[nimz]

The reason is that in some trials of Delamanid, a rare but serious side effect called QT prolongation was uncovered. To monitor for this side effect, a patient needs to have regular EKGs done. Since Delamanid is not approved in many countries yet (including the US), Otsuka wants to eliminate even the smallest chance of a patient complication which could delay their approval process. So they call for full hospitalization while the patient is on the drug.

For brand new drugs that cure critical illnesses, the FDA has a compassionate use program (officially called Expanded Access) which allows drugs that are not fully approved yet to be used immediately by very ill patients. Otsuka requiring months of hospitalization to monitor for a rare side effect was just wrong and against the spirit of compassionate use access IMO. This side-effect is easily and cheaply monitored by regular EKGs at a local primary care doctor or urgent care clinic.

[refurb]

Considering QT prolongation has resulted in several drugs being pulled off the market, I’m not sure I blame the Pharma company for being cautious.

They are putting the use of the drug entirely in the hands of people who might not have the resources or training needed. If the use of the drug resulted in several deaths, it could significantly delay approval or kill the program entirely.

[nimz]

True that they're being cautious to protect their investment in the drug. In the case of TB, it's taken seriously enough that the drugs used to treat MDR/XDR TB are taken under close medical supervision, especially brand new drugs like Bedaquiline and Delamanid. They are usually administered under DOT (directly observed therapy) where a patient is required by law to take the drugs daily in front of a witness, or with lower-risk patients they use VDOT (Video Directly Observed Therapy) where patients can use an app to video record themselves taking the medicine daily and submit the evidence to the local Department of Health. In cases where patients don't comply to treatment, they can be arrested and isolated due to the public health risk.

[refurb]

Protecting their investment sure, but it can extend beyond that to impact clinical perceptions of the drug such that it limits use unnecessarily (regardless of whether Otsuka markets it).

I was tangentially involved in a clinical development program that offered access through compassionate use. The folks who received the drug were truly out of options. As a result several died. Investigation determine the drug was not the cause. That didn’t stop the rumors from swirling and several physicians pulling out of the clinical trials and actively discouraging patients from taking part. Probably extended the development by at least 2 years (it was eventually approved) and as a result many people who could of benefited from it had to wait, which likely resulted in a number of deaths that could have been avoided.

[sschueller]

So QT should be paying for the hospital stay and the drug if they are using this data. Drug trials aren't free either.

[AlexDragusin]

Couldn't some sort of waiver be signed in presence of various witnesses and properly notarized etc work in these cases instead? That way the company would not be held liable and the patient, by his own choice, can get access to the drug when he needs it.

[markdown]

That only takes care of liability, not the PR cost of having people die due to a side-effect of your drug.