A version of this story was originally published by member station WAMU.

With the GOP fully in control of the federal government for the first time since 2006, Congressional Republicans are taking their first steps to assert their power over the District of Columbia’s local government.

After an impassioned debate, the House Oversight and Government Reform Committee voted Monday evening to block a D.C. law giving District physicians the right to prescribe lethal medication to terminally ill patients who have less than six months to live.

Committee chair Jason Chaffetz and other Republican conservatives have argued that the D.C. law should be nullified because it runs counter to ethical prohibitions against suicide. Most of the Republicans on the committee framed their opposition as a “pro-life” stance, with a number expressing concerns that the D.C. law could leave vulnerable dying patients at the mercy of physicians and relatives eager to hasten their exit.

“I worry that assisted suicide will create a marketplace for death,” Chaffetz said.

Rep. Darrell Issa R-Calif., who represents one of six states that already have enacted legislation similar to D.C.’s right-to-die bill, was the only Republican to vote against the measure. Issa argued that given Congress’ failure to stop such legislation nationally, he didn’t think interference in D.C. affairs was justified.

In a statement after the committee vote, Mayor Bowser called it “a signal to DC residents that Congress has zero respect or concern for their will or the will of their elected officials.”

Though the District gained an elected mayor and legislature in 1973, Congress retained broad authority over the city. That included the right to disapprove of bills — or, in simpler words, block bills passed by the D.C. Council from taking effect. But that right has been used sparingly: Congress has only formally blocked three D.C. bills over the last 45 years.

The panel voted 22-14 in favor of sending to the House floor the bill to the House floor. However that may be as far as it goes.

Unless conservative Republican backers of the manage to get the committee-passed “resolution of disapproval” through the House and Senate and signed by President Donald Trump by the end of the week — an exceedingly uphill battle given the normal pace of Congress — the law appears likely to take effect as passed by D.C. Council and signed into law by Mayor Muriel Bowser.

The bill becomes law if it is not blocked within 30 legislative working days of being sent Congress. By the D.C. City Council’s calculations, time is up for opponents of the Death With Dignity bill on or about Saturday.

That leaves an almost impossibly narrow window for floor votes in the House and Senate. Moreover, it is not even certain that Trump would sign a disapproval resolution. White House press secretary Sean Spicer refused to say what the president might do when asked about the Death With Dignity law at a press briefing last week.

The audience at the committee meeting, which lasted until past 7:30, included activists in the right-to-die movement, including at least one who considers herself a potential beneficiary: Mary Klein, a 69-year-old D.C. resident who is dying of cancer. In an earlier interview with WAMU, Klein described the measure’s passage, by an 11-2 D.C. Council vote in November, as “a great relief.” Dan Diaz, the widower of Brittany Maynard, a young woman who wrote about her decision to end her life after a long battle with brain cancer, also turned up “to support D.C.,” he said.

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In response to outrage from patients and lawmakers, Marathon Pharmaceuticals has delayed the launch of an $89,000 drug for Duchenne muscular dystrophy.

The company had announced the annual list price for Emflaza, which is a steroid, after the Food and Drug Administration approved the drug Thursday.

Emflaza is approved as an orphan drug, which means it is intended to treat a rare disease. Duchenne is an inherited disorder that causes muscles to become weak. There is no cure for the condition, which mainly affects boys, but some drugs, including Emflaza, are used to lessen symptoms.

For years, many American patients have imported deflazacort, the generic version of Emflaza, for about $1,200 a year. But because the medicine wasn’t approved in the U.S., the cost of the medicine wasn’t typically covered by insurers.

That contrast in price between became a flash point Monday as Sen. Bernie Sanders, I-Vt., and Rep. Elijah Cummings, D-Md., sent a letter to Marathon on Monday morning demanding answers about the $89,000 price for a drug that isn’t new. It has been used routinely by Duchenne patients in the U.S. since at least 2005.

“We believe Marathon is abusing our nation’s ‘orphan drug’ program, which grants companies seven years of market exclusivity to encourage research into new treatments for rare diseases — not to provide companies like Marathon with lucrative market exclusivity rights for drugs that have been available for decades,” Sanders and Cummings wrote.

