FDA seeks to improve access to experimental drugs

[OzarkDem]

FDA is revising its guidelines to allow seriously ill patients to have access to drugs in Phase 1 Clinical trials when they aren't eligible for the trials. Is this a real benefit for patients or a way to help drug manufacturers recoup costs on drugs not approved for sale to the public?

http://www.alertnet.org/thenews/newsdesk/N11221752.htm

[tridim]

1. But if you want to smoke some pot for chemotherapy side effects you go to jail

The hypocrisy of the FDA is just stunning.

[Tight_rope]

3. My thoughts exactly...The FDA is just a bunch of hypocrisy!

[depakid]

12. People in public health no longer take the FDA at face value

It's a classic example of a captured agency.

[OzarkDem]

5. Get someone to sponsor

a Phase 1 clinical trial to demonstrate its benefits. Once that happens, anyone who is seriously ill can apply with the researcher to have access to the drug without being in the trial.

[flamingyouth]

11. Yep

Exactly. :thumbsup:

[sweetheart]

2. Street drugs aren't cheap enough

The FDA's strategy is working brilliantly. 40 million people smoke cannabis in north america and the
drug is getting cheaper year on year. The same goes for cocaine, halucinogens and the opiates.

A round of applause for the FDA who's dimwitted tenure has seen unregulated experimental drugs
spread out to hundreds of millions of people at fire sale prices. (CIA, DEA and the other assholes,
stand up and take a bow)

[Lance_Boyle]

4. This is a good thing, not a moneymaker for the drugcos.

The Standard of Care in many segments of Oncology and Infectious Disease treatment *is* experimental. This allows those who would not be eligible to receive the experimental drugs in clinical trials to receive them outside of those trials, and allows collection of (at least anecdotal) evidence on non-study populations. The downside is that those persons receiving these drugs outside of the study context will likely not be protected to the same extent that clinical trial participants are. It is not permissible, for instance, for clinical trial sponsors to say "we will not pay you a dime in the event this experimental drug maims you." Instead they have to say things like "we do not have a set policy to pay you in the event the experimental drug maims you." But they must leave the door open to paying if, for instance, they lose a lawsuit that says they must. I wonder if those receiving these drugs outside the aegis of a clinical trial will have their rights protected in this way?

[OzarkDem]

7. That's a big question, esp for Phase I trials

The potential for legal liability in these situations is great. The potential for harm to patients is also very great. I'm assuming there must be some way they will address the legal issues. Smaller research companies couldn't bear the cost of a lawsuit company and most big pharma companies don't back smaller, cutting edge companies who have drugs in Phase I trials because of the risk that the treatment won't pan out.

I'm really surprised they did this.

[Warpy]

6. No, it's a way to give desperately ill people

a little hope and maybe even a cure. They lowered the barriers for a lot of AIDS drugs back in the late 80s and that has continued.

If you're close to death, you're willing to try anything. You know that if it doesn't work, nothing will change and medical science will advance a little. If it does work, you'll live a little longer. It's a win-win situation for the sick.

[OzarkDem]

8. But

What if the drug is toxic and you end up dying from the treatment instead of your disease? And what of the issue of patients wanting to take experimental drugs that aren't applicable to their disease?

One hates to see people who are considered ineligible for a trial (perhaps their system is too weak, their heart damaged, etc.) hasten their demise with an experimental drug. Its a double edged sword. And there's no evidence it actually advances research in any way.

[Lance_Boyle]

10. Often times clinical trial entry criteria are rather arbitrary,

or done for convenience rather than justice. For instance, the erectile dysfunction medications available now were available in clinical trials (through phase III) only for heterosexual men in monogamous relationships. Other times the restrictions are more understandable, if still unjust. I saw a phase II trial of a novel compound for treatment of sepsis prohibit pregnant women from participation. Nevermind that a ridiculously high percentage of sepsis patients die from it, and that in terms of efficacy the standard of care was indistinguishable from "no intervention." The sponsor worried (rightfully so) that the compound might harm the fetus. The IRB opined that a mother dying of sepsis would likely be no better for the fetus, and the trial was held in limbo for months on end. I wondered in that case whether the prohibition against the sponsor exclupating itself from responsibility for fetal harm might have been justifiably waived. I still wonder.

[OzarkDem]

14. I see your point

The same POV prevailed for years with chemotherapy and pregnant women until trials showed drugs had no effect on the fetus.

But clinical trials for cancer treatments are very rigorous and exclusions are often based on risks to patients (cardiotoxicity, etc.)

And what happens if the patients who are eligible for clinical trials opt to get the experimental treatment outside of a trial instead of risk being randomized to an arm that won't give them the treatment they want? It could interfere with the ability of researchers to design quality trials, not to mention make accrual much more difficult.

Hopefully the FDA is taking these things into account. They do have some very good consumer reviewers on board (a friend of mine is one) so hopefully they'll have some input into the final decisions on regs.

[Warpy]

18. I've also seen trial criteria waived for some patients in the past

and experimental drugs given as a last ditch attempt to keep them alive. Examples are DHPG (later Gancyclovir) in terminal AIDS patients and propafenone for patients with drug resistant ventricular dysrhythmias. It was probably illegal at the time, but the drug company R&D people apparently were willing to risk it, as we were in the trenches.

[Warpy]

13. So WHAT?

Remember, these are people who are desperately ill with no other treatment.

They already know they are going to die. They should have been told the treatment itself could kill them. It's called informed consent.

I know I'd do it. There would be as much to learn from my death as from my cure.

[OzarkDem]

15. Sometimes hard for families to understand

particularly if a loved one's life is cut short because of a treatment that was unlikely to help them in the first place. The patient may (or may not) be understanding when they only get the negative side effects and none of the benefits. Familiy members certainly don't understand it.

It may not be a popular role, but someone has to advocate for the rights of people who are desperately ill and may make bad choices based on false hopes.

[Warpy]

16. Anything that advances knowledge

done by someone who's facing certain death is hardly a bad choice, even if it does end his life a few weeks or even days ahead of schedule. THAT is what's so hard for some families to understand, that the patient was ready to let go, giving his life meaning by advancing medical knowledge, even if the family was not.

As for "unlikely to help him in the first place," that's a severe misunderstanding of what clinical trials are all about. The drug in question has already been given to healthy volunteers and the side effects noted. Nobody does fishing expeditions with sick people, giving them random substances to see if something works. These are all drugs that have worked in animals modeling the same disease, been tolerated well by healthy volunteers, and are all ready to be tested on the sick. This just widens the pool, allowing the very sick to skip the possibility of getting placebos instead of the active compound. The double blind studies are done on the less ill.

[Betsy Ross]

9. Legal access does not make trial drugs accessible to most

A friend in a chemo trial was told that the medication would be $15,000 per bi-monthly dose outside the trial program. Only the very rich could have access to such drugs.

[Warpy]

17. As long as the drugs are experimental

the compassionate use of them for the desperately ill can be written off in the testing budget.