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Derek Lowe The 2002 Model

Dbl%20new%20portrait%20B%26W.png After 10 years of blogging. . .

Derek Lowe, an Arkansan by birth, got his BA from Hendrix College and his PhD in organic chemistry from Duke before spending time in Germany on a Humboldt Fellowship on his post-doc. He's worked for several major pharmaceutical companies since 1989 on drug discovery projects against schizophrenia, Alzheimer's, diabetes, osteoporosis and other diseases. To contact Derek email him directly: Twitter: Dereklowe

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March 11, 2014

Compassionate Use: An Especially Tough Case

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Posted by Derek

Update: Chimerix says this evening that they will make their drug available to the boy in question as part of a new 20-patient open-label trial, after discussions with the FDA. This might have been the best way out of this, if it gives the company a better regulatory path forward at the same time. My guess, though, is that the company's position was becoming impossible to maintain no matter what.

Many of you will have seen the stories of a dying 7-year-old whose parents are seeking compassionate use access to a drug being developed by Chimerix. It's hard reading for a parent, or for anyone.

But I can do no better than echo John Carroll's editorial here What it comes down to, as far as I can see, is that a company this size will go bankrupt if it tries to deal with all these requests. So under the current system, we have a choice: let small companies try to discover drugs like this, without granting access, or wipe them out by making them grant it. Even for large companies, it's rough, as I wrote about here. I don't have a good solution.

Comments (58) + TrackBacks (0) | Category: Drug Development


1. Anon on March 11, 2014 1:09 PM writes...

What I never understand is why a parent just doesn't buy the molecule from a chemical vendor. It's available on the milligram scale for a few hundred dollars. Even if it would be malpractice or illegal they would still save their son. It would be worth a shot. You could probably find the dosing regimen in the current clinical trial literature.

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2. Anonymous on March 11, 2014 1:21 PM writes...

Pharma companies don't seem to have thought this out very well. Compassionate use is best handled by independent experts. Perhaps the solution is for companies to let someone like Quintiles organize Compassionate Use Advisory Committees of physicians. All requests would be judged by the independent committee and someone else would deal with the regulatory paperwork and data collection. The company could set aside some amount of drug on a yearly basis and from there, stay out of it.

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3. Anon on March 11, 2014 1:23 PM writes...

I don't understand how these programs work... could they provide the drug to the patient at cost?

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4. biotechtoreador on March 11, 2014 1:42 PM writes...

I believe the FDA does permit cost recovery for drugs in compassionate use (which one could play abt with using accounting tricks). This doesn't help company in long term.

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5. bigredbruce on March 11, 2014 2:01 PM writes...

I think you need to have a 3rd party entity handle "the compassionate use" distribution/admin burden. That seems like a legitimate role of government, or perhaps an independent charitable organization like a Gates Foundation if you are an anti-government type. But clearly the system won't work if a company is going to go bankrupt or at least suffer financial hardship to make the drug available. They have enough problems getting to market. When I originally saw story, I thought the reason was going to be that the drug company didn't want a bad outcome on the record for an uncontrolled trial of their product. But I see there is a real financial issue even before you get to that point.

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6. steve on March 11, 2014 2:06 PM writes...

I just don't buy it. The manufacture of a small molecule drug like this isn't cost limiting; the major costs of a Ph3 are the clinical trial costs, not the manufacturing. If the company has the resources to fund a Ph3 they can have the resources to supply the drug.

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7. Curious Wavefunction on March 11, 2014 2:13 PM writes...

Sounds like a classic moral dilemma: Do you save one now and preclude saving more later or do you sacrifice one now and maximize the probability of saving more later? Tough choice, my sympathies to the parents and I wish them and their child all the best. Can't see how I could have done anything different.

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8. Anonymous on March 11, 2014 2:16 PM writes...

I'm sorry but I have to call bs on the cost argument. Yes, it might "cost" a lot of money based on the "price the market will bear" post approval, but the cost of manufacturing a simple small molecule is peanuts and is not a factor.

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9. biotechtoreador on March 11, 2014 2:26 PM writes...

It's not a cost argument it's a 'why would I put my kid in a randomized trial where s/he may get a placebo when I can just buy the drug and make sure s/he gets it".

I don't see an adequate answer that satisfies both the desire to help people near term and to also have a sustainable orphan drug industry.

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10. Anonymous on March 11, 2014 2:35 PM writes...

"Chimerix, the company that manufactures Brincidofovir, has given hundreds of patients emergency access to the medication in the past, but they have since stopped this practice saying ‘they cannot afford it,’ according to Johnson Jr. However, Chimerix has received more than $72 million in federal funding to develop Brincidofovir."

