Disruptive innovation in the biopharma industry

“Disruptive innovation” is a concept made popular by the business community that is starting to catch on in the biopharmaceutical industry. During the Partnering for Cures panel “Beyond business as usual: Disrupting the biopharma business model,” a distinguished group of panelists discussed both the problems with the current bio-pharma business model and possible disruptive solutions that would make the system more effective.

Moderator Gautam Jaggi of Ernst & Young’s Global Life Sciences Center started off the conversation by stating that as big pharma is experiencing ever increasing development times and costs, “The time has literally never been better to make changes to the R&D system through disruption.”

Tomasz Sablinski of Transparency Life Sciences and Celtic Therapeutics pointed out that it is not the people involved in R&D that are causing the problems, such as high failure rates and increasing costs, but something inherent in the playing field: “These people are smart as individuals, but there is something about the system that causes smart people to create poor results.”

All panelists agreed that biopharma companies need to figure out more quickly which targets will not work (also known as “fast fail”). As Stephen Marc Paul of Weill Cornell Medical College noted, “Last stage attrition is killing these companies. We need to figure out earlier in the process which drugs will not work so that we can create a pipeline of late-stage molecules with a higher probability of success.” 

The sharing of ideas and data is another theme that kept surfacing in the discussion. Representing patient advocacy groups, Kathy Giusti of the Multiple Myeloma Research Foundation stated that “Patients are investors too, and they can put pressure on academic centers to make their data public.” According to Giusti, this is just one example of how patient groups can be a disruptive force when their passion and sense of urgency is given a seat at the table.

Bernard Munos of InnoThink noted that sometimes “The translational challenges might be too big for one company alone; the science is just too tough. Companies can wear themselves out. Companies should try to work with their competitors and join forces.”

Ben Shapiro of PureTech Ventures believes that real disruption will come when big industry realizes that “Pharma’s role should be to invest in the best ideas out there” and that instead of doing their own internal research, pharma companies should be focused on stepping in where venture capital has left a hole and finding the best idea from the outside.

If biopharma companies would take a more nimble and innovative approach to finding and sharing the best ideas, both from inside and outside their company walls, we would see progress accelerate, panelists agreed.

Turning patients into partners

Bonnie Addario is a lung cancer survivor with an extensive family history of the disease. When she was diagnosed, she wanted to do something to help further the search for a cure. She thought she might hold some fundraisers and turn the money over to her oncologist. Instead, she formed two foundations that advocate, fundraise, and perform research.

That should be the modern model of a patient advocacy group, according to the Partnering for Cures panel discussion, “Powered by Patients.” The panel looked at the new breed of patient groups, weighed their importance in the process, and shared best practices of bringing patients into the R&D arena.

Patient advocacy groups are powered by the people who have the diseases or their family members, making them “wonderfully, positively motivating,” said National Institutes of Health Director Francis Collins. Families often get involved to expedite slow or nonexistent movement on disease research. Addario noted that when she was diagnosed, the survival rate for lung cancer hadn’t changed in 40 years. When Scott Johnson of the Myelin Repair Foundation was diagnosed with multiple sclerosis, he was told there would probably be a treatment within the next 25 years. But, 25 years later, there wasn’t.

Johnson looked into the medical research ecosystem and realized there was no cohesive manager of the research and translation process. For him, it made sense for a nonprofit to step into that role. “A nonprofit has more freedom to operate, fewer constraints,” he explained. Nonprofits can be the convener and manager of the process. His organization began by convening partners in academia, developing a research plan, and working closely with their own scientific team on research. The next step was to bring in pharmaceutical companies. Johnson said his organization does the work to validate and de-risk discoveries made in academia so they will be up to industry standards and will be able to be replicated. “We know we have to give them compelling enough data to get their interest,” he said of pharma companies.

Other stakeholders say the involvement of patient groups brings value to the research and translation process. Dennis Urbaniak of Sanofi reported that his company is changing the focus of how it runs its business to better serve communities in different disease areas. He said they want to listen to people who live with the diseases they treat and bring them into R&D by crowdsourcing questions on care to learn how patients actually want to be cared for.

“Most scientists welcome the opportunity to have that connection with what they are working on,” explained Collins. “They may have never been in the same room with someone who has the condition. It is enormously inspiring.”

N. Anthony Coles of Onyx Pharmaceuticals works with the Multiple Myeloma Research Foundation (MMRF) on a variety of projects, including leveraging MMRF’s clinical trials network to collaborate with patients and get information to them about trials, speeding up the enrollment process.

Onyx also invites patients to visit the company two or three times a year, “so we don’t ever lose sight of who we work for,” Coles said. “The most successful patient engagement groups are led by someone with personal experience,” he said, adding the hope that everyone who touches healthcare decisions has the kind of “transformational experience” through meeting patients he had in dealing with his son’s illness.

Patients want to contribute, stated Addario, but they don’t feel valued. “Until we invite them to our tables, to our board rooms, to the places where we are making decisions about their lives, they won’t feel valued,” she said.

She encouraged stakeholders to view patients in a new way. “We have to start to look at patients as someone who has the solution within their body. The biomarkers are there. All the keys to open the door are there in the patient,” she said.

The Patient-Centered Outcomes Research Institute was created as a provision of the Affordable Care Act to look further downstream in the translational pipeline, said Collins, and to figure out what works in the real world and in the minds of patients. “We want to collect information in a way that captures the patient experience and compares it across studies,” he said.

Lastly, Collins’ wish list included open access of information. “We can all go faster if we have the information in front of us, if we aren’t wasting our time doing experiments that already failed,” he said. He is encouraged by the strong movement to ensure that research publications are available to those who need them, and he is optimistic about the amount of clinical trial information available on clinicaltrials.gov.

Patient groups bring value to the ecosystem that can’t be ignored. To conclude the panel, moderator Susan Dentzer of Health Affairs summed up the panelists’ final sentiments: “If you are not bringing patients into your organization, you’re not just a dinosaur, you’re extinct.”