. — [CNBC, FDA, Inside Health Policy, BioSpace]
In Case You Missed It
A new study found that just 2.5% of nearly 10,000 patents on small-molecule drugs cite government funding, despite the fact that 99% of drugs approved from 2010 to 2019 originated through NIH-funded research. It’s yet another example of a rigged system: Taxpayers fund the science, but pharma patents the profits. — [Nature Biotech, JAMA, Endpoints]
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Patients Joins HHS for a Drug Prices Roundtable
Last week, five P4AD Patient Advocates participated in a roundtable with HHS Secretary Kennedy and NIH Director Bhattacharya, focused on the burden of high drug prices and the administration’s exploration of a ‘Most Favored Nation’ (MFN) strategy. Emily, Jackie, Janet, Melissa, and Sarah shared their stories with the Secretary, the NIH Director, and HHS staff about the sacrifices they have had to make to afford their lifesaving and lifesustaining medications. MFN pricing has the potential to be a powerful tool to lower drug prices if it’s designed with patients at the center and works in tandem with the historic Medicare Negotiation Program. As the administration shapes its policy, centering patient stories independent from industry influence is critical to ensure the framework will meaningfully lower prices in the U.S., without unnecessarily raising them abroad. — [LinkedIn, YouTube, White House]
Lowering Drug Prices Doesn’t Hurt Innovation
A new report from Bentley University found that research and development (R&D) spending by 134 drug companies hit record levels after the passage of the Inflation Reduction Act, increasing from $211 billion in the 18 months before the law to $247 billion in the 18 months following its passage. This directly contradicts Big Pharma’s fearmongering that the Medicare Negotiation Program would hurt investment in new drug development. In reality, the industry has experienced steady R&D investment spending and continued strong revenue. While drug manufacturers continue to spread misinformation, 88% of Americans support Medicare negotiation to rein in pharma’s unchecked pricing power. The bottom line: the IRA has coincided with a period of record-high investment in the sector. Don’t be misled by Pharma’s cries to the contrary. — [Bentley University, Lever News, Commonwealth, Arnold Ventures]
Tariffs Are Back. And They’re Still a Bad Idea.
Tariffs are back in the news this week, after President Trump announced plans to impose rates of up to 200% on pharmaceutical products “very soon”, with new details expected at the end of the month. P4AD is cautioning that tariffs won’t hold drug companies accountable; they’ll just increase the risk of drug shortages and raise prices for patients as manufacturers pass any new costs onto patients. Price hikes will hit long before any potential benefits from reshoring or increased investment in U.S. manufacturing materialize. At a time when 1 in 3 Americans already can’t afford their prescription drugs, the last thing patients need is another policy that will make medicines even more expensive. — [The Hill, Bloomberg, Reuters]
In Case You Missed It
In preparation for the third round of Medicare negotiation, P4AD submitted comments to CMS on the cycle’s new draft guidance. P4AD will continue fighting to protect the program’s implementation as it begins to deliver savings for 9 million Medicare recipients in 2026. — [Bloomberg Law, CMS]
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Pharma Handouts, Not Patient Relief: Congress Passes The ORPHAN Cures Act
Today, the House passed a reconciliation bill that includes a nearly $5 billion giveaway to the pharmaceutical industry: the ORPHAN Cures Act, just a day after the Senate passed the same legislation, which now heads to the President’s desk. By including ORPHAN Cures, the reconciliation bill is ensuring drugs that should be included in Medicare negotiation — a program that’s supported by 86% of Americans on both sides of the aisle — won’t see lower prices in the coming years. It’s deeply disappointing that for many members of Congress, appeasing industry interests seems to come before standing with the nine in ten Americans demanding lawmakers lower drug prices and the millions of patients forced to make impossible choices because of their prescription costs. The reconciliation bill may have undermined historic affordability progress, but P4ADNow, alongside our community of patients, will continue fighting implementation of ORPHAN Cures and working to protect the Medicare negotiation program from any other attacks. — [Arnold Ventures, CSRxP, POLITICO Pro, CNBC, POLITICO Pro, Healthcare Finance, Fierce Pharma, Common Dreams, Nation of Change]
PhRMA Panics as “Most Favored Nation” Pricing Gains Traction
