Big Pharma: Adopting Orphans and Genomics
Okay fine… Big pharma aren’t taking parentless children out of foster homes and raising them as their own. As with all headlines, it’s a bit of a double-entendre, but you may find it interesting nonetheless.
It may seem surprising (even counter-intuitive) to learn that big pharmaceutical companies are ramping up their R&D departments to find treatments and cures (orphan drugs) for rare diseases. These are conditions that affect fewer than 5 in 10,000 of the general population in the EU or fewer than 200,000 in the US. This just doesn’t seem economically viable, but pharmaceutical companies are jostling all over each other to get a piece of the action in rare diseases.
While the term ‘orphan’ drugs may not be too familiar, the term ‘blockbuster’ drugs might be more so. These are extremely popular drugs (e.g. Vioxx, Lipitor, Zoloft) that generate annual sales of at least $1 billion and are commonly used to treat common medical problems like high cholesterol, diabetes and cancer. These are dependent on extremely high volumes of prescription sales with Pfizer’s Lipitor (atorvastatin) still generating US $12 billion per year, being the best selling drug of all time. This has meant that Pfizer’s and many other pharmaceutical companies’ business models have exclusively relied on the profits a handful of blockbuster drugs. But things are changing.
The great ‘patent apocalypse’ is already upon us. Patents are expiring faster than they are being replaced, meaning generics from competitors are going to eat into the profits of big pharma, as the blockbuster drugs go off patent. They need to find proprietary technologies and new business models to create new revenue streams. An example of this is the shift in the pharmaceutical business model to concentrate increasingly on rare diseases and an increase in the development of orphan drugs.
Orphan drugs are defined as pharmaceutical agents developed to treat medical conditions which, because they are so rare, would not be commercially viable to produce without government assistance. This trend towards orphan drug development was initiated by the 1983 Orphan Drug Act, the orphan medicinal products regulation by the European Parliament, and other such laws internationally. These laws have provided incentives to rare disease drug developers including seven years of market exclusivity in the US, and ten years in Europe (which may be further extended for paediatric use); reduced or waived regulatory fees and tax credits. These have been very successful so far: the Food and Drug Administration has approved more than 500 drugs for rare diseases since the US law came into force, compared with fewer than ten such products in the decade before that.
This has meant orphan drug development has become not only a reality for big pharma but has become increasingly profitable. Compared to control companies, those that market orphan drugs are five times more profitable and have 10–15% higher market value. The global orphan drugs market is expected to reach US $176 billion by 2020, and account for 19% of total branded prescription drug sales.
While proving a new profitable business model, only around 600 orphan drugs have been approved, meaning the overwhelming majority of the 7000 classified rare diseases are still without any true treatment options. However, emerging science and technology is changing this.
Genomics is driving a new era of scientific discovery and drug development, especially for orphan drugs in rare diseases.
Studying the genetic profiles of patients helps scientists predict whether a particular process in the body is involved with a disease, allowing better targeted research. Harnessing genomic sequencing and genomics big data analysis will dramatically improve the success rate of discovering new medicines and accelerate orphan drug development, meaning new, potentially curative treatments for rare diseases becoming a reality.
This brings us to Genomes.io. We aim to supercharge rare disease research and pharmaceutical development by accelerating the process of bringing life-improving and potentially curative treatments to those rare disease patients that need it most. To do so, we generate secure and participant-owned genomic databases for rare disease patient organisations and connect these with researchers from academia and industry. However, in a more responsible and sustainable model that ensures:
1. Secure data storage
2. Privacy-preserving and transparent data sharing
3. Fully informed, repeat data access consent
4. Equitable compensation for participants
In this way, rare disease research and development can move further faster, without compromising the privacy and ownership rights of individuals’ most valuable piece of personal information.
If you represent a rare disease organisation, orphan drug R&D organisation, or are otherwise interested, please do get in touch at email@example.com. Alternatively, we’d love to hear from you through our Discord, Twitter, Facebook and Telegram!