No one doubts that the drug industry’s traditional model for developing new cures is badly broken. Fewer exciting new medicines are reaching patients these days, even as spending on research and development has risen and blockbuster drugs that have long been the backbone of pharmaceutical profits have lost their patent protection. A widening gap divides the discovery of promising new laboratory compounds from the ability to turn them into innovative therapies. A similar gap separates recent scientific gains in understanding genes from the creation of new drugs that use this knowledge to fight disease.
Meanwhile, new ways of finding cures more effectively and delivering them to patients faster have been springing up to meet these challenges. These innovative models provide alternatives to the industry’s traditional way of doing things — which has been to keep most research in-house and jealously guard research data, compounds and other intellectual property. The new approaches are more collaborative as researchers and clinicians from nonprofits, universities, private industry and public agencies pool their resources to create new therapies.
These developments mark the start of what could become an industry-wide transformation of the war against disease. In our special report we examine key aspects of this transformation.
Companies are turning to new methods of developing drugs as blockbusters lose patent protection. These models include partnerships with universities and the sharing of once closely guarded warehouses of compounds. A key focus of the research is "translational medicine" — whose goal is to replace traditional one-size-fits-all drugs with personalized treatments for patients with specific genetic profiles.
A decade after pharmaceutical companies took heat for their reluctance to make HIV/AIDS drugs widely available to impoverished African nations, the industry has changed its approach on pricing and access to drugs. Now, advocates for social responsibility in global health are focusing on how companies decide which drugs they will develop and how they manage operations in the developing world. New approaches include sharing patented compounds with companies developing treatments for tropical diseases, and rewarding companies that develop treatments for neglected Third World diseases.
Regulators must walk a fine line between providing access to new, cutting-edge therapies and protecting the public from drugs that might be dangerous. The U.S. Food and Drug Administration, the world’s largest medical regulatory agency, aims to speed up the process of reviewing new drugs but still faces criticism. Global guidelines for drug applications can help smooth over differences, while frequent communication between drug companies and regulators can help resolve issues in the best interests of the public.
Philanthropies and nonprofits are creating new models for drug development that cut against the grain of traditional for-profit drug discovery. Success stories range from bringing a meningitis vaccine to sub-Saharan Africa in record time to accelerating the start of clinical trials for a promising new cancer treatment. Open-source research is a key part of new models, and public and private projects are under way as well. Such initiatives may serve as templates for future drug development.