Thanks to a few bad actors and plenty of media bias, drug development and the pharmaceutical industry is often thought of by consumers as a cold, heartless industry that, rather than being patient-focused, puts profit and revenues ahead of treating and curing diseases effectively. While there certainly a few stories that support such claims, drug discovery and development is, at its fundamental levels, an earnest enterprise where the ultimate aim is to research, develop and commercialize therapies that effectively treat or cure a disease. There is no better example of this than the cystic fibrosis community, where academic research, drug developers, the media, and grass-roots organizations come together to create a powerful synergy that is fueling the next generation of CF therapies. According to a recent article in GEN, the cystic fibrosis research support model is giving rise to an emerging trend in philanthropic drug development.
Dr. Moharem El Gihani, author of the piece, notes that: “The importance of venture philanthropy can also be gauged by its ability to fill the funding gap in areas of research that have hitherto been deemed too risky (from a technical or business case perspective) for pharmaceutical companies to invest in. A notable example being the Cystic Fibrosis Foundation contributing $75 million towards the early stage development of Kalydeco, Vertex Pharmaceuticals’ drug for the treatment of cystic fibrosis.”
BioNews Texas has highlighted in a recent article the major resources being poured into the successful development of Kalydeco, which, in combination with another drug, could substantially improve quality of life and outcomes for cystic fibrosis patients. Kalydeco is currently in trials, with Vertex planning on publishing much-anticipated results this summer.
The fact that a well-established, well-supported organization like the Cystic Fibrosis Foundation has backed Vertex in their endeavors to find a viable therapy for CF should not only be a ringing endorsement to the cystic fibrosis community that the drug developer is on the right track and working in the best interest of patients, it should also point to the fact that, in spite of the fact that there is a tendency to demonize the pharma industry, the public’ health’s greatest hope for therapies and cures lie with the companies who are seeking to profit from developing these novel therapies. A case in point is Gilead’s new Hepatitis C treatment, which is being heralded as a true cure for the disease. At present, there is a debate over the drug’s affordability. In the end, however, the discussion among insurance companies and government entities will not be whether or not the Hepatitis C cure can be afforded, but rather how it can be afforded.
In contrast, cystic fibrosis will most likely never have this same problem — the patient population is far too small for any high-priced cystic fibrosis therapy to be a major public health concern. What is necessary, however, is that a productive relationship continues to exist between drug developers and the rest of a disease community. If this trust is broken, then the much-needed support for funding a cure for a disease like cystic fibrosis could be in jeopardy.