Have you ever wondered how treatment is developed for the rarest of diseases, otherwise known as orphan diseases? Well, it can be a tricky process, but it’s possible! The drugs or medical devices used to treat rare ailments, which are defined as affecting less than 1 in 2,000 people in Europe or less than 200,000 individuals in the United States, are called orphan drugs. Successfully developing an orphan drug that helps those who don’t yet have an effective treatment for their disease can be very rewarding. Nonetheless, the process presents a few challenges. To find out more, keep reading.
The Pool of Possible Patients for Clinical Trials Is Very Small
To ensure a drug works properly, it needs to be tested on those afflicted with what the drug is seeking to treat. Since orphan diseases are extremely rare, this poses an immense problem when it comes to conducting clinical trials. Often times the patients for these drugs are widely spread out geographically and research teams belonging to different centres are involved. This ends up significantly hiking up costs as a result.
What can make this problem even trickier is that volunteers for clinical trials should be adults. However, among the top 350 rare diseases, 27 per cent of patients do not survive until their first birthday. Additionally, identical symptoms are required for proper comparative testing, but the progression of many orphan diseases can vary significantly from patient to patient, thus further narrowing down the possible pool of trial candidates.
Clinical Testing Is Already a Lengthy Process, and Orphan Drug Testing Can Take Even Longer
As those with clinical research training know, clinical trials are a lengthy process. The purpose of trials is to ascertain the effects of the drug and determine its safety. Typically, this is carried out in three phases with an additional post-marketing phase. In respective order, they assess safety and interaction with the human body, efficacy, comparison with other treatments or placebo, and long-term effects. Only one third of tested drugs might pass just the second phase alone.
On average, it takes ten years to get to the marketing phase of a new drug after the discovery of a novel molecule, and it may cost multiple tens of millions of dollars. As already noted, because a large portion of the testing is dependent on having a sufficient amount of trial patients, this process can become even lengthier if participants are difficult to find, as is the case with orphan drug testing.
Further Challenges Can Face Those with Clinical Research Training
Professionals with a clinical research diploma known that there are additional challenges to creating orphan drugs. Not much scientific research is published on rare diseases and as a result little is known about their natural history.
Additionally, a sponsor is required so that funding is available to develop a new drug. This is usually an institution or private financier that initiates the research, is responsible for it, and is subject to associated legal obligations. Since it is not expected that the high costs associated with developing an orphan drug will be recovered, finding a sponsor can be especially tricky.
For all these reasons, effective therapies are not available for more than 95 per cent of patients affected by orphan diseases. It’s a challenge that needs addressing, and could be an important area for improvement that you help tackle throughout your career in clinical research.
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