As a leftist disabled woman, this post is a misleading mischaracterisation of the 21st Century Cures Act, and frankly, typical of the kind of anti-Big Pharma rhetoric favoured by the abled left, who have the luxury of ignoring the fact that many of their fellow progressives would have no quality of life to speak of without Big Pharma.

To be clear, there are substantial problems with this bill — in particular the reduction of funding for the NIH and for solutions related to opioid care (though it is unclear to me the extent to which that opioid funding was meant to translate into the meaningful pain management alternatives needed by patients, rather than substance abuse initiatives preferred by politicians).

But a major feature of this bill that Senator Warren has expressed concern with is the introduction of so-called ‘real-world evidence’ to speed post-approval indications of drugs currently used in off-label prescribing. She has characterised this as “legalised fraud”.

As a person with a rare disease, who has responded incredibly poorly to first-, second- and third-line treatments, I am actually very pleased to see legislation that will make effective treatments more available to people like me.

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