Why Senator Warren is wrong about the 21st Century Cures Act


As a person with a rare disease who has responded very poorly to first- and second-line treatments, I’m incredibly dismayed to see Sen. Warren characterising off-label prescribing practices as ‘legalised fraud’. I am similarly frustrated to see that disability charities have been campaigning against this bill, ostensibly on grounds similar to the concerns Warren raises in this speech. While there are certainly some problems with the bill — in particular, the reduction of funding to the NIH — this strikes me as an instance where the disability community at large is in the dark as to the challenges faced by some of its members.

The continued perception of randomised controlled trials as the ‘gold standard’ for evaluation of drug safety and efficacy is something of a cultural artefact — scientific practices are heavily biased toward the use of evidence that is thoroughly controlled, while tending to disregard the ways in which this control erases ‘real-world’ variability. But what this means is that a system that accommodates only large-scale, heavily controlled sources of data is going to fail to accommodate ‘rare’ events, such as rare diseases, and rare presentations of disease. For example, in the case of neurological sleep disorders, most of which are considered rare, this creates enormous barriers to accessing care because the majority of treatments are ‘off-label’. This status has a variety of consequences; most notably, treatments are often not covered by insurance or social security benefits (or in the case of the UK by state-funded care), and because they are not included amongst the indications in pharmaceutical dispensaries, a majority of clinical practitioners, including specialists, are often unfamiliar with them.

Contrary to Warren’s characterisation of real-world evidence and off-label use as a nefarious agenda of Big Pharma, these are in fact prescribing practices that are already ongoing for rare and treatment-unresponsive diseases - but which are only accessible to the lucky few who are able to either pursue treatment at a major research centre, or who have a physician who is dedicated and open enough to listen to what patients have learned from their peers and do a bit of their own research. The use of ‘real-world evidence’ for post-approval studies of new indications would make these already-ongoing prescribing practices accessible to a larger portion of the patient population.

Moreover, from a theoretical perspective, a move toward the inclusion of ‘real-world’ evidence, which I take to refer to case reports / series, holds promise for me as making room for individuated patient experience within the systemic infrastructure of healthcare itself.

I would encourage people to look into the treatment options available to patients with Idiopathic Hypersomnia, for example, to develop a better understanding of why the real-world evidence component of the 21st Century Cures Act could in fact prove invaluable to some members of the disability community.

Full text of the Act is available here.

Disability-led design & health justice. Director of Communications for The Disabled List. They / theirs. Tip jar: paypal.me/alexhaagaard

Disability-led design & health justice. Director of Communications for The Disabled List. They / theirs. Tip jar: paypal.me/alexhaagaard