[nimz]

This advancement is especially promising because access to Delamanid (a drug similar to Pretomanid) is very difficult for patients in many countries. The company that developed Delamanid, Otsuka Pharmaceutical, has been extremely restrictive in allowing access to it.

I know someone critically ill who was petitioning Otsuka for access to Delamanid under compassionate use, but Otsuka would only agree to give access if the patient stayed in a hospital for 4-6 months. This is just a crafty way for pharma companies to refuse access since they know the cost of months of hospital stay is not affordable or practical. Luckily in this instance, TB Alliance and others fought with Otsuka to get this patient access to the drug.

In many high-need countries (India/Africa/China etc), desperate doctors frequently just lie to Otsuka about the patient staying in the hospital in order to get access to the drug.

Opening access to Pretomanid is a great advancement!

[mkl]

Why do they want extended hospital stays? Is it to boost their success statistics or something?

[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.

[gdy]

To make sure TB is cured and avoid developing a new drug-resistant strain?

[closingbracket]

I'm going to step in here as a reminder that Africa has more than 54 countries.

[asavadatti]

While you are not wrong, India (1.32B) and China (1.41B) both have larger populations than Africa (1.25B) so it makes sense to to group these together (as regions, not countries)

[anthony_doan]

This is why I refuse to apply to pharma companies for work or war companies.

I think they're unethical and morally bankrupt.