August 2013 Archives

Has a third barrier to market access emerged to hinder the pharmaceutical Industry?

First, it was regulators that stood in the way of pharmaceutical products getting to market. The past decade or so has been a fallow period for the pharmaceutical industry in terms of the number of NMEs approved by regulators in the US and other major markets. However the past two years have seen an uptick that suggests that the industry might be, at long last, reaping the rewards of its investments in R&D and licensing to bring products to regulators with the data necessary to receive approval. While increasing its success at getting over the regulatory hurdles, the industry has faced increasing challenges in getting someone to actually pay for its products once approved for sale.

The second hurdle has been - and continues to be - payers who refuse to reimburse for products that do not demonstrate both economic and clinical value compared to the existing standard of care (see NICE, CMS lowering reimbursement rates, step-through programs instituted by private payers, etc.). Companies are only just beginning to realize the importance of market access as a goal rather than a corporate function that negotiates prices and rebates with a formulary manager. Building the capabilities to deliver the data that supports a product's value proposition to payers (as well as physicians and patients) has been a long, and in some cases, painful journey.

Even as pharmaceutical companies come to terms with the needs for CER and RWE in support of efforts to market their products, they may need to deal with an additional access barrier in certain markets: The patent office.

While pharmaceutical companies had to defend their patents against challenges from generic manufacturers in the past, they have not had to demonstrate any benefit for their products in order to receive and maintain patents. The sole criteria for being granted a patent was that the product was novel - not that it worked or that it was even safe.

In recent months the Indian patent office has refused to grant or has rescinded patents on pharmaceutical products on the grounds that they were not innovative enough. In addition to recent revocations of patents for two Allergan eye drugs (Ganfort and Combigen) the Indian patent office has rescinded patents on Pfizer's cancer drug Sutent, Roche's Hep C drug Pegasys, and a Merck asthma medication.

While this trend has not yet extended to other emerging markets, the Indian market is one of the highly anticipated engines of future growth for the industry. Pharmaceutical companies will need to demonstrate economic and clinical evidence of their product's innovation in order to persuade yet another stakeholder - the patent office - that the product is of sufficient benefit to the Indian population to be granted and to hold onto a patent.

The good news is that the same capabilities and data that are needed to convince payer stakeholders can be leveraged with the patent office. This additional hurdle simply provides added urgency for companies to undertake a fundamental rethinking of market access from the days when the regulator presented the largest barrier to entry.

A few months ago I asked: What is happening to health care in America? My answer was that no one really knows. Since then, events have reinforced this conclusion.

(1) The health care "law" is a moving target. President Obama unilaterally delayed the ACA's employer mandate by a year. His administration also delayed by one year the law's limits on patients' out-of-pocket costs. Now, to quell uproar among Congressional members and their staff, the Office of Personnel Management is planning to give extra money to those higher-income government employees set to lose their subsidized health coverage as they shift from the Federal Employees Health Benefits Program to coverage under Obamacare. These employees currently have generous coverage but, absent this revision, won't qualify for subsidies under the ACA. Without these subsidies they warn of a "brain drain." Will this stand? We don't know.

(2) You will note that it is the Democrats who are complaining the loudest to Obama about losing their subsidized health insurance. These are the very same Democrats who voted for the health care law just a few years ago. Now, if there was any doubt, it's clear that the lawmakers who voted on Obamacare had not read or had not understood what they were voting on.

(3) Obamacare's affects may vary widely between one person and another and between one company and another. For instance, a pharmaceutical company that markets hypertension or cholesterol drugs might see a different outcome from a pharmaceutical company developing and selling medicines for multiple sclerosis. The former therapeutic areas have room for higher patient treatment rates while most MS patients are already being treated. Anyone who says they understand how this will play out must first understand it from all such sides. There is no one answer.

(4) The 906 pages of the Affordable Care Act are being converted into tens of thousands of pages of "real" laws by the government regulators who are responsible for implementing the law. Who really knows what these regulators will come up with? I don't. They may not either.

I love hate mail

The following exchange is a result of an op-ed from July 2012 where I debunked the utter nonsense that naturally occuring and plant-based drugs are better and safer. Someone took exception. Couldn't resist.

"You're just joshing."

No I'm not.

"Several Native Nations herbal healing traditions observe the benefits of the use of white willow bark for pain relief".

Big deal. The Chinese used to use elemental mercury as a laxative. Because something was used 5000 years ago, this means that it is somehow superior to what we use today? Can you say "Luddite?" And FYI, aspirin was invented to mitigate the stomach problems that salicylic acid was causing. Plus it actually works. And salicylic acid is used to remove warts.

"One would think the practice would have been discarded generations ago if the conditions associated with salicin had been widespread."

It should have been. Yet some people are so ideologically blind that they cannot accept the fact that medicine is just a *little* better off than it was back then.

"oh, that's right, you indirectly group Native Nations herbalists with ignorant Europeans who believed in a flat Earth, an Earth-centered solar system and alchemy's base metal to precious metal conversion myth."

Nice logic. Because people were scientifically ignorant 2000 years ago, that means that they are now. If I read you right, since the majority of people on earth didn't accept Copernicus 600 years ago, that means you shouldn't take an aspirin. Give me a few years and I'll try to figure out your logic. Make that a few centuries.

"Don't forget the "medical practitioners" who believed in bleeding for gout, who administered liquid mercury for a wide variety of medical complaints, and who refused to observe hygiene as a basic protection against bacterial infection until a blind researcher stumbled on the discovery of the association of certain strains of bacteria with certain disease processes."

You need to fact check. I don't think John Snow (who put 2 and 2 together, solving the 1850's cholera epidemic) was blind. But he may have needed reading glasses.

