Pharma R&D Today
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Sources of Innovation for Drugs of the Future
Posted on June 6th, 2016 by Jeffrey Paul, PhD in Pharma R&D
Innovative drug targets are necessary if precision medicine is to become the norm. Collaboration will be the enabler in developing novel drug therapies: between academia, with its discovery engine; biotechnology, with its platform specialization; and pharma, with its resources and expertise.
Precision, personalized or targeted medicine denotes chosen therapies, based on an individual’s genetic, epigenetic and environmental factors, which deliver the right drug to the right patient at the right dose. Some futurists believe there could even be a money back guarantee if the individualized prescription fails to perform. Who will provide the expertise to discover novel therapies meeting the high hurdles set by regulatory and payers?
Academia, biotechnology and pharma companies, which each provide innovation and future collaborations via open innovation, will provide a much-needed infusion of new ideas. To allow or encourage open innovation, a fresh look at patents and intellectual property is also needed.
Academia is the engine of basic discovery in the health sciences. Academic research provides the fundamental understanding of molecular mechanisms and their relationship to physiological / pathophysiological processes leading to potential drug targets. Immunologic (proteins), small-molecules, genetic and epigenetic approaches are the source of new drug targets. Funding for academic research comes primarily from the NIH. In order to advance a career in academia, one must share information via publishing in peer-reviewed journals or else perish. Several academic institutions have established drug development institutes, which have the mission of discovery and generating lead drug compounds.
Biotechnology (including drug and diagnostic) companies are dedicated primarily to a single platform of R&D. In many cases, the platform was originally developed by an academician who chose to commercialize the discovery by opening a biotechnology company. As an example, Professor Greg Verdine of Harvard has formed a new biotech, WaVe, based on synthesizing therapeutic nucleotides. Pfizer has provided them with about $900 million as part of the collaboration. Other examples of a technology based on a single platform are Raindance and Regulus, which aim to develop genetic diagnostics and microRNA as therapeutics, respectively.
Biotechs are passionate and single-minded about their platforms. Their innovative technologies and lead compound(s) receive all the available resources necessary to move the compound forward to the next phase of development or external licensing. There is very little decision-making other than how to get the lead compound to the next phase of commercialization. If a platform does not succeed, the biotech will close and reinvent itself with a new platform and a new name.
Since we are discussing drugs for disease (i.e. drugs to help grandma), it should be stressed that the progression from an innovative idea to product commercialization is via validation and the demonstration of quality and reproducibility. Regulatory oversight is the nature of our drug development and diagnostic business. Nascent platforms are typically not quite ready for commercialization — therefore, biotechs must provide operational legs by scaling-up, optimizing processes and developing quality specifications. The unfortunate recent example of Theranos, a diagnostic start-up with an innovative technology, clearly illustrates the importance of the need for validation and quality measures, as regulatory inspections uncovered quality deficiencies at their centers.
Pharma companies have focused their innovation energies on improving speed/cost efficiencies, de-risking methods, developing novel clinical designs and strategic quantitative decision-making. Pharma also has vast chemical libraries and provides the expertise in medicinal chemistry required to synthesize small drugable compounds. However, with the use of therapeutic proteins and nucleotides, which interact with immune, genomic and transcriptomic systems, traditional drug synthesis may not be utilized. Pharma has been looking externally to academia and biotech companies for providing lead drug compounds. As biotechs are passionate about their platform and lead molecules, pharma is generally impartial or agnostic about their leads. Many pharma companies with large pipelines decide the fate of their compounds based on mathematical models to calculate the probability for meeting criteria.
Mergers and acquisitions (M&A) are also used by pharma to obtain drug candidates. To increase efficiencies, pharma has outsourced much of their clinical trial expertise to CROs. Christensen (of disruptive innovation fame) warns that too much outsourcing of key expert competencies leads to “dis-integration” and leaves the pharma company vulnerable and without a competitive, innovative edge in R&D.
The arena to develop complex future drugs will be created by the transparent collaboration and sharing of resources between academia, biotechnology and pharma. Open innovation, first introduced by Henry Chesbrough, describes how innovation occurs with the sharing of internal and external resources. Importantly, for open innovation to work, the concept of intellectual property needs to be re-defined. When one collaborates by working externally, competitive information and project control is liberalized. Developing a new drug or a novel bio-process is very complex, requiring multifunctional expertise, not as simple as keeping the secret Coca-Cola recipe in a secured vault. With open innovation, there is shared de-risking and transparency. Examples of open innovation are seen in precompetitive programs involving Lilly; and centers of excellence at Novartis; and Pfizer.
Finally, the NIH can play a very important role in promoting collaboration. From a recent Oracle blog discussing precision medicine: The NIH needs to embrace a little bit of Silicon Valley’s culture and operate, at least in part, like an incubator that fosters highly scalable businesses that validate and promote PM;
 Open Innovation: The New Imperative for Creating and Profiting from Technology; Henry Chesbrough; Harvard Business Press; 2006.
 The Innovator’s Prescription; Clayton M. Christensen; McGraw Hill; 2009
All opinions shared in this post are the author’s own.
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Jeffrey Paul, PhD
Principal at JPharm Consulting
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