This Week in Drug Pricing: Fighting ORPHAN Cures in the Senate, The Tide Continues to Turn Against PBMs, Supporting Transparency in Drug Advertisements on Capitol Hill, and ICYMI
Welcome to the Week in Review.
Fighting ORPHAN Cures in the Senate
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.
P4ADNOW urges Congress to Reject The Pharma-Backed Bill and Protect Key Drug Pricing Reforms
WASHINGTON, D.C. — Today, the House Energy and Commerce Committee advancedthe ORPHAN Cures Act(H.R. 946) through their markup. This legislation, driven predominantly by the pharmaceutical industry and its allies, 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 take effect in January. Patients For Affordable Drugs Now urges Congress to stop progression of this harmful bill.
“The overwhelmingly popular Medicare negotiation program is on the verge of delivering savings to nine million patients, and the last thing patients and the taxpayers need is a new loophole for the pharmaceutical industry to exploit,” said Merith Basey, Executive Director of P4ADNOW. “The Orphan Cures Act is yet another industry handout designed to protect corporate monopolies while undermining widely popular drug pricing reforms and overdue relief for patients”
An orphan drug designation applies to a treatment for conditions affecting fewer than 200,000 people in the United States. However, many drugs with multiple orphan indications reach much larger patient populations and are highly profitable for pharmaceutical manufacturers. The industry has spent millions of dollars misleading patients by falsely claiming that allowing multi-orphan drugs to remain eligible for price negotiation will harm innovation. The reality is the 2022 prescription drug law maintains all existing incentives for the development of orphan drugs, and only a small percentage of drugs approved for more than one orphan disease gross over $200 million under Medicare each year, the threshold for negotiation eligibility. Instead, the ORPHAN Cures Act will ensure prices are kept artificially high, unjustly undermining the victories that patients fought for.
Americans are already paying 3 to 8 times more than people in other wealthy nations pay for the exact same brand-name drugs, and 1 in 3 report difficulty affording their prescriptions.
Medicare negotiation is projected to save taxpayers $100 billion by 2031 — and thousands of P4ADNOW patient advocates have sent letters and taken digital action demanding Congress vote NO on ORPHAN Cures.
What’s next:The ORPHAN Cures Act is expected to move as part of a larger package advancing through Congress in the coming days. While the path forward is still taking shape. P4ADNOW will continue to fight to safeguard patient savings every step of the way, and we urge Congress to do the same.
You can find a one-pager fact sheet on the bill at the link here.
### 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.
WASHINGTON, D.C. – Patients For Affordable Drugs Now Executive Director Merith Basey issued the following statement in response to President Trump’s new Executive Order (EO) on drug pricing:
“We completely agree with President Trump that Americans shouldn’t be paying the highest drug prices in the world for the exact same brand-name prescription drugs. And we broadly support international reference pricing — including ‘Most Favored Nation’ style policies — that, if well designed, could help rein in inflated drug prices and deliver savings for patients.
This Executive Order is a step in the right direction, but without additional guardrails, it leaves room for pharmaceutical companies to continue gaming the system at the expense of patients. The reality is: drug companies set high prices in the U.S. because U.S. policy lets them – unlike other high income countries that negotiate lower prices.
With nearly nine in ten Americans demanding lower prices and greater accountability from drugmakers, the administration can pursue an even better deal, in line with other high income nations, including through the popular Medicare Negotiation program.
While outstanding questions remain, P4AD is committed to working to ensure this executive order is implemented in a way that delivers lower drug prices for patients. We’ll continue to monitor implementation closely and urge the administration to stay focused on patient-centered reforms that hold the true driver of high drug prices accountable: drug corporations.”
BACKGROUND
Several aspects of the EO raise immediate questions:
It’s unclear what immediate actions the administration will take — or how it defines “aggressive” actions that may follow if manufacturers choose not to voluntarily comply with MFN pricing targets.
The EO offers limited details about how HHS will enforce these targets or what mechanisms it would use to encourage compliance in the absence of new legislation or clearly defined regulatory authority.
While the order outlines a direct-to-consumer model at MFN prices, it does not provide information on how such a system would operate, whether it would be integrated with public or private insurance, or how HHS would establish and oversee it.
The EO also proposes revisiting drug importation policies, which could face logistical, regulatory, and trade challenges — particularly given the supply limitations in countries like Canada.
