Due to the unique characteristics of rare diseases, developing medications for them might be difficult. The rarity of a condition makes drug development difficult. Due to a paucity of people, well-designed clinical trials to demonstrate efficacy can be difficult to conduct, and studies substantial enough to determine major hazards are nearly impossible to do. When no other options are available, patients may be ready to risk side effects in exchange for potential benefits, but the benefit-to-harm balance is difficult to quantify and may be unfavourable in some situations. Clinical pharmacology, a translational discipline, is a critical component of drug development, including therapeutic development for rare diseases. Health administrators have financial challenges when it comes to innovative pharmaceuticals, particularly orphan drugs. Individual rare disorders impact a small number of people, yet they collectively affect a significant portion of the population. They require our assistance.