The pharmaceutical industry is panicking over a potential “Most Favored Nation” pricing policy. In an op-ed for STAT News, PhRMA CEO Stephen Ubl rolled out the industry’s usual fearmongering and blame-shifting to argue against aligning U.S. drug prices (which are the highest of anywhere in the world) with those in other wealthy nations. He even cites data from No Patient Left Behind, one of the pharma-funded front groups we exposed in our 2023 “Hidden Hand” report, to push the narrative that patients abroad are “freeloading.” Here’s the truth: drug companies already make huge profits in countries with lower prices. They want to keep the U.S. as their cash cow, charging Americans four to eight times more for brand-name drugs than patients elsewhere. MFN pricing threatens that status quo, which is exactly why pharma is fighting it. Aligning prices with peer nations is a path toward real relief for patients, but we must ensure it’s done right, without letting pharma raise prices abroad to protect their margins. Don’t let their talking points distract from the real problem: drug corporations, not other countries, are driving high prices in the U.S., and increasing prices internationally wouldn’t do anything to lower prices at home. — [STAT News, P4AD, HHS]
In Case You Missed It
On Monday, the FTC and DOJ held their first of three listening sessions examining how drug companies use tactics like pay-for-delay, product hopping, and rebating to block competition and keep prices high. Drug companies abuse the patent system to extend their monopolies and maximize profits, and the U.S. desperately needs increased competition and transparency if we want to see lower prices. According to the FDA, introducing one generic drug into the market reduces drug prices by 39%, while six or more result in a staggering 95% reduction in price. That’s why P4ADNow continues to fight for legislation to reduce patent abuses by drug corporations. — [FTC, FDA]
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WASHINGTON, D.C. — Today, the House passed a reconciliation bill that includes a nearly $5 billion giveaway to the pharmaceutical industry: the ORPHAN Cures Act. Despite outrage from tens of thousands of patients on Medicare to halt its progress, ORPHAN Cures was included in the reconciliation bill, was passed by both chambers this week, and now heads to the President’s desk. Its inclusion defies the will of American patients and, if implemented, will diminish the savings due to be generated for patients via the historic and wildly popular Medicare negotiation program.
“When nine in ten Americans want Congress to go further to lower prescription drug prices, there is no good reason to hand Big Pharma a new loophole to exploit — and stick taxpayers with a $5 billion bill,” said Merith Basey, Executive Director of Patients For Affordable Drugs Now. “This unnecessary carveout keeps prices high on drugs that should be negotiated, weakening Medicare negotiation before it even begins to deliver savings. It sends a clear message to the 1 in 3 Americans struggling to afford their prescriptions: a majority of Congress chose Big Pharma over patients. Patients across all 50 States fought too hard for the historic progress we’ve made to watch this legislation undermine it at the last moment. We will keep fighting the implementation of ORPHAN Cures and any bills like it that would weaken Medicare negotiation, a program supported by 86% of Americans across party lines.”
The ORPHAN Cures Act creates a new loophole allowing some of the most profitable drugs with multiple orphan indications to avoid Medicare price negotiation, even as these drugs generate massive sales. The CBO estimates this carveout would cost taxpayers nearly $5 billion over the next decade while weakening the Medicare Negotiation Program just as it’s set to deliver savings to 9 million seniors starting in January.
P4ADNow patient advocates have sent 19,545 letters to Congress, met with Members of Congress, spoken out publicly, and talked to the media demanding lawmakers reject the ORPHAN Cures Act. While this bill now heads to the President’s desk, Patients For Affordable Drugs Now will continue to fight its implementation, protect the Medicare negotiation program, and work to secure lower prices for patients who need relief now more than ever.
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Patients For Affordable Drugs Now, is the only national, patient advocacy organization focused exclusively on policies to lower drug prices. We empower and mobilize patients and allies, hold accountable those in power, and fight to shape and achieve system-changing policies that make prescription drugs affordable for all people in the United States. P4ADNow is bipartisan and does not accept funding from organizations that profit from the development or distribution of prescription drugs. To learn more visit; PatientsForAffordableDrugsNOW.org.