The EO does not specify which drugs would be impacted or how the proposal would interact with the existing Medicare Drug Price Negotiation Program under the Inflation Reduction Act.
### 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.
Growing Momentum Around International Reference Pricing
Senators Josh Hawley (R-MO) and Peter Welch (D-VT) unveiled a bipartisan bill that would bar drug companies from charging Americans more than the international average price for a specific prescription drug, adding to growing momentum around international reference pricing. Meanwhile, the Trump administration is considering a separate proposal that would tie Medicaid drug payments to foreign prices as part of broader budget reconciliation legislation, with new reports that Trump is expected to sign an executive order pursuing the initiative as soon as Monday. P4AD supports the principle that Americans shouldn’t be paying up to eight times more than patients in other countries, and we’re encouraged to see bipartisan efforts to lower drug prices through mechanisms such as international reference pricing. These proposals have the potential to deliver real relief for patients, but ultimately, the devil will be in the details. We’ll be watching closely to ensure any final plan truly lowers prices without restricting access here or abroad. — [POLITICO, Stat News, Axios, Endpoints News, Bloomberg, POLITICO]
P4AD Holds Community Town Hall
On Tuesday, we hosted a live Town Hall to answer questions from our community and shared the latest in our fight to stop pharma, its lobbyists, and allies from rolling back the progress we’ve made to lower drug prices. We covered everything from the threats to Medicare negotiation, to the potential harm of tariffs, to recent competition bills progressing through Congress, to efforts to reform the PBM system. Thank you to our community members who joined us live and to those of you who wrote to us with close to 600 questions! If you missed it, check out the video here!
Drug Spending Hits Record High As Patients Struggle to Afford Medications
According to Axios’ coverage of a new IQVIA report, Americans’ out-of-pocket spending on prescription drugs has surged 25% over the last five years, reaching a historic high of $98 billion last year. Even though total net medicine spending rose 11.4% last year, hitting a massive $487 billion, more than one-quarter of new prescriptions went unfilled — largely because they aren’t covered by insurance. It’s no surprise that nearly 90% of voters, including 89% of Trump voters, say prescription drug prices are too high, with nearly 60% of Americans reporting struggling to afford their medications in the last 12 months. Americans – who pay between four and eight times more than people in other wealthy countries – are urgently demanding that the White House and Congress do more to address this crisis of high drug prices. — [Axios, Arnold Ventures]
ICYMI
In a powerful report for ProPublica, David Armstrong reflects on the cancer drug Revlimid — the same drug that helped treat his own multiple myeloma — and the industry tactics that kept its price high for years. The piece, which includes reflections from P4AD’s David Mitchell, who is also a multiple myeloma patient, captures both the human stakes and the corporate strategies that put profit above patients.
In an exclusive first reported by The Hill, P4ADNow and AARP sent a joint letter to top House committee leaders urging them to reject the EPIC Act (H.R. 1492) — a pharmaceutical industry-backed bill that would delay Medicare drug price negotiation for small-molecule drugs from 9 to 13 years. The move comes as lawmakers consider what to include in the upcoming reconciliation package, where the drug industry is pushing for the bill’s inclusion. The letter argues that if Congress wants to “align” timelines for biologics and pills, as President Trump recently suggested, it should do so at 9 years, not 13, to protect savings for patients and taxpayers. Blocking earlier negotiation would mean drugs like Eliquis and Jardiance, which were negotiated in the first round, would not be eligible, costing Americans billions in higher prices. – [P4ADNow, The Hill, Axios, AARP]
Pharma-Backed Patent Bills Return
Yesterday, Senators Thom Tillis and Chris Coons reintroduced two harmful bills — the PREVAIL Act and PERA — that would entrench pharma monopolies and block lower-cost competition. P4ADNow strongly opposes both bills, which would weaken key patent oversight tools and expand patent protections that keep drug prices high. Patients helped defeat these harmful proposals in the last Congress, and are ready to do it again. With 78% of voters saying drug companies are focused on profits over people, advancing these bills isn’t just bad policy — it’s bad politics. – [P4ADNow, Arnold Ventures]
Pharma Opposes Tariffs —They Prefer to Hike Prices Themselves
