Improving Patient Access-Rapid Approval and Patient Assistance Programs

Antoni Ribas, MD, PhD: I think for the increased number of breakthrough therapies, we’re getting better at developing drugs and targeting the right things in the cancer and the immune system. There are so many new agents being labeled with breakthrough activity because early on in their clinical development, they have evidence of activity in a place of critical unmet medical need. So, I think it’s a good thing that the FDA is allowing these studies to focus on that unmet medical need and bring a new agent to the clinic, to the FDA, and to the American people as soon as possible.

We’re losing a lot by not treating people and having therapies that are not working when we know there’s a therapy in clinical development that could work for those patients. It should not be that patients have to come only to UCLA or an academic center to be treated. The majority of patients are not treated in our centers. And those won’t only get access to breakthrough therapies if they’re approved. So, I applaud the FDA for this program. It does have its shortcomings. The approvals come earlier with less data, and then we have to go a little bit backwards on trying to see what are the true benefits of that agent and how does it compare to other prior agents. The majority of breakthrough designations are based on single arm studies, but then there’s always a randomized study that’s being planned, because the final approval in the United States and the approval in many other regulatory agencies around the world would require a randomized study. I think, eventually, we will get all of the information. It’s just that with breakthrough designation, it gets to patients earlier.

The question of clinical trial endpoints for immunotherapies has been something that’s been debated a lot, because sometimes we don’t have an objective response. There may still be benefit, because the tumor goes through a process that doesn’t qualify in a subjective response or may go through a period of initial progression followed by a response. Changing the RECIST criteria to have confirmation of progression—what people call IR, or an immune related RECIST criteria—is a good way to do this, because it allows to recapture a patient who had initial progression followed by a response. I think we have to ask our therapies to work. By work, it means that the patient, who had the tumor that was growing, had the tumor stop growing and then, a long time period after, the tumor is under control. There’s no endpoint for that, but that would be a durable response. I think those kind of effects should be captured in clinical trials and should be brought to the agencies.

In my clinic, we enroll all of the patients, who are on standard-of-care immunotherapies, in the programs that the companies have set up to help with approvals and out-of-pocket cost. These are very expensive agents, and having a percentage as co-pay would be limiting for many patients, that would decrease the number of patients that get agents that have been demonstrated to benefit in that indication. We don’t want that. The two approved from Bristol-Myers and from Merck have good programs that enroll the patients, but I haven’t heard of patients coming back and saying, “The out-of-pocket costs as co-pay are too expensive.” So, I know they’ve been very efficient with those programs to help patients get the treatments.

I work in California where there’s a whole bunch of programs, and it’s hard to track any single one. I think every one of my patients seems to have a different approval process and a different way of getting access to a drug. But, the patient assistant programs from Merck and Bristol-Myers are really helping patients get the treatments.