For rare diseases with small patient groups and little bargaining power, the price of drugs is often less dictated by cost of production and market value than dictated by whim, and the limits on peoples’ perception of the value of a human life.

To put the spotlight on one drug, Spinraza, one could make a case that greed sometimes supersedes the desire to help, for many who work in the medical industry.

Spinraza, the trade name for nusinersen, is a drug used to treat a rare infant neurological disorder, spinal muscular atrophy, a degenerative disease that often leads to death before the age of four. This disease is generally caused by a genetic defect in which the patients are unable to produce the protein coded for by a gene called SMN (survival motor neuron), without which, those cells, which control movement, will ultimately degrade and die. Over time, this causes most patients to lose motor functions, eventually requiring artificial breathing in order to survive.

With a patient base of around 25,000 in the US, and treatment cycles that usually call for a limited number of doses, usually, six in the first year for patients with infantile SMA, and three in the following years, pharmaceutical groups claim that high per-patient costs are justified in order to recoup development costs, and fund further development.

However, the drug was developed, partially using public resources, and was approved by the FDA for treatment in December 2006. The stake holders then sold the license for this drug to Biogen, a co-developer, for $75 million USD, in addition to milestone payments for additional government approvals, as well as future royalties.

This drug is an oligo-nucleotide drug, far more complex to produce than a simple pharmaceutical compound, but the list price put on this drug was extraordinary, $125,000 per unit, especially when considered that the development of this drug wasn’t entirely private: it relied on resources from government funded laboratories, as well as seed grants from a patient advocacy non-profit group. It should also be noted however, that the patient-advocacy group, CureSMA, who provided seed funding and agreed to the license sale, is actually not exclusively patient oriented, and public records indicate that it receives some of its funds directly from pharmaceutical corporations.

Biogen publicly described their decision making on the pricing of this drug, and is on record saying:

“a number of important factors, including its clinical value, its impact to patients and the health care system as a whole, and the need for Biogen to fund further research and development to make the next innovation possible.”

A number of countries, however, disagree as to whether its clinical value justifies this high price. To be clear, this is a treatment, not a cure, and primarily exists to improve quality of life, and extend the lives of patients as much as possible. In the initial clinical studies, the infantile SMA patients who received nusinersen were 47% less likely to have died, or required permanent respiratory assistance than the control group, over the course of the study, still a marked improvement. The costs, though, have still been assessed by some to outweigh the potential clinical benefits. Therefore, while some countries, including the USA (some state Medicare programs and private insurers), and Taiwan (and its respective national health insurance program) have approved payment for this drug, while others, including Denmark and Australia have rejected public payments for the drug labeling Spinraza’s clinical outcome as not cost-effective.

The U.S, European, Canadian, Brazilian and now Japanese regulatory agencies have all approved treatment for those with the means to pay. Biogen has stated that they offer the drug for free in those countries whose governments have not yet approved the drug for treatment.

Meanwhile, the National Centre for Pharmacoeconomics of Ireland suggested a 10 fold price reduction for infantile SMA in order to bring the drug into cost effectiveness.

Biogen, as of January reported stellar earnings, whilst still bemoaning the recouping development costs for a drug partially produced using public resources. The drug caused 15.3 percent surge in Biogen’s 2017 fourth quarter revenue, bringing in $363 million dollars, with an expected 2018 revenue of between $12.7 to $13 billion USD.

Meanwhile, Biogen is already facing pressure from what appears to be a newer, better drug on the horizon. AVXS-101, a single treatment gene therapy drug, whose preliminary effectiveness is already impressive, is being shopped by Novartis pharmaceuticals.

A gene therapy drug like this would effectively replace the disfunctional genes that misencode for SMN, and the change would hopefully be permanent and more effective. One doctor who worked on the gene therapy trial this year stated, “I’ve been doing clinical trials for over 40 years, and the success we had here was far more profound than we’ve ever seen before.” Whereas Spinraza showed improvements in basic motor function, these latest studies have shown some patients regain the ability to walk, and even to speak.

As new drugs for horrible diseases incrementally, and sometimes monumentally improve, new pricing questions will emerge. This is especially the case with drugs for life-threatening diseases, priced out of reach for out of pocket coverage, to the point when even national insurers will deny coverage. It makes the question for some all too real, how much is a child’s life ultimately worth?

Staff writer: Ari B