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John Horton

John Horton

Posted: November 7, 2010 11:19 AM

For cancer patients, what is the "acceptable risk" that a drug will pose safety risks or won't work properly? That's one of the thorny questions addressed by the FDA last week in a two-day, open-to-the-public meeting about "biosimilar" drugs -- second-generation versions of drugs made from living organisms, or "biologics."

Developing medicines from living organisms sounds like a plot directly from a B-grade Halloween movie. Heard of insulin? One type, Humulin, was the first biologic marketed, in 1982. Since then, the FDA has approved dozens of biologics for a wide range of uses, such as Abraxane, a chemical wrapped in human proteins, for breast cancer. Studies are continuing on novel products such as Desmoteplase, made from vampire bat saliva, for stroke victims.

Biologics are different from typical chemical prescription drugs in important ways. Generic drug companies can make virtually identical copies of non-biologics like Lipitor and Prozac. Once the original drug maker's exclusivity period expires, generic drug makers can apply for FDA approval, piggybacking on the original company's safety and effectiveness studies, rather than spend millions to develop their own. As a result, the company can offer generic drugs at a fraction of the original cost, allowing patients to save dramatically for medicines of equivalent safety and efficacy.

Yet biologics are different: just as no two living things are precisely identical, it isn't possible to create a truly identical "generic" version of a biologic drug. At most, a second-generation drug maker can create a version that is similar to, but not the same, as the original -- thus the term "biosimilar." But depending on how similar it is, a "biosimilar" may or may not be equally as safe and effective as the original biologic.

This begs the question before the FDA: When it comes to granting FDA-approval to a biosimilar, allowing the drug maker to bypass much of the FDA approval process for biologics, how similar is "similar enough" to keep patients safe?

As a matter of common sense, the FDA's process for biosimilars shouldn't sacrifice patient safety. After all, biologics are more complex than other pharmaceuticals, implying an enhanced need for scrutiny. But against the backdrop of the burgeoning biologics market, some biosimilar drug companies are arguing for more lenient safety requirements.

In a recent White Paper funded by an Indian biosimilar drug maker, the authors criticize Europe's strict requirements, advocating for India's laxer model. The authors point out that the relaxed regulatory requirements mean that some of the world's best, most effective drugs are widely available in India at a much lower price. True, but so are the world's worst, least effective drugs: India is a haven for the manufacture, sale and transit of expired, adulterated and unregulated medicines, including those sold by fly-by-night "rogue" Internet pharmacies. For example, India does not require biosimilar drug makers to evaluate virus clearance, or test whether the drug provokes an immune response. (This would seem to be desirable for a drug derived from vampire bat saliva.)

But perhaps most troublingly, the paper's authors argue that the economic downturn should prompt us to relax our safety standards more than we would if patients were not having a hard time making ends meet. They suggest that as long as the biosimilar drug can be sold more cheaply than the original, there is a level of "acceptable risk" to a patient that is justified by making the drug more widely accessible at a lower cost.

The notion that some adverse reactions can be viewed as an "acceptable risk" begs the questions: Which patients shall we put at risk? What kind of risk is acceptable? At what cost? Is it really good policy to lower safety standards in order to ensure greater access to medicines that may be more dangerous?

Biosimilars hold enormous promise: life-saving medications accessible by a greater number of Americans at a reduced cost. But if the biosimilars fail to have the same efficacy and safety as the original, it's the patient who may pay the biggest price. And if our medical system begins to view patients as mere numbers we will have already slid too far down the proverbial slippery slope.

The reason that generic (non-biologic) drugs are seen as safe and reliable alternatives to more-expensive brand medications is that the FDA has, by and large, held generic drug makers to the same high standards as the original manufacturer, refusing to dilute safety requirements. In developing a process and standards for biosimilar drugs, the FDA should hold biosimilar drug makers to those same high standards.

 
 
 
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Caroline Sutherland
Hay House Author, Speaker & Health Expert
07:30 PM on 11/08/2010
The public pays a high price for “trusting†drug companies. Now, “trusting†the FDA to slip on standards of biosimilar drugs is a double crime to the patient, the consumer. Don’t even get me started on the FDA standards. Think about it Food and Drugs – approved by same company! We all know Food-like-substances in packages, bags, bottles and cans give the Drug companies so much more business due to the very poor, addictive health (illnesses) they cause.
~ Dr. Theresa Ramsey, http://www.DrRamsey.com, @DrRamsey
09:34 AM on 11/08/2010
A thorny and interesting issue. Biologics are tricky to duplicate, to the point of impossibility in some instances, resulting in the position expressed in this article. On the other hand, I am instinctively skeptical of any position, however seemingly straightforward, providing big pharma with additional economic advantage - like they don't have enough already. Here, the non-approval of generic "biosimilars" will function as a de facto continuation of the preexisting drug patent, thereby shielding the biologic developers from competition and (artificially?) keeping prices high. Which way to go? What's the best choice for the FDA?