Marathon said FDA approval would help more patients get the drug.

“Our goal in commercializing Emflaza all along has been to make it available to that broader set of patients who prior to FDA approval have not had access to the therapy,” said a statement attributed to Marathon CEO Jeffrey Aronin that was read Monday to a meeting of parents, patients and advocates in Washington, D.C. “We are pausing our launch, which has not yet taken place. We have not sold any new product and will pause that process.”

But patients and their families cried foul.

“What you’re doing is robbing my insurance company,” said Dana Edwards, a mother from New Jersey whose 12-year-old has taken deflazacort, the generic version of the drug, since he was 5.

Pat Furlong, the president and founder of Parent Project Muscular Dystrophy, which sponsored the Monday conference, read the statement to an outraged crowd in a conference room at the Mayflower Hotel.

“Since last week’s approval, they’ve heard from all of us,” he said. Furlong told the audience that complaints from patients helped to prompt Marathon’s action.

The company, Furlong read, will continue to offer patients an expanded access program, which allows about 800 patients to receive the drug from the company. More can join that program free, and patients can continue importing the drug from Canada or “wherever they are getting it,” the statement said.

In January, Kaiser Health News and NPR published an investigation that found the orphan drug law intended to help desperate patients is being manipulated by drugmakers for financial gain.

Republican Rep. Robert Aderholt, the chairman of the appropriations subcommittee that funds the FDA, called Marathon’s announcement a “tipping point.” And he said the astronomical increase in deflazacort’s price “makes me question whether the current construct of how the FDA approves orphan drugs does more harm than good if companies have found a way to game the system.”

At Monday’s meeting, Marathon executives presented a slideshow on financial assistance for patients, as well as details on the drug’s expected availability. Emflaza was set to hit the market by mid-March, said Eric Messner, vice president of sales and marketing for Marathon.

Messner said the drug would cost $50,000 to $54,000 after rebates and discounts to insurers. “I know there has been a lot of discussion about that. How did we arrive at the price?” Messner said.

An audience member interrupted Messner asking if the company had talked to patients and families about the price.

He offered assurance that they did and said there would be an ongoing dialogue with patient groups. “We expect that patients will experience a low or zero out-of-pocket experience,” he said.

Late Monday, Marathon released an official statement and open letter to the Duchenne community, promising that “price should not be a barrier” for patients. “Put simply we expect patients will pay a standard copay of typically $20 or less per prescription.

Edwards, the New Jersey mother, said she hopes President Trump will fulfill his vow to bring down high drug prices.

Kaiser Health News’ coverage of prescription drug development, costs and pricing is supported in part by theLaura and John Arnold Foundation.

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Every night, Jason Early attaches a catheter in his chest to a machine by his bed that, over the course of nine hours while he sleeps, removes his blood from his body, cleanses it and returns it because his kidneys are no longer able to do the job.

It’s been about 18 months since the 28-year-old Dallas resident started getting dialysis after his kidneys failed as a complication from the Type 1 diabetes with which he was diagnosed as a child.

Like many patients with end-stage renal disease, Early, who is completing a bachelor’s degree in finance at the University of North Texas at Dallas, turned to a charity for financial assistance to cover his health insurance costs.

Such “third-party payments” by nonprofit groups, health care providers and others are controversial. The federal government has expressed concern that health care providers and organizations they’re affiliated with might be inappropriately “steering” patients to marketplace plans instead of Medicare or Medicaid, for which they are often eligible. The public programs reimburse for the dialysis services at lower rates than most private plans. The efforts by charities have also long been a sore spot with health insurers, who say they encourage sick patients who have expensive health care needs to opt for private coverage.

Insurers suffered a setback recently when a federal judge temporarily blocked a new rule from the Department of Health and Human Services that was set to go into effect Jan. 13. It would require that dialysis centers inform insurers if the centers are making premium payments either directly or indirectly through a third party for people covered by marketplace plans. Insurers would then have the option of accepting or denying the payments.

