There are those who say that, even after proving to be a gold mine, orphan drug development may not yet be totally understood by big pharmaceutical companies. That’s mainly because of the necessary strategic shift – I’d say an organic shift though – to a patient-centric focus, which makes the differences between the traditional drug and the orphan drug business models so vast.
That’s the opinion of biotechs who have been navigating the path of orphan drug development quite promisingly, like Shire and BioMarin. They are firm in stating that big pharma does not have the necessary mindset to deploy the level of urgency and innovation, aside from the degree of risk, to develop rare disease therapies successfully and continuously.
But then, let’s look at Novartis and what they are doing in the space. They are supposed to maintain the #1 ranking in orphan drug sales by 2018, with not less than 5 among the 30 best-selling orphan drugs in the market worldwide, according to EvaluatePharma. How would Shire and BioMarin explain that? Is that all about dealing with the challenge of social work and proximity with the patient population or can it be tackled with a bold scientific and procedural approach?
When speaking with John Orloff, Novartis’ Chief Medical Officer, and listening to his previous presentations at the World Orphan Drug Congress USA, I realized that Novartis has an organic and extended indication approach to orphan drug development, which is structurally different from what other big pharma like Pfizer and GSK are doing with a totally dedicated orphan drugs business unit. Besides, the Swiss drug maker has a strong commitment and comprehensive strategy towards gene therapy and supply chain, which enhances the way new first-in-class therapies for rare diseases are developed. Add to that the big effort – here agreeing that it’s not easy for big pharma to incorporate the patient centricity aspect to the development process – of successfully planning and budgeting clinical trials, excelling in study design and endpoint definition, deploying a patient – not site – recruitment approach and mainly making EVERY data point count.
Here’s a great article I read about these different perspectives.