Orphan Drugs

When Prometic started research and development into therapeutics for rare and orphan diseases in 1989 there was a clear mission to respond to the unmet public health need. Around the same time, the FDA and NIH had been pushing initiatives for the pharmaceutical industry to address the lack of drugs for small patient populations which ultimately lead to the Orphan Drug Act (ODA) and the Rare Disease Act (RDA). Concurrently, patients suffering from rare diseases were becoming representatives for a new patient advocacy movement which has now become a global Initiative. These advocacy groups have been imperative in facilitating clinical trials, generating awareness and ultimately increasing the likelihood of a correct diagnosis. Strategic examples of patient groups if we’re allowed?

Since then, research into rare disease has rapidly evolved. Advances in technology and science have made it more cost effective and faster to find associations between genetic mutations and rare diseases. As a result, the pharmaceutical industry has shifted considerably in favor of including rare diseases as a drug discovery strategy. With this increased attention, more and more rare diseases have been identified, and it seems as a collective they are not so rare at all, affecting an estimated 25 million people in the USA and 30 million in the EU. To date, around 6,000 rare diseases have been identified. In the U.S., they are defined as conditions that affect fewer than 200,000 Americans and in the E.U., disorders occurring in fewer than one in 2,000 Europeans fall into this category.

In general the pharmaceutical industry has seen an increase in the price tag associated with drug R&D over the last decade however despite higher costs, rare diseases continue to have huge investments due to the initiatives that the ODA can provide. Before the Orphan Drug Act had passed there were as few as 10 drugs recorded entering the market for rare diseases but by 2017 over 450 products treating 668 orphan indications had been approved by the FDA. The EU shortly followed with a similar policy in 2000 which increased incentives for pharmaceutical companies to focus on orphan diseases such as market exclusivity, reduced regulatory fees and subsidies for clinical trials.

In today’s pharmaceutical market there is still a lot of scope for R&D into rare and orphan diseases as patients and the families of those affected by extremely rare diseases still do not have any available or sustainable treatments. Prometic is working to bridge this gap with a rich pipeline of therapeutics to address this need while collaborating with patient advocacy groups to ensure that the industry is able to best serve the individuals.