In granting the preliminary injunction in late January, U.S. District Court Judge Amos Mazzant in Sherman, Texas, criticized the government’s administrative process for establishing the regulation and said it hadn’t considered the benefits of private individual insurance or the fact that the rule would leave thousands of patients without coverage.

Insurers were not pleased. “Inappropriate steering and third-party payments increases costs for all consumers, and it risks harm to patients who are often eligible for public coverage options,” said Kristine Grow, a spokesperson for America’s Health Insurance Plans, an industry group. “We continue to urge [the Department of Health and Human Services] to prohibit these payments when there is alternative coverage for patients.”

But patient advocates were delighted. “We thought this was an important win for dialysis patients because it not only spoke to the procedural elements of the rule but to the substance, the potential of dialysis patients to have their coverage taken away,” said Hrant Jamgochian, the chief executive of Dialysis Patient Citizens, an advocacy group that was the lead plaintiff in the lawsuit.

Premium assistance has been critical to improving Early’s quality of life. “Without the [American Kidney Fund] assistance I would be living to pay my medical costs,” he said. “They give me an opportunity to get a breather from medical costs so that I can live my life outside of my illness.”

Although some patients, such as Early, are able to undergo dialysis at home, many must spend four hours at a dialysis center three times a week. The process is debilitating and time-consuming.

Many people lose their employer-sponsored health insurance because they are unable to work. Medicare is often an alternative because under the law people with end-stage renal disease – even those younger than 65 — are generally eligible for coverage.

Others may sign up for private coverage on the exchanges. Some may qualify for Medicaid.

Advocates for kidney patients say coverage on the individual market is better than Medicare for some people. In marketplace plans, the maximum amount that someone can be required to pay out-of-pocket for covered services in 2017 is $7,150. But there’s no cap on beneficiaries’ spending in Medicare, and patients are on the hook for 20 percent of the cost for doctor visits and other outpatient services such as dialysis. Supplemental “Medigap” plans can help cover out-of-pocket costs, but 23 states don’t require insurers to sell those plans to people with end-stage renal disease who aren’t yet 65.

Last year, Early bought an Aetna silver plan with a $1,500 deductible and a $6,000 out-of-pocket maximum on the Texas health insurance exchange. The American Kidney Fund paid the $359 monthly premium. The policy covered all of his diabetes drugs and equipment. By the end of the first month of dialysis copayments, Early reached his spending maximum and the plan paid everything after that.

But Aetna exited several marketplaces in 2017, including Texas, and Early signed up for Medicare in January. Now he’s responsible for a $134 monthly Part B premium, $65 for his prescription drug plan and $300 a month in copays for drugs.

Medicare doesn’t cover the insulin pump he uses, so he’ll have to pay $300 monthly out of pocket for that too. At the moment he’s also paying $478 each month for the basic Medigap “A” supplemental plan available in Texas to patients younger than 65 with end-stage renal disease. The Medigap plan covers his 20 percent coinsurance payments for dialysis and other outpatient care.

He said he’s been told that the American Kidney Fund, a charity that provides assistance to about 20 percent of the more than 450,000 people who are on dialysis and receives funding from dialysis providers, will start picking up his Medigap premiums soon. He hopes so.

“I miss my Aetna plan,” he said.

Sixty percent of the people who receive premium assistance from the American Kidney Fund get help with their Medicare or Medigap plans rather than marketplace or other private coverage, said LaVarne Burton, the president and CEO. Even though the judge’s order doesn’t apply to people in public plans, she hopes it will discourage insurers that are increasingly erecting barriers to third-party payment.

“We want to protect the ability that these individuals have to make the choice that meets their needs,” Burton said.
Kaiser Health News is an editorially independent news service that is part of the nonpartisan Henry J. Kaiser Family Foundation. You can follow Michelle Andrews on Twitter: @mandrews110.

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Anti-abortion rights protesters gathered outside Planned Parenthood clinics across the country Saturday, in a series of rallies calling on politicians to end federal funding for the century-old organization. The activists planned to picket outside roughly 200 Planned Parenthood clinics — but at many of those locations, counterprotesters were there to meet them.

