Patient Registries in Orphan Drug Research
An orphan disease is defined as a condition that affects less than 200,000 people all over the world. Due to its limited presence, it is often not feasible nor economically viable to develop orphan drugs without government assistance. However, these diseases do exist and patient require medication and treatment for these diseases. To do that, the scope of patient registries in orphan drug research should be explored to make productive advances even with these types of restraints.
Challenges in Orphan Drug Research
Orphan drugs are therapies that are developed specifically to cater to rare diseases that affect just 6-8% of the world’s population. Orphan drug development has been an obstacle in the medical field due to a number of reasons. These include the low prevalence of the disease, the severity of the disease, a small population of patients, heterogeneity in the patient population, challenges in patient recruitment and limited repository of knowledge about the natural history of the disease.
While these roadblocks do exist, there needs to be a way to successfully navigate around them as orphan drug research and development is still a very important need that must be addressed. There are approximately 5000 to 8000 rare diseases, and the bigger problem is that the diversity of the diseases is vast. So the recourse required to develope this treatments is similar to the effort that must be taken for ailments that affect hundreds of thousands of patients.
Role of Patient Registries in Orphan Drug Research
All the factors listed above hamper the research and development of orphan drugs. However, rare patient disease registries can be a useful tool in understanding and monitoring the course of the disease and in providing information that can then be used in clinical trial design.
There is immense potential for patent registries in orphan drug research and development in that they can provide great insight into the natural history of the disease as well as the variability of the patient population. A rare patient registry is a useful tool when scarce information is available, which is a significant impediment in orphan drug development.
In addition, a rare patient registry helps to provide a database of the previous information that can help inform decisions about sample sizes for new new trials. Since the budget and profitability of rare disease drugs may already be low, this could help in sample size calculations for new clinical trials.
However, for the patient registry to be useful for this purpose, the focus needs to be on collecting the right kind of information so that the appropriate outcomes measures can be corrected right from the beginning of the patient registry program. In recent years, several rare disease global networks have been established that support drug development and technological advancements that have led to tremendous improvements in orphan drug development.
The Right Patient Registry Software Solution
To bring about successful results, public and private funding should be directed towards the establishment of rare disease registries monitored by healthcare professionals and patients. These registries would help in working towards treating and potentially curing rare diseases. The right patient registry software solution will help overcome the obstacles faced by orphan drug development for more productive results.
