Orphan Drug Development Requires Unique Strategies

Posted Monday July 02, 2012 by Melissa

I recently read  a paper entitled Strategies for the Sustainable Development and Delivery of Drugs for Rare Diseases by Kristen Cardinal and Chris Meier of the London Business School. The paper won a competition created by GlaxoSmithKline called Rare Diseases competition: a sustainable model for the pharmaceutical industry.

from Strategies for the Sustainable Development and Delivery of Drugs for Rare Diseases by Kristen Cardinal & Chris Meier

I found some interesting analyses in the article and found it worthy of its role as the winner of the competition.

It described the four challenges faced by the pharmaceutical industry with respect to rare disease as:

1. The medical challenge: Due to the small number of patients, best practice for diagnosis and treatment is often difficult to establish – In some cases, physicians may be unaware that the disease even exists. For example, SPTCL (subcutaneous panniculitis-like T-cell lymphoma, a rare type of cancer) is frequently misdiagnosed as panniculitis (a skin disease) due to similar symptoms. As a result, individuals suffering from rare diseases are often treated incorrectly.
2. The scientific challenge: For any disease, both diagnosis and treatment require a clear understanding of how the illness ‘works’ – i.e. an insight into the physiological processes that underlie disease progression. However in the case of many rare diseases, the small number of available patients limits the scientific research of the condition. For example, identifying the genetic drivers of Costello Syndrome (a developmental disorder) took several decades mainly due to the small number of patients available for study. As a result, the biomedical basis of many rare diseases is poorly understood.
3. The clinical challenge: Before a new treatment or therapy can be sold on the market, its efficacy has to be demonstrated in clinical trials. To show the beneficial effects of a therapy in a statistically significant manner, testing of hundreds or thousands of patients is required. For many rare diseases, the number of available patients is not sufficient for such large-scale clinical trials. For example, StemCells Inc. (a Biotechnology company) recently discontinued clinical studies of a new treatment for Batten disease, a rare neurodegenerative disorder, due to an insufficient number of suitable patients.
4. The commercial challenge: Since most rare diseases have few patients, the commercial potential of medicines for these conditions is often limited. For example, some rare oncology indications affect only a few hundred patients, making the development of dedicated drugs commercially challenging.

Another interesting portion was the highlight of the importance for pharmaceutical companies to participate in the development of diagnostic products for the disease. This is in line with patient interest as well. If patients can be diagnosed earlier, they can receive therapy earlier and hopefully prevent irreversible damage from their condition. This also allows for revenues for the pharma company from their treatment product for patients who might otherwise not be diagnosed from traditional methods such as symptom aggregation.

Finally, it also discussed the importance of collaboration – collaboration between the pharma companies and academia, charities and other pharma companies. Collaboration was a topic discussed at length at the recent World Orphan Drug Congress. Specifically, patient populations would like to see common data sets and sharing for patient registries, also as a method incredibly useful for furthering research at faster pace – biomarkers, drug metabolism and pharmacokinetics (DMPK), absorption and distribution (ADME).

I encourage you to read the entire paper as I think it is helpful for the patient, patient organization, and pharma perspectives.

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