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A pharmaceutical industry focus on orphan drug development could lead to better treatment options for the smaller patient population.

By Anonymous on Aug 28, 2012

A recent Thomas Reuters study which investigated the value of orphan drugs from 1990 to 2030 stated that rare diseases have the potential to generate as much profit as mainstream drugs. With around 250 new rare diseases discovered every year, it is no surprise that this market is worth $637 million- only $1 million more than a matched control group of non-orphaned drugs. The report continues to state that this market is more economically viable for pharmaceutical companies as it has smaller and cheaper timelines for development, better government incentives and terms on exclusivity.

As well as the obvious increased profit for the pharmaceutical industry, a move towards orphan drug development could begin to meet the medical needs of the smaller patient population. About 40% of orphan drugs are used to treat cancer, the biggest rare disease category, in these small patient groups.

Developing one ‘blockbuster’ drug for cancer has been a struggle for the industry as cancer has always been prone to many genetic anomalies. With so many anomalies still unknown, patients with rarer cancer conditions have difficulties finding a treatment to suit their individual needs. Plus, a University of Rochester Medical study demonstrated that definitive studies to support the true effectiveness of various cancer therapies is still lacking. The Thomson Reuters study provides support for the pharmaceutical industry to invest more in R&D for these rarer diseases, so that these types of patients will be provided with better treatment options for their specific medical needs. 

In the UK, the 2012 National Cancer Patient Experience highlighted that although more cancer patients were receiving better treatment choices, it is not always the case for rarer cancer types. The Thomson Reuters research is encouraging to pharma companies considering looking into patients with rarer diseases and could ensure they receive the same consideration and services from the healthcare industry as the generic patient population. In addition, it could not only inspire better medication but also more diverse therapies as pharmaceutical companies look to develop holistic treatment programmes for patients who find it harder than others to maintain the normality of life after diagnosis.     

To conclude, the pharmaceutical industry can clearly profit from meeting unmet, rarer medical needs when developing orphan drugs. Following on from the Thomas Reuters research, companies should evaluate their current business models and consider adapting it to the changing healthcare landscape and the yearly growth of rarer diseases. Aswell as the payer sector, pharma would benefit from taking the time to create greater dialogue with patients and doctors- those with hands on experience of rarer diseases- so that their next new drug can meet as many individual needs as possible.