Seems like a cost argument to me.

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11. steve on March 11, 2014 2:38 PM writes...

It's been posited as a cost argument everywhere I've seen it, which is BS as I said and Anonymous said. I looked up Chimerix' Ph3 on clinical - it's for patients undergoing hematopoietic stem cell transplants and the age cutoff is 18-89. They could give it to a 7-year old and not in anyway compromise their patient accrual. The simple way to handle this is to give it under compassionate use to any patient not eligible for their trial and for everyone else enroll them in the trial. How much money from investors, etc. are they losing by the horrible publicity they're engendering with this policy?

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12. Anonymous on March 11, 2014 2:41 PM writes...

"The company would have to dish out $50,000 per compassionate-use patient, since insurance doesn't usually pay for experimental drugs, Moch said."

50000$? Sure. That's what a simple small molecule cost to manufacture per two week dose.

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13. Am I Lloyd peptide on March 11, 2014 2:41 PM writes...

I don't think the issue is the manufacturing cost although that may well be part of it. The real issue as was pointed out by a commenter on that CNN article is legal liability and the future of the drug. Compassionate use patients are already in a precarious condition. What if a patient dies when he is on the drug? Even if this might have happened anyway, his death will undoubtedly be linked to the drug. This may well lead the FDA to reject it and endanger the lives of thousands of future patients with the disease. It's a tough moral choice and very unfortunate but I can see the logic.

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14. Anonymous on March 11, 2014 2:42 PM writes...

It's also an infrastructure/regulatory cost issue, they said if it was just one patient, it wouldn't be an issue, but hundreds of individual IND's all with potential for things to go south is a TON of risk for the company and a huge amount of work for the small staff. If like (5) said, the FDA or another agency was to take on the burden of individual IND's and so forth it would be fine because like everybody has said, manufacturing cost is negligible.

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15. Anonymous on March 11, 2014 2:46 PM writes...


Those are reasonable concerns. Claiming it cost 50000$ per patient as you try and feel out the market is not.

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16. alig on March 11, 2014 3:13 PM writes...

It seems like a total of 26 patients have been treated with the type of virus this boy has. Four of the 26 still died within 8 weeks.
From a Chimerix abstract in Feb 2014: A total of 26 subjects in Study 202 (n=14) and Study 350 (n=12) received BCV BIW based on AdV viremia at screening. Subjects ranged from 7 months to 68 years of age and all but one were HCT recipients. AdV was detected at the central laboratory in 21 subjects at the time of first dose. Baseline viremia (BL) ranged from 100 (the lower limit of detection for the assay, LLOD) to 2.2 x 107 copies/mL (median 3700). AdV viremia decreased to ≤LLOD for 67% (14 of 21) subjects within the first week of therapy; an additional 4 subjects (18 of 21, 86%) reached ≤LLOD at some time during treatment. High levels of AdV viremia (>1000 c/mL) detected in 14 subjects decreased to ≤LLOD in 79% (11 of 14) within a median of 9 days of BCV, and with a mean decrease of 1.8 log10c/mL. Observed all-cause mortality during a median 8 weeks (range 3 to 47) of follow-up was 15% (4 of 26). Two of the 4 subjects who died had undetectable AdV viremia at the time of death

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17. steve on March 11, 2014 3:19 PM writes...

There is no liability for the company under compassionate use; if it's approved by FDA the company is absolved of any liability issues. The infrastructure issue can be resolved by hiring one person in charge of processing or by contracting it out.

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18. steve on March 11, 2014 3:22 PM writes...

It's also hard to understand why the infrastructure issue would be worse while their in Ph3 than it was when they offered it while the drug was in Ph2.

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19. steve on March 11, 2014 3:27 PM writes...

sorry - "they're in Ph3" *&%! spellchecker!

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20. Ellie on March 11, 2014 3:36 PM writes...

The impression I got from the article was that the cost issue was because if they provide it to one child, they will have to provide it to potentially hundreds of other children who also desperately need it.

It's just such a sad story. They can't provide it, but of course the parents will fight tooth and nail to save their son.

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21. M on March 11, 2014 4:00 PM writes...

I think the major part of the cost is not supplying the drug itself. The FDA requires all kinds of monitoring, paperwork, reporting, and other regulatory hoops to jump through for a drug company to comply with the complex compassionate use regulations. That's where the $50000 per patient goes.

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22. rtw on March 11, 2014 4:17 PM writes...

hum... I posted a comment earlier on this topic and for the first time ever in the several years posting here I received message about the post being reviewed. It hasn't turned up yet today. Am I being censored?