The group #ProtestPP, which organized the rallies, says demonstrations have been planned in at least 45 states, according to ABC News. From Detroit to Los Angeles, from St. Louis to St. Paul, The Associated Press reports that protesters have gathered with signs reading “Pray to End Abortion” and “Defend Life.”

“Why should tax dollars be given to somewhere where they’re doing killings of babies,” anti-abortion demonstrator Marisol Pfaff asked St. Louis Public Radio’s Camille Philips. Pfaff and scores of other protesters gathered Saturday outside the only clinic in Missouri that provides abortions.

Planned Parenthood — which provides a wide range of health care services for women, including abortion — receives more than $500 million a year from the federal government, about 75 percent of which comes from Medicaid.

According to Planned Parenthood, about 3 percent of the services it provided in 2014 were abortion-related. The group says the vast bulk of its services instead go toward contraception and the prevention and treatment of sexually transmitted diseases. It also provides cancer screenings and other women’s services.

That’s why Sharon Caldwell and a large group of other counterprotesters came out to the St. Louis clinic to rebut the claims of the anti-abortion rally Saturday. Caldwell tells Phillips the organization provided invaluable help for her after she was raped.

“They were the only people who cared,” Caldwell says. “They were just like, ‘A terrible thing happened. Here’s how we support you.’ “

The dueling rallies in St. Louis offer just a microcosm of the flurry of protests and counterprotests in cities across the U.S.

In St. Paul, Minn., The Minneapolis Star-Tribune describes a scene in which thousands of protesters and counterprotesters faced each other on the same pavement just a dozen feet apart, held back by parallel crowd barriers. The Planned Parenthood supporters — who far outnumbered their anti-abortion counterparts, according to the paper — shouted chants of “Defund Pence” and “Tax the church!”

Meanwhile, in Denver, the balance between Planned Parenthood’s opponents and supporters was reversed — with abortion rights opponents outnumbering the counterdemonstrators outside a clinic. Colorado Public Radio’s Sam Brasch reports that Planned Parenthood officials actually asked supporters to stay away, fearing conflict would only make patients even feel more unwelcome.

Leslie Hanks, an anti-abortion demonstrator in Denver, told Brasch he hopes President Trump “does the right thing and I hope that Planned Parenthood is defunded, but that’s the very least that should happen.”

Anti-abortion activists have been re-energized with a new, much more sympathetic administration having moved into the White House. Vice President Mike Pence addressed the annual March for Life last month — a historic first in the major anti-abortion rally’s decades-long history — and President Trump has blocked U.S. aid money from supporting groups that provide or “promote” abortion in other countries.

Still, Planned Parenthood supporters remain undeterred.

“All across the country, Planned Parenthood supporters are taking it upon themselves to organize in their communities on their own,” Kelley Robinson,of Planned Parenthood Action Fund Support, said in a statement.

“Saturday, and every day, Planned Parenthood advocates and activists show that they refuse to be intimidated and they won’t back down.”

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Patients in Alexandria, La., were the friendliest people Dr. Muhammad Tauseef ever worked with. They’d drive long distances to see him, and often bring gifts.

“It’s a small town, so they will sometimes bring you chickens, bring you eggs, bring you homemade cakes,” he says.

One woman even brought him a puppy.

“That was really nice,” he says.

Tauseef was born and raised in Pakistan. After going to medical school there, he applied to come to the U.S. to train as a pediatrician.

It’s a path thousands of foreign-born medical students follow every year — a path that’s been around for more than half a century. And, like most foreign-born physicians, Tauseef came on a J1 visa. That meant after training he had two options: return to Pakistan or work for three years in an area the U.S. government has identified as having a provider shortage.

He chose to work with mostly uninsured kids at a pediatric practice in Alexandria. “That was a challenge,” he says. “But it was rewarding as well because you are taking care of people who there aren’t many to take care for.”

And the U.S. medical system depends on doctors like Tauseef, says Dr. Andrew Gurman, president of the American Medical Association. He worries that if President Trump’s executive order on immigration takes effect, it will mean parts of the country that desperately need medical care may not have a doctor.

