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The Future of Rare Disease Research

By Renee Breiten
On April 1, 2020

March was Rare Disease Month at Pharm-Olam. We started off by reviewing an article that was published in Nature (read it here). From there, we examined the field of rare disease research (click here). We went on to look at the specific drug classes and development platforms that researchers use in rare disease research. We covered small molecules (access it here), antibody therapy (available here), protein replacement therapies (read it here), oligonucleotide therapies (read it here), gene therapies (click here to read it), and drug repurposing (available here).

As the month of March comes to an end, we want to conclude our salute to rare disease with a look at the future of rare disease research.

 

The State of Rare Disease Research

Right now, the state of rare disease research is very different depending on which therapeutic area the specific condition concerns. More than 33% of orphan drug approvals center on indications within oncology. At the same time, fewer than 5% of rare diseases have an approved treatment. One issue is that researching cures for specific conditions may not always have a direct payoff for sponsor organizations creating the treatments. A relatively small number of afflicted patients means that the market for any drug developed is also small.

That said, some of that is changing thanks to new technologies. As science begins to understand the molecular, genetic, or phenotypic presentations of different conditions, the pathway to developing treatments becomes much more direct. It is possible that several indications may positively be impacted by drugs that target the same pathophysiological mechanism. To this end, there are initiatives set up to help move those efforts along as well as build upon the successes of related therapies.

Efforts like the Rare Disease Registry (RaDaR) are good examples. By consolidating the patterns of drug responses, researchers can avoid duplicate efforts and build on earlier studies. Together, they save time and money as well as develop meaningful treatments. However, the right researchers need to be involved.

 

Rare Disease Research at Pharm-Olam

In order to study rare diseases with this effect, researchers need to have a firm and complete understanding of the underlying mechanisms involved. Rare disease is a core focus at Pharm-Olam and one we know well. Roughly 88% of our PMs and 92% of CRAs have experience in rare diseases. We leverage this knowledge to improve our knowledge base. To this end, our study execution repository for Rare Disease is continually evolving. We capture lessons learned from our clinical studies to leverage insights and develop tools to improve rare disease study execution and decision making. 

 

Moving Forward in Rare Disease

March may be almost over and the world has other concerns. The recent coronavirus outbreak, commonly known as COVID-19, has consumed media in the biopharma industry and beyond. While the pandemic needs to be a priority as people around the world work to flatten the curve, the seriousness of rare diseases and the importance of structured research in the field continues. 

While this article concludes our dedicated coverage to the topic, Pharm-Olam is committed to excellence in rare disease research. If rare disease research is on your radar, connect with us. Whether you follow our social media efforts or start a conversation about our rare disease CRO services, Pharm-Olam is helping create a healthier world by going the distance in rare disease research.

Sources

  • Tambuyzer, E., Vandendriessche, B., Austin, C.P. et al. Publisher Correction: Therapies for rare diseases: therapeutic modalities, progress and challenges ahead. Nat Rev Drug Discov (2020). https://doi.org/10.1038/s41573-019-0059-7

Tags: Rare & Orphan Disease, Pharm-Olam Insights

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