Innovation in Clinical Trials to Meet Increased Patient, Provider and Payer Demands for Safe, Effective and Cost-Effective Personalized Therapies
Despite significant advances in medical treatments to reduce risk of major cardiovascular events such as cardiac death, nonfatal myocardial infarction, embolic stroke, unstable angina and the need for revascularization procedures, the number of Americans experiencing cardiovascular complications and death are on the rise.
Improved treatment modalities are being offset by an aging population, higher rates of obesity and diabetes, poor medical education and poor adherence to ever more complex treatment regimens.
The number and speed of new treatments development has slowed and there have been a number of late-stage development failures and set-backs, the most notable being the CETP class of agents, designed to increase the levels of HDL (good) cholesterol. The therapies that have been recently approved by health authorities are either considered very expensive (i.e. PCSK9 class of antibodies to lower LDL cholesterol) or have limited improvements in efficacy or both. As a consequence, the patient benefits of these treatments and adoption rates have been disappointing and resultant investment returns to shareholders, have not met expectations.
Change is needed in the manner in which innovation is defined, the speed and cost of identifying at-risk populations and the individualization of therapeutic intervention.
The use of “Real-World Evidence” (RWE) from large deidentified patient electronic medical records can help quantify at-risk populations and support determination of interventional study hypotheses and potential benefits. This can accelerate the development of novel agents, reduce the cost of these programs and support the cost-effectiveness of the resultant studies.
Similarly, RWE analyses can help better target therapies to populations most at need and most likely to benefit from the therapeutic intervention. This is particularly important as the complexity and cost of care in these patients continues to increase and is not on a sustainable path.
These observations will be highlighting using the “at risk” patient group with well-controlled LDL levels while on statin treatment, with high triglycerides, and other cardiovascular risk factors. Use of RWE and randomized, blinded placebo-controlled clinical studies together provide powerful research tools to determine the value, magnitude and benefits of the experimental treatment.
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