STATEMENT: Patients For Affordable Drugs Now Urges Congress to Reject Revival of Pharma-Backed ORPHAN Cures Act
WASHINGTON, D.C. — Just days after patients won a critical victory to keep the ORPHAN Cures Act out of the Senate reconciliation bill text, lawmakers have reinserted this harmful, pharma-backed proposal into the proposed final text. P4ADNow is calling on Congress to remove the ORPHAN Cures Act once and for all and protect the historic Medicare negotiation program that patients fought to secure.
“Patients are infuriated to see the Senate cave to Big Pharma by reviving the ORPHAN Cures Act at the eleventh hour,” said Merith Basey, Executive Director of Patients For Affordable Drugs Now. “This is a blatant giveaway to the pharmaceutical industry that would keep drug prices high for patients while draining $5 billion in taxpayer dollars. We call on lawmakers to remove this unnecessary provision immediately and stand with an overwhelming majority of Americans who want the Medicare Negotiation program to go further. Medicare negotiation will deliver huge savings for seniors and taxpayers; this bill would undermine that progress.”
The ORPHAN Cures Act would create a new loophole allowing some of the most profitable drugs with multiple orphan indications to avoid price negotiation under Medicare, even as they generate massive sales. The CBO estimates this carveout would cost taxpayers nearly $5 billion over the next decade while diminishing the popular and effective Medicare negotiation program that is poised to deliver savings to millions of seniors starting next year.
P4ADNow patient advocates have sent over 13,000 letters to Congress demanding lawmakers reject the ORPHAN Cures Act, met with legislative offices, spoke out publicly, and talked to the media to make it clear that patients should not have to pay more just to pad pharma’s bottom line. Once again, patients are mobilizing to push back against this dangerous provision.
P4ADNOW urges Congress to protect patients and taxpayers by removing the ORPHAN Cures Act from the reconciliation package now.
### 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.
Investors- and Lawmakers – Aren’t Buying Pharma’s “Warnings”
New reporting from POLITICO reveals that lawmakers from both parties have been snapping up stocks in the 13 largest U.S. pharma companies in recent weeks — all while the drug industry ramps up its warnings that Inflation Reduction Act reforms and Most-Favored-Nation pricing momentum will hurt innovation and slash profits. The takeaway? Behind the industry’s sky-is-falling rhetoric, Wall Street and Washington still see pharma as a safe bet. No surprise, given that analysts have long noted that drugmakers could lose $1 trillion in revenue over a decade and remain the most profitable industry sector. The gap between the industry’s doomsday messaging and investor behavior is hard to ignore, and it’s patients who are left to slip through the cracks. — [POLITICO, West Health]
Pharma Front Groups Working Overtime For Big Drug Companies
It’s been a month since P4AD releasedThe Rampant Reach of Pharma’s Hidden Hand, exposing six major pharma-funded front groups masquerading as patient advocates while quietly pushing Big Pharma’s agenda. Since then, these groups have continued their efforts to protect drug industry profits. The Center for Medicine in the Public Interest (CMPI) — whose entire board has pharmaceutical industry ties — is now pushing a new report blaming high U.S. drug prices on so-called “global freeriding”, a classic pharma talking point that deflects blame and distorts reality. Meanwhile, Pacific Research Institute (PRI) President and CEO Sally Pipes is parroting similar messaging in a new op-ed, dismissing Most Favored Nation (MFN) as “a distraction” — no surprise given PRI’s deep financial ties to pharma. The problem isn’t that other countries pay too little, it’s that we’ve failed to hold pharmaceutical corporations accountable at home. The drug industry is one of the most profitable industries in the world, and its network of front groups continues to spread misinformation to protect its bottom line. Patients and policymakers deserve to know whose interests these groups serve. — [P4AD, CMPI, Newsmax, West Health]
P4AD Patient Advocate Speaks Up in New Op-Ed
