Increasingly, the development of new pharmaceuticals utilize genetic information to stratify patient subpopulations and/or predict efficacy and adverse events – the development paradigm of personalized medicine. A recent report by Thomas Reuters (“Spotlight On … Personalized Medicine, A Pharma Matters Report,” the “Report”) summarizes the technical, regulatory and economic challenges the pharmaceutical industry faces as they move from the traditional drug development model to a personalized approach. Historically, the development of new molecular entities rely on developing drugs with annual sales over $1.0 billion. In contrast, personalized medicine will develop drugs and therapies targeted to smaller patient populations with significantly reduced annual sales. The Report also underscores the benefits of personalized medicine and highlights progress the pharmaceutical industry has made toward providing targeted therapies.
Benefits and Progress in Personalized Medicine
- Elucidating Disease Complexity: Personalized medicine depends on the analysis and characterization of large data sets of genetic information. The Report notes that the knowledge developed through this research will impact future research and development by improving the systematic characterization of disease mechanisms and pathway cascades that are vital to directing the discovery and development of new treatments.
- Identifying Novel Targets: Prior to the genomic revolution, only 500 drug targets existed to which the majority of drugs are directed. Sequencing and characterization of the human genome has identified over 22,000 drug targets for the development of novel therapies.
- Moderating Research & Development Costs And Timelines: Relying on the Boston Consulting Group, the Report notes that nearly $300 million can be saved by pharmaceutical companies by incorporating personalized medicine technologies into their R & D process. As an example, a major pharmaceutical company was reported to save approximately $9.5 million by incorporating predictive genomic markers into a redesigned $10 million clinical trial.
- Project Prioritization: Integration of the tenets of personalized medicine can improve management of drug development pipelines by focusing resources into rapid development of drug candidates with the best potential for success. For example, the integration of molecular-based data with bioinformatics and data mining can facilitate the pipeline decision-making process by providing increased information about the pharmacological, proof-of-concept, and toxicity profile of a potential drug or therapy, thereby allowing resources to be directed to compounds with higher probability of success.
- Buttressing Regulatory Submissions: Clinical studies that are tied to genomics can provide improved and efficient regulatory assessment of drug candidates.
- Commercial Viability of Personalized Therapies: A major disincentive to developing personalized therapies is the perceived lower return on investment that targeted drugs will provide because of smaller patient populations and therefore lower sales. However, the Report indicates that personalized therapies that are effective with minimal side effects are likely to drive repeated prescribing and patient compliance, favoring premium pricing and faster adoption thereby increasing sales and return on investment.
Personalized Medicine Today
The Report lists that over 50 drugs having a predetermined genetic marker are in development. Quoting a study conducted by the Tufts Center for the Study of Drug Development, the Report notes that more than 90 percent of pharmaceutical companies now utilize at least some genomic-derived targets in their drug discovery program.
In addition, the Report lists several notable collaborative efforts to develop personalized therapies, but cautioned that it is unclear from the available information if the development of a targeted therapy drove the deals. However, the Report notes an emerging trend to re-purpose fully-launched therapies as personalized products to cut development costs and improve efficiency.