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indiscriminant use of tools, technologies and plat- forms in the DDD process was not the panacea for lackluster or stagnant productivity. We suggested that a more focused and integrated implementa- tion of such TTPs should result in a significantly improved output of therapeutic drug products2. Alas, we were wrong in our optimistic pro-
nouncements! In 1938 the USA Food, Drug and Cosmetic Act was enacted and heralded the advent of the ‘New Drug Application’ (NDA) and the ‘New Molecular Entity’ (NME) descriptors. These pivotal designations along with ‘NDAs filed’ and ‘NMEs approved’ have subsequently served as pro- ductivity indicators of the pharmaceutical industry. We have noted previously, that since the 1950s there have been perceptible downward trends of NDA filings as well as NME approvals by the FDA2. This downward spiral continued during the period 1997-2007 (Figure 1), even when there was a concerted effort by pharmaceutical companies to utilise more TTPs in the DDD process. The single year exception for productivity was in 2004, when both novel drugs approved and therapeutic drug filings increased. This was, coincidentally the year that the FDA introduced the new designation of Biologic License Applications (BLAs) and New Therapeutic Biologics (NTBs). After we wrote our article predicting the positive impact of technolo- gies on the DDD process2, NDA/BLA filings and NME/NTB approvals did not change significantly from 2008-17 (Figure 1), with the exception of 2017. Thus our pronouncement that the strategic utilisation of TTPs alone should improve therapeu- tic drug product output really did not materialise. In the past, third-party service providers to phar-
maceutical companies have developed such TTPs. Pharmaceutical companies then attempted to incorporate these TTPs in-house, but with limited success as discussed above. More recently there has been some re-evaluation of TTP incorporation and concomitant use in the DDD process. A small num- ber of companies such as CombinatoRx (see below) attempted to create their own in-house, proprietary,
integrated platform-DDD (PD3)
approach. However, there was significant criticism of such efforts. It was suggested that most small pharma/biopharma companies did not have the bandwidth to carry out such an approach, since it would lead to dilution of effort, poor tactical and strategic decision-making and a lack of focus. More recently, a number of companies have not only ignored such criticism, but also forged ahead with an innovative, new model. In this article we discuss the issues facing such companies, and how to implement in-house PD3 efforts to populate
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individual company drug pipeline. We discuss the trials, tribulations and successes of the PD3 com- pany Moderna Therapeutics, to highlight issues associated with this approach.
Platform technologies and Drug Discovery and Development The words tool, technology and platform are often used interchangeably. However, in the biotech and DDD sectors a single tool is a component item (think hammer on a carpenter’s tool-belt), which when combined together constitute a technology (think carpenter’s tool-belt). A combination of technologies, when applied in a cohesive manner, can be labelled as a platform (think the carpenter him/herself complete with tool-belt, safety glasses, mode of transportation etc). In this article we use such relationships to provide a general perspective on what constitutes a platform, whether it is tech- nological, biological, molecular or digital in nature.
Problems of individual technology and platform implementation The original efforts by pharmaceutical companies to integrate TTPs into the DDD process were com- promised by flawed implementation strategies. Pharmaceutical companies would attempt to bring such TTPs in-house from third-party development companies. However, most pharmaceutical man- agement teams responsible for such decisions were often isolated and unaware of the complexities associated with technology development and implementation cycles. The global pipeline of TTPs was a complicated mishmash of ‘products’ at vari- ous stages of developmental maturity, and the rush to acquire them was often fraught with internal pressures and misunderstandings. This often resulted in the consideration and adoption of TTPs that were traversing various parts of the Technology Development Cycle2. The Technology Hype Cycle (THC) compound-
ed the flawed decision-making process of which TTP to adopt2,3. The THC describes the enthusi- asm that often greets new technologies and the subsequent disenchantment that follows. It con- sists of five stages: i) Technology trigger: product launch produces excitement; ii) Peak of inflated expectations: a ‘frenzy of publicity’ generates over- enthusiastic assessment and unrealistic expecta- tions; iii) Trough of disillusionment: expectations have not been met and advocates plus most gener- al, interested parties abandon the technology/plat- form; iv) Slope of enlightenment: some businesses continue to evaluate and develop the technology/
Drug Discovery World Fall 2018
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