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23. MDACC Grad on March 11, 2014 4:17 PM writes...

What if this could be offered with zero liability for the company regarding both the patient/family suing and that the FDA can not use that data against the company in any way. Additionally, the FDA (or another agency like the CMS) would give the company a fully transferable credit that can be used for any FDA transaction (like an NDA) or possibly a tax break? The value of this credit would then either be made anonymous, or slightly inflated, to prevent a commercial partner or CMS for using it as a basis for valuing the product as less.

Another option would be that if a company hits a certain threshold they would be issued a voucher, similar to FDASIA?

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24. Betsy on March 11, 2014 4:21 PM writes...

This is such a difficult situation, and I understand where both sides are coming from. But Chimerix is making a huge PR mistake by not being more vocal about WHY they cannot take on compassionate use cases. My Twitter & FB feeds are FULL of people ranting about Chimerix, calling for their CEO to step down, etc. Chimerix hasn't posted a single response on their Twitter account, and the only evidence they've made any statement at all was a report from this poor child's mother. I do believe Chimerix has valid reasons for their stance, but they need to get out there and explain them to the public. Failure to do so will forever link their name to this kid's death.

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25. weirdo on March 11, 2014 4:23 PM writes...

Isn't this a pro-drug of a marketed i.v. antiviral? Can't they put the child on that, without jumping through any hoops?

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26. Am I Lloyd peptide on March 11, 2014 4:35 PM writes...

#17: Absolved? I don't think so. If the patient dies then the company has some explaining to do, and it might be impossible to prove that the death wasn't caused by the drug. What's stopping the FDA from pulling the plug on it then?

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27. exGlaxoid on March 11, 2014 4:36 PM writes...

If there is no cost issue, then I don't see why someone here can't whip some up in their lab and send it to the family. Other than the legal and regulatory issues, which would be the same for the company. I see both sides, but I certainly would agree that if I was in their family I would find a way to obtain some even if they just hired a contract company to make some as a "research sample", and it happened to get lost somewhere. I am sure that people have gotten drugs that way before, it certainly happens often for non-FDA regulated drugs (eg meth, coke, etc), which seem easy enough to obtain.

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28. fyi on March 11, 2014 4:56 PM writes...

Just getting some chemist to whip up a batch will not help. The drug substance cidofovir is available and probably being given to him already. Unfortunately that may be part of the poor child's problem as there are severe kidney tox issues and poor absorption. CMX-001 is a complex formulation of cidofovir that will hopefully alleviate some of these issues. CMRX is a very small company of very limited resources that really needs to focus on getting the proper trials completed. It is also targeted right now for prevention of the infection, or for very new infections. This is an established case and may not be the best use of limited supply anyway.

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29. Anonymous on March 11, 2014 5:50 PM writes...

The system is sick. The country is sick.

By the way, how come I saw this news on, yeah, that Russian website!

The US Presstitutes like NBC, CNN, ABS, CBS will never mention a story like this.

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30. watcher on March 11, 2014 5:58 PM writes...

The problem here is with the media as much as the company.

But, what does the CEO make in income & bonus? How much do the board members and the scientific advisors get paid? Cut these expenses to the company, and more other things can be done.

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31. SteveM on March 11, 2014 7:37 PM writes...

Re: #28 fyi FYI some more: A search indicates that CMX-001 is not a formulation of cidofovir, but rather a long chain phosphate ester of that parent molecule.

This site does not allow web addresses in comments. But if you do a Google search on "brincidofovir structure images" it pops up one specific image of both molecules.

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32. great unknown on March 11, 2014 8:19 PM writes...

I sympathized with the "damned if they do, damned if they don't" logic in Derek's post. Until I saw this:

If this is true, what can the justification be?

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34. SteveM on March 11, 2014 9:01 PM writes...

Re: #32 Anonymous. Thanks for the link. How did you post it? When I try to post a comment containing a link, the system holds the comment for review but it never appears.

Appreciate any advice.

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35. steve on March 11, 2014 10:16 PM writes...

As they should have. To recap, since many posted without reading the earlier responses:
1) Cost of the drug is trivial, should not prevent the company from supplying it;
2) The company supplied it when they were in Ph2 so there's no reason they couldn't have supplied it in Ph3; the infrastructure arguments don't differ;
3) The clinical trial is restricted to patients 18-89 so they could give compassionate use to patients outside that demographic as with Josh;
4) The publicity is going to make their problem much worse than it would ever had been if they'd just quietly supplied the drug.

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36. David on March 11, 2014 10:57 PM writes...

