"Over the last few months, we have continued to execute our development strategy for Firdapse® (amifampridine phosphate) and further our mission to serve the LEMS and CMS patient communities. We were pleased by our recent receipt of a Special Protocol Assessment agreement with the FDA for the protocol design, clinical endpoints, and statistical analysis approach to be taken in our upcoming, second Phase 3 study evaluating Firdapse for the symptomatic treatment of LEMS. We believe that our receipt of the SPA provides us with a clearly defined development and regulatory pathway to complete the development of this product. We were also gratified to have been granted orphan drug designation for Firdapse for the treatment of myasthenia gravis. Finally, we have launched our new website to make it easier for patients to access information about obtaining Firdapse at no cost through our expanded access program, which is another step in our continuing efforts to serve LEMS and CMS patients."
The net result of ongoing cost pressures and a precipitous decline in R&D productivity will be an unprecedented decline in the share of the overall healthcare profit pool captured by the largest research-based pharmaceutical companies, while lower-margin sectors, like generic manufacturers, will increase their share. The critical question for these companies is how to evolve their business model in order to thrive in a world of shifting profit pools by focusing on outcomes rather than by simply generating more inputs—more products, more procedures and ultimately more cost to the payers.