Rising Cost of Drug Development isn’t Slowing Down Researchers Working to Uncover Novel Therapies for Rare Diseases

Every day the scientific community makes progress in the study of rare disease. Research in the field has advanced exponentially in recent years and we’re starting to see more treatments which help reverse the effects of diseases and more cures. This is in contrast to many past developments which have simply treated symptoms. It’s incredible. Except for one thing- the cost of these new novel therapies. The more advanced the treatment, the more expensive it tends to be. Someone has to deal with this cost and for most patients, paying the price out-of-pocket is an unfathomable consideration.

For instance, Spinraza, a treatment for spinal muscular atrophy costs 750,000 dollars the first year and 375,000 dollars each year after. Thankfully, 24 of 28 states in the EU have agreed to cover the cost of this therapy for patients.

In total, about 10% of Europe’s GDP is spent on healthcare costs. However, orphan drug costs equate to only 15% of healthcare expenditures. For the pharmaceutical agencies themselves, that percentage is between 3 and 5.

The World Orphan Drug Congress

The issue is that governments are struggling to keep up with the cost of the promising new therapies being laid on the table.

At the World Orphan Drug Congress held in Barcelona, Spain last November, 500 pharmaceutical companies, patient advocates, and researchers came together to discuss the issue.

Ségolene Aymé, founder of Orphanet, explained that the cost of FDA approved cancer drugs at the time of their approval has moved from just 50 dollars in 1965 to 50,000 dollars in 2016. For orphan drugs, this number is even greater.

How nations are dealing with the costs varies greatly. In Belgium, 100% of drugs used for treating 11 of the most common rare diseases are reimbursed for patients. In France, 91% are covered, and in Sweden, just a mere 25% are reimbursed.

Despite the issue of payment, researchers aren’t slowing down in their work. Ultimately, they understand there are real lives at stake. They’ve made it clear their goal is to continue to produce tangible results for patients.

“It just comes down to getting the regulators and payers on board and up to speed. We need to continue that vein of partnership to keep up with the pace of scientific innovation.”

You can read more about this issue and the future of rare disease drug development here.


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