Sickle cell disease (SCD) is a hereditary blood disorder that, in the United States, primarily affects people of African descent. SCD is associated with pain, which is often excruciating, and life-threatening complications.
Current guidelines from the National Heart, Lung, and Blood Institute recommend opioids for pain management. Concerns about addiction and fear of being stigmatized as drug abusers by some healthcare providers are among the reasons the sickle cell community expressed a strong desire to limit opioid use. Most use complementary medicine techniques to reduce the need for these drugs. During Tiers I and II, a question that focused on the use of alternative techniques plus pain medication versus opioids alone to manage pain consistently rose to the top of our list of concerns. Concerns about physical health and difficulties communicating with healthcare providers were also of great import. Those who had participated in a chronic disease self-management program spoke highly of its benefit in this regard.
During Tier III, we plan to further refine our questions, and ultimately design a scientifically robust comparative effectiveness research (CER) study proposal. We will accomplish this by continuing to conduct systematic literature reviews and increasing research/clinical partner involvement. Increased participation from these partners is critical to inform the design of the proposed study and to identify barriers to its implementation in a real-world clinical setting. We will continue to engage the patient community through meetings, presentations, etc., to educate them on CER and PCORI methodology and seek their input on question revisions and study design.
*All proposed projects, including requested budgets and project periods, are approved subject to a programmatic and budget review by PCORI staff and the negotiation of a formal award contract.