Keynote: Genetic Therapies Will Explode, Better Access to Data Will Drive Value

June 2022 Vol 13, No 6

With a much better understanding of the biology of cancer and other diseases, genetic medicine is entering “a whole new ballgame,” said Scott Gottlieb, MD, former director of the FDA, in a keynote address.

Over the past 5 years, there have been 20 FDA approvals of novel genetic medicine technologies, and this is just the beginning, he said, as 5% of phase 3 clinical trials in progress are testing such technologies. “We are in the early innings,” he said.

Genetic medicine will be transformed much in the same way that monoclonal antibodies made their way from the cancer arena to indications in primary care (eg, hypercholesterolemia), said Dr Gottlieb.

In the next couple of decades, genetic therapies “are going to become pretty mainstream therapies for more routine conditions,” he said. “Modifying DNA to cure disease, preventing proteins from being made in the first place, or enabling the production of proteins that were not there before to try to intervene in disease, [and] retrain the immune system to kill cells. Obviously, the big application is in oncology. Because all the proteins in our body need to be coded for by DNA and RNA, the genetic medicines can theoretically reach any protein.”

Gene therapies have higher odds of success than the industry average, with small interfering RNA-targeted therapies having a rate of success as high as 10 times the average.

“By targeting the genetic features of disease, you have more certainty up front that you’re going to have a more certain outcome on the back end,” said Dr Gottlieb. Novel treatment targets are also increasing, from about 1300 in 2012 to about 1600 in 2020, owing to a better understanding of the biology of the disease.

Insurance coverage of these novel therapies will continue to be an issue, even when they deliver an enormous amount of value, he predicted. Unlike treatment of some other chronic diseases, delays in access to genetic therapies for intractable diseases can cause disability that may never be overcome.

The Future of Value-Based Payments

Seema Verma, MPH, former chief of the Centers for Medicare & Medicaid Services (CMS), continued the keynote session on the importance of value-based care.

“There are a lot of different flavors of value-based care,” she said. “To me, it means that providers have skin in the game or financial incentives around lowering costs and increasing quality, keeping patients healthy, and focusing on improving outcomes.”

When providers are taking risk, a lot of administrative requirements can be lifted to reduce provider burden, such as eliminating prior authorization requirements and allowing providers to have more freedom to use innovative technology and treatments to improve quality. It also provides strong incentives to address the social determinants of health, such as assistance with housing or transportation, which “may do more to keep a patient out of an ER than actually giving them a prescription,” said Ms Verma.

Value-based care can be used to focus on specific public health issues by adjusting quality metrics, such as the inclusion of health equity, she said.

New developments will support the transition to value- based care, with the key one being access to data, which will “benefit patients and providers in many incalculable ways,” she said. “New regulations require that patients have access to their electronic medical record, and the new rules make it clear that providers can’t deny access to patient records. It has to be given to them in an electronic format, and hospitals also have to proactively inform their patients’ other providers when there’s a hospital admission, discharge, or transfer. CMS payers, Medicaid Managed Care, Medicare Advantage, and all of the Exchange plans are required to give their patients access to all of their claims data.”

These developments have major implications for patient care and value-based care in particular, she said. Having comprehensive data, whether through claims data or medical records, provides a treasure trove of data with the potential to reduce redundancies, identify care gaps, and improve overall care. “Having a complete medical history is a huge step forward in efficiency,” she said. “The collective data provide a lot of information that could transfer care delivery beyond an individual patient transaction. With large-scale analytics and artificial intelligence, we can understand the triggers and common traits of patients with high costs and poor outcomes, and hopefully address them on the front end.”

Complete medical data can also accelerate the development of innovative drugs and treatments along with more personalized medical care. Going forward, ensuring interoperability across the entire health system, including public health, will be critical for enhancing disease surveillance as well as responding to any future pandemic.

Access to pricing data is also being enhanced. Insurance companies will have to provide their customers with price estimators based on the customer’s individual situation and will be required to publicly post their negotiated rates, including those for drugs, for all providers. “It’s going to take market innovators to figure out how to leverage this data tsunami to make an impact,” said Ms Verma. “It’ll be a challenge to bring the data together from all the different portals in 1 place to combine price and quality data to make it meaningful for patients and providers.”

More data should lead to more development in virtual care, with the resultant opportunities to lower costs and improve access and quality by providing more in-home care.

“Efforts around remote or in-home care are enhanced by a lot of the new technology that can monitor patient vitals, patient activity, medication adherence, and even new at-home diagnostic testing,” she said. “Collectively, these tools have the power to prevent emergency room visits, reduce hospitalizations and also post–acute care stays. The new technology will be transformative and may be able to help patients stay in their homes longer and avoid expensive long-term care. Certainly, telehealth and new data available through price transparency and interoperability are significant changes for our healthcare system and will support the transition to value.”

More development into value-based drug payment is expected. As with medical services, there is a movement toward tying payment to outcomes. “This is important for several reasons,” she said. “First, manufacturers are setting very high price tags…approaching a million dollars. Those manufacturers, in my view, need to have some skin in the game. Right now, they’re getting paid whether a drug works or not, but a value-based payment could tie their payment to many different types of outcomes. Value-based drug pricing could become especially important with the number of breakthrough drugs and devices being approved by the FDA, where data on efficacy are limited.”

The FDA has indicated that by 2025 it will be approving 10 to 20 cell and gene therapy products yearly. While these medicines are certain to be groundbreaking, the drugs will have little impact if few patients have access to them. Value-based drug reimbursement will not guarantee lower prices, she said, and may actually increase launch prices, “but it does offer more negotiating power to payers and providers that are taking risk and requires manufacturers to have more accountability,” said Ms Verma.

Value-based payment arrangements could provide payers an assurance of cost-effectiveness and allow them to consider lifting requirements for prior authorization or other forms of utilization management for high-cost medicines, thereby increasing access for patients and relieving burden on providers.

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Last modified: November 15, 2022

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