Rare Disease - Episode 12

Episode 12 - Recognizing the Strengths and Weaknesses of Current Clinical Practice Guidelines

As Patrick F. Fogarty, MD, briefly discusses the improvements in the length of time of which new factor 8 and factor 9 protein products extend treatment for hemophilia patients and stresses why it is important for clinicians to work together to ensure that their patients have access to these longer-acting agents, the panelists consider issues that should be addressed by managed care professionals and clinicians.

From an economic standpoint, Hugh Fatodu, RPh, MBA, explains that longer-acting agents lead to more product waste and states that a large amount of unused product sits inside the patient’s home. However, he believes that specialty pharmacies can help improve the amount of inventory that is provided to patients.

Maria Lopes, MD, MS, and Mr Fatodu explain that to improve the amount of protein product administered to the patient and to reduce the amount of product waste, it is key to determine how much of the drug is necessary to the patient and develop a fair balance plan.

Dr Lopes and Mr Fatodu explain that the end result of practicing fair balance should minimize waste by recommending lower dosage requirements, yet should ensure that patients do not have bleeding episodes.