Pharmacogenetic (PGx) testing involves the analysis of genes known to affect response to medications. The field has been projected as a leading application of personalized or precision medicine, bu...

Platelet-rich plasma (PRP) injections are often used for the treatment of knee osteoarthritis (OA), despite clinical value and cost-effectiveness not being definitely established. PRP injections are considered as a potential means of reducing pain and improving function in patients with knee OA, in the hope of delaying or avoiding the need for surgical intervention. Centers that offer PRP injections usually charge patients out of pocket and directly market services. Therefore, the purpose of this study was to quantify the current (1) prices and (2) marketed clinical efficacy of autologous PRP injections for knee OA. A prospective cross-sectional study was performed based on 286 centers identified in the United States offering PRP injections for knee OA. A total of 179 (73.4%) centers were successfully contacted via e-mail or phone, using a simulated 52-year-old male patient with knee OA. Scripted questions were asked by the simulated patient to determine the current marketed prices and clinical efficacy, either reported as “good results” or “symptomatic improvement,” claimed by each treating center. The mean price for a single unilateral knee same-day PRP injection was $714 with a standard deviation of $144 (95% confidence interval [CI]: $691–737, n = 153). The mean claim of clinical efficacy was 76% with a standard deviation of 11% (95% CI: 73.5–78.3%, n = 84). Out of the 84 clinics, 10 claimed “90 to 100% efficacy,” 27 claimed “80 to 90%,” 29 claimed “70 to 80%,” 9 claimed “60 to 70%,” 8 claimed “50 to 60%,” and 1 claimed “40 to 60%.” These findings provide a unique perspective on the PRP market for the treatment of knee OA that is valuable to physicians and health care providers in providing better education to patients on the associated costs and purported clinical benefits of PRP injections.

Background: Increased understanding of the molecular causes of disease has begun to fulfill the promise of precision medicine with the development of targeted drugs, particularly for serious diseases with unmet needs. The drug approval regulatory process is a critical component to the continued growth of precision medicine drugs and devices. To facilitate the development and approval process of drugs for serious unmet needs, four expedited approval programs have been developed in the US: priority review, accelerated approval, fast track, and breakthrough therapy programs. Methods: To determine if expedited approval programs are fulfilling the intended goals, we reviewed drug approvals by the US Food and Drug Administration (FDA) between 2011 and 2017 for new molecular entities (NMEs). Results: From 2011 through 2017, the FDA approved 250 NMEs, ranging from 27 approvals in 2013 to 46 in 2017. The NME approvals spanned 22 different disease classes; almost one-third of all NMEs were for oncology treatments. Conclusions: As these pathways are utilized more, additional legislative changes may be needed to re-align incentives to promote continued development of innovative drugs for serious unmet needs in a safe, efficacious, and affordable manner.