innovation.org - Newsletters

Realities of Biopharmaceutical Research & Development
13 September, 2010 -
It is well-known that researching and developing new medicines is a long, high-risk, and expensive venture. In fact, it takes an average of 10 to 15 years to bring a drug from discovery to approval[i], most drug candidates fail along the way and, on average, each new medicine costs over $1 billion.[ii] But what goes on in the R&D process that makes it so hard, and what drives researchers to spend years relentlessly studying one medicine?



Several recent articles give insights about the complexity and uncertainty of biopharmaceutical R&D, and also what inspires researchers to pursue new advances despite the long odds.



•Ideas for new medicines come from unexpected places, and R&D projects often take unexpected turns. A recent Wall Street Journal story followed the history of a promising new multiple sclerosis drug called fingolimod currently under review at the FDA.[iii] The medicine was originally tested for use in transplant patients.



“Fingolimod comes from an idea hatched a quarter-century ago by Tetsuro Fujita, a Kyoto University pharmacology professor who had investigated bitter plants used in traditional Asian medicine…. Dr. Fujita assembled a team from his university and two Japanese companies to sift through the various fungal products. They found a potent immunosuppressant in a particular kind of winter-insect-summer-plant, called Isaria sinclairii.



[….] “After tests, Switzerland-based Novartis dropped the idea of using the medicine for transplant patients because it was no more effective than existing agents. But the company discovered that fingolimod had a powerful effect in multiple sclerosis, reducing the number of relapses in which patients may experience vision problems, fatigue, numbness and other symptoms. Trials in thousands of patients followed.”

•Progress requires collaboration, especially as modern research becomes more complex and data-rich.
A recent article in the New York Times told the story of an ongoing collaboration between government, biopharmaceutical companies, imaging companies, universities, and non-profits to share data on Alzheimer’s disease (AD).[iv] The collaboration has led to progress in early diagnosis of the disease and over 100 studies on new drugs to fight AD.



“'We wanted to get out of what I called 19th-century drug development — give a drug and hope it does something,' Dr. [William] Potter [an Eli Lilly researcher] recalled in an interview on Thursday. “What was needed was to find some way of seeing what was happening in the brain as Alzheimer’s progressed and asking if experimental drugs could alter that progression.'


“Scientists were looking for biomarkers, but they were not getting very far.


“'The problem in the field was that you had many different scientists in many different universities doing their own research with their own patients and with their own methods,' said Dr. Michael W. Weiner of the San Francisco Department of Veterans Affairs, who directs ADNI. “…. What was needed was to get everyone together and to get a common data set.'


“But that would require a huge effort. No company could do it alone, and neither could individual researchers.”


•Developing a plan, or protocol, for a clinical trial is very complex and full of uncertainty.
A recent article in the New Yorker by Malcom Gladwell follows the ups and downs of a small biotech company working on a new cancer treatment.[v] After testing it in several types of cancer they found that it had benefit against melanoma, one of the most complex and difficult to treat cancers.

“Synta was faced with a dilemma. Given melanoma’s variability, the company would ideally have done half a dozen or more versions of its Phase 2 trial: low-LDH, high-LDH, early-stage, late-stage, prior-chemo, chemo-naïve, multi-drug, single-drug. There was no way, though, that they could afford to do that many trials with seventy patients in each treatment arm…. But not even big firms can do multiple Phase 2 trials on a single disease – not when trials cost more than a hundred thousand dollars per patients and not when, in the pursuit of serendipity, they are simultaneously testing that same experimental drug on two or three other kinds of cancer. So Synta compromised.”

•An idea gives researchers the spark, but empathy for patients is the inspiration.
Malcom Gladwell’s article quotes a researcher at a biotech company talking about the failure of a promising cancer drug:


“The patient was just out of college. He had and I.V. in his arm. You went to the hospital and you sat next to some kid whose only wish was not to die, and it was impossible not to get emotionally involved. In physics, failure was disappointing. In drug development, failure was heartbreaking.”


These observations just scratch the surface of what it is like to research and develop new medicines but they provide a window into the long, intellectually-challenging journey that researchers take, with the ultimate goal of making a difference for patients. Today there are nearly 3,000 new medicines in development.[vi]

PHRMA | Disease is our enemy. Working to save lives is our job.
------------------------------

[i] J. A. DiMasi, “New Drug Development in U.S. 1963–1999,” Clinical Pharmacology & Therapeutics 69, no. 5 (2001): 286–296; M. Dickson and J. P. Gagnon, “Key Factors in the Rising Cost of New Drug Discovery and Development,” Nature Reviews Drug Discovery 3 (May 2004): 417–429; J. A. DiMasi, R. W. Hansen, and H. G. Grabowski, “The Price of Innovation: New Estimates of Drug Development Costs,” Journal of Health Economics 22 (2003): 151–185.
[ii] J. A. DiMasi and H. G. Grabowski, “The Cost of Biopharmaceutical R&D: Is Biotech Different?” Managerial and Decision Economics 28 (2007): 469–479.
[iii] P. Landers, “MS Drug’s Epic Journey From Folklore to Lab: Research into Ancient Chinese Fungus that Propagates Inside Insects Yields Potential Relief for Multiple Sclerosis,” Wall Street Journal, 22 June 2010, http://online.wsj.com/article/SB10001424052748704256304575320714138159240.html?mod=WSJ_hps_sections_health.
[iv] G. Kolata, “Rare Sharing of Data Leads to Progress on Alzheimer’s,” New York Times, 12 August 2010, http://www.nytimes.com/2010/08/13/health/research/13alzheimer.html?pagewanted=1&_r=1&th&emc=th.
[v] M. Gladwell, “Annals of Innovation: The Treatment: Why Is It So Difficult to Develop Drugs for Cancer?” New Yorker, 17 May 2010, p. 68-77.
[vi] Adis R&D Insight Database, 13 August 2010.

innovation.org - Newsletters