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Challenges in the rare diseases’ space

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Rare diseases are a large group of diseases that affect a very small percentage of the population. They first became known as orphan, because pharma companies were quite uninterested in “adopting” them. Approximately 7,000 rare diseases have been identified until today, with 1 in 17 people being affected at some point during their lives. In the past few years, there has been a great number of advances in the rare disease space; however, a huge gap remains, as only a small 10% of those have a recognized treatment.

 

High development costs

It has been quite challenging for companies to cover the huge costs that come along with the drug development process of a potential treatment for a rare disease. A near-sighted solution to this problem is for developed drugs to have high price tags; however, raising the price of the medications will not only create a huge burden on patients but will make the treatments practically unavailable for the few who need them.

 

Challenging clinical trials

There are certain challenges in conducting clinical trials for rare diseases, starting with patient enrollment. There are not enough patients (by definition!) who meet the inclusion and exclusion criteria and are willing and capable to participate until the end of a trial in this space. It has not been uncommon for companies to conduct a separate trial for a few patients, or even only one, who could not participate in the main trial due to geographical reasons.

The clinical trial design for a potential rare disease drug is another difficult process for pharma. The companies struggle to design trials that could meet their goals, without this having an effect on patient enrollment. There are also certain challenges related to the trial data, as the rare disease patient populations are usually quite diverse in their clinical presentations and histories, something which puts an extra strain on ensuring the quality of the results.

 

Potential solutions to challenges in the rare diseases’ space

 

What the future holds

Science evolves rapidly, with more and more targeted therapies under development, covering a wide range of genetic rare diseases; many of these treatments will soon appear on the market. By 2024, the rare disease market is predicted to take more than 18% of prescription sales. 

As disease awareness is also growing, more patients are going to be diagnosed, broadening the eligible patient population for these treatments. Despite the aforementioned important challenges, it seems that pharma still sees opportunities in the rare disease space, and luckily for patients, keeps pursuing them rather enthusiastically.

#rarediseases #rarediseasechallenge #orphandiseases #rarediseaseawareness

 

Sources

https://www.nature.com/articles/s41436-019-0616-9 

https://www.abpi.org.uk/new-medicines/rare-diseases-facing-the-three-challenges/#c0c7ec25 

https://pharmaphorum.com/r-d/views-analysis-r-d/biggest-challenges-rare-disease/ 

https://www.ema.europa.eu/en/human-regulatory/overview/orphan-designation-overview

http://www.pharmatimes.com/web_exclusives/The_rare_disease_challenge_1323505 

https://www.iconplc.com/insights/blog/2019/10/24/4-challenges-to-successful-rare-disease-drug-development/ 

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