I, for once, would love to see the media and the rest of the ranters put their money where their mouth is, and pay fully for the course of treatment.
it's very easy to demand SOMEONE ELSE pay for whatever it is. this is, imo, a simple solution.

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37. matt on March 11, 2014 11:08 PM writes...

This seems like a ready-made campaign for Kickstarter: start a foundation for small-company compassionate-use drugs. The foundation pays (perhaps occasionally audited) expenses of producing the drugs, may track as necessary the patient outcomes, handles the needed liability paperwork. If charities and all the Internet outcry are willing to pony up, problem solved, right?

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38. petros on March 12, 2014 3:40 AM writes...

re Matt

And how much would it cost to produce a sufficient quality of both novel drug and formulate it appropriately while complying with all GMP/GCP requirements?

It would almost never be a trivial matter to achieve

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39. simpl on March 12, 2014 8:24 AM writes...

fyi has it right in #28, it is not directly a cost problem, but restricted amounts of many R&D substances can continue into the first years after launch: think of penicillin. I've worked on several developments where batch planning, tox. or clinical studies were pushed around to optimise timing to available substance. But some of the more successful ones also had their planning in phase II and III revamped for additional uses - including additional indications, additional studies in more countries, WHO projects and compassionate use cases. There is always compassionate use for life-saving drugs if they improve survival, too, so you add that into your substance budget.
Also, some of the doctors were quite pushy, which is probably a good way to make things happen.

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40. Anonymous on March 12, 2014 8:33 AM writes...

#21 is spot on. That $50,000 figure sounds about right. It isn't really the cost of the molecule itself, it's that getting FDA approval for compassionate use exceptions involves RED TAPE. RED TAPE costs $$$. So yeah, $50,000 for FDA approval isn't far fetched.

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41. steve on March 12, 2014 9:06 AM writes...

Again, hard to believe. From FDA:
The paperwork reporting responsibilities a sponsor must submit for a single patient IND or single patient use under an existing IND is modest. If a patient is treated under an existing IND, the sponsor must collect and report adverse reactions and include such events in its annual reports. A single investigator wanting to treat a patient will refer to the commercial IND for most information and will have to provide additional information about the patient to be treated to obtain informed consent and local IRB approval. Exactly what to do is described in the oncology part of FDA's website and the Agency's role in the process.

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42. Troy on March 12, 2014 9:08 AM writes...

Brincidofovir is the prodrug of Cidofovir, which is marketed as Vistide by Gilead. Why doesn't the boy just get Cidofovir? It should be the same mechanism of action, and if it is life-threatening, why not go with something that is already on the market?

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43. Troy on March 12, 2014 9:10 AM writes...

Brincidofovir is the prodrug of Cidofovir, which is marketed as Vistide by Gilead. Why doesn't the boy just get Cidofovir? It should be the same mechanism of action, and if it is life-threatening, why not go with something that is already on the market?

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44. sepisp on March 12, 2014 9:56 AM writes...

The public wants to have immediate access to unproven treatments while demanding thoroughly proven, 100% safe treatments. Have the cake and eat it too.

IMHO, the current policies are over-cautious. Rare fatal, but idiopathic events should be acceptable. For instance nimesulide was pulled from the market because of a couple of deaths in liver-compromised patients. This is absurd - what bout the rest of the patient population that *isn't* liver-compromised? Speaking of livers, while nimesulide isn't, the main alternative, paracetamol, is virulently hepatotoxic if overdosed.

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45. steve on March 12, 2014 10:21 AM writes...

Troy makes a great point. Wonder why the company didn't suggest this? There's already human data for this indication.

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46. Anon on March 12, 2014 10:27 AM writes...

Interesting how the stories written now say that "the ceo caved" and use language that implies he was greedy and holding back his own private stash of the drug. The company is clearly going out of their way to help this kid and are still getting bad press.

If a dog barks for 15 minutes you finally let it in the house just to shut it up, the next time its just going to bark for 20 minutes.

Note to self: don't found a startup that pursues ground breaking treatments

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47. Anonymous on March 12, 2014 10:36 AM writes...

@45: Precisely. This is the biggest question of this whole saga.

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48. Triazine on March 12, 2014 11:19 AM writes...

@Troy and @Steve: CNN says he was already treated with Brincidofovir ( but it nearly shut down his kidneys, which is why his doctors were looking to Cidofovir.

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49. Anonymous on March 12, 2014 11:20 AM writes...

@42, 45, 47, etc., wondering why they didn't give him cidofovir...they did. Note this sentence from the original CNN article: "They gave him an antiviral drug, an intravenous form of brincidofovir, but it ravaged his kidneys." The 'intravenous form of brincidofovir' they're referring to is cidofovir (Vistide).

