Rare diseases often go unnoticed, but affect millions of people every year. The problem is that developing treatments for such diseases takes a long time, including years of investment and research before they can get a breakthrough.
What is Orphan Disease Research?
Orphan diseases are medical conditions that affect very few people, usually fewer than 200 thousand people a year in the US, which is also the reason why there’s little to no research on these diseases. A lot of pharmaceutical companies do not invest a lot of time and energy in this, as not a lot of people are affected by this. Orphan disease research focuses on understanding these rare conditions and developing treatments and medications specifically for them, also known as orphan drugs. Governments all around the world provide incentives like tax benefits or faster approvals for companies to invest in this space.
Why Does it Matter?
Even though each disease affects a small group of people, the combined effect is detrimental. There are about 7000 rare diseases around the world, and about 400 million people who are affected by orphan diseases. About 95% of these diseases don’t have cures, which, for a lot of families and patients, means that there are a lot of uncertainties and a lack of hope. Orphan disease research gives them a glimmer of hope and the reassurance that not all is lost if they’re afflicted with a disease like that.
Role of Technology and Innovation
Over the past few years, technology has transformed how researchers approach rare diseases. Tools like gene sequencing, artificial intelligence, and digital health platforms help scientists identify the core problem faster and create personalised therapy solutions. Companies like Allucent help bring these innovations to life and provide the expertise and flexibility needed to enhance rare disease studies.
The future of Orphan Disease Research
The next few years are looking pretty hopeful for the orphan disease community. A lot more investment, better technology, and more global collaboration combined will lead to accelerated progress. Personalized medicine is also going to be a key part of the healthcare industry in general, where the treatment plan is tailored to each patient’s genetic makeup. We can also expect faster clinical trials through more digital and decentralised platforms, global data sharing to help with identifying the patient and what their diagnosis is, and more partnerships between small-scale biotech firms and CROs to help facilitate smarter and faster studies. Governments will most likely increase the amount of incentives to encourage companies to invest in rare disease research groups, with advocacy groups continuing to raise awareness and increase funding for these types of conditions.
Orphan disease research isn’t just about finding cures, it’s about giving those families who’ve lost hope something to look forward to. When science and technology come together, the future of orphan disease research looks better than ever.
Lynn Martelli is an editor at Readability. She received her MFA in Creative Writing from Antioch University and has worked as an editor for over 10 years. Lynn has edited a wide variety of books, including fiction, non-fiction, memoirs, and more. In her free time, Lynn enjoys reading, writing, and spending time with her family and friends.
