Before he was chairman of Oyster Point Pharmaceuticals, Michael Ackermann, PhD, worked with a team developing an implantable device. It looked promising, but it was expensive. The team decided to abandon the program, realizing that it was no longer realistic to think the device would receive a high-dollar CPT code to cover the cost of the procedure.
“Where I think that’s really relevant today is that we need to remember we’re not developing for what the market is now, we’re developing for the market that we’re going to be launching into, and the market that we’re going to be competing in for years to come,” Dr. Ackermann said during the breakfast breakout session “Evolution of the Dry Eye Market” at OIS@AAO 2018.
That was one of the lessons for moving the dry eye space forward that Dr. Ackermann and other experts shared based on their experiences.
Caroline Blackie, OD, PhD, medical director, ocular surface disease at Johnson & Johnson Vision Care, said successful innovation can provide an opportunity to change the way medicine is practiced, to have a profound effect on patients’ lives.
In a previous position at TearScience, she saw that there was a significant lack of clarity over not only why meibomian gland dysfunction should be seen as important, but also even what it was. The company realized that it was extremely unlikely that doctors would physically manipulate the gland to evacuate stagnant content, for rehabilitation. So, the firm innovated a device that would perform that function.
What no one expected was that because the technology worked, a number of other device treatments now exist with an add-on manual expression of the gland. “So the base point,” she maintained, “is that when you innovate something that truly works, you have the opportunity to change the face of medicine.”
Christian Roesky, MD, PhD, managing director and CEO at Novaliq, said his group encountered resistance to its decision to try to redefine cyclosporine for dry eye disease. People didn’t believe its use could be improved after 10 years on the market, but eventually the treatment succeeded. “We need headwinds,” Dr. Roesky explained. “If we just follow trends, we will not explore something new.”
Clinical Trial Design
Saying clinical trial design is extremely critical in dry eye disease, session moderator Preeya Gupta, MD, of Duke University Eye Center, asked the panelists for key issues related to clinical trial design.
Dr. Blackie stated that clinical trial design, whether for a diagnostic or therapeutic product candidate, comes down to knowing three things:
- What you’re doing.
- Why you’re doing it.
- How to measure it.
She said the “why” is usually to try to elevate and potentially transform the patient experience. But for the “what” and “how,” there needs to be extreme clarity about what component or components of the ocular surface system are to be affected and perturbed.
“As we think about the dry eye patient and how we collect that patient, if we think about the system in general it becomes overwhelmingly complex,” Dr. Blackie continued. “We really need to titrate and drill down to what components of that system we’re looking to address so that we can home in on the appropriate metric.”
She also said it’s important to work closely with the appropriate regulatory body to be sure the metric is aligned with other validated metrics, to be set up for success when communicating the data.
Dr. Roesky believes it’s important to spend time and effort on Phase II clinical trials, and not just hunting endpoints or regulatory approval. “There’s so much that we don’t know,” he stressed. “So, we have a commitment that in all our trials we have exploratory – investigations, endpoints, devices. We need to learn.”
Dr. Ackermann agrees with giving appropriate time to Phase II, to truly understand how the product works, and the way that it impacts the disease, before moving on to Phase III. “Once you optimize the mechanism of action,” he said, “you’re at least going to do the best that you can do for that particular product, and then you can layer in all the other endpoints that maybe are more global and less driven by your business.”
Quinton Oswald, CEO of Notal Vision, added that for venture-backed companies, venture capitalists sometimes think that the chemistry, manufacturing, and controls phase can be done at the end. But that’s not the case, he said. A company should start early on with its supply chain because products can fail at the approval level due to lack of control. “Push very hard on your boards to allow you to invest in that specific area,” Oswald said.
For questions about this article, please contact Steve Lenier at Steve@healthegy.com.