Tag Archive for: rare disease

An FDA advisory committee voted Wednesday that Ipsen’s fibrodysplasia ossificans progressive candidate palovarotene was effective and the benefits of the drug outweighed its risks.

The pharma industry is facing an opportunity to overcome rare and orphan disease patient resistance and build long-term relationships through support programs specifically designed to best serve these populations and improve patient outcomes.

“There could be around 10 total regulatory decisions this year on cell and gene therapies, including up to five decisions on gene therapies for rare genetic diseases alone,” said Stephen Majors, director of public affairs at the Alliance for Regenerative Medicine.

Chinook has a leading compound designed to treat IgA Nephropathy, or IgAN, a rare disease that can lead to kidney failure in young adults which has attracted a range of developers and is already the target of drug candidate developed by Novartis.

As the number of rare disease drugs on the market exponentially increases, marketers will have to ramp up efforts to persuade payers of the value of these often costly treatments.

Although the bulk of gene therapy research is still focused on rare disease, recent launches show that certain chronic conditions could be successfully treated with this technology.

Med Ad News spoke with Mike Czuba and Sharlene Jenner of AbelsonTaylor about their agency’s approach to getting the right message to the right patient in the rare disease space.

Without question, there has been significant progress in pinpointing treatments and accelerating rare disease diagnosis, largely due to the precision of data-driven commercial strategies and the speed of digital clinical trials.

Traditional pharmaceutical sales and marketing approaches will be inefficient when trying to reach such a small population. To maximize efficiency and effectiveness of commercial efforts, commercial leaders must take a creative approach to their targeting strategy.

When you hear the phrase “gene therapy,” what comes to mind? A revolutionary cure for an exotic disease? A risky experimental approach that messes with nature? Something in between?