The U.S. is experiencing a drug pricing crisis, and Latino communities are among those hit the hardest. 11% of U.S. adults have been unable to afford or access quality healthcare — its highest level since 2021 — and the most notable increases have been among Hispanic adults, rising 8% to 18%. This week, the Latino Newsletter published an op-ed from P4AD patient advocate Sonia Vega about her struggles to afford the Ozempic needed to manage her diabetes. Despite Ozempic costing less than $5 to manufacture, Sonia’s “out-of-pocket cost is over $1,000 a month” — and she’s not alone. P4AD is committed to fighting for equitable and affordable medication access in the Latino community, because no one should be forced to skip prescription doses or avoid treatment just to make ends meet. You can read Sonia’s full op-ed at the link here. — [Gallup, Latino Newsletter, CNBC]
Patient Victory: ORPHAN Cures Act Excluded from Reconciliation Text
This week, patients scored a key victory as the Senate Finance Committee released reconciliation text that excluded the harmful ORPHAN Cures Act – a pharma-backed proposal that would have weakened the Medicare Negotiation Program and protected drug company profits at the expense of patients. Over the past several weeks, P4ADNOW patient advocates mobilized to oppose the provision: sending 13,326 letters to Congress, meeting with lawmakers, and telling their powerful stories – including an Alabama patient advocate, Beth, who spoke with her senators. Beth takes Jakafi, an orphan drug that costs $17,500 every month, and she spoke out about how the Orphan Cures Act would exempt Jakafi and other orphan drugs from eventual price negotiations. Last Thursday, P4ADNOW and AARP joined forces to send a letter to Senate offices opposing the inclusion of ORPHAN. We’re proud to stand with the patients who made this happen, and we’ll keep fighting to ensure no version of ORPHAN resurfaces and every patient can get the medications they need at prices they can afford. — [P4ADNow, InsideHealthPolicy, POLITICO, Endpoints, Fierce Pharma, FirstWord Pharma, AInvest, Global Genes, The Pharma Letter]
I-MAK Releases New Report on Pharma Patent Abuse
On Tuesday, I-MAK published a new report chronicling how drug companies continue to manipulate the patent system to extend monopolies and keep drug prices artificially high. The report, which is part of I-MAK’s Overpatented, Overpriced series, focuses on the top-selling blood thinner Eliquis and the blockbuster family of diabetes and weight-loss drugs, including Ozempic, Rybelsus, and Wegovy — all of which are included in the second round of Medicare negotiations. By stacking patents to block competition, the report found that Bristol Myers Squibb and Pfizer have raked in an estimated additional $50.7 billion off Eliquis alone. Novo Nordisk is projected to make a staggering $166 billion over five years in additional profits by extending monopolies on its GLP-1 drugs. The report reinforces why patent reform is one of P4AD’s top priorities — and why Congress must act to stop pharmaceutical companies from using monopoly tactics to keep drug prices high for patients. — [I-MAK]
In Case You Missed It
The administration’s termination of over $1 billion in National Institute of Health (NIH) research grants was overruled by a federal judge this week. The NIH plays a critical role in drug development. In fact, 99 percent of drugs that received FDA approval between 2010 and 2019 received federal funding during their R&D. — [NIH, NBC News, Reuters, The Hill, Axios]
WASHINGTON, D.C. — Patients For Affordable Drugs Now is applauding a patient-driven victory as the harmful ORPHAN Cures Act was excluded from the reconciliation text released by the Senate Finance Committee today – protecting the Medicare Negotiation Program from a pharmaceutical industry carveout that would have driven up drug prices and padded Big Pharma’s profits at a time when one in three Americans still cannot afford their prescription drugs.
“The exclusion of the ORPHAN Cures Act from the Senate Finance Committee’s reconciliation text is a victory powered by patient advocacy and a direct rejection of the pharmaceutical industry’s greed-driven agenda,” said Merith Basey, Executive Director of P4ADNOW. “This decision helps protect the popular and effective Medicare Negotiation program and its promise of lower prescription drug prices for millions of Americans on Medicare. We remain vigilant against any effort to revive this harmful proposal, or anything like it, and will continue fighting to ensure every patient can get the medications they need at prices they can afford.”
