I wanted to take just a short minute so crystallize a thought. As I read stories on the FDA and drug approval, I am struck by the way consumers/patient groups are characterized. There is an impression that is given that the industry pushes drugs on reluctant patients. Perhaps that is true for many drugs but not for the issues that face many patients with rare disease.
Quoting from Fran Hawthorne’s book “Inside the FDA”
The FDA has to focus on Type I mistakes, because the risks of allowing a dangerous drug on the market are simply so much higher than the risks of not allowing a good one. The first type of risk is that the status quo will be disrupted and people will die much sooner than they otherwise would. The second type is simply that people will not gain an improvement — that the status quo will remain.
For context, Hawthorne uses Type I vs. Type II errors to differentiate sins of commission vs sins of omission. This statement is justifying the need to tilt toward being conservative because being too aggressive puts a large, reasonably healthy population at risk. However, this argument breaks down with the risk of death without treatment is 100%. That is true for Sanfilippo and for so many other rare diseases.
I have to be honest, this mistake is our own. The industry and patient groups have allowed this faulty reasoning to persist and we haven’t made our case: to regulators, lawmakers and apparently pundits and the media. Applying a blanket set of rules based on reasoning that applies to the general population is killing people with rare disease – bottom line.
Parents of children with Sanfilippo are beating down the doors to get access to treatment (trust me, I know first hand). Patient advocacy groups, Industry, parents and patients have to get together and get better at telling our story. As Dr. William Gahl from NIH explains, “Patients with rare disease want to be protected but they don’t want to be protected to death”.
Until we do a good job of conveying this message, we only have ourselves to blame.