Preparing Patients and the Health System for Curative Therapies in the Pipeline

Curative therapies represent a conundrum for healthcare. On the one hand, there is the ability to cure patients of potentially life-threatening diseases or diseases that would require costly treatments for the duration of a patient’s life, but on the other hand, these therapies are very expensive and might require novel financing mechanisms that the US healthcare system is not equipped to handle.

During “Paying for Cures: Ensuring patient access and system sustainability,” a 1-day event in Washington, DC, panelists discussed how the system can catch up with the science by examining ways to pay for products that have high upfront costs with benefits that accrue over time.

In the first session of the day, speakers representing patients, providers, and a health system discussed the current situation and what high-cost cures could mean for both patients and the system.

Marc Boutin, JD, chief executive officer of the National Health Council, highlighted what a unique situation curative therapies put people in. The patient advocacy business model is more than 300 years old, and advocacy has always had a huge focus on finding a cure.

“And now, for the first time, we’re actually talking about cures, [which] upends the entire dynamic for people with chronic conditions,” he said.

There is a huge amount of excitement, but there is also caution, because patient advocacy groups recognize that patients with chronic and life-threatening diseases have been the targets of snake oil for centuries, Boutin explained. Even if the cures a true and real, patients are worried, now, about access. He noted that it used to be parents with a genetic disease worried about their children getting the disease and not having a treatment option, but now these patients worry that there is a cure but they won’t be able to get access for their children.

He also noted that there are 3 core things that patients are interested in for treatments: survival, how they feel, and their ability to function. The challenge is that patients weigh those things differently.

Anish Goel, PhD, who has lived with beta-thalassemia his whole life, added that the importance patients place on those 3 issues will also change a different points in their life. For Goel, a cure is important, but not crucial—he has responded well to treatment and can continue living his life if a cure never comes along. But he acknowledged that isn’t the case for all patients with beta-thalassemia. Some of them do not respond well, and they are holding out hope for a cure.

He added that how patients place importance on a cure can also impact clinical trials. For instance, there are patients who are responding well to the current treatment who will not sign up for a trial because “they don’t want to mess with a good thing.”

As a result, the evidence base for many curative therapies is small, because the clinical trials haven’t been very large, said Murray Ross, PhD, vice president at Kaiser Permanente. He also discussed the challenges with payment. Right now, his system has little engagement in pay-for-performance in this area. Some of the innovation that is happening today is a bridge to the next innovation, which might come just a few years from now. In pay-for-performance, the challenge becomes identifying the performance metric, which can be complex.

The panel was followed by Colin M. Young, PhD, director of drug development pipeline research at the Massachusetts Institute of Technology FoCUS (Financing and Reimbursement of Cures in the US) Project, who looked at the drug development pipeline for cell and gene therapies and what impact those therapies could have if they come to market. His group approached forecasting by looking at progress rates, success rates, eligible patient populations, adoption inhibitors, and pricing assumptions.

In hematological cancers, Young’s group forecasted that 20 to 25 cell or gene therapy products will be approved by 2030, mostly in acute and chronic leukemias, B-cell lymphomas, or multiple myeloma. They assumed a price point of $400,000 and approximately 22,000 to 28,000 patients treated annually (based on the number of relapsed or refractory patients out of the combined incidence of 150,000 patients). The annual direct cost would be between $9 billion and $11 billion.

In solid tumors, which hasn’t had any success to date, the group only forecasted 1-2 products would be approved by 2030. The number of eligible patients would vary depending on the cancer and the antigen targeted. For instance, epidermal growth factor receptor is expressed in 50,000 patients with non—small cell lung cancer compared with just 1500 patients with anal cancer. Still, the group estimated between 8000 and 12,000 patients would be treated annually by 2030 with an annual direct cost of $3 billion to $5 billion.

In gene therapies, the diseases addressed can be broken down into 3 categories: ultra orphan, orphan, and high-prevalence conditions. Young’s group forecasted 6 to 7 ophthalmology, 5 to 6 hematology, 3 to 4 neurology, and 2 to 3 metabolic drugs would be approved by 2030. In addition, they estimated 6 to 7 drugs in other conditions, such as hereditary angioedema or cardiomyopathy. So far, there has been just 1 success—Luxturna for inherited vision loss. The total eligible patients will be sensitive to the success of trials for higher prevalence conditions, but the group predicted 12,000 to 15,000 patients treated annually by 2030.

Ultimately, Young pointed out, there are a number of factors that his group cannot forecast, such as unpredictable changes in success and progress rates. Instead, when the group observes changes, it will have to do a new forecast. Some things driving changes include patient availability, evolution through shared knowledge, quantum leap events (ie, successes in high prevalence areas), and offsetting costs.

“We know a lot of these diseases have therapies right now which are expensive,” Young said. When they look at the cost of a hemophilia A gene therapy, it may be $1 million or even $2 million, but the cost of that one-time treatment means not having to pay $300,000 a year for the rest of the patient’s life.

Overall, his group is looking at 425 drugs in active trials for 214 diseases with 40 to 60 expected approvals by 2030. Those approved drugs would treat between 40,000 and 55,000 patients with an implied drug cost of $20 billion to $25 billion.