Over the past several weeks, P4ADNOW patient advocates sent over 13k letters to the Hill, met with congressional offices, and spoke out publicly to oppose the ORPHAN Cures Act – legislation that would have allowed some of the most profitable drugs with multiple orphan indications to avoid price negotiation, despite being widely used and costing Medicare hundreds of millions of dollars each year. Last week, P4ADNow and AARP sent a joint letter to the Senate opposing ORPHAN Cures.
An “orphan” drug designation is meant to incentivize treatments for rare diseases affecting fewer than 200,000 people. But too often, pharmaceutical companies abuse the system by stacking multiple orphan indications to extend monopolies and keep prices high for as long as possible. The ORPHAN Cures Act would have enabled those abuses while weakening Medicare negotiation. To make matters worse, the Congressional Budget Office (CBO) found that this would cost the federal government nearly $5 billion over the next 10 years, an unnecessary gift to drug companies at the direct expense of patients and taxpayers.
Thanks to the leadership of champions in Congress and the groundswell of advocacy from patients nationwide, this proposal has been removed.
### 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.
P4ADNOW and AARP Send Letter on OPRHAN Cures to Senate
To safeguard hard-fought and cost-saving drug price reforms, P4ADNow and AARP sent a joint letter to all Senators to oppose the inclusion of the ORPHAN Cures Act in the reconciliation bill. At a time when nine in ten Americans want further action from Congress to lower the price of prescription drugs and an overwhelming majority support giving Medicare the power to negotiate lower drug prices for ALL drugs, including this policy in the reconciliation package would be a step backward. The Congressional Budget Office (CBO) found that the ORPHAN Cures Act would increase federal spending by $4.87 billion over the next 10 years. That’s billions of dollars going straight into the pockets of big drug companies. We’re urging Congress to put patients first rather than bend to the whims of greedy drug manufacturers. — [P4ADNow, AV, CBO]
Most Favored Nation: One Month Out
This week marked 30 days since President Trump issued his executive order to align U.S. drug prices with other high-income nations through the “Most-Favored-Nation” (MFN) approach. In the U.S., drug companies charge Americans between four and eight times what patients in other high-income countries pay for the very same brand-name drugs because the current system lets them. If done right, international referencing pricing could help lower prices in the U.S. But as expected, Big Pharma is already pushing back hard. Pfizer and other drugmakers met with the administration, but failed to make any commitments to lower prices. Instead, Pfizer CEO and PhRMA Board Chair Albert Bourla said he hopes prices in Europe increase, even threatening to pull drugs from some countries if they don’t pay more. We’ve said it before: any proposal that seeks to raise prices abroad is a nonstarter. The real problem is that the U.S. has failed to rein in the pharmaceutical industry. Americans know who the drivers of high drug prices are, and pharma’s recent comments make it even clearer where their priorities lie. — [White House, P4ADNow, Quartz, Reuters]
Spotlight on Cell and Gene Therapies
The heads of HHS, FDA, NIH, and CMS came together for a roundtable discussion on the future of cell and gene therapies, where NIH Director Jay Bhattacharya emphasized the importance of making these life-saving treatments both scalable and affordable. Treatments like these have been critical for patients like Janet Kerrigan, whose CAR-T therapy put her multiple myeloma into remission. But no one should be saddled with the enormous price tags these innovations currently carry, which can reach nearly $3 million for disorders like sickle cell. Lowering this cost is a priority, and we’re encouraged to hear that affordability might also be on the radar for federal leaders as they chart the path forward. — [Endpoints News, P4AD, Ortho]
In Case You Missed It
On Monday, the FDA announced it would be reinstating the internal team responsible for advancing generic drug approvals, reversing its controversial decision to dismantle the office earlier this year. As Endpoints News noted, the team played a key role in driving the agency to a record number of new generic drug approvals before its elimination in April. — [BioSpace, Endpoints News, Pink Sheet]
