Biotech Values

[ghmm]

BMRN:

a) by implying that placebo patients are being harmed by being given placebo you are making the almost explicit assumption that the drug is more beneficial than harmful (and that the FDA's rules are arbitrary and capricious). I would point out that, as most on this board know, this is generally not the case - most drugs, even those with moderately good ph2s (a 'reasonable' post hoc), turn out to be more harmful than beneficial (they have meaningful AEs and fail their Ph3 efficacy endpoints).

You "..."'d over a key part of my point . Those trials may be the right trials to do but I doubt any public company will initiate and complete them I don't see how its even practical in this patient population given the limited efficacy that we more or less agree these therapies may provide (OK you think may not even exists).

I am not assuming the drug is more beneficial I am assuming the burden placed on a DMD family to participate for 2 years (actually much longer when you consider the full duration of the study especially enrollment time) is too great.

b) you are effectively prioritizing the choice of the current patient over the harm to many years of future patients. There is not much doubt that as policy this will physically harm many more people more than it helps.

I would say your definition of harm is much much different than mine. If a drug is proven safe I would accept marginal efficacy and do not see this as harm. Your definition seems to include a hypothetical that an approved drug prevents future approvals in that disease. I think it just raises the bar slightly (actually if it were up to me I would be very lenient in approving therapies). I wouldn't want to evaluate treating patients today on an actuarial type system where the benefit to current people with a disease is compared to a potentially greater benefit treated with a future drug at a further time point (Should we only approve cures then?). I suspect doing so would necessitate discounting the non-existent future therapy to the point where a marginal therapy today is better than the potential future therapy.

If BMRN is successful with getting their Batten drug approved on 1 open label trial I think it may help open the door for flexibility outside of an RCT at least in rare diseases.