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Anna Törner: ”Orphan Designation – the "petite robe noire" of drug development”

It is easy to cling to various regulatory incentives, like orphan designation, and other expedited pathways, without understanding what they truly mean or whether they are indeed right (or wrong) for the current project, Anna Törner writes in a column. 

Now I'm dipping my toes into French, which I'm not all too familiar with. The concept of the 'petite robe noir,' invented by Coco Chanel in the 20th century, boils down to having a versatile little black dress in your wardrobe that's almost always the perfect choice. This 'little black one' is the saving grace, helping us navigate through the chaos of an overflowing wardrobe that, during the hour of need, provides absolutely nothing valuable.

An Orphan Designation (OD) is the early confirmation by authorities that achieving 'Orphan Status' is possible at the time of approval. This confirmation typically garners significant interest, capturing the attention of both those immersed in the scientific aspects of the project and financiers. From a regulatory perspective OD provides the project with a distinct identity. Moreover, there exist numerous written guidelines that provide guidance and structure. Additionally, there is also the possibility of obtaining comprehensive regulatory advice, such as from COMP at EMA (1), at minimal or no cost.

The little black dress is rarely spot-on, but every now and then, it becomes the hidden centerpiece

So, what is the crux of the matter here? Well, OD is the possible promise of orphan drug status later, upon approval. An OD is based on limited data and an assumed significant benefit. To get your drug approved as an orphan drug, you must be able to demonstrate benefits related to efficacy, safety or patient management, a significant benefit. If you succeed here, you can expect exclusivity for at least 10 years and restrict other players from pursuing the same indication. Essentially, an OD merely demands some promising data and thoughtful reasoning, saying it is likely that the drug can demonstrate these benefits in future clinical trials.

Financial stakeholders often applaud OD. They can readily smell short development timelines and reduced costs. As many of us know, an orphan program typically requires just one pivotal study at a standard significance level of 5%. But what good is that if the disease is extremely rare? Personally, I have worked on phase II projects across more than 50 sites, expecting an average of just one patient per site.

So, what is this column really about? Essentially, I notice that it is easy to cling to various regulatory incentives, like OD, and other expedited pathways, without understanding what they truly mean or whether they are indeed right (or wrong) for the current project. The thing is, we hold onto words and terms that we recognize and can refer to, instead of delving into the more complex medical and regulatory reasoning that would create greater value in the project.

Finally, the little black dress is rarely spot-on, but every now and then, it becomes the hidden centerpiece in the wardrobe, stealing the spotlight at the party. Orphan Designation works a bit like that too. Often, it's just a 'check the box' exercise early in the development process, not carrying much weight. It's more about confirming something we might already know. However, occasionally, it hits the mark – providing direction and momentum. Executed correctly, Orphan Status, i.e., when the product hits the market, becomes the ultimate trump card.


Footnote 1. COMP is likely to be replaced by other advisory bodies.

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