As the Senate considers the House-passed reconciliation package, P4ADNow is urging lawmakers to reject the inclusion of the ORPHAN Cures Act — an unnecessary Pharma handout that would weaken the effective and popular Medicare Negotiation Program. At a time when nine in ten Americans want further action from Congress to lower drug prices and an overwhelming majority support giving Medicare the power to negotiate lower drug prices for every drug, including this policy that would reduce the number of drugs eligible for negotiation would be a harmful step backward. The Congressional Budget Office (CBO) found that the bill would cost taxpayers $4.8 billion over the next 10 years. That’s nearly $5 billion in lost savings — not to support innovation or new treatments, but to allow drug companies to keep prices high for longer at the expense of patients. Americans know that Pharma is to blame for their high drug prices, and over the coming days, P4AD patient advocates who would be impacted by this legislation will be speaking to their Senators to express their strong opposition to the ORPHAN Cures Act. — [Axios, CMS, CBO]
The Tide Continues to Turn Against PBMs
Arkansas is taking on PBMs with its first-in-the-nation law barring PBMs from owning pharmacies in the state. The profitability of PBMs has risen in recent years as a result of vertical mergers between PBMs, insurance companies, and pharmacies, leading to higher prices for patients. Arkansas’ law represents a major step toward dismantling these vertically integrated monopolies that contribute to keeping prices artificially high. While PBMs claim to be utilizing their bargaining power on behalf of patients, they’ve simultaneously fought to ensure their rebate practices stay hidden from view. As Axios reported, “the ramifications are nationwide, with many other states weighing new restrictions, including prohibitions on steering business to affiliated pharmacies.” — [Axios, Axios, Reuters, Pharmacy Times]
Supporting Transparency in Drug Advertisements on Capitol Hill
On Thursday, Reps. Dave Taylor (R-OH) and Jan Schakowsky (D-IL) introduced the Drug-Price Transparency for Consumers Act of 2025, companion legislation to the Senate version that would mandate pharmaceutical companies disclose the cost of their drugs in all direct-to-consumer (DTC) advertising. This joins the growing trend against DTC Pharma advertising, after Senators Josh Hawley (R-MO) and Jeanne Shaheen (D-NH) introduced the bipartisan and P4ADNow-endorsed No Handouts for Drug Advertisements Act last month. The U.S. is one of only two countries that allows DTC advertising for pharmaceutical drugs, and the least patients deserve is transparency on the cost of what’s being advertised to them. P4ADNow endorsed this bill, and is supportive of efforts to bolster transparency at all steps of the pharmaceutical process. — [Washington Examiner]
In Case You Missed It
This week, P4AD attended Latino Magazine’s LATINO 100 luncheon. Latino patients are more likely to report difficulty affording prescription medications, and Gallup polling earlier this year found that Hispanic adults are reporting the greatest increase in lack of ability to afford or access quality healthcare. P4ADNow is working closely with organizations and groups who serve the Latino community as part of our push to expand our community in the fight to lower drug prices.
WASHINGTON, D.C. — Today, the House passed a reconciliation package that includes theORPHAN Cures Act(H.R. 946), a pharma-backed provision that would create new loopholes in the Medicare Drug Price Negotiation Program — just months before the first lowered prices take effect. Patients For Affordable Drugs Now strongly opposes the inclusion of this policy, which serves the interests of drug corporations at the expense of patients and taxpayers.
“In a giveaway to Big Pharma, the House just voted to weaken one of the most effective and popular drug pricing reforms in decades — ignoring the will of nine in ten Americans who are demanding Congress do more to lower prescription drug costs,” said Merith Basey, Executive Director of P4ADNOW. “By letting high-cost drugs with multiple orphan uses avoid negotiation and extending the timeline before negotiation for other medications, lawmakers have handed pharmaceutical corporations a new and unnecessary way to protect monopoly pricing — all while 1 in 3 Americans can’t afford their medications.”
Despite industry claims, the ORPHAN Cures Act does nothing to advance innovation. The 2022 prescription drug law already safeguards orphan drug development incentives. Instead, this bill keeps U.S. prices high while other nations pay far less. According to the CBO, the bill would cost taxpayers at least $5 billion over the next 10 years — a massive giveaway to the pharmaceutical industry at the expense of patients.
P4ADNOW patient advocates have mobilized in opposition to this bill, sending thousands of letters to Congress urging its rejection. The fight is not over — the reconciliation package now heads to the Senate, where P4ADNOW will continue working to protect patient savings and block harmful industry carveouts.
