How Creative Licensing Can Improve Patient Access
By Tyler Menichiello, contributing editor
Despite all the advances being made in our industry, the cost of cell and gene therapies is still prohibitive to many. Take CSL Behring’s hemophilia B gene therapy, Hemgenix, for example, which costs $3.5 million per dose, making it the world’s most expensive drug. Not every healthcare system can support multi-million-dollar therapies for patients. In fact, even in markets like the U.S., insurance plans may struggle to cover these costs as the number of available therapies — and thus, eligible patients — grows. As life altering and lifesaving as they may be, these innovations mean nothing if they don’t reach the patients who need them.
Lowering the cost of these therapies is critical for improving accessibility and addressing health disparities — not only here in the U.S., but around the world. Two organizations committed to this mission, Caring Cross and the University of California’s Innovative Genomics Institute (IGI), worked together on IGI’s recently published white paper, Making Genetic Therapies Affordable And Accessible. One of the report’s key points is the role that technology transfer offices (TTO) play in improving the accessibility of gene therapies. TTOs, which manage universities’ intellectual property (IP) and licensing agreements, serve as academia’s gateway to commercialization. The report argues that TTOs can and should include provisions for accessibility in their licensing agreements.
To learn more about how TTOs can play a key role in making cell and gene therapies more accessible, I met with the scientific director of Caring Cross, Dr. Rimas Orentas, and director of IP at IGI, Dr. Susan Abrahamson. Joining us were members from University of California, Berkeley’s (UC Berkeley) TTO, the Office of IP And Industry Research Alliances (IPIRA), including assistant vice chancellor, Dr. Carol Mimura, and director of IP, Dr. Laleh Shayesteh.
Public-Private Partnerships: Doing Well By Doing Good
Therapies and platforms licensed from academia are often the result of publicly funded research (e.g., NIH grants) — “they didn’t come out of the companies first,” Orentas says. The mutual goal of UC Berkeley’s Socially Responsible Licensing Program (SRLP) and Caring Cross is the “equitable use of these technologies” that were developed, in part, through public funding. According to Mimura, this can be accomplished by devising public-private partnerships early on in the research collaboration stage — years before an invention has been developed — to bring innovations and inventions to the developing world. She says these collaborations are a “win-win” for all parties.
Mimura says companies that license and commercialize Berkeley technologies under its SRLP often employ a “dual-commercialization approach.” These companies make a profit selling licensed products in countries with patent rights where patients can pay to subsidize sales (either at-cost or given away for free) in under-developed target countries without patent rights. “They won’t make a profit in one licensed field-of-use, but they will in another,” she explains.
One example of a dual-commercialization approach she points to is Sanofi’s semisynthetic artemisinin, the low-cost antimalaria drug jointly developed by UC Berkeley, Amyris Biotechnologies, the Institute for OneWorld Health (iOWH), and Sanofi. Grant money from the Bill & Melinda Gates Foundation allowed non-profit iOWH to foster product development in a three-way partnership with UC Berkeley and for-profit Amyris. Amyris (spun out of UC Berkeley) received funding to develop this semisynthetic artemisinin (which they would not profit from) and the associated platform technology from which they could later commercialize with products outside of malaria treatment.
“We found a way to work with others to de-risk the technology over seven years so that when the partnership delivered a technical solution to Sanofi — a genetically modified yeast that makes semisynthetic artemisinin — they could then commercialize artemisinin for artemisinin-based combination malaria therapies (ACTs).” In this case, Mimura says, the goal of the partnership and the Bill and Melinda Gates Foundation was realized. Namely, low-cost ACT ingredients to treat malaria.
“Sanofi would not have licensed such an unproven research discovery directly from the university,” she explains. “The risk-to-reward analysis would have been too high. Knowing how many years of R&D would have been required to produce a product without any guarantee of success, Sanofi would have chosen other projects to invest in. However, receiving a de-risked product in a sublicense, even with price restrictions, was acceptable.”
Sanofi was playing the long game. “They can go back to these countries in sub-Saharan Africa and Asia to sell their profitable drugs later after increasing the health quotient of these countries by treating an endemic disease like malaria.”
This malaria case highlights the power of TTO research and licensing agreements in setting provisions for accessibility. Essentially, the agreement was “if you do this one project that will not make you any money and develop your platform, you can sell related products using the platform under the same license for a profit, and we’ll give you the malaria rights on a royalty-free basis,” Mimura explains. This approach of “doing well by doing good” is referred to as Creative Capitalism (termed by Bill Gates) — the combination of two (oppositional) driving forces in human nature: self-interest and altruism.
Equity Starts Upstream
The overarching message is clear: TTOs have the power to incentivize Creative Capitalism by crafting “strings-attached” research and licensing deals.
“We worked on a plan where if a licensee takes a compound and the compound or drug gets FDA clearance, they have to come back to us and provide a plan for affordable access to people in various countries, including those in the U.S. who cannot afford these medications,” Shayesteh explains. You can see an example of this affordable access provision on page nine of this sample licensing agreement. Shayesteh tells me IPIRA keeps its licensing templates online, and encourages any interested universities to reference them or reach out to IPIRA directly for more information.
The success of semisynthetic artemisinin and other cases from Berkeley shine bright as examples of mission-oriented development and mutually beneficial public-private partnerships. By establishing these kinds of provisions in research agreements and licensing deals, TTOs can play a significant role in bringing biotechnologies out of universities to underserved people. In this way, they can improve global accessibility to emerging therapies.
“I think we’re all sort of seeing the writing on the wall when we’re talking about $3 million, one-time treatments,” Abrahamson says. “I think something is going to happen here; I mean, it has to.” She says UC Berkeley is aiming to be a leader in this space and do what it can to lead the way, and she hopes other universities follow suit. If university TTOs can improve the part of the process they’re involved in — early technology licensing — “then hopefully, progress can follow.”