A dry drug pipeline

SDN Members don't see this ad. (About Ads)

Originally Posted by genesis09

Ever heard of thalidomide? Thalidomide is why proof of efficacy is required and sulfa elixir is why proof of safety is required. We do not want history to repeat itself.

wouldn't thalidomide be proof of why safety is required? correct me on my history

Originally Posted by Argentium

wouldn't thalidomide be proof of why safety is required? correct me on my history

The thalidomide issue led to Kefauver-Harris Amendment. That law requires manufacturer's the drug is effective for its indication. Thalidomide was not approved in the U.S. in the 1950s. It never proved its safety.

The Sulfa Elixir event led to the Food, Drug, and Cosmetic Act of 1938, and that required proof of safety.

In the Eschenbach editorial, it was not that efficacy would never be tested. Instead, a drug would be marketable during the clinical trial phases. Instead of having to wait through 10 years of clinical trials, a patient, with a doctor's prescription, could use the drug once it received approval from the FDA as being relatively safe. Clinical trials would still be performed, but the company could market its drug and patients would have access to the medications. This would not be the wild-west of drug use, with everyone taking all sorts of drugs for whatever the marketers decided was the biggest market. Patients would still need a doctor's approval for the prescription. There is the potential for abuse (by companies and doctors) and misuse (by patients), but hopefully, a patient would only try these unproven drugs as a last resort and after failing to respond on multiple rounds of standard treatment.

There would be risks, costs and liabilities would have to be worked out, but perhaps such an idea, or a variation of it, could decrease the costs of developing drugs such that drugs with new mechanisms of action for common illnesses and drugs for illnesses with no treatment and small patient populations could be pursued.

Originally Posted by gadlo

In the Eschenbach editorial, it was not that efficacy would never be tested. Instead, a drug would be marketable during the clinical trial phases. Instead of having to wait through 10 years of clinical trials, a patient, with a doctor's prescription, could use the drug once it received approval from the FDA as being relatively safe. Clinical trials would still be performed, but the company could market its drug and patients would have access to the medications. This would not be the wild-west of drug use, with everyone taking all sorts of drugs for whatever the marketers decided was the biggest market. Patients would still need a doctor's approval for the prescription. There is the potential for abuse (by companies and doctors) and misuse (by patients), but hopefully, a patient would only try these unproven drugs as a last resort and after failing to respond on multiple rounds of standard treatment.

There would be risks, costs and liabilities would have to be worked out, but perhaps such an idea, or a variation of it, could decrease the costs of developing drugs such that drugs with new mechanisms of action for common illnesses and drugs for illnesses with no treatment and small patient populations could be pursued.

what is the timespan of phase 3 efficacy/safety RCTs relative to the rest of the drug development pipeline? what is stopping treatment refractory patients from enrolling in these trials? aren't phase 3 trials usually where we see the crazy random side effects where FDA blocks further development? do existing safety-only trials have the sample size or duration to see serious side effects? do you really think that big pharma wouldn't try to abuse such a system? how would an MD even know what drug to write for if it's only completed safety testing? how would they know it's safe AND effective for a condition? big risk for liability. huge.

maybe if big pharma moved their reader's digest/daytime TV advertising budget to the organic chemists, biologists, and pharmacologists, we wouldn't be in this dilemma.

Isn't there a compassionate use clause or something for cases of terminal illness without effective treatment that allows for drugs to be used without the usual process? Maybe I am thinking of something else.

Originally Posted by gadlo

Is this insane?

Yes

Originally Posted by psychoandy

maybe if big pharma moved their reader's digest/daytime TV advertising budget to the organic chemists, biologists, and pharmacologists, we wouldn't be in this dilemma.

And lose there 18% profits a year when fortune 500 companies only make 9%. Surely you jest. They will just keep cutting the headcount of those pesky scientists to keep there bottom line up until the patents run out and ........ ooops. Ruh Roh.

Originally Posted by owlegrad

Isn't there a compassionate use clause or something for cases of terminal illness without effective treatment that allows for drugs to be used without the usual process? Maybe I am thinking of something else.

For some drugs yes, from what I've seen its usually with treatments that were once approved, but have since have had that revoked.

Originally Posted by Brock Landers

Yes

I heard about this proposal a few months ago. I immediately discounted it as being a ridiculous idea. Some companies are sleazy enough that they'd market their products for anything. Some companies are fly-by-night. They'd set up shop, start selling some risky product, and simply go bankrupt once someone got hurt and sued. There are all sorts of unscrupulous (and perhaps mentally unstable) doctors who could start prescribing anything for everything!

So on its face. This seems like a mess! Then, a few days ago, I read about the patients with amyotrophic lateral sclerosis or Lou Gherig's disease who were using an unproven drug because they couldn't get into a clinical trial for a drug derived from the aforementioned drug. My grandmother died of ALS when she was 71. It was terrible. There was nothing that anyone could do for her. She had to slowly suffer for years with ever decreasing quality of life until she succumbed to this disease.