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50. Lu on March 12, 2014 11:52 AM writes...

The boy is only 7 and already has 4 cancer diagnoses. His organs are severely damaged from all the disease and treatments. The prognosis is...not good. And all this anger looks misdirected.
Americans need to learn to let it go.
I'm starting to draft my advance directive.

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51. steve on March 12, 2014 12:01 PM writes...

@Triazine, thanks for the info; I think you switched the two compounds but that's interesting and sheds a different light on the whole controversy. Would the kidney tox of Brincidofovir really be different than that of Cidofovir? Even if it's a different route of administration, don't you need to achieve the same Cmax to be effective?

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52. Anonymous on March 12, 2014 12:13 PM writes...

@51 (Even if it's a different route of administration, don't you need to achieve the same Cmax to be effective?):

Not necessarily. I've never looked at the PK of this drug, but toxicity may be associated with Cmax while efficacy may be associated with overall exposure (AUC). The reason for developing the prodrug brincidofovir may be a good way to get high overall exposure over time without a toxicity-inducing spike.

An example that comes to mind: Vyvanse as a prodrug of amphetamine. With Vyvanse, you can't reach the same Cmax as you can with straight amphetamine. (In this case, high Cmax is being avoided for a different reason--abuse potential, not toxicity--but the case still holds.)

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53. qetzal on March 12, 2014 12:53 PM writes...

Here's an excerpt from the publication on the Phase II study of CMX001 (Marty et al., 2013 NEJM 369:1227):

CMX001 is an orally bioavailable lipid acyclic nucleoside phosphonate that is absorbed in the small intestine and transported throughout the body as a phospholipid. It crosses target cell membranes by means of facilitated and passive diffusion and has a long intracellular half-life. CMX001 is converted intracellularly to cidofovir diphosphate after cleavage of its lipid moiety and phosphorylation by intracellular kinases. Unlike cidofovir, CMX001 is not a substrate of organic ion transporter 1, is not concentrated in renal proximal tubules, and is unlikely to have renal toxicity.

So there are a few possible reasons why the prodrug is more potent/less toxic than cidofovir itself, and why it might work in the boy when cidofovir itself did not.

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54. Design Monkey on March 14, 2014 8:15 AM writes...

This calls just for a little bit of American entrepreneurship.

Organize custom synthesis company which offers synthesis services. Anybody wants an experimental drug thingy, not yet approved by FDA - go and order synthesis of it. This way synthesis company doesn't sell brincidofovir or anything itself (that would be extremely evil violation of patents), it sells only synthesis services of what customer ordered. End product will have analytical certificates of it's purity and also a label "Not for human use". If customer decides to do something inappropriate and illegal with ordered product - like eats it - that's his own responsibility, he can not sue anybody, but itself. Also this way customer pays for synthesis himself, instead of making crapstorm in media, that company has to give it him for free and with dozen of dancing doctors in addition, just because he wants it.

Of course this model will not work with biologicals, and will need a bit of ironing of legal issues (Better call Saul!), like possibly setting up a separate consulting company which consults (but does not advice anything that's violates medical practice regulations, God forbid) about what customer wants to order.

In short, if one wants non-approved drug - take costs and responsibilities yourself.

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55. Deep Lurker on March 14, 2014 10:01 AM writes...

@41 "The paperwork reporting responsibilities a sponsor must submit for a single patient IND or single patient use under an existing IND is modest."

And your reason to believe that this sort of self-serving statement from the FDA is accurate and truthful is?

OK, it might be that the FDA sincerely believes that a $50,000 cost for their reporting requirements is "modest." But if so, their statement is truthful in a Jedi sort of way, "from a certain point of view."

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56. steve on March 14, 2014 6:22 PM writes...

And your reason for assuming the $50K cost is accurate and truthful? Since the company already has an IND (they're in Ph3) all FDA asks is that adverse effects be reported in their annual report. Sounds like "modest" fits that description much better than "$50K/patient" (wherever that figure came from...)

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57. Anonymous on March 15, 2014 4:46 PM writes...

#1, #54, i've pondered this issue myself. my father will be paying $10k for the first month of treatment with a small molecule inhibitor in a last ditch effort against his cancer that his insurance would not cover. i believe his doctor proposed the option based on positive results from a single other patient with a similar profile who had managed to get into a trial. in his case the money is there, but what if the price had been 50k or 100k, just for a shot in the dark?

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58. joeylawn on March 21, 2014 4:10 PM writes...


The boy seems to be doing better.

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