The Trump administration’s investigation into pharmaceutical imports has raised the possibility of a 25% tariff on drugs and ingredients. A new PhRMA-commissioned report warns tariffs could raise U.S. drug prices by nearly 13% and cost Americans $51 billion annually — but let’s be clear: drug companies set drug prices, and while we agree tariffs would drive up costs and threaten access, Big Pharma’s warning is more about protecting its bottom line than protecting patients. As P4AD’s Merith Basey told The Lancet Oncology, it’s patients who would suffer the most, facing higher prices, potential shortages, and threats to the progress made under Medicare drug price negotiation. – [Reuters, Consumer Affairs, Pharmaphorum, The Lancet Oncology]
Pharma’s Scapegoat: Inside the $31M Campaign to Shift Blame
A new Wall Street Journal article reveals how Big Pharma’s $31 million lobbying blitz last year, plus another $13 million in Q1 2025, helped shift blame for high drug prices onto pharmacy benefit managers (PBMs). From funding minority-led nonprofits to underwriting conservative media campaigns, the piece exposes how drug companies are shaping the narrative to deflect scrutiny. It’s a calculated strategy that makes strange bedfellows — and while P4AD supports genuine reforms to rein in PBMs, it’s clear the drug industry is more focused on shifting blame and protecting profits than fixing the system. – [The Wall Street Journal]
ICYMI
P4AD released an open letter to our community – on the 100th day of President Trump’s second term – reaffirming our commitment to lowering drug prices. While Pharma ramps up lobbying and pushes carveouts like the EPIC Act, patients are organizing, fighting back, and driving progress. The fight continues.
PREVAIL and PERA Bills Would Undermine Reforms, Extend Monopolies, and Keep Drug Prices High
WASHINGTON, D.C. — Patients For Affordable Drugs Now strongly opposes the reintroduction of two harmful patent bills — the PREVAIL Act and the Patent Eligibility Restoration Act — which would further enable abuse of the patent system and prolong monopolies on brand-name medications.
“These dangerous bills would further rig the patent system in favor of the pharmaceutical industry,” said Merith Basey, Executive Director of Patients For Affordable Drugs Now. “At a time when one in three Americans can’t afford their prescriptions, Congress should be working day and night to lower prices, but instead, Senators Tillis and Coons are siding with Big Pharma and helping the industry further entrench its power at the expense of patients”.
While nearly 90% of Americans say prescription drug prices are too high — and more than 9 in 10 voters across party lines believe Congress must act to lower them — these proposals move in the wrong direction. Today, 29 million Americans are considered “cost desperate,” struggling to afford the medications and care they need. Yet instead of reining in Big Pharma, Congress is poised to expand its power — even as nearly two-thirds of voters view pharmaceutical companies unfavorably and see them as one of the top drivers of high health care costs. A full 78% of voters believe drug companies are focused on profits, not patients. Advancing these bills would not only undermine popular reforms but also stifle competition and hand even more control to an industry voters know is rigging the system against them.
BACKGROUND
Americans already pay between four and eight times what people in other wealthy nations pay for the exact same brand-name drugs.
The PREVAIL Act would restrict key tools used to challenge weak or abusive pharmaceutical patents, weakening oversight and limiting the ability of the U.S. Patent and Trademark Office to ensure patent quality.
The PERA Act would open the door to even broader patent protections for drugs, diagnostics, and other products, further locking in monopolies and blocking competition.
Between 2005 and 2015, 74 percent of the new drug patents issued were for drugs already on the market. Of the roughly 100 best-selling drugs in another study, nearly 80 percent obtained an additional patent to extend their monopoly period.
In September 2024, P4ADNow sent a letter to all the members of the Committee urging them to vote against S. 2220.
In the House: H.R. 6986 to address patent thickets; H.R. 9070, the Affordable Prescriptions for Patients Act; H.R. 1717, the Interagency Patent Coordination and Improvement Act; and H.R. 5429, the Medication Affordability and Patent Integrity Act.
In the Senate: S. 142, to ban pay-for-delay agreements; S. 148 and S. 1067, to reform the citizen petition process; and S. 79, to establish a task force between USPTO and FDA to improve communication in the implementation of each agency’s patent-related activities.
The Senate has already unanimously passed an amended version of S. 150. These Senate bills are estimated to save taxpayers $4.5 billion over the next 10 years.
### 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.
