The US has faced numerous issues involving prescription drugs, especially with rising costs and accessibility of life-saving and required medications. One class action lawsuit (Wilkins et. al. v. Genzyme Corporation (n/k/Sanofi) against Genzyme (now owned by and known as Sanofi, the eighth largest pharmaceutical company in the world) alleges the company intentionally misled patients with Fabry disease as to the safety and efficacy of their product in the early 2010s. Specifically, the case concerns Genzyme’s patent on Fabrazyme, one of the only effective enzyme replacement therapies available in the US to help treat Fabry disease. Fabry disease is a lethal genetic disease which most commonly affects men. The disease prevents the enzyme alpha-galactosidase-A from effectively breaking down lipids and can cause strokes, heart failure, and kidney failure. Research estimates about 4,000 men in the US have Fabry disease. An ongoing lawsuit alleges that the Fabrazyme drug became contaminated and that Genzyme misled patients as to the safety of the drug due to the contamination and sent patients an ineffective lower-than-recommended dose due to a supply shortage. The plaintiffs allege that these actions of Genzyme accelerated the progression of the disease.
The class, including four children, alleges that the batches of Fabrazyme became infected with a vesivirus and that Genzyme provided low doses which caused the Fabry disease to progress. Plaintiffs alleged that due to defective Fabrazyme injections, their Fabry disease progressed, causing symptoms ranging from adverse mental health effects like depression to allergic reactions, and from the development of hematological cancer to heat intolerance, among many other life-altering, severe symptoms. Due to the progression of the Fabry disease, patients also experience shortened life expectancy. When Fabrazyme is administered in the proper dosage, it stops the progression of the disease, allowing those with Fabry disease to live a closer-to-average lifespan. The class alleges that around July 2009, Fabrazyme became contaminated with Vesivirus 2117 in its manufacturing facility in Massachusetts. Specifically, the virus targeted Chinese hamster ovary cells being grown and collected in the facility. The class further asserts that Genzyme continued selling this defective Fabrazyme to patients under the misrepresentation that the vesivirus was not harmful to humans. However, experts and scientific research has shown that vesiviruses can present threats to human health, such as by killing human embryo cells. Additionally, Allen Black who represented plaintiffs in this litigation explained that the vesivirus is considered a “Category B biodefense pathogen,” in the same category as salmonella and hepatitis A. Black further stated: “The only reason the vesivirus was not known to cause disease in humans was because it was never injected into a human until it was injected into my clients.” As a result of an inspection performed by the Food and Drug Administration, the Fabrazyme contamination was discovered, and Genzyme was ordered to pay more than $175 million and be monitored by government oversight for almost a decade.
The complaint further alleges that Genzyme provided low doses (one-third to one-tenth of a regular dose) of Fabrazyme to patients with Fabry disease from 2009 to 2012, going against the FDA’s approved usage and dosage for the drug, in response to a drug shortage. Genzyme told its patients that the shortage would last only six to eight weeks. The shortage did not end until over two years later. Because Fabry disease is terminal without proper treatment, the low doses provided to patients allegedly caused the disease to progress faster than it would have with the approved dose given every two weeks. Following research completed by the European Medicines Agency, low dosing with Fabrazyme was effectively banned in Europe because it was shown to cause accelerated disease progression. One member of the class reported a loss of 80% of her hearing due to the low dosing recommended by Genzyme. Additionally, Black explained that some of the members of the class have now died from Fabry disease. Further, Black argued that “if the drug had been triaged at full dose for at least some people, some Americans would have had a therapeutic benefit instead of 100 percent useless treatment for everyone.”
The plaintiffs claim that Genzyme outlined a contingency plan in the event of a Fabrazyme shortage, providing favorable treatment to markets in Europe. Specifically, the class alleges that the “business value” of the market in Europe necessitated Genzyme’s favorable treatment of its European market, placing the low dosage sacrifices solely on the shoulders of US patients. The alleged rationale for this action was that another drug, Replagal, was readily available in Europe, but not in US markets. The plaintiffs argue that Genzyme did not want to lose its market share in Europe where patients could easily switch to another drug, while US customers were left without an accessible alternative and were left to ration their treatments.
In an unsealed complaint from a lawsuit (Schubert v. Genzyme) , the plaintiffs alleged that through the discovery process, they uncovered information regarding the Australian medical regulatory authority. Specifically, that complaint contended that Australia considered whether, in an effort to save patients money on treatment, Fabrazyme would still work if patients took a lower dose of 0.2 mg/kg. The complaint claimed that Genzyme “warned that reducing that dose across the board would have significant clinical consequences for patients, with the expectation that many would suffer irreversible harm as a result of insufficient dosing… and treatment at a higher dose is necessary and may be life-saving.”