"You appear to debunk the people's chemist's assertions about aspirin by selectively attacking his advertising mis-statement that Mother Nature is the source of aspirin, and his hyperbole about the chemical giant, Bayer's motives for patenting its creation, aspirin. Granted: aspirin is sourced from plants".

No it's not. Aspitin is synthesized from salicylic acid, which is in plants. There is NO aspirin in plants.

"Granted: Bayer is legally able to patent the processes it developed for synthesizing aspirin from plant life. Common sense."

Absolutely, except for the common sense part. The patent for aspirin ran out 15 million years ago, so I have no idea what you are talking about. Nor do you, it seems.

"however, as a debunker who surely is interested in balancing both sides of the argument."

No I'm not. I'm interested in the truth. Science is not a democracy. And if I had to spend my time responding to every crackpot conspiracy theory. I would simply not have time to take care of more important priorities in my life, such as sorting lint and collecting Sponge Bob memorabilia.

"why no discussion of the people's chemist's scientifically justifiable attack on industrially derived aspirin's horrifying range of documented side effects?'

1) Because I didn't feel like it. 2) If aspirin is so bad, please explain why Americans consume 80 billion pills per year, yet the zombie apocalypse has yet to happen. Except on The Walking Dead, which is just plain awesome. (P.S.-should you choose to watch it, I'm just letting you know that it isn't real. And if you somehow believe it *is* real--not unlikely, given what I've read from you--please feel free to blame the pharmaceutical industry, which we all know is responsible for the assassination of Archduke Ferdinand of Austria, traffic on the Cross Bronx Expressway and the price of pork bellies on the Chicago Mercantile Exchange.

"internal bleeding, potentially deadly ulceration of tissue in the lining of the stomach and small intestine, Reyes syndrome in flu and chickenpox sufferers who have been administered aspirin, aspirin allergy-induced asthma."

I guess that explains why the lifespan of Americans is going up every year.

"According to the Mayo Clinic, "Side effects and complications of taking aspirin include:
1) Stroke caused by a burst blood vessel. While daily aspirin can help prevent a clot-related stroke, it may increase your risk of a bleeding stroke (hemorrhagic stroke).
2) Gastrointestinal bleeding. Daily aspirin use increases your risk of developing a stomach ulcer. And, if you have a bleeding ulcer or bleeding anywhere else in your gastrointestinal tract, taking aspirin will cause it to bleed more, perhaps to a life-threatening extent.
3) Allergic reaction. If you're allergic to aspirin, taking any amount of aspirin can trigger a serious allergic reaction.
4) Ringing in the ears (tinnitus) and hearing loss. Too much aspirin (overdosing) can cause tinnitus and eventual hearing loss in some people.
Do you accept the authority of the Mayo Clinic, Josh?"

I sure do. But you don't. You are taking all the potential side effects of a drug (and this applies to *any* drug). Failing to take into account both the risks and benefits of a drug is de facto proof of an illogical and ignorant analysis. Such a trite mindset. According to your "logic" there isn't any drug in on earth that should be taken, unless it comes from some weed or whatever.

Normally I wouldn't bother to respond to this, but 1) I'm in a bad mood today 2) your comments were so obnoxious that I just couldn't resist 3) Just in case we have any future encounters, don't try to out-obnoxious me. It's like pulling a sword on Zorro.

I mean this with love.

As if the FDA doesn't have enough to do.

Yes, they sure do, but this didn't stop them from issuing a warning last week that is so ridiculous that it gave me a headache.

But, if you believe the FDA, I'd better learn to live with that headache, because if I happen to take anything to relieve it I could end up taking an unplanned dirt nap. At least that's their message. Which will no doubt scare the hell out of many people, and for absolutely no reason.

But it also provides a great opportunity to examine the data behind this warning and recognize how absurd it really is.

For reasons that are entirely unclear, the FDA decided that acetaminophen (Tylenol) must now carry an additional warning that the drug can cause Stevens-Johnson Syndrome, a very serious (and potentially fatal) condition where epidermal cells die, causing the skin to slough off the body. SJS is an acute dermatological emergency.

There are multiple causes for this rare condition, including viral and bacterial infections, UV light or radiation exposure, but most cases are thought to be caused by allergic responses to certain drugs.The drugs associated with SJS include antibiotics (especially sulfa drugs), non-steroidal anti-inflammatory drugs (NSAIDs), and anti-seizure medications. And now acetaminophen is on the list. But why?

The FDA warning notes that there have been a whopping 107 cases of SJS (and 12 deaths) over a 43-year period that have been associated with acetaminophen use. Since Americans swallow about 30 billion acetaminophen-containing pills each year, a little math tells you that 1.3 trillion of them have been consumed during this time frame. And they are worried about 12 deaths over 43 years from 1.3 trillion pills? This is probably about the same relative risk as choking on a tuba.

So, what should Americans should do with this information? Should we stop taking Tylenol and switch to Advil? Probably not, since Advil (ibuprofen) is an NSAID--a class that is also linked to SJS. Should we simply live in pain because there is a barely minuscule chance that someone will have a bad reaction to a drug?

No, we shouldn't. Which is why the FDA warning is both ludicrous and harmful. They are telling people that if you take acetaminophen you need to be on the lookout for the symptoms of SJS. This is certainly helpful, since otherwise I wouldn't even consider seeking medical help if skin started falling off my body.

This is nothing but a useless and counterproductive scare. The label change will cause some people to avoid the drug, but will have absolutely no benefit. This is hyper precaution at its worst.

I'm more than willing to take my chances with the pills, but if you give in to the FDA's baseless warning and chuck all your pain relievers, it's no skin off my back.

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