Improving Treatments for Rare Disease Patients

Before 1983, there were few treatments for rare diseases. Various obstacles stood in the way, including little representation for patients, few incentives, and a lack of resources. Fortunately, the Orphan Drug Act was passed in 1983, offering incentives to pharmaceutical and biotechnology companies to develop rare disease treatments. While there have been improvements since then, organizations are still working to improve the treatment landscape for rare disease patients.

What’s Changed

Since the introduction of the Orphan Drug Act, there has been massive growth in the number of rare disease drugs approved. The incentives that the act offers are a large part of this: grants, tax breaks, market exclusivity, and more. They have led to the approval of 373 drugs for 468 designations as of 2014, which can be compared to only 38 drugs in 1983.

America is not the only country who has introduced legislation to spur rare disease research and treatment development. In fact, clinical trials that focus on rare disease have increased by 88% over the past decade in Europe due to legislative changes.

While legislation has played a large role in growing and improving drug development for rare diseases, it is not the only factor. New knowledge on genomics has allowed medical professionals to make tremendous improvements in treatments, especially because 80% of rare diseases stem from a genetic cause. Scientists have been doing innovative and hard work to see these improvements.

Looking Forward

Academic institutions, along with large pharmaceutical companies, play a large role in the innovation of the treatment process. While smaller companies often try to participate in this process, they face challenges like a lack of resources and experience. As rare disease research often requires a lot of money and knowledge, it becomes clear that smaller companies can have a difficult time.

PharmaMedic Consultancy (PMC) aims to help these companies. They support their efforts to help them enter the rare disease field. One of their major services is bridging the gap between stakeholders, both scientific and commercial, and creating relationships with patients, regulators, medical professionals, and opinion leaders. Essentially, they take care of the medical affairs so that these companies can focus on the research itself.

Improvements in scientific knowledge, developments in technology, and legislative changes have allowed for more rare disease research and treatments. Hopefully, PMC and other companies will lead to even more.

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