According to a story from globenewswire.com, the biopharmaceutical company Orphazyme has officially endorsed a set of guidelines that outline how rare disease patient advocacy organizations and drug companies should interact and work together during the process of developing new treatments. These two actors have to potential to share a mutually beneficial relationship that can lead to the development of more effective therapies for rare disease.
Orphazyme has chosen to orient its developmental focus towards life-threatening and debilitating rare diseases. So far, much of the company’s development has been targeted at a group of rare, genetic disorders called lysosomal storage diseases. The company’s leading drug candidate, called arimoclomol, is being designed to treat four different rare diseases: inclusion body myositis, Niemann-Pick disease type C, Gaucher disease, and amyotrophic lateral sclerosis.
The endorsement of these new guidelines is not exactly a surprise, as the company has worked closely with patient advocacy groups during the development of its products. The company has had a close relationship with Niemann-Pick UK, for example. Orphayzyme and NPUK have worked together for nearly a decade.
“The knowledge and experience shared by these groups have been instrumental in guiding our path.” – Anders Hinsby, CEO of Orphazyme
These guidelines were first published in the Orphanet Journal of Rare Diseases earlier this year. The guidelines highlight four different areas of opportunity where patient groups and drug companies intersect.
- For patient advocacy groups, this means finding companies that are interested in developing therapies that are relevant to the group. The rare disease drug field is still a limited one, and compared to most other field of development, rare disease treatments are a high risk, high cost project that does not appeal to all developers.
- Once this has been achieved, engagement between patients and the biopharmaceutical company is the next step in the process. Feedback from patients is facilitated by the organization and is ideally done in a group setting as opposed to on an individual basis. This allows for a diverse array of experiences and feedback for the company.
- Another area in which patient advocacy groups can help drug developers is through financial support of research and development costs, which can often be steep when developing rare disease therapies. This must be done carefully to allow the patient advocacy group to maintain autonomy.
- Support and communications during clinical trials is also essential, as patient groups can help determine how clinical trials are designed and get in contact with interested patients who would be interested in participating.
You can check out the full guidelines here.