### 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.
Trump Revives Global Drug Pricing Debate
This week, President Trump issued an Executive Order (EO) focused on addressing the high cost of prescription drugs. One key provision aims to align U.S. prices with those in other high-income nations — a concept referred to as “Most Favored Nation” (MFN). Right now, Americans pay the highest drug prices in the world for two main reasons: U.S. policy allows drug companies to set launch prices and raise them at will, and unlike other countries that negotiate prices for nearly all prescription drugs, the U.S. only recently began limited negotiation for a small set of expensive drugs through Medicare. That’s why international reference pricing frameworks like MFN are worth exploring — but only if they’re done right. P4ADNow supports pursuing a better deal for Americans — including through the widely popular Medicare Negotiation program — however, any MFN policy must meaningfully lower prices for U.S. patients, without driving up costs or limiting access abroad, and should include strong guardrails to prevent drug companies from exploiting new loopholes. Check out P4ADNow’s Executive Director Merith Basey’s interview on the EO on Univision. — [New York Times, Forbes, Epoch Times, Citeline, Healthcare Finance, STAT News, Endpoints News, Reuters, POLITICO, Washington Post, Bloomberg Law, USA Today]
Another Win for Patients and Another Warning For Big Pharma In Court
Patients netted a massive victory this week, after the U.S. Court of Appeals for the Third Circuit rejected AstraZeneca’s challenge to the Medicare Drug Price Negotiation Program in a unanimous ruling — affirming that Medicare can and should be negotiating lower drug prices on behalf of seniors and people with disabilities. This ruling marks the first appellate decision evaluating and upholding the negotiation program’s core framework, and it serves as a warning to other drug companies attempting to protect their outrageous profits through the court. As Michael Lieberman of Fairmark Partners — the firm representing P4AD in amicus briefs — told Inside Health Policy, the decision is a “positive sign for the government regarding how the Third Circuit might rule in other cases.” To date, P4AD has submitted four amicus briefs on behalf of patients and has signed onto seven amicus briefs in our campaign to challenge Big Pharma’s attempts to undermine Medicare negotiation through the U.S. legal system. — [Endpoints News, Inside Health Policy, P4AD, Pink Sheet, Bloomberg Law, Reuters, FirstWord Pharma, Law360]
The ORPHAN Cures Act Would Undermine Patient Savings
On Wednesday, the House Energy and Commerce Committee advanced the ORPHAN Cures Act (H.R. 946), which was included in a larger reconciliation bill largely focused on Medicaid. Driven predominantly by the pharmaceutical industry and its allies, ORPHAN Cures would create an unnecessary loophole in the Medicare Negotiation Program, allowing even more drugs to avoid price negotiation — including those indicated for multiple rare diseases — right before the first round of negotiated prices takes effect in January. Medicare negotiation is on the verge of delivering $1.5 billion in savings to nine million patients and $6 billion in taxpayer savings, so the last thing Americans need is a new loophole for the pharmaceutical industry to exploit. Thousands of P4ADNow patient advocates have sent letters demanding Congress vote no on ORPHAN Cures, and P4ADNow is urging Congress to side with patients and stop the progression of this harmful bill. — [Congress.gov, CMS]
Endorsing the No Handouts for Drug Advertisements Act
On Wednesday, Senator Jeanne Shaheen (D-NH) and Senator Josh Hawley (R-MO) introduced the No Handouts for Drug Advertisements Act to eliminate a tax break that fuels the flood of prescription drug ads designed to boost profits for Big Pharma and drive up prices for patients. The U.S. is one of just two countries that even allows direct-to-consumer (DTC) drug advertising — there’s no reason taxpayers should be footing the bill for it. We’re proud to support Senators Shaheen and Hawley’s common-sense reform to put patients before pharma profits. — [Inside Health Policy]
ICYMI
Patients For Affordable Drugs’ founder and president, David Mitchell, has been included in Washingtonian’s Washington DC’s 500 Most Influential People of 2025 for the third year in a row. David’s commitment to reforming the drug price system without taking a penny from industry has made him one of the most trusted and influential voices on this issue.
