While the both the EMA and FDA (Office of Orphan Products) has seen a substantial and consistent rise in orphan designation requests, and consequently an increase in the orphan designations for which they approve, the figures are not equivalent.
In the United States for instance, the OOPD designated 354 of the 466 applications received in 2015. In 2016, the OOPD received 110 more applications, setting a new record from the previous year, yet designated fewer (333). These numbers show designated applications dropping a whopping 20%.
Why are less (%) applications getting designated to date?
With more applications being sought after, there are bound to be more mistakes. There are several in fact that we frequently see with applications. Here, I have described the most common reasons.
Claiming disease subsets that are not medically plausible
While there is precedence in both the US and Europe for approvals based on subsets of diseases effecting larger populations (>200,000 and >5 in 10,000, respectively), there are rare circumstances where this is applicable, proving to be quite challenging when considering medical plausibility. There are ways to justify these subsets however they are not made clear in any guidance.
Inaccurately calculating prevalence
In some case, there is an evident lack of existing literature and investigators working on the same disease creating challenges in defining the population. A common mistake in these cases is to submit on incidence instead of prevalence.
Submitting inadequate scientific rationale to support the use of the product in the proposed indication
The data required to support the use of the product in the indication must meet the criteria of the ODA. Whether nonclinical or clinical data is presented, the OOPD does expect to see sufficient and applicable animal or human data.
Orphan requests that include subjective language
At times when asked to review orphan designations we find Sponsors attempting to use subjective language in the application requests. These may be words such as refractory, debilitating, life-threatening, etc. all of which will raise concerns at the OOPD. The real question is “what is the disease?”
How do I know this?
At RareMoon, we have a 100% approval rating on both FDA and EMA orphan applications. We have the expertise and experience to ensure every application is written in proper regulatory language and backed my accurate feasibility valuations and due diligence.
Often, we are asked to advise when Sponsors have received an abeyance letter which also allows us to:
- see first-hand the justification from the OOPD;
- review the application, assess feasibility, and assess whether the OOPD requests are attainable; and
- fix it, or break the news that the application (disease, plausibility, etc.) is not justifiable.
Having gained insight through our experience, working and interacting with the OOPD, ex-FDA Directors and on dozens of orphan applications gives our Clients the utmost confidence in our team. If you are looking to gain an orphan designation, are in need of strategic advice, orphan regulatory expertise, or other regulatory application support, I invite you to have a conversation with us. I am confident our call will be insightful and provide ideas to help you achieve your goals.
