The 'Most Expensive Medicine in the World' and the False Dilemmas

Uncertainties are inherent to all emerging technologies

In the article O “remédio mais caro do mundo” e os dilemas para o SUS e o STF[1], published in the Brazilian newspaper Folha de S. Paulo on August 26, Daniel Wei Liang Wang et al. propose a broad debate with society about the price of Zolgenzma. The drug is indicated for the treatment of spinal muscular atrophy (SMA), a rare disease that causes progressive muscle paralysis. As many have noted, starting with the price is a skillful way to end this discussion.

Aldous Huxley said that "sometimes it is necessary to re-establish the obvious". It is worth remembering that Zolgensma is on the list of ‘curative gene therapies’. It is, therefore, endowed with a strong component of disruptive innovation, with the potential to cure, which is something that is not trivial. It is the result of the ‘biotechnological revolution’, which started in the mid-1970s.

Everything indicates that these types of gene therapies are here to stay. Until January 2017, there were no less than 925 new therapies of this kind in the industry's pipeline, for 209 indications. Although very expensive, these therapies are often aimed at patients with rare (and ultra-rare) diseases, who are often classified as having unmet medical needs and potentially fatal illnesses.

In theory, the small number of people affected by specific rare or ultra-rare diseases would make it impossible to put their budgetary impact in general terms. But, if we cumulatively consider the cost of all these therapies in the "pipeline", sustaining such high-cost treatments in the current medical system would be significantly challenging.

Thus, a recommendation should be made that the authors discuss the chronic underfunding of the Sistema Único de Saúde (SUS), Brazil's publicly funded health care system. I do not believe that they are fundamentally opposed to the expensive technological advances of biomedicine. If so, they would be Luddites and, as such, they would only have to enter the ‘time machine’ to stop the infamous biotechnological revolution.

Health economics experts are not usually Luddites. Instead, they believe that economic assessments, such as the aforementioned cost-effectiveness analysis, would need to change, in part or completely. This would guarantee a minimally fair and detailed decision-making process about the desirability of such therapies.

The article also discussed whether the effects of such expensive treatments would persist in the long term. The advent of these therapies is recent. Such doubt also affects the most accurate estimate of its real value and adequate price. These are uncertainties inherent in any emerging technologies.

It would be advisable for the authors of the article to direct their admonitions to regulatory bodies, commissions, and the government's legal apparatus. With the "us versus them" argument, they point the finger, between the lines, at the rare patients: the eternal cannon bushings of electoral projects of the questionable search for efficiency, which is an often unsuccessful feat. They never address them as recipients; always as targets.

What are the Executive and Legislative branches of our government doing to face the challenges created by these innovations that are already knocking on our door? Henry Ford, the inventor of the automobile, has a witty phrase attributed to him: "if I asked people what they wanted, they would say 'faster horses'". The authors should ask the government: "automobiles or faster horses?"

Op-ed published at Folha de S. Paulo, on 2020, September 13th.

Cláudio Cordovil Oliveira is Researcher at the Sergio Arouca National School of Health (ENSP / Fiocruz) and member of the international consortium of researchers “Social Pharmaceutical Innovation - Spin” ( FAPESP)


[1] WANG, Daniel Wei Liang et al. O remédio mais caro do mundo e os dilemas para o SUS e o STF. Sao Paulo. 2020 August 26th.

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