Standardization in Orphan Drug Development

Although new treatments for orphan diseases are widely discussed in the scientific and pharmaceutical industries, these treatments are difficult to successfully realize.

We have spoken about the reasons for this and what the scientific and medical community can do about it with experts from Maastricht University Medical Centre, Greenwood Genetic Centre,, Newron Pharmaceuticals and Parexel.

Rare Diseases Affect Multiple Organs

In patients suffering from a rare disease, it is often not only one system in the body that is affected, but multiple. This makes orphan diseases much harder to treat. Consequently, treating clinicians are forced to coordinate a variety of therapies, specialists and other medical services.

A Lack of Clinical Data

While patients and families struggle with these issues, scientists and doctors are confronted with a lack of clinical data surrounding orphan diseases Рmissing biomarkers and insufficiently robust data while scrutiny from regulators is increasing.

To complicate the situation even more, there is no standardized system across the EU for the assessment, approval and reimbursement of orphan drugs, meaning that certain treatments may have been approved and are reimbursed in one country, whereas in another country a patient may not have access to the treatment.  

Therefore, there is a pressing need for development of standardized methodologies and novel health economic models so that more and better therapies for rare diseases can be made available to patients.

Creating a Burden of Disease Report

Newron Pharmaceuticals and Parexel International together lead an initiative that represents the first practical attempt for a standardized methodology in order to provide the payers and
healthcare policy makers with the information they seek to make informed decisions.

In developing and conducting an International Burden of Disease study for a severe neurodegenerative orphan disease called Rett syndrome, they will compare and contrast physician and caregiver views of symptoms and their impact; identify satisfaction with current disease management, and characterize treatment patterns for the Rett patient population.

Watch our video on orphan diseases and listen to experts discuss the societal impact, the burden on patients, their families and communities and an opportunity to develop a common approach to rare drug development.

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