I would add a slightly diffrent approach. This article presupposes that the FDA should be the sole - sole - gatekeeper of pharmaceuticals in the US. Such is conventional wisdom. Little-known fact: the FDA is a disaster, maddeningly feckless and slow on the front end (drug approvals), and disastrously weak on the back end (enforcement). The recent egg scandal exposed the gaps in FDA enforcement capabilities, and the Administration is almost as weak on Drugs as it is on Food.

So, should we leave the "biosimilars" decision solely in the FDA's hands? Or should we allow individual providers and - more importantly to my mind - patients, once informed of the costs, risks and benefits, acquire these biosimilars for their own use, perhaps on an emergency or even last-resort basis?
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onionboy
Blessed are the Cheese Makers
04:31 PM on 11/08/2010
I don't think we can expect most patients to be able to make a properly informed decision about the safety and efficacy of some "biosimilar" biologic agent. It's very complex and we'll likely see any skew the result of advertising dollars spent rather than actual research that exists. That's pretty much how it works with drugs today. You'll be seeing the same with devices soon ("Doctor, I want the DuraLine artificial disk. It will let me do horseback riding again. If you won't give it to me, I'll find a surgeon who will.").

There is a way to resolve this, but it's going to take more government intervention into pharma and providers, not less. More transparency and scrutiny. If some consider that the greater evil, then they'll need to take the risk that goes with it.
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09:21 AM on 11/08/2010
This is such a tough issue.

There are real risks, and you do have to be very cautious. On the other hand, some of the activity on this front is pharma trying to protect its market share. And doing comparative evaluations on the products is expensive, and pharma-funded research can be skewed (not always, but there is the potential).
09:12 AM on 11/08/2010
FDA should definitely stick to the same safety standards for biosimilar products. There can be no 'acceptable risk' for any patient.

However, at the same time, let us also not rob the 'generic' movement of its credit. The generic movement has made many drugs available at affordable cost to the masses. The original leflunomide is still 10 times costlier than the generic version years after its introduction in the market. Prices of original biologics have come down (to the extent of 50% !) after introduction of the generics.
07:54 AM on 11/08/2010
Every medical intervention (or non-intervention) carries potential risks and benefits. The main job of medical providers is to weigh that balance in an individual patient. Having said that, safety and efficacy data should be required for every pharmaceutical product on the market so that the patients and providers can make informed decisions.
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William Diaz
Passive-Aggressive word salad tossed here!
07:09 AM on 11/08/2010
Most of the costs of name brand drugs, be they 'biologicals' or straight up pharmaceuticals is marketing. I dont have the reference here with me, but I was shocked when I read what percentage of a drug's cost went into ads, hiring cheerleaders to schmooze doctors and providing medical shills with huge honorariums. The cost for merchandising ended up being more than 2 times the cost of drug development, I do remember that...

There is no reason for the FDA to change the regime for safety and efficacy, but there is no reason to to do clinical trial all over again. Clinical trials of drugs are hugely important and necessary, but the cost of that is properly borne by the company to which the patent is assigned originally. The reason the drug companies want to make the process of selling generic biologicals more difficult is to bouy the prices of their own drugs after the patent expires. Just a way to milk a few more million out of the captive medical audience.

I am a scientist, I work with biologicals (my current grant has it written into one of the aims), and my wife does as well. In addition to that, I am in insulin dependant diabetic and use them myself.

Being in endocrinology gives me a unique perspective on the utility of such compounds, but being a patient gives me nothing but contempt for drug companies.

Have a great day!
04:59 AM on 11/08/2010
.
one of the major dangers of biologics is that adverse effects, some of which can be
extremely serious and cumulative, do not show up quickly--sometimes even taking
years to manifest... Even the smallest change in a complex biological molecule
can profoundly alter the safety and efficacy profile. .Most biologicals have a mode
of action that works on the human immune system, which is extremely delicate
and far from completely understood in all its ramifications
You can be assured that the OEM biologicals as approved by the FDA have been
thoroughly and exhaustively tested and reviewed for years, and that the risk/reward
in use of these OEM drugs is as indicated in the labeling. Most biologicals, because
of expense of development, are only developed for extremely serious conditions for
which "chemical" drugs are ineffective, and virtually all biologicals must be used with
extreme care and caution because of inherent risks--

the concept of a "generic" biological is a fallacy, and such biologicals
are not in any sense whatsoever the same as "generic" drugs.
.
02:30 AM on 11/08/2010
I spent ten years doing research on the manufacturing process of biological drugs for a major biotech company. The molecules are very complex, and the process for making them is extremely complicated. The process is also very sensitive (i.e. easy to screw up). It's very difficult to successfully transfer the manufacturing process from one site to another WITHIN THE SAME COMPANY.

This article is right. There is no way for a generic manufacturer to make the exact same product. In fact, since all of the product quality assays are proprietary to the company who developed them, there is no way for the generic company to perform the same tests on their product that were done for the original drug. Chemical drugs have a very clearly defined structure that is relatively easily duplicated. Biologic drugs aren't anything like that.