I am not talking about a zero sum game here, where we'd open the flood gates and let anyone do anything. We would have to think long and hard to determine who is responsible for costs, liabilities, patient protections, etc. Efficacy would still be determined and controls would have to be put in place to ensure patient safety. Our current approach is extremely costly and time consuming. This limits the type of drugs which are pursued and the time until which patients have access to medications that they may desperately need. This would not be a simple solution, but aspects of it are realistic and compassionate.

I was not asking if you thought that this proposal was insane. I was asking if you thought that patients, who have no other options and were going to die from their disease, were insane for trying an unproven drug. This does not seem like insanity, but rather, desperation. We prevent them in the name of safety, but this is misguided. Who are we to deny them the right to try to save their own lives?

Originally Posted by Argentium

wouldn't thalidomide be proof of why safety is required? correct me on my history

Originally Posted by genesis09

The thalidomide issue led to Kefauver-Harris Amendment. That law requires manufacturer's the drug is effective for its indication. Thalidomide was not approved in the U.S. in the 1950s. It never proved its safety.

The Sulfa Elixir event led to the Food, Drug, and Cosmetic Act of 1938, and that required proof of safety.

I never understood that either. I know that incident lead to the amendment, but I don't see how it was an issue of efficacy. It was just another safety concern. I can see a case where you were giving a drug for an indication, and people continued to die without benefit- that would be a case for efficacy.

Originally Posted by gadlo

In the Eschenbach editorial, it was not that efficacy would never be tested. Instead, a drug would be marketable during the clinical trial phases. Instead of having to wait through 10 years of clinical trials, a patient, with a doctor's prescription, could use the drug once it received approval from the FDA as being relatively safe. Clinical trials would still be performed, but the company could market its drug and patients would have access to the medications. This would not be the wild-west of drug use, with everyone taking all sorts of drugs for whatever the marketers decided was the biggest market. Patients would still need a doctor's approval for the prescription. There is the potential for abuse (by companies and doctors) and misuse (by patients), but hopefully, a patient would only try these unproven drugs as a last resort and after failing to respond on multiple rounds of standard treatment.

There would be risks, costs and liabilities would have to be worked out, but perhaps such an idea, or a variation of it, could decrease the costs of developing drugs such that drugs with new mechanisms of action for common illnesses and drugs for illnesses with no treatment and small patient populations could be pursued.

I do understand the idea, but it seems that you would end up with more lawsuits against drug companies. Think how many commercials you hear for lawyers about Darvon and Avandia. Those drugs have been around for a while, and still had post-marketing surveillance issues. If you shorten the clinical period, you're going to shorten the ability to see these effects. Orphan and refractory conditions I can see an exception, but coming out with some crazy new molecule for DM or HTN and using because "well it doesn't make you die, so try it!" should really not be the standard we use.

As for Qnexa, why don't physicians just prescribe it as two separate products? I don't see there being anything significant with the controlled release other than easier compliance, although I'll admit I haven't really read up on it. Theoretically you use phentermine 15mg qam and topiramate 50mg bid instead of Qnexa 15/92 and you've just saved a few hundred $. I can't see the 92mg vs 100mg being anything other than another Pharma tactic (Aricept 23, Androgel 1.62%). Feel free to enlighten me here.

Originally Posted by owlegrad

Isn't there a compassionate use clause or something for cases of terminal illness without effective treatment that allows for drugs to be used without the usual process? Maybe I am thinking of something else.

Compassionate use can be utilized for drugs in development or marketed drugs. Drug manufacturers give the drug away for free if the patient meets specific guidelines

Originally Posted by psychoandy

what is the timespan of phase 3 efficacy/safety RCTs relative to the rest of the drug development pipeline? what is stopping treatment refractory patients from enrolling in these trials? aren't phase 3 trials usually where we see the crazy random side effects where FDA blocks further development? do existing safety-only trials have the sample size or duration to see serious side effects? do you really think that big pharma wouldn't try to abuse such a system? how would an MD even know what drug to write for if it's only completed safety testing? how would they know it's safe AND effective for a condition? big risk for liability. huge.

maybe if big pharma moved their reader's digest/daytime TV advertising budget to the organic chemists, biologists, and pharmacologists, we wouldn't be in this dilemma.

Without a huge paradigm shift in drug development, investing more money in organic chemists, biologists, and pharmacologists most likely won't result in higher drug approval rates. The bottom line is that the human body is so complex that its difficult to predict what a drug will do from a theoretical point of view - especially if the science isn't well understood (ex. cns).

Also marketing budgets are down as well. There hasn't been big DTC spend in a while for a new product