Gus Gardner, senior vice president, Epidemiology, HEOR and patient insights at Optum®, shares his perspectives on how research as a care option is poised to change the health care landscape.
Before delving into the various benefits of research as a care option, it’s a good idea to take a look at the current health care landscape and types of patient populations that are particularly in need of expanded options for care.
Aging population:
Around 72 million Americans will be over the age of 65 by 2030, and they will have an expectation of living 20 or more years beyond that age.1 For this demographic, cancer is fast approaching heart disease as a leading cause of death (27.7% heart disease; 22.1% cancer).1
These elderly individuals will be demanding treatment options beyond the usual and customary, such as the opportunity to participate in clinical trials as a care option. The goal is not only to extend life, but to improve quality of life and functional health as long as possible.
Rare diseases:
In the United States, a rare disease is a condition that affects fewer than 200,000 people.2
According to the National Institutes of Health (NIH), there are more than 7,000 rare diseases affecting between 25 million and 30 million Americans. (~1 in 10).2 Only about 5 percent of these conditions have treatment options of any kind, except for symptom management.3
These populations are often willing to try investigational and off-label treatments to mitigate their symptoms.
Terminal illnesses:
A terminal illness is considered an irreversible condition that will, in the near future, result in a state from which a person is unlikely to recover. Some of these terminal illnesses include cancer, multiple organ failures or massive heart attacks.4
It is estimated that more than 1 million Americans die of illnesses that have treatments in development every year, yet fewer than 3 percent gain access to clinical trials. Only 1,200 individuals a year are granted compassionate use exceptions.5
A growing number of these patients and their providers are asking that they be considered for access to investigational treatments, regardless of where they live.
Understanding the across-the-board benefits
For patients, participating in research as a care option means having the ability to participate in clinical trials from a local medical practice setting, where they can receive their regular care.
Being part of a clinical trial could also mean access to no-cost investigational treatments and potential reimbursement for expenses associated in trial participation.
For providers, it offers the benefit of expanding trial access to their real-world patient populations, enabling the community practice setting to become an essential investigational site.
This means keeping their patients “at home” rather than having them go to regional medical centers or university research hospitals.
For payers, research as a care option offers the potential for improved patient outcomes and more efficient delivery of care. It has the potential to favorably shape the cost curve for payers as well.
And for life sciences sponsors, it offers quicker recruitment and more streamlined data collection. This may ultimately help increase speed to market over competitors.
Understanding how research as a care option can change the health care landscape
There are five key ways research as a care option is poised to change the way we approach not only clinical trials, but patient care overall. They include:
1. Access to the newest therapies
As health care in America continues to move in the direction of patient-centered care, patients will expect more than usual and customary physician preference for care, especially when it comes to treatment options for end-of-life interventions.
2. Making the health care system work better for everyone
This expanded access is important to everyone, not just those in the life sciences industries. This is another deliberate step toward achieving the “Triple Aim” of better outcomes, delivered efficiently and at a lower cost.
3. Compression of the treatment discovery cycle
Under legacy drug discovery models, it can take years to navigate the current linear drug discovery process. Even finding enough participants to power a study can take months to years.
Today, however, Optum has the ability to analyze data from our distributed “network of networks” and build a cohort sufficient to power a study. This includes finding patients and investigators, matched to study inclusion and exclusion criteria — all from a laptop computer.
4. Reduction in regulatory restraints
Although the FDA is vigorously supporting this approach, no guidance has yet been published. What we expect to see is incremental movement in the direction of pRCTs (pragmatic randomized controlled trials) to a fully evolved research model where essentially all evidence for regulatory approvals will be done in near-real time using real-world evidence.
5. Localization of research options
The localization phenomenon that takes research from the regional medical center and university research hospital setting back into the community health care system will benefit everyone in the health care ecosystem — most importantly the patients. (Provided these patients meet eligibility requirements.)
References:
- Centers for Disease Control and Prevention. The State of Aging and Health in America 2013. Atlanta, GA: Centers for Disease Control and Prevention, US Dept of Health and Human Services; 2013. Accessed 8/17/18.
- National Institutes of Health. FAQs about rare diseases. Accessed 8/20/18.
- Rare Disease Day. Accessed 7/23/18.
- American Cancer Society. Frequently asked questions: What is a terminal condition? Accessed 8/20/18.
- Right To Try Act. FAQs. Accessed 7/23/18.
- Gottlieb S. Statement from FDA Commissioner Scott Gottlieb, M.D., on the signing of the Right to Try Act. May 30, 2018.
