Since the Orphan Drug Act of 1983, over 1700 investigational therapies have entered the research pipeline.  But of those only 320 have been FDA approved for marketing.  The therapies don’t usually fail because they lack efficacy or because they are unsafe.  They fail because their sponsors cannot afford to maintain the manufacturing and clinical development programs through the arduous process of target-specific FDA approval.   Dozens, if not hundreds, of potentially life saving therapies sit on the shelves of biopharmaceutical companies because the costs to get them approved for the people they could save might exceed the revenues earned once commercialized.

The central problem faced by for-profit drug producers is that their strategies are limited to only those paths which ensure reasonable investment returns for their shareholders.   This narrows the scope of new drug development and imperils existing drug programs when changes in the profitably outlook come to surface.   Drug programs of shareholder-owned companies face questionable future revenue generation in the cases of rare diseases, patent expiration, information asymmetries, and - in the case of biologics- high operating costs.   And, although progress is being made to add transparency and consistency to the drug approval process, FDA investigatory requirements are often impossible to predict, and small companies routinely burn through their program budgets before their drugs can get to Phase 3 trial.

In the July 2005 issue of Health Affairs, Dr. Victoria Hale, founder of Institute for OneWorld Health, outlined her model for the role of non profit drug companies in addressing global health crises.   She cites the ability of non profits to garner intellectual property and other production resources at cost or cost plus, whereas for-profit suppliers would be loathe to collaborate with each other without near-prohibitive fee arrangements.   In the case of assuming a discontinued drug program, a non profit can partner with the patent-holding company and take advantage of the infrastructure, manufacturing protocols, and regulatory paperwork that is already completed.  Additionally, a nonprofit drug company can also act as a public-private partnership and bring multiple for-profit parties together into collaboration to expeditiously move toward drug production, without necessarily doing any of the development or production in-house.

Dr. Hale’s model cites several successful partnerships, including the Malaria Vaccine Initiative (MVI) collaboration with Glaxo SmithKline to develop a specific product, Medicines for Malaria Venture and GSK undertaking broad vector research together, and continuing work by the Global Alliance for TB Drug Development and the International AIDS Vaccine Initiative.

There is no reason to believe the essential parts of this model cannot work globally, for all diseases that may be unprofitable to address from a share-holder’s perspective.  For treating sufferers in both the developing world and in Western countries, for-profit drug producers face many of the same uncertainties, and the opportunities for non-profit solutions to needed drugs are significant.

Partnership for Compassionate Use Therapies is committed to the production of potentially helpful experimental drugs to treat sufferers of otherwise untreatable terminal illnesses.  Often these are orphan diseases that affect smaller populations than malaria and black fever.   Nevertheless, the horrible demise awaiting sufferers of motor neuron diseases and untreated carcinomas present a unique and compelling case to make new therapies available as soon as possible.