“International medical graduates have been a resource to provide medical care to areas that don’t otherwise have access to physicians,” he says. “With the current uncertainty about those physicians’ immigration status, we don’t know whether or not these areas are going to receive care.”

According to the AMA, today there are about 280,000 international medical graduates in the U.S. That’s about 1 in 4 doctors practicing here. Some are U.S. citizens who’ve gone abroad for medical school, but most aren’t.

“They don’t all have permanent visas, and so a lot of them are concerned about what their status is going to be, whether they can stay, whether they can go home to visit family and still come back, and the communities they serve have similar questions,” he says.

And the care provided by the graduates of foreign medical schools is, by and large, top notch. A study published Feb. 3 in the journal The BMJ, formerly The British Medical Journal, shows Medicare patients treated by doctors with degrees from non-U.S. medical schools get just as good care — and sometimes better — than those treated by graduates of American medical schools.

The immigration uncertainty is hitting medical schools at a tough time. Dr. Salahuddin Kazi is in charge of recruiting top students from across the world for the University of Texas Southwestern residency program.

“Typically we have 3,000 people applying for our 61 positions — of those 3,000, at least half of them are international medical graduates,” he says.

Applicants find out their program match in March and usually start working in June. That gives them about 90 days to get a visa. Kazi worries this year that won’t be long enough and that students from countries included in the travel ban won’t be admitted.

“That would create hardship for the hospital, for us and for our remaining residents,” he says. “They’ll have to pick up more shifts or give up vacation.”

Pediatrician Tauseef left Louisiana two years ago but continues to care for low-income patients at Los Barrios Unidos Community Clinic in Dallas. Six of the 30 physicians who work at this clinic are from other countries.

Tauseef says they’re all educated to do the same thing. “As a physician, being a foreign medical graduate, U.S. medical graduate, a Muslim doctor, a non-Muslim, we are trained to look for signs and symptoms,” he says. “We do not look at anybody’s color; we are not trained to look at anybody’s religion or ethnicity.”

Tauseef, who has been in America for 13 years, says he will apply for U.S. citizenship in March.

This story is part of a reporting partnership with NPR, KERA and Kaiser Health News.

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The confirmation of Tom Price, the orthopedic surgeon-turned-Georgia congressman, as secretary of Health and Human Services on Friday represents the latest victory in the ascendancy of a little-known but powerful group of conservative physicians in Congress — the GOP Doctors Caucus.

During the Obama administration, the caucus regularly sought to overturn the Affordable Care Act, and it’s now expected to play a major role determining the Trump administration’s plans for replacement.

Robert Doherty, a lobbyist for the American College of Physicians, the professional organization for internal medicine doctors, says the GOP Doctors Caucus has gained importance with Republicans’ rise to power. “As political circumstances have changed, they have grown more essential,” Doherty says.

“They will have considerable influence over the discussion on repeal and replace legislation,” he says.

Price’s supporters have touted his medical degree as an important credential for his new position, but Price and the caucus members are hardly representative of America’s physicians in 2017.

The “trust us, we’re doctors” refrain of the caucus obscures its conservative agenda, critics say.

“Their views are driven more by political affiliation,” says Mona Mangat, an allergist-immunologist and chair of Doctors for America, a 16,000-member organization that favors the current health law. “It doesn’t make me feel great. Doctors outside of Congress do not support their views.”

For example, while the American College of Obstetrics and Gynecology has worked to protect access to abortion, the three obstetrician-gynecologists in the 16-member House caucus are anti-abortion and oppose the ACA provision that provides prescription contraception without copays.

While a third of the U.S. medical profession is now female, 15 of the 16 members of the GOP caucus are male, and only eight of them are doctors.

The other eight members are from other health professions, including a registered nurse, a pharmacist and a dentist. The nurse, Rep. Diane Black of Tennessee, is the only woman.

On the Senate side, there are three physicians, all of them Republican and male: Sen. John Barrasso, an orthopedic surgeon from Wyoming; Sen. Bill Cassidy, a gastroenterologist from Louisiana; and Sen. Rand Paul, an ophthalmologist from Kentucky.

While 52 percent of American physicians today identify as Democrats, just two out of the 14 doctors in Congress are Democrats, Reps. Ami Bera and Raul Ruiz, both of California.

About 55 percent of physicians say they voted for Hillary Clinton and only 26 percent voted for Donald Trump, according to a survey by Medscape in December.

Meanwhile, national surveys show doctors are almost evenly split on support for the health law, mirroring the general public. And a survey published in the New England Journal of Medicine in January found almost half of primary care doctors liked the law, while only 15 percent wanted it repealed.

Rep. Michael Burgess, R-Texas, a caucus member first elected in 2003, is one of the longest serving doctors in Congress. He says the anti-Obamacare Republican physicians do represent the views of the profession.

“Doctors tend to be fairly conservative and are fairly tight with their dollars, and that the vast proportion of doctors in Congress [are] Republican is not an accident,” he says.

Price’s ascendency is in some ways also a triumph for the American Medical Association, which has long sought to beef up its influence over national health policy. Less than 25 percent of practicing physicians are in the AMA, the organization says.

Price is an alumnus of a boot camp the AMA runs in Washington each winter for physicians contemplating a run for office. Price is one of four members of the caucus who went through the candidate school. In December, the AMA immediately endorsed the Price nomination, a move that led thousands of doctors who feared Price would overturn the health law to sign protest petitions.

Even without Price, Congress will have several GOP physicians in leadership spots in both the House and Senate.

Those include Rep. Phil Roe of Tennessee, the caucus co-chair, who also chairs the House Veterans Affairs Committee, and Burgess, who chairs the House Energy and Commerce subcommittee on health. Sen. Cassidy sits on both the Finance and the Health, Education, Labor and Pension Committees. Sen. Barrasso chairs the Senate Republican Policy Committee.

Roe acknowledges that his caucus will have newfound influence. Among his goals in molding an ACA replacement are to kill the requirement that most people buy health insurance (called the individual mandate) as well as to kill the requirement that 10 essential benefits, such as maternity and mental health care, must be in each health plan.

He says the caucus will probably not introduce its own bill, but rather evaluate and support other bills. The caucus could be a kingmaker in that role. “If we came out publicly and said we cannot support this bill, it fails,” Roe says.

The GOP Doctors Caucus has played a prominent role in health matters before.

For example, in 2015, when former House Speaker John Boehner needed help to permanently repeal a Medicare payment formula that threatened physicians with double-digit annual fee cuts, he turned to the GOP Doctors Caucus. It got behind a system to pay doctors based on performance — the so-called “doc fix.”

“When the speaker had a unified doctors’ agreement in his coat pocket, he could go to Minority Leader Nancy Pelosi and show that, and that had a lot to do with how we got this passed,” Roe says.

But not all doctors are unified behind the caucus. Ruiz, one of the two physicians in the House who are Democrats, says he worries because few doctors in Congress are minorities or primary care doctors.

Ruiz, an emergency room physician who was elected in 2012, says he is wary about Price leading HHS because he is concerned Price’s policies would increase the number of Americans without insurance.

Indeed, many doctors feel the caucus’ proposals will not reflect their views — or medical wisdom.

“My general feeling whenever I see any of their names, is that of contempt,” says Don McCanne, a family medicine physician in California who is past president of the Physicians for a National Health Program. “The fact that they all signed on to repeal of ACA while supporting policies that would leave so many worse off demonstrated to me that they did not represent the traditional Hippocratic traditions which place the patient first.”

This story was produced through a collaboration between NPR and Kaiser Health News, an editorially independent program of theHenry J. Kaiser Family Foundation. Follow Phil Galewitz on Twitter:@philgalewitz.

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Republican Sen. Chuck Grassley, chairman of the Senate Judiciary Committee, has opened an inquiry into potential abuses of the Orphan Drug Act that may have contributed to high prices on commonly used drugs.

In a statement, Grassley said the inquiry is “based on reporting from Kaiser Health News” and strong consumer concern about high drug prices.

“My staff is meeting with interested groups and other Senate staff to get their views on the extent of the problem and how we might fix it,” Grassley wrote.

A six-month Kaiser Health News investigation published in January found that the orphan drug program intended to help desperate patients is being manipulated by drugmakers. While the companies aren’t breaking the law, they are using the Orphan Drug Act to secure lucrative incentives and gain monopoly control of rare disease markets where drugs often command astronomical price tags.

KHN’s investigation, which was published and aired by NPR, found that many drugs that now have orphan status aren’t entirely new. More than 70 were drugs first approved by the Food and Drug Administration for mass market use. Those include cholesterol blockbuster Crestor, Abilify for psychiatric disorders and rheumatoid arthritis drug Humira, the best-selling drug in the world.

Others are drugs that have received multiple exclusivity periods for two or more rare conditions. About 80 drugs fall into this latter category, including cancer drug Gleevec and wrinkle-fighting drug Botox.

Before the Orphan Drug Act passed, drugs for rare diseases were often abandoned during development, hence the name orphan. The patient populations were simply too small for the drugs to be financially viable for companies, they said.

Grassley, the senior senator from Iowa, is well positioned to call for changes in the law’s incentives. The Judiciary Committee he oversees has jurisdiction over anticompetitive and patent-related issues. Grassley made headlines late last year calling for hearings to demand that drugmaker Mylan justify its price hikes for the lifesaving EpiPen.

Grassley’s office has reached out to staff members of the Senate Health, Education, Labor and Pensions committee, which has jurisdiction over the FDA. When asked this week about a possible update to the law, the committee lauded the Orphan Drug Act for helping millions of families and said it “will continue oversight of the law to ensure it is working as intended.”

But there are questions over whether the law is working as intended. Dr. Robert Califf, who left his post last month as commissioner of the FDA, said it’s time to review the orphan drug program and the incentives offered to corporations.

While the law has been very successful in bringing effective treatments to patients with rare disease, Califf said, it’s also the case that when a program “reaches a mature phase it’s important to reassess it.”

Califf went on to say, “It’s understandable that whenever you put a financial incentive in place in the United States, people will try to figure out how to take advantage of it — almost all of them within the law. So, I’m not talking about illegal activity but … there is a game of cat and mouse that goes on.”

Government incentives for orphan drug development are needed because the research involved is costly, risky and uncertain, Anne Pritchett, vice president of policy and research at PhRMA, the drug industry’s lobbying group, said in an interview in late 2016. This week, a PhRMA spokeswoman said the group hadn’t heard of any movement on changing or updating the Orphan Drug Act.

Dr. Martin Makary, a professor at Johns Hopkins University School of Medicine and a vocal critic of the high prices of orphan drugs, has suggested that drugmakers should repay some of the federal incentive money once a drug reaches a $1 billion in annual sales.

Former U.S. Rep. Henry Waxman, D-Calif., who pioneered the Orphan Drug Act in the 1980s, thinks Congress should tackle prescription drug pricing on a variety of fronts, and that an update to the Orphan Drug Act should be part of the solution.

But Peter Saltonstall, president of the National Organization for Rare Disorders, said his group has heard “no energy” for changing the law now.

“I think the Orphan Drug Act, at least from our perspective, has been successful,” Saltonstall said, adding that there are other drug pricing concerns that could be addressed by Congress.

Today, more than 450 orphan drugs have been approved. Obtaining orphan designation for a product is a popular business strategy for manufacturers. Drugs approved to treat rare diseases made up 40 percent of new drugs approved by the FDA in 2016 and nearly 50 percent of new drugs approved in 2015. The drugs are also expensive, at $111,820 for a year’s treatment with an orphan drug, on average, in 2014 compared with $23,331 for a mass market drug.

Kaiser Health Newsis an editorially independent newsroom that is part of the nonpartisan Henry J. Kaiser Family Foundation.KHN’s coverage of prescription drug development, costs and pricing is supported in part by theLaura and John Arnold Foundation.

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