The development, production and sale of drugs in the United States is a complex landscape that involves intellectual property and stringent federal regulations.
But according to scientists at Colorado State University, the status quo of the US pharmaceutical market may soon change its mind. This is partly due to a growing community of do-it-yourself biohackers who are disrupting business as usual for drug discovery, development and distribution.
A piece of perspective of September 13th in Trends in biotechnology frames these emerging problems and predicts how the US pharmaceutical industry and regulatory environment will have to change in response. The authors of the document are Jean Peccoud, the Chair of Abell in Synthetic Biology in the Department of Chemical and Biological Engineering; Jenna Gallegos, postdoctoral researcher at the Department of Chemical and Biological Engineering; and collaborators of Bio-Link Australia, Woodrow Wilson International Center for Scholars and Boston University School of Public Health. Peccoud is also a member of the faculty of the CSU School of Biomedical Engineering.
While the prices of many drugs continue to rise, new models of funding, research and development of drugs have emerged as part of what the authors call a "new ecosystem of innovation". In particular, this ecosystem involves the production of drugs on smaller and more personalized scales.
"Key drugs are still too expensive," Peccoud said. "People do not have access to them, so what if they can do it on their own?" What do we do about it? "
The authors use the Open Insulin Project based in California as a case study on how the bio-do-it-yourself movement could shape the future of medicine. Founded in 2015, the creators of the project are seeking to increase competition in the insulin market by developing and releasing an open source protocol for the production of unpatented insulin.
Why does the Open Insulin Project exist in the first place? Insulin is 100 years old, but remains prohibitive for many patients, with some uninsured patients paying up to $ 400 per month for this life-saving drug. People are angry and, in some cases, people die from lack of access to insulin at affordable prices.
The US regulatory system today favors existing producers of biological drugs such as insulin. Biologists are drugs made by a living organism, as opposed to small molecule drugs, such as aspirin, which are chemically synthesized. When producing unpatented small molecule drugs, a competitive company only has to demonstrate that it has made pure aspirin. But biological drugs are more difficult to control for quality; so competing companies have to do costly human trials - not worth the price of $ 250 million if they can not even ask for a patent to recover their investments.
While the structure of human insulin is no longer protected by patent, the handful of large insulin-producing companies continues to market "insulin analogues" that are supposedly better varieties of normal insulin. The authors point out that the medical benefits of these new versions of insulin are questionable.
This is where biohackers come in. If the Open Insulin Project succeeds in developing a "home-made" insulin protocol, it should avoid violating the patented insulin production methods. But biohackers producing insulin for personal use should not worry about patent infringement, even if security considerations are certainly at stake.
"It would be prudent for regulators to involve patients and innovators in community bio-labs to design an adaptive supervision that promotes an ethos of accountability," say the authors.
The real challenge for the biohackers of the Open Insulin Project, if they wish to allow competing pharmaceutical companies to sell insulin without a patent, will be regulated, the authors say. The success of the project will be severely limited by the cost of regulatory approvals, which include demonstration of biological consistency, safety and possibly effectiveness.
If these new models of insulin production are successful, they will depend on alternative approval processes, the authors say. If the biosimilarity of biohacked insulin could be confirmed without clinical studies, the cost of development would decrease significantly. This could be achieved through a small-scale and decentralized production. And technology already exists; microbioreactors that are able to produce small doses of organic products have been developed in university laboratories.
"In short, the status quo must change," Gallegos said. "We can not face problems if we do not recognize them".