Orphan Biosimilars
Interchangeables for the treatment of rare diseases
Rare diseases tremendously lack affordable treatment options
Orphan drugs have been neglected by the biosimilar industry for a long time
Although there is no commonly accepted definition of the term rare or orphan disease, it is generally understood as a life-threatening pathology with low prevalence for which no, or no satisfactory, treatment options are available. Low prevalence is rated individually in different countries and usually varies from 1-6 patients out of 10.000 residents. To date, about 7.000 rare diseases have been discovered, with up to 250 being additionally spotted every year. 1
World-wide, 350 mio people are estimated to suffer from a rare disease, including 25-30 mio US and 30 mio EU residents. 1, 2
Orphan drugs are (bio)pharmaceuticals specifically developed for the treatment, prevention, or diagnosis of rare diseases. Incentive structures implemented by industrial state governments paved the way for a clear trend towards the development of orphan medicines. This positive effect however comes along with serious impacts on the access to and affordability of orphan medicines:
The lack of alternative treatment technologies and absence of competition from (bio)generics imposes the risk of manufacturer monopolization, resulting in extraordinarily high price tags, and making orphan drugs some of the most expensive (bio)pharmaceuticals in industry.
With >60% biologics account for the majority of the global orphan drug market.
Patient cost-sharing programs in industrialized countries as well as insufficient reimbursement by the governments limit the access to orphan medicines. A developed orphan drug for example is officially unavailable for 10-30% of EU patients. 3 This development is likely to deteriorate considering increased financial pressure on national healthcare systems.
In emerging countries, especially those lacking specific orphan drug legislations, the majority of patients can neither access nor afford adequate orphan drug therapies due to insufficient market availability and unaffordable out-of-pocket costs.
At Valerius, we are positioned to fill the need for affordable orphan medicines by leading the development of orphan drug biosimilars.
1 National Institutes of Health, Frequently asked questions about rare diseases [Online]
2 World Health Organization, Priority Medicines for Europe and the World "A Public Health Approach to Innovation", Background Paper 6.19 Rare Diseases (March 2013)
3 Bignami F, Eurordis survey on orphan drugs availability in Europe (July 2007)