----- Original Message ----- 
From: "Deborah Harrell" <[EMAIL PROTECTED]>
To: "Killer Bs Discussion" <[email protected]>
Sent: Monday, September 12, 2005 9:35 PM
Subject: Re: Sources of Drug Innovation
>
> But _discovering_ that drug can be in the course of
> basic or esoteric research, or even seredipitously.

It can be, The existance of this type of discovery is not in dispute.  The
prevelance is what I consider important.  For example, if 5% of drugs are
discovered in nature and 5% are discovered duing basic research, with 90%
discovered by various drug companies, then the effects of proposed policies
would be quite different from a situation where drug discovery was
distributed 1/3, 1/3, 1/3%. I appreciate the work you went to finding your
example, but that example doesn't address this question.  Indeed, I'd guess
that if, say, 20% of the drugs came from searches of naturally existing
compounds, then the vauge wording in the report would be replaced by more
precise wording.



> Naturally-occuring chemical compounds were a large
> source of many 'new' drugs in past decades (think
> antibiotics esp.), and in oncology is still very
> important:


This may be a good point to raise my second point. I'm not sure what you
mean by discovering...and I'm starting to think that this is very much at
the core of how we see things differently.  The nature of experimental
research varies with the maturity of a scientific field.  For example, high
energy physics, for a long time, was a matter of cranking up the energy
another factor of 2 or 5 or 10 and looking at the intereractions.  Charm
was found this way: there was a narrow resonence that was found by two
groups once the energies at the accelerators were high enough.  Careful
work is also needed, of course, and a bit of luck selecting the area in
which to run your experiment.  But, for this type of work, the connotations
of the word "discovery" works fairly well.

In the middle of the 20th century, I think this word worked fairly well
with medicine too.  It was the age of "medical miricles."  A few pills
could beat diseases that had been plauging humanity for millenia.  My mom
is very much a product of that time: she expects doctors to provide pills
to fix her every ill and has a hard time accepting that not eating properly
and drinking enough fluid is the cause of most of her ills. Simply by
looking with a bit of care, numerous drugs were found.

As a field matures, the "low hanging fruit" is fairly well picked over, and
a new regiem begins. New features are not really discovered in the same
sense as they were in the infancy of the field.  Now, development is much
more guided.

(BTW, I realize that I'm traveling far afield from the origional question,
but I hope that you can see that I'm trying to address a fundamental
question that underlies our perceptions: how does the process of research
and development work?)

At this point, the drop-off in the productivity in simple careful looking
is significant.  Now, detailed investigation is required.  The possibile
candidates for detailed investigation are typically infinite (well at least
infinite in terms of practical considerations).  What is already known is
used to rank potential candidates for investigation.

It appears to me that you lump this work into "development."  This limits
"discovery" to a small part of scientific research.  Most of what is called
"research" is guided detailed investigation.  It isn't simple development
because there are too many unknowns for that.  Indeed, this type of
investigation usually requires a very well trained intuition....because of
the mix of uncertainy and knowledge.

If that is what your catagories mean, then you do include most of what
others call research into "development."  If not, then I think it is fair
to say that drug companies have most of the discoveries of new drugs.

Let me comment on numbers a bit below.

>This program is
> becoming increasingly important in the treatment of
> cancer, as a number of important anticancer agents
> have been derived from natural products, including
> plant-derived agents (such as the vinca alkaloids,
> taxanes, and topoisomerase I inhibitors),
> microbe-derived agents (such as the anthracyclines,
> epothilones, and rapamycin analogues), and
> marine-derived agents (such as bryostatin and
> dolastatin 10).
> Dr. Cragg described the NCI's process for establishing
> collaborations with source countries to develop
> programs to screen potential anticancer agents.
> Currently, such collaborations exist with over 15
> countries, including Australia, Brazil, China, Costa
> Rica, Fiji, New Zealand, and Pakistan.

The obvious question I want to ask is "what is the denomenator?"  How many
new drugs do we have in for oncology in the last 30 years?  If this number
is a score or two, then these reports show a significant fraction of drugs
are created this way.  If this number is in the hundreds, then this report
is consistant with other information that I've been given.


> This program is an excellent example of how global
> cooperation can succeed in the discovery and
> development of new anticancer agents.

It appears that the claim in this  report is that the process they are
describing has _some_ meaningful contribution to fighting cancer with
drugs. I have no problem with that.


> Offering the Japanese perspective, Dr. Nagahiro Saijo
> from the National Cancer Center Hospital in Japan
> described the current state of anticancer research in
> Japan. Over the past few years, research has greatly
> improved largely because of the creation of the
> Japanese Clinical Oncology Group (JCOG) by the
> government. Dr Saijo also discussed Japan's
> contribution to cancer drug development including the
> contribution in the development of irinotecan, ZD1839,
> tegafur, and other agents...

