FAQ's

Orphan designation is typically the very first interaction that a biotech company developing a drug for a rare disease will have with FDA or EMA. 

A company that is able to demonstrate "promise" that its drug is effective for treating a rare disease or condition using clinical data or data from animal models of the human rare disease, can submit an application for orphan designation to the FDA or EMA. 

Biotech companies can reap more value  from submitting their applications sooner rather than later for the following reasons: 

1. Early application increases the asset’s value, thus improving the terms sheets a biotech company can expect with potential investors.

2. Once orphan designation is received, a biotech company can collect tax credits for clinical trial expenses incurred in the USA.  These tax credits likewise impact term sheets with investors. 

3. Receiving FDA or EMA orphan designation demonstrates a company's competence in regulatory interactions and enhances the credibility of its drug development program. 

According to the 1983 Orphan Drug Act, companies that received  orphan drugs designations are entitled to: 

1. Market exclusivity for seven years following marketing authorization. This means you and you alone can market your product for the treatment of the designated rare disease. This form of market protection is better than a patent because it relies upon the FDA's promise in contrast to the uncertain success of patent attorneys (not sure what that means. May need better phrasing). This strong exclusivity has yielded high price points for orphan drugs (median 100,000 USD per patient per year).

2. Eligibility to receive tax credits upon orphan designation, underscoring the importance of early application.

3. Exemption from the PDUFA Fee. This  can yield roughly 3.5 million dollars in savings.

4. Exemption from the Pediatric Research Equity act (PREA). The complications of pediatric drug development can be substantial.

Historically, orphan drug development requires fewer clinical trials and their approval pathways can be faster provided they meet eligibility criteria and follow the appropriate regulatory pathway. 

Prior to 1983, there were only 12 drugs for patients with rare diseases.  By 2024, more than 1,200 have been approved.

It takes on average 3 weeks to finalize an application and 3 months for the FDA to review it.  We typically  budget 10 months for EMA applications to meet their process requirements which are different from the FDA's.

No! Animal model data of the human rare disease in animals who received your product showing evidence of  change such as smaller tumors,  longer survival rates, better histology at necropsy, among others)  compared to diseased animals who did not receive the orphan product are 100% satisfactory.

As the previous director of the Office of Orphan Products (OOPD),  I was the final authority deciding the fate of 1,400 orphan drug designation applications. Like my predecessors and my successors, roughly 30% of all first time applicants were designated. With repeated resubmission this number gets up to a little better than half all applicants. 

In contrast, from my regulatory consulting office, more  than 90% of all applicants are awarded. Why? Not because I receive any special treatment from my ex employees, no. Rather, I will only accept clients who are as committed to success in developing their orphan products as we are in acquiring orphan designation . My advice and our team's support will guide them throughout the regulatory process in writing an application that meets approval requirements. Basically,  I offer my clients access to my unique experience as a trusted advisor who shares their ambition to develop drugs for patients with rare diseases. 

In the conduct of two consultancies over 8 years, I have personally written over 800 orphan designation applications, certainly more than anyone else on the planet. You can do it yourself and you can probably hire someone cheaper. But if you want it done right at a fair price (remember, without orphan designation every other dollar spent is wasted), then you should hire me.

Our initial 30-minute conversation is free. Of course, you get what you pay for. But we love to advise, to teach, and to opine. Our business model is to charge for tangible products (i.e. written applications) that advance our client’s assets value, typically by 10x to 100x our fee for these written deliverables.

It's as different as Europeans are from Americans, that's a lot! The European application is about 3 times in length, is responsive to four criteria instead of FD's two, is a multi-stage process of application validation, pre submission meetings and ultimately the rendering of an opinion by the Committee on Orphan Medicinal Products (COMP). It takes around 10 months. 

In my former capacity as OOPD director, I participated in COMP meetings every six months and likewise my European colleagues visited me at FDA headquarters. For the years 2012 through 2017 in my first consultancy,  I submitted more applications for European designation than any European entity. As an orphan regulatory expert,  the European system is both foreign yet very familiar to me and my team at Only Orphans Cote. 

 Absolutely! Half our work is orphan designation both at FDA and EMA, the other half is getting orphan drugs through the review division as marketed products; Pre IND Meeting, Type A/B/C/D Meetings, Breakthrough Therapy Designation, Fast Track Designation, INTERACT Meetings, OOPD Meetings, IND Submission, QIDPD, RMAT. At Only Orphans Cote, we do everything to get your orphan drug licensed and marketed in the US.  

The Rare Pediatric Disease Designation is an affirmation by FDA that in the event a company receives marketing authorization of its product for the treatment of a pediatric rare disease, the company  will indeed receive the Priority Review Voucher (PRV). The PRV is a special incentive because they market for roughly 100 million dollars. Therefore, the RPDD likewise increases your asset’s value because it demonstrates to your investors that upon marketing, a revenue stream can be expected not only from drug sales but also from this important one time sale of the PRV.

Fast track Designation is a very old - at least 3 decades - system of administrative expediency which FDA extends to products with an unmet medical need. While it is only available for products that have an open IND, no clinical data per se is required for applying for fast track designation. The review of applications for fast track designation is fairly criteria-based and therefore award of this designation is reasonably predictable and more often than not, positive.

In contrast, Breakthrough Therapy Designation is a much newer system that embraces all the benefits of Fast Track Designation and awards extra support  in the form of extra meetings and interdisciplinary meetings. Breakthrough Therapy Designation requires the submission of clinical data that identify exactly why this therapy offers a breakthrough in comparison to currently available therapies. The award of Breakthrough Therapy Designation expounds upon breakthroughs criteria, which can be difficult to interpret by many and will require an expert guidance to enhance its chances of success against what many describe as a highly subjective guidance by existing processes. A minority of Breakthrough Therapy Designation applications are awarded the designation and an award is quite unpredictable. Nevertheless, companies that receive breakthrough therapy designation benefit greatly as the biotech marketplace recognizes the value of FDA’s affirmation of “breakthrough-ness”. Retrospective studies have demonstrated that recipients of Breakthrough Therapy Designations are faster to the clinic, faster through the clinic, and more likely to be rapidly acquired by other pharma companies.

Only me. I am well supported by a team of business development associates and regulatory affairs associates who manage the administration of meetings and contracