ARTICLE IN BRIEF
With two new high-priced drugs being introduced to the market, neurologists and pharmacoeconomics experts examine the high costs of therapies and what can be done about them.
As yet more neurologic drugs hit the market with hefty price tags, a new position statement from the American Academy of Neurology says that steps need to be taken to contain costs and ensure that patients with neurologic conditions have access to the medications they need.
The AAN position paper, released in March, calls for federal agencies to negotiate contracts with manufacturers of Medicare Part D drugs; proposals to promote transparency in how drugs prices are set, including disclosure of prices paid by insurers and consumers; and allowance for the reimportation of high-quality drugs from Canada when prices for those prescriptions are lower than in the United States.
Nicholas E. Johnson, MD, chair of the AAN Government Relations Committee, which co-wrote the position statement, said patients with neurologic conditions are being especially hard hit by rising drug costs. He noted, for instance, that the cost of glatiramer acetate (Copaxone) to treat multiple sclerosis (MS) increased 84 percent from 2010 to 2015. [For more about the AAN position statement, see “The AAN Position on High-Price Drugs.”]
“Prices are going up substantially across the board no matter what area of neurology you are looking at,” Dr. Johnson, assistant professor of neurology at the University of Utah, told Neurology Today.
Two new neurologic drugs, deflazacort for Duchenne muscular dystrophy and nusinersen for spinal muscular atrophy, have quickly become the latest examples of that trend.
Developed under the Orphan Drug Act, the two drugs have raised questions about whether federal policies intended to promote treatments for rare diseases are also allowing manufacturers to maximize patents and profits at the expense of patients.
Deflazacort, approved by the Food and Drug Administration in February, has been expected to have a price tag of $89,000 annually, despite the fact that it has been sold for years in Europe for a fraction of that amount.
In response, in part, to the criticism about its drug pricing, Jeff Aronin, chairman and chief executive officer of Marathon Pharmaceuticals, posted in an “open letter” online to the Duchenne community in February that the company had decided to delay their launch efforts in order to meet with Duchenne community leaders to discuss concerns about pricing; maintain its expanded access program for patients receiving the drug; and continue to offer that option for patients receiving deflazacort from other sources.
On March 3, Senators Patty Murray and Al Franken, among others, sent Aronin a letter requesting specific information about the rationale behind its pricing methodology and the company's specific plans to work with pharmacy benefit managers, among other initiatives, to make the drug more affordable and accessible to patients.
Nearly two weeks later, on March 16, Marathon released a statement that it had sold the drug to another pharmaceutical drug, PTC Therapeutics; at press time, information about the cost of the drug was not available.
Nusinersen, which gained FDA approval in December, is even more costly. The drug, the first approved medication for spinal muscular atrophy, is priced at $750,000 for the first year of treatment, followed by a price of $375,000 annually.
“The price of nusinersen was determined through a rigorous and thoughtful process that evaluated a range of information and strived to achieve an appropriate balance among three key pricing principles — clinical value, impact to the healthcare system, and stakeholder returns,” said Ligia Del Bianco, a spokesperson for Biogen, the drug's maker.
She said the company already has made progress in getting some commercial and state Medicaid plans to cover the drug and has set up a program to help patients get access to it.
Robert C. Griggs, MD, FAAN, professor of neurology, medicine, pediatrics, pathology & laboratory medicine at the University of Rochester and a lead investigator on the clinical trial that led to deflazacort's approval, said that while the Orphan Drug Act has delivered on its promise to promote the development of treatments for rare diseases, the legislation has had the unintended consequence of driving up drug prices, in part because manufacturers can get extended market exclusivity for their products even if the drug in question is not a new product.
“I think it is really important for patients to get the best treatment they need,” Dr. Griggs said, but he added that doesn't mean that there should be no consideration to costs.
He said he favors “price plus policies,” which would allow drug companies to recoup their development costs and make a profit, though with “some limits on how much drugs can be marked up.”
Dr. Griggs said more research into the cost-effectiveness of drugs and head-to-head comparisons of drugs would also help control costs. He is doing an NIH-funded study with three to five years of follow-up that will compare the use of deflazacort to the much lower-priced corticosteroid, prednisone, to see which agent has the most benefit to muscle strength and function and the fewest side effects.
A COMPLEX MARKETPLACE
Michael Fischer, MD, associate professor of medicine at Harvard Medical School and an internist in the division of pharmacoepidemiology and pharmacoeconomics at Brigham & Women's Hospital, said the issue of drug pricing is complex and confusing because the normal rules of a competitive marketplace don't apply.
“There is no real competition,” he said. “The system essentially allows companies to charge whatever the market will bear.”
He said doctors may be reluctant to discuss drug prices with their patients, in part because a lack of pricing transparency on the part of drug companies and insurers makes it hard to explain why a drug costs what it does. Also, talking money and whether something is affordable are sensitive topics for many people.
But Dr. Fischer said costs should be discussed along with all the other benefits and risks of the drug being recommended because price can be a barrier to care.
Physicians “need to look carefully at the evidence and give the patient our best evidence-based opinion on whether we think a medication will help or not. We shouldn't feel compelled to recommend a medication enthusiastically just because it is new,” he said.
Aaron E. Miller, MD, FAAN, professor of neurology at the Icahn School of Medicine at Mount Sinai and medical director of the Corinne Goldsmith Dickinson Center for Multiple Sclerosis, said he is very open about discussing drug prices with his patients.
“We have seen an astronomical increase in the price of every disease-modifying MS drug,” he said, even for drugs such as glatiramer acetate that have been around for 20 years.
“It's gotten to the point where I say to the patient, ‘Let's figure out which drug we want to get you on and then let's see if we can get it for you.’”
Dr. Miller said he was pleased to see the AAN's position statement on drug pricing and is optimistic that pressure from patients and physicians will bring about some needed reforms.
The next case example on drug pricing is coming soon. Dr. Miller said the MS community is waiting to see what Genentech will charge for the MS drug ocrelizumab when it comes to market.
“We're hoping the company will be reasonable,” he said. Approval for the drug had been expected at the end of last year, but at press time, the FDA had delayed the decision pending more information on good manufacturing practices from the sponsoring manufacturer.
THE AAN POSITION ON HIGH-PRICE DRUGS
The timing of the release of the new AAN position statement is ideal given that there is a new administration in Washington and public sentiment is strong on the need to rein in drug prices, Nicholas E. Johnson, MD, chair of the AAN Governmental Relations Committee, which co-wrote the statement, told Neurology Today.
While new drugs priced at tens or even hundreds of thousands of dollars have helped create a sense of urgency on the issue, the AAN's position paper expressed concern about three distinct trends in drug pricing and said that different solutions will be required to address each of them:
- A massive increase in the pricing of previously low-cost generic drugs used to treat common disorders without obvious increases in the cost of production or distribution
- A massive increase in the pricing for high-priced generic and brand-name drugs used to treat serious disorders that are not protected by the Orphan Drug Act
- The high cost of new medications used to treat rare disorders as defined by the Orphan Drug Act.
“Drugs that treat complex, chronic conditions like Parkinson's disease, epilepsy, and migraine, and specialty drugs which may require special handling or administration, such as those used for multiple sclerosis, are particularly expensive,” the statement said. “Spending on specialty medications has increased by $54 billion since 2011 and now accounts for more than 70 percent of all prescription spending growth.”
The position paper said that the AAN would support proposals that would prohibit direct-to-consumer advertising of prescription drugs, a move supported by the American Medical Association's House of Delegates.
On the issue of reimportation, the statement cited the example of glatiramer acetate, which it said on average costs MS patients in the US 287 percent more for a monthly prescription compared to what they would pay in Switzerland.