Pillhead

the personal views of a doctor in industry

Posts Tagged ‘R&D

A Walk in the Valley of Death

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The NIH has announced quite loudly that it is taking a bold step in helping to find cures for rare and neglected diseases (NIH Announces New Program to Develop Therapeutics for Rare and Neglected Diseases, NIH News). So what do the numbers look like?

 

According to a recent NIH press release, the atrition rate in the pre-clinical phase of development is so bad that researchers call it the “valley of death”! They think 80-90% of compounds fail to make it to human testing; some inside industry think that even this is low-balling it, and that number is closer to 90% (The NIH Takes the Plunge, In the Pipeline).

 

Some more numbers from the NIH press release:

  • “NIH estimates that, in total, more than 6,800 rare diseases afflict more than 25 million Americans. However, effective pharmacologic treatments exist for only about 200 of these illnesses.”

  • “Studies suggest that it currently takes more than a dozen years … to take a potential drug from discovery to the marketplace”

  • “it takes two to four years of work and $10 million, on average, to move a potential medicine though this preclinical process”

The WSJ reports that, “Stephen Groft, the director of the rare diseases office, said the program is starting with $24 million in funding this year with expectation of receiving the same amount each year until 2013”

  • USD24 million for 4 years is an investment of USD92 million;
  • Each drug costs USD10 million “on average” to get through pre-clinical development, and so there is enough money to develop about 10 candidates;
  • 90% of these candidates will fail, leaving just one successful compound;
  • With the timelines given, this compound would come to market in 2020;
  • According to Steven Paul, President at Lilly Research Laboratories the chance of getting from phase 2 trials to market is 1 in 8 (Blockbuster Drugs and Innovation) – I cannot find the ph1 attrition rate;

 

This program has about a 12% chance of resulting in one effective treatment in 2020 for one out of the 6600 untreated rare diseases.

 

It is a step in the right direction, but let’s not get carried away.

 

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Written by Pillhead

May 22, 2009 at 3:33 pm

Sales of New Drugs – no more blockbusters?

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An article in Forbes (The Value Of New Drugs Is Dropping) cast an eye over the initial sales of new drugs launched in the last decade. The article looked at data from IMS, and found that drugs launched in 2008 sold only a tenth as much in their first 6 months on the market as did drugs launched in the banner year of 1999. Despite the good news that the FDA approved more drugs in 2008 than in 2007, “new medicines contributed much less than 0.5% to the growth of the global market, another way in which the first half of 2008 constituted six of the weakest months in a decade”

At first sight, this article appears to fit our gut feeling that the “low hanging fruit” are taken and few blockbuster drugs remain. It also fits and confirms the results of the drive espoused by several firms for more drugs of middle revenue.

But looking at the way this article presents data looks a little like cherry picking to me. The period of study is 1998 to 2008, but one of the big comparisons is made with 1999 a “banner year”. And using “six” as a statistical cutoff is odd: “2008 constituted six of the weakest months in a decade”?

How bad is it really looking for us from this data?

first 6 month sales for new drugs

first 6 month sales for new drugs

If there is a trend here, and I do not see a clear one, then it is equally valid to say that the last three years have seen a return to form. Never since the hayday of the late 90’s, when pharma launched Singulair, Vioxx, Viagra and Avandia, have we seen sales at these levels. 

I do not think this would be an accurate refection, but it is important not to see what we want to see in the data. 

What I do see in the graph is a remarkably stable picture of first 6 month sales in the region of 5-10 million USD. It would be interesting to look deeper to see why. 

There is certainly no strong indication here that the drugs launched more recently are any different from those 10 years ago. For that conclusion, we need more data.

Written by Pillhead

February 23, 2009 at 1:22 pm

Blockbuster Drugs and Innovation

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Sir James Black is a nobel prize winner for medicine and inventor of both propranolol (at ICI) and cimetidine (at Smith Kline and French). In an era when entire R&D divisions of big pharmaceutical firms have trouble coming up with a single drug of this caliber, a man credited with two of them must be taken seriously.

In a fascinating interview with the FT (An acute talent for innovation), he noted that we are an industry with “a reputation for development” but that “it keeps making promises”. I found this rather revealing in an elegantly understated way.

Our reputation for discovery is a deserved one. It talks to our past where significant headway was made against significant diseases. The interesting part is that Sir James feels that today we keep making promises that we do not keep. He is, in part, talking about the inflated sales projections for some of our pipeline products.

To emphasize just how off base our predictions can be, Sir James points out that the sales forecasts for propranolol and cimetidine (£250,000 and £5m respectively) were actually off by orders of magnitude. I know of another drug that was off by three orders of magnitude. Clearly even when we have a drug ready for market, even when it is in fact of the caliber to be a blockbuster, industry can be fairly clueless.

According to Steven Paul, President at Lilly Research Laboratories (WSJ, Big Pharma R&D: Things Are Tough All Over), only one in eight drugs in phase II trials will make it to market. It does not need pointing out that making it to market is no guarantee of commercial success. The idea of prospectively making bold predictions based on such unlikely bets is odd, both in the sense that otherwise intelligent people make them, but more so perhaps that nobody notes that this is daft.

As Sir James notes, “very few of the drugs classified as blockbusters retrospectively were designed in that way.”

Written by Pillhead

February 15, 2009 at 7:12 am

Posted in Pipeline

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