The Patient-Driven Drug Pipeline

Excerpts from the book

Confronting Complexity

X-Events, Resilience, and Human Progress


John L. Casti

Roger D. Jones

Michael J. Pennock



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The new-drug pipeline is a mere shadow of its former blockbusting self. Despite the fact that R&D expenditures have been increasing over the last several years, the number of drugs in the pipeline has been decreasing. There have been many proposed explanations for this:

(i) a diminished margin for improvement that escalates the level of difficulty in proving drug benefits

(ii) genomics and other new science have not yet reached their full potential

(iii) mergers and other business arrangements have decreased candidates

(iv) easy targets are the focus as chronic diseases are harder to study

(v) failure rates have not improved

(vi) rapidly escalating costs and complexity decreases willingness/ability to bring many candidates forward into the clinic

The bottom line, however, is that, despite tremendous financial incentive, the current traditional R&D process has failed to deliver significant new drugs in the last few years. The dried-up pipeline has led to a patent cliff in which many branded drugs have gone off patent with little in the pipeline to replace them. While the supply has diminished, the demand for new treatments has not.

Business experiments outside the traditional drug-development process have emerged to address this problem. They are taking advantage of the increased availability of information and increased abilities for communication and interaction that have occurred in the last few years. I will focus on one of these experiments, the Michael J. Fox Foundation for Parkinson’s Disease Research (MJFF), to illustrate.

The MJFF tries to open a bottleneck in the drug development process, the gap between basic research and translational research that converts scientific findings into potential cures for human diseases. Scientists tend to be a maverick lot, randomly looking under this stone and that for anything new and interesting—I can say this lovingly because I was a professional scientist for more than twenty years. The culture of testing new ideas to develop into cures is a more directed process that is driven by the need to help desperate patients. Many potentially valuable ideas do not make it across this cultural divide.

The MJFF attempts to shepherd ideas across this divide by forming teams of scientists and business people. Funding is provided to the scientists under the condition that they are subject to deadlines, milestones, and team-generated priorities. Unpromising ideas are pruned and priorities are reset periodically. The responsibility of the business people is to get the surviving ideas into the clinical-trial process. The MJFF also makes sure that the team gets the scientific tools they need to get the job done. These include biomarkers, pre-clinical models, and reagents. The foundation generates assistance and funding from the patient community. Finally, the MJFF attempts to break down scientific silos and increase communication in the scientific community by making introductions and sponsoring workshops.

Traditionally the directions of new-drug development have been driven by the pharmaceutical industry. We are now seeing that individuals through patient-driven organizations may be having an effect with MJFF being an example. It is difficult to tell from the MJFF website what has been the real impact of their efforts, but what is clear is that patient communities now have the tools to influence the supply of treatments in the healthcare market place. The new interconnectedness of the 21st century allows not just individuals, but complementary communities of basic scientists, patients, business people, and drug developers to interact to identify and remove traditional obstacles in the delivery of healthcare.

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