Spinal Muscular Atrophy Researchers Identify Spinraza Ethical Challenges

Although Spinraza has the potential to change the course of spinal muscular atrophy, a number of ethical challenges revolve around its use, a group of American researchers argues.

In an article in the journal JAMA Pediatrics, they maintained that the healthcare system needs to address six issues to ensure that SMA patients “benefit from treatment, are protected from harm, and are treated fairly.” The title of the piece is “Ethical Challenges Confronted When Providing Nusinersen Treatment for Spinal Muscular Atrophy,”

The group, led by Dr. Alyssa M. Burgart of Stanford University, said the biggest challenge is cost. Spinraza’s pricetag is $750,000 the first year, and $375,000 each year thereafter.

A cost that hefty can make insurers reluctant to cover the treatment, the group said.

It also creates the possibility of hospitals bearing partial or full costs if an insurer refuses to re-imburse them. In fact, this is a major reason why some hospitals are not offering Spinraza (nusinersen) treatments, the team wrote.

A second ethical challenge is the possibility that people with similar levels of disease severity will receive different treatments because of cost. This generates “concerns for the just distribution of healthcare,” the scientists wrote.

While unequal health insurance coverage is an inherent flaw of the U.S. healthcare system, Spinraza’s enormous cost makes the problem particularly daunting.

Healthcare insurers and hospitals are not the only ones bearing the cost burden, the team wrote. The lifelong cost of the therapy may force families to opt out of treatment or become impoverished.

The second ethical challenge is dealing with limited information on Spinraza’s long-term effectiveness. Since Spinraza trials involved small patient samples and were relatively short term, the verdict is out on whether most patients will benefit long term.

It is also not clear if the improvements seen in the trials will translate into improvements in muscle strength and function when doctors treat patients.  

While patients and their families may be prepared to accept these uncertainties, health insurers may not be. This may lead to situations in which insurers approve a treatment only if patients can prove with arbitrarily determined measurements that it is effective. Limited access to such testing may further disadvantage a patient, the team argued.

In addition, there is no agreement on what a treatment benefit is. This has a bearing on a third ethical issue — informed consent. If a treatment is failing to provide benefits, a doctor may decide that it should be dropped.

Since there is no consensus on what a treatment benefit is, patients, families and physicians may find themselves holding differing views on the issue. The best way to deal with this is to discuss it before a patient starts treatment, the team contended.

“Although these complex discussions occur at the bedside, institutions should ensure that clinicians [doctors] and patients have the support they need while facing prognostic uncertainty,” they wrote.

The fourth ethical challenge is how to allocate treatment. Granting access to everyone who is eligible will likely lead to treatment delays. The question is: Who gets treatment first.

As an example, doctors may need to use special procedures to administer a spinal tap drug, including sedating a patient and having them hospitalized afterward.

Researchers agree that Spinraza should be started as soon as possible. Logistical obstacles could cause delays that make it less effective.

Since drug administration resources are limited, hospitals may need to come up with ways to decide who gets priority access to treatment. These could include allocation criteria, case by case circumstances, or even lotteries. Each method has pros and cons in terms of fairness and other considerations.

Availability of treatment centers, which also affects therapy allocation, is the fifth ethical challenge revolving around Spinraza. While it is possible to open more centers with the expertise to administer Spinraza, this solution is also linked to cost.

Some centers may choose not to offer Spinraza or to develop the expertise needed to administer it, leaving just a few centers to tackle patients’ needs.

“Such a system may increase overall wait times and strain the participating centers in an unsustainable and unfair fashion,” the team argued. “Patients who live far from a participating center may not have the resources or clinical stability to travel, creating further disequilibrium of justice.”

The sixth ethical challenge is ensuring transparent communication between patients, families, doctors and others in the SMA community about these issues.

On the one hand, patients and families need reliable information about a medical center’s care processes to make treatment decisions. This means the centers need to be up-front about how they allocate resources. The transparency will help patients obtain Spinraza at a center that offers the most advantages for their situation.  

“As we pass through the initiation phase of nusinersen treatment among prevalent and newly diagnosed patients, pressure must be applied to reduce the cost and its effect on access, learn more details of medication benefit and safety, and examine different ways to initiate treatment and manage clinical workflows for this therapy,” the researchers concluded.