This indicates that Japan has done something

> >From a pharma:
> ...The final address of the session was delivered by
> Dr. David Parkinson, from Novartis Pharmaceuticals,
> who discussed global drug development from the
> perspective of a global pharmaceutical company. Dr.
> Parkinson noted that although the opportunities are
> enormous, there are significant challenges to global
> drug development. Some of the more major threats
> facing the pharmaceutical industry include the
> increase in the cost of research and development
> without an attendant improvement in productivity (ie,
> the number of agents approved) the longer development
> time without a corresponding decrease in development
> risk; and the increasingly conservative regulatory
> climate coupled with the increasingly difficult
> reimbursement setting.
> The current reality in industry-sponsored clinical
> trials is that the majority of registration trials are
> focused on the United States and Europe. The changing
> reality, however, is that as the number of new agents
> increases, there is an increased need for patients in
> clinical studies that cannot be met within the United
> States. Thus, the interest in drug development
> globally is increasing within the pharmaceutical
> industry, particularly because there is an increased
> capability of countries outside of the United States
> to perform these trials.



> Some of the factors to consider in the move to truly
> global trials are: How common is the disease in
> various parts of the world? Is there a uniformly
> accepted standard treatment? Are there likely to be
> differences in the magnitude of response or in the
> toxicities within certain populations?
>
> > That development takes years, IIRC 5
> > years is a goal now for fast tracked drugs, but it
> > represents only a
> > fraction of what drug companies do in R&D.  Unlike
> > most companies now, drug companies actually do R.
> >
> > They need to find the candidates in the first
> > place....few candidates are
> > handed to them by universities or are found by
> > happenstance. They have to
> > search for candidates.  This search is done by trial
> > and error sometimes,
> > and it is now guided by computer programs that take
> > knowledge of chemical
> > reactions and models likely results of various
> > chemical combinations.  It
> > is much more research than what I do, for example.
> > It is actual trailblazing research.

> The article I cited deals with cancer research/drugs
> only; I will look for others, frex antibiotics and
> antihypertensives, heart drugs etc.

I hope you understand why I don't think the percentage of drugs that come
from various sources is a function  can be adressed by citing specific
examples.


> That's a teeny bit of exaggeration...   ;)
> I already acknowledgeded the pharmas near-undisputed
> in the drug development arena, and no-where did I call
> it "just" or "merely."

OK, but the word "development" has a very specific meaning in Research and
Development.  Development usually indicates that there is very little
that's really new involved.  For example, one colleague of mine at a former
company, a man with a great track record of innovations, complained that we
no longer did research and development; we only did development.

That's part of a trend that I've seen in industry.  Drug companies have a
far higher research budget than most industries.  The oil industry, for
example, has gone from having a number of fairly impressive research
facilities to having virtually none...during the time I've worked in it.

In a highly competitive climate, with minimal profit margins, research is
seen as a luxury.  It doesn't really pay to do it.

>But I will not call 'me-too'
> drugs that have no significant advantage over
> predecessors "significant discoveries" either.

No, those aren't; I agree.  One problem that I see is that the climate is
shifting away from doing industry research into drugs, because the risks
are starting to outweigh the potential rewards.  Gautam's point that a
company would be a fool to work on a new AIDs drug is a case in point.  I
know of  a start-up that had a lot of venture capital tied up in a single
promising new AIDs drug.  They were able to get the capital because of the
big potential payday.

Now, the potential payday must be balanced against the bad publicity a
company receives from making large margins on drugs needed by dying people.
So far, they've addressed this by mostly getting these margins in the US;
accepting smaller margins in Europe and Canada.  If the potential for a big
payday is lost, then it is reasonable to assume that venture capitalists
will lose most of their interest in startup drug companies.

One could argue that there is a tremendous amount of inefficiency in this;
that the money going to take folks to Pebble Beach and the bonuses paid to
salesmen, and the profits going to shareholders is not money spent on
research or production.  That's absolutely true.  Drug companies are the
fattest companies I know of.  They were shocked at the thought of a 4%
layoff when it looked like some sort of price control on drugs were
forthcoming.  Having suffered through multiple 50% and >50% layoffs, I
think of a 4% layoff as barely touching the deadwood.

But, the other point is that more efficient companies with smaller profit
margins do (with a few exceptions) little or no research.  It's all
development...and that is done on a minimalist basis. Instead of having a
department, one hires a consultant part time for a few years or even a few
months to do the job.  This is extremely efficient, but it cuts out
virtually all research.

Universities do fundamental research, as do government labs.  But, this is
usually not very directed towards a practical goal. For most scientists,
the real sexy research is unknown territory....high energy physics was that
when I obtained my Phd.  Less sexy is fundamental research in more
developed fields.  Useful results are the least sexy of all.  In many ways,
scientists need to have their arms twisted to do work that seems mundane to
them.  Inventing useful drugs is not fundamental....it's really not as sexy
as knowing more about how things fundamentally work.

The question I think we have to face is what we will do if drug companies
stop doing research.  This would be their most logical course of action if
US prices on patented drugs are cut to, say, Canadian levels.  Creating "me
too" drugs would, I think, still be profitable, but we might very well face
an overwhelming drop in true innovation at drug companies.

Given that likely outcome, we need to ask how expensive and how efficient
it would be to have governments take up the slack.  One way to answer this
is to look at the number of truly new drugs that have come out under
government patents over the last 20 years vs. the number that have come out
under private patents.

Dan M.




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