At an oral argument in May 2023, the plaintiffs argued that both types of conduct on their own were enough to cause injury. If the low dose was the sole action taken by Genzyme to ration Fabrazyme, this would have been enough to result in individuals’ injuries. Additionally, the contamination of Fabrazyme with the vesivirus also would have been enough on its own to cause injury. Yet, the class claims that both of these worked in tandem to cause increased injury to patients needing Fabrazyme. Furthermore, the class is suing to get the money back that Genzyme billed Medicare and Medicaid charged for the reduced dose. Black estimates that, with compounded interest over a decade, the money damages are around $2 billion.
Black summed up the argument by stating:
I argue that a drug company cannot pick the survivors of a drug shortage based on market share instead of medical need. It is tortious conduct for a corporation to make these decisions under principles of negligence and breach of fiduciary duty. During a critical drug shortage, patients are vulnerable, so the company should act with the highest duty of care to preserve health, safety and lives. Self-dealing by sending the drug to customers who don’t absolutely need it in order to maximize “market share” is not only immoral but illegal.
The Court of Appeals for the First Circuit issued an opinion on February 15, allowing the plaintiffs to continue their lawsuit against Genzyme (Sanofi). Previously, a lower court held that the lawsuit was filed too late and that the plaintiffs lacked standing, or grounds to sue. On a purely procedural basis, the First Circuit opinion concluded that “the district court incorrectly dismissed plaintiffs’ claims for lack of standing.” The court also upheld the dismissal of four of the plaintiffs’ claims, explaining that those four “waited far too long before filing this lawsuit.” Black explained he may petition the First Circuit for a rehearing en banc in regard to the four plaintiffs, which would mean the entire panel of judges on the court would hear the case instead of a panel of only three judges. The court returned the case back to the district court in order to answer two questions: whether the remaining claims withstand Genzyme’s statute of limitations defense (that the plaintiffs filed the lawsuit too late) and whether the complaint meets the requirements under procedural rule 12(b)(6) to state a claim upon which relief can be granted.
It is illegal under US law for a pharmaceutical company to market a drug for an unapproved, untested off-label use, such as for a reduced dose. US law allows the federal government to “march in” on a drug that has been federally funded in times of great need. The Bayh-Dole Act, enacted in 1980, provides federal agencies this power in situations when a medication is under a government-funded patent, specifically when necessary to provide for public health. However, the government has never exercised these march- in rights. One example of when the government was asked to exercise its march-in rights was with the drug Norvir, which boosts the effectiveness of other HIV/AIDS treatments. In 2012, the National Institute of Health (NIH) declined to exercise its march-in rights to allow a generic version of Norvir because the price was prohibitive to people needing treatment. Essentially, the NIH declined to use its march-in rights because it felt that the high price of drugs is a power left solely to the legislative branch, namely Congress, rather than federal agencies, which are a part of the executive branch.
In December 2023, the Biden administration put out a document seeking to clarify when march-in rights are appropriate. In this guidance document, now available for public comment, criterion two lays out the framework an agency needs to use when considering march-in rights for a health product when the health or safety needs “are not reasonably satisfied” by a company. Specifically, the document lists questions an agency should ask to determine if there is a health and safety need, including, “What is necessary to resolve the health or safety need?” This answer could be a greater quantity of a pharmaceutical drug and/or more options to access similar products, like creating a generic version of a drug, among others. Another factor specifically targets prescription drug prices, asks the agency to consider if the price is “extreme, unjustified, and exploitative of a health or safety need.” This question is especially poignant as the US faces much higher prescription drug costs when compared to similarly-situated countries. For example, a year’s supply of Fabrazyme can cost an individual more than $600,000. One study found that US prescription drug prices are more than 2.5 times more expensive than other countries. Additionally, a CDC report from 2023 found that an alarming 8 percent of US adults took less than the prescribed dose of a prescription drug due to the high cost of their treatments.
The House Ways and Means Committee recently held a hearing on chronic drug shortages, where more than 250 medications are facing a shortage in the US, exacerbated by reliance on foreign markets. The Federal Trade Commission also announced it will be investigating a chemotherapy shortage to determine whether pharmaceutical drug companies have hindered the sale of generic versions of drugs on the market.
The issue of prescription drug accessibility intersects across price, availability and efficacy. The class action lawsuit against Genzyme alleges serious misconduct against a population with a rare genetic disease. Guidance issued by the Biden administration shows promise to encourage the federal government to use the never-before tool of march-in rights for government-funded patents, especially in an area as consequential as rare prescription drug efficacy and accessibility.