Bottom line: As someone with expertise in the field, I would not allow anyone in my family to take a biosimilar unless it was thoroughly tested for safety and efficacy. Biosimilars cannot be proven to be equivalent without that. Each one is a unique drug in and of itself.
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tomteboda
01:42 AM on 11/08/2010
All drug treatments have a level of "acceptable risk". Simply read the side effects on any over the counter or prescription medication to get a handle on common "acceptable risk" levels.
09:20 PM on 11/07/2010
FRAMED WRONG. It's pharma propaganda.

"biosimilar" = genetically modified.

The REAL question should genetically mutated biologics be ALLOWED to even exist, and the answer is a resounding NO.

These mutated "biosimilars" ONLY exist for the sake of extending profit, and we do not know the long term primary and secondary effects so the best thing is a complete moratorium. The precautionary principle.
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SilentSolidarity
So what do you need? Besides a miracle.
10:14 PM on 11/07/2010
""biosimilar" = genetically modified."
This is by far the worst comment I have ever read.

ALL MEDICINE is the result of biotechnology, and requires genetic engineering. If you oppose it, better start searching for some leafs in the rainforests of Congo in case you get sick.

Biologics are genetically modified organisms that target the sick areas of the body. If you don't like it, you can continue to use the chemicals they mix up that cause several side effects.
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cable1977
Against logic there is no armor like ignorance
11:27 PM on 11/07/2010
"Biologics are genetically modified organisms that target the sick areas of the body. If you don't like it, you can continue to use the chemicals they mix up that cause several side effects. "

That's not entirely accurate. Biologics aren't necessarily genetically modified anything and certainly don't have to be an organism. An antibody is a biologic. Other large proteins would also be considered biologics. And biologics can have just as many side effects as a small molecule pharmaceutical.
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MagicManDoneIt
When facts are lacking. Just say...
03:43 AM on 11/08/2010
Well, at least you haven't devolved into unfounded, paranoid conspiracy theory. Keep up the reasonable tone.
Genders
Love, Tolerance, Enlightenment
01:45 PM on 11/08/2010
They may be wrong, but there is every reason to think the big Pharm are conspiring to make big bucks and questionable treatments for sometime questionable disease. Don't be naive. That's why we need strong Republic regulation of the Pharm companies.
08:54 PM on 11/07/2010
I take Orencia IV's. The cost with infusion is about $40,000 a year.

There is a different rheumatoid disease modifier that killed the woman next to a friend getting IV's. So obviously there are issues of both cost and safety.

Most of us that take these toxic meds don't have a choice (who in their right mind would take then otherwise?), but we need safety as well, as long as it's not just a Big pharma stall.
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SparkyGump
It's time for the party of NO to Go
08:25 PM on 11/07/2010
I'm on Humira so this concerns me. I guess I'll find out if they're risky as I'm sure Medco will make me switch.
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01:49 AM on 11/08/2010
You are always welcome to pay out of pocket.
06:45 AM on 11/08/2010
I'm on Humira as well. Paying out of pocket is not possible... we'd be talking $1600 a month last time I looked into it.
06:22 PM on 11/07/2010
I agree that standards should be high. Relaxing standards is no guarantee that prices will be low since patients are not going to haggle with their life. Furthermore people expect a certain level of quality and betraying that trust would be unacceptable.
09:21 PM on 11/07/2010
high standards lol thats a good one. high profits is all that matters. your needs take a back seat. welcome to repug america.
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ResearchGuy
Writer,etc.
06:06 PM on 11/07/2010
IMHO, all drugs are a safety risk. Read the full prescribing information, never trust the advertising, and be especially cautious about any prescription drug advertised direct to consumer or pushed by a doc who may have been compensated by drug companies (as nearly all are in one way or another now).
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cable1977
Against logic there is no armor like ignorance
11:30 PM on 11/07/2010
Everything you do is a safety risk.

Getting in the car in the morning and driving to work is a huge safety risk. But then again, so is taking public transportation. Even walking down the street is a safety risk. Eating and drinking are both safety risks.

Why is it that people have such a poor understanding of risk? One doesn't just consider the risk, but also the benefit in order to evaluate if the risk is worthwhile.
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tomteboda
01:44 AM on 11/08/2010
According to the CDC, motor vehicle deaths account for more than 1/5 of the deaths of children. I'm surprised that no one ever argues it is child endangerment to put a youngster in a car.
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liberaldawg
What are you looking at?
04:40 PM on 11/07/2010
I've used enbrel for 6 years and has been a miracle drug for controlling my arthritis. However, since I am unemployed, I cannot afford the almost $1500 a month.
schatsie
banks are more dangerous than standing armies
08:43 PM on 11/07/2010
Check Canadian drug prices....I was on Tamoxifen when it was $100 per month in the States and 11 per month in Canada....
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01:48 AM on 11/08/2010
Tamoxifen is dirt cheap here. Look at Costco's drug pricing list online and ask your pharmacy to price match. Fun fact: almost no online "Canadian pharmacy" is actually located in Canada, and they are not strictly regulated.

Enbrel is significantly more than tamoxifen, even if you look at warehouse costs.

Liberaldawg, have you looked into a patient assistance program from the manufacturer of Enbrel?