The next generation of therapies built upon the science of stem cells and genetics will require new types of partnerships. If there is promise that these technologies can redefine how we understand and treat disease, there is already a certainty that the alliances and partnerships required to bring these therapies to patients are also beyond traditional models for commercialization.
Clearly, these are not pills in a box sold off the shelf to millions of people, says Brock Reeve, Executive Director, Harvard Stem Cell Institute. Instead these therapies involve complex treatments that rely upon platforms and individualized approaches requiring multiple different skill sets than those found in many traditional product development and marketing teams.
Brock Reeve, Executive Director, Harvard Stem Cell Institute
At BioPharm America™ in Boston on Sept 22, 2014, Reeve will lead a panel discussion around the topic, “What type of partnerships will lead to successful advanced therapy and regenerative medicine programs?” Panelists include Eric Halioua, Co-Founder and CEO of Promethera Biosciences, and Frederick Jones, Director at Broadview Ventures.
In the traditional route to commercialization for biotech-pharma, a deal begins with a small company developing science from academia to the point where a larger company steps in to buy the property.
“One of the things that is happening in our space, particularly because of the nature of the science, is that there are multiple parties involved that are playing a larger role than they do in the traditional transaction space,” said Reeve. Because gene-based or cell-based therapies target specific patient populations or are more complex to bring into the clinic, disease foundations, clinicians and hospitals treating those populations are often much closer to the user and the product than are the traditional biotech companies.
As a result, biopharma companies, the academic community and disease foundations are exploring different organizational and business models to help answer the question of how their different areas of interest intersect in the role of accelerating drug discovery and development and how they can work together more creatively.
“Nonprofit foundations are no longer simply writing grants to academic researchers. They are intimately involved at all stages of the discovery as well as the development process,” Reeve said.
“In addition, there are the patients themselves who assemble, sometimes through the disease foundations, sometimes through organizing as an interest group with other patients with the same disease and sharing information about scientific studies or current treatments or clinical trials. The organization PatientsLikeMe is a very good example of people who are very active in the treatment of their own disease. They are so current on the state of the science that they will know the informal results of studies before the sponsoring organizations do or ask if they can enroll in a given clinical trial before the trial is even started,” he said.
The power of patient groups in shaping the landscape for pharma companies was demonstrated in the case of drugs for HIV/AIDS where it was the patient population that pushed to make drugs available at accessible prices for a greater number of people, said Reeve. In orphan diseases, foundations become actively involved not only with academics but with companies and with the FDA, about what the regulatory process should be for a new therapeutic, what the clinical trial process might look like and what it might be worth to that population.
“They can pave the way, helping to set up clinical networks and accelerate the whole clinical and regulatory process,” he said.
Meanwhile, pharma companies are aggressively trying to figure out how to work differently with early-stage science. Reeve points to the Johnson & Johnson Innovation Centers. A similar strategy is being pursued by Pfizer with its Centers for Therapeutic Innovation, while other big pharma companies have announced new early-stage discovery programs or are beefing up corporate venture groups. Academic institutions have also introduced new approaches to break down interdisciplinary barriers and to move science farther down the development path, by forming groups such as Harvard University has done with the Harvard Stem Cell Institute, the Broad Institute, and the Wyss Institute.
New partnerships will also be required for the delivery of cell or gene-based therapies. Even though many refer to allogeneic products as “off-the-shelf” they are not as “off-the-shelf” as typical pharmaceutical products. Deciding what to keep in-house and what to outsource is a challenge and many firms rely closely on third party manufacturers. Shipping and supply chain logistics are also complex with many stringent requirements to ensure product viability.
In the case of autologous therapy, where cells are taken from an individual patient and, with or without processing, are injected or placed back into the same patient, the question arises of how local production needs to be. For example, does individual batch production need to occur in a remote facility or can it happen on site? In some cases, physicians prefer that the cells never leave the very floor or suite of the hospital where the patient is being treated so that they can be assured of the control over the process.
“You need to involve the clinicians to be able to do this at scale,” Reeve said. “Even in local, closed-loop systems there are multiple stages, each demanding a highly specialized expertise and know-how.”
The session at BioPharm America will not spell out a prescription for working in these new fields so much as provoke questions. For people who are focused on commercialization, the session will provide a chance to raise the curtain on next-generation therapies to explore trade-offs and see how the complexity plays out in different examples. For those BioPharm America participants who are deep in the science of this field, the session provides an opportunity to look up from the bench and hear the larger issues to be confronted as the science advances.
“The audience will hear about the roles of the different players and especially those that patient groups, hospitals and clinicians can play, not only in the delivery, but in the development of these therapies. Typically companies have not thought about what it takes to bring all these participants on board, including regulatory and reimbursement authorities, in order to be successful. We will share experiences and perspectives so that we can collectively improve our success rate in bringing products to market.”
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