Precision Medicine will need to get out of the pharma silo that is based on symptoms

Welcome to the digital era of biology (and to this modest blog I started in early 2005).

To cure many diseases, like cancer or cystic fibrosis, we will need to target genes (mutations, for ex.), not organs! I am convinced that the future of replacement medicine (organ transplant) is genomics (the science of the human genome). In 10 years we will be replacing (modifying) genes; not organs!

Anticipating the $100 genome era and the P4™ medicine revolution. P4 Medicine (Predictive, Personalized, Preventive, & Participatory): Catalyzing a Revolution from Reactive to Proactive Medicine.

After low-cost airlines (Ryanair, Easyjet ...) comes "low-cost" participatory medicine. Some of my readers have recently christened this long-lasting, clumsy attempt at e-writing of mine "THE LOW-COSTE INNOVATION BLOG". I am an
early adopter of scientific MOOCs. My name's Catherine Coste. I've earned myself four MIT digital diplomas: 7.00x, 7.28x1, 7.28.x2 and 7QBWx. Instructor of 7.00x: Eric Lander PhD.

Upcoming books: Airpocalypse, a medical thriller (action taking place in Beijing) 2017; Jesus CRISPR Superstar, a sci-fi -- French title: La Passion du CRISPR (2018).

I love Genomics. Would you rather donate your data, or... your vital organs?

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Concernant les fichiers son ou audio (audio files) sur ce blog : ce sont des fichiers Windows ; pour les lire sur Mac, il faut les ouvrir avec VLC (

"Despite Spending $50 Billion Per Year In R&D, Pharma's New Drugs Less Effective Than Drugs Developed 40 Years Ago"

"Keep calm and don't take drugs"
"TechDirt" did read Philippe Even MD PhD's books about "Big Pharma"! ;-)

"Despite Spending USD 50 Billion Per Year In R&D, Pharma's New Drugs Less Effective Than Drugs Developed 40 Years Ago": link to the article (July 7, 2013, Techdirt)

... from the the-future-is-yesterday! dept

More bad news has arrived for consumers. The drugs don't work. Or, they don't work as well as they used to. Despite the exorbitant prices charged for new medications and despite the industry's claims that expensive R&D efforts are driving these prices up, the fact remains that newer drugs cost more and do less, riding a decades-long slide from peak potency.
Research published on Monday showed that the effectiveness of new drugs, as measured by comparing the response of patients on those treatments to those taking a placebo, has plummeted since the 1970s...

The new study in the journal Health Affairs examined 315 clinical trials that compared a drug to a placebo and were published in four of the world's top medical journals (BMJ, Journal of the American Medical Association, Lancet and New England Journal of Medicine) from 1966 to 2010. The drugs targeted the full range of human ills, from cardiovascular disease and infections to cancer, mental disorders and respiratory illness.
In the early years, drugs easily beat the placebo: They were, on average, 4.5 times as effective, where effectiveness means how well they lowered blood pressure, vanquished tumors, lifted depression or did whatever else they were intended to.

But the trend line was inexorably downhill, found Dr Mark Olfson of Columbia University and statistician Steven Marcus of the University of Pennsylvania. By the 1980s drugs were less than four times better; by the 1990s, twice as good, and by the 2000s just 36 percent better than a placebo. Since older drugs were much superior to placebo and newer ones only slightly so, that means older drugs were generally more effective than newer ones.
If this disappointing (albeit somewhat unsurprising) news wasn't damaging enough for an industry many people have developed a healthy distrust for, the passing of the national healthcare plan will make it even worse.
The law established an independent research institute to compare the effectiveness of different treatments for the same condition. That way, patients as well as private insurers and government programs such as Medicare can stop paying for less effective therapies. If the new analysis is correct, then "comparative effectiveness research" could conclude that older drugs, which are more likely to be generics, are better than pricey new brand names that deliver the most profits for drugmakers.
Well, this part is good news for consumers, or at least beneficiaries of the new health plan. If the most effective drug is also the cheapest, everyone wins... almost. Pharmaceutical companies won't be happy, but this really is their own fault. They often tout the (often inflated) high dollar cost of R&D but fail to mention this outlay is routinely outweighed by marketing and administration costs.

There are a few reasons effectiveness may have declined over the past forty years, not all of which are tied to self-interest and profit chasing. One possible factor is that the low-hanging fruit of the pharmaceutical world was plucked first, generating effective medications for simpler ailments. It also could be that those volunteering for clinical trials are increasingly people not having success with currently available drugs. Another factor mentioned in the article is the fact that the quality of clinical trials has increased over the years and the additional scrutiny to detail has narrowed the definition of success.

Despite these factors, many scientists feel there is an underlying truth to the overall claim that older drugs just work better.
While experts agree that tougher trials and similar factors explain some of the decline in drugs' reported effectiveness, "something real is going on here," said Olfson. "Physicians keep saying that many of the new things just aren't working as well," and therefore prescribe antidepressant drugs called tricyclics (developed in the 1950s) instead of SSRIs (from the 1980s), or diuretics (invented in the 1920s) for high blood pressure instead of newer anti-hypertensives.
Then there's this:
"The way the drug regulatory system is set up, even if you have just a small advance, if you market it right it can be very profitable," said [Dr. Aaron] Kesselheim.
The profits-over-effectiveness factor cannot be denied. Marketing budgets routinely meet or exceed R&D budgets because they have to. Pharmaceutical companies are not nearly as interested in breakthrough medication as they are in pushing minor variations or incremental advances. [See also: this "Brain Candy" clip.] Without a ton of marketing, these interchangeable drugs will never find purchasers.
From 2000-2007, 667 new drugs were approved by the FDA. Of those, only 75 (11%) were new molecules that were much better than what we already had. In fact, over 80% of all drugs approved were no better than what we already had. Those are "me-too" drugs. Why do the pharmaceutical companies spend so much on marketing? Because you have to really promote drugs that really have no benefit over others that already exist. You have to convince people to buy those.

You know what needs no promotion? Awesome new drugs that save lives. When was the last time you saw a commercial for chemotherapy? For epinephrine? For steroids? Those drugs need no promotion - doctors just know to use them. But I bet all of you know about Nexium. Or Cialis.
Taking this route has worked for years, at least in terms of profitability. There's no reason to change it now. The results of this research paint a rather unflattering picture of the pharmaceutical industry, but as usual, its representatives seem blithely unconcerned.
The drug industry says it isn't worried. "Our sector is not concerned about objective, high-quality patient-centered comparative effectiveness research," said PhRMA's Burkholder. "We believe the substantial value of our products will continue to be demonstrated."
Frankly, I'm inclined to believe that first sentence. The sector doesn't seem to care what research says about its new, expensive, ineffective drugs. It already has plenty of compliant doctors, paying customers and an exploitable patent system in its corner. That makes the second sentence extraneous. If the market's tied up, the industry doesn't really need to worry about demonstrating value. All it needs to do is maintain course.

1 commentaire:

Low-Coste Innovation Blog a dit…

"La loi de transparence qui oblige à déclarer un agenda cadeau (s'il dépasse 10€) mais dispense de déclarer le colloque aux Bahamas (si l'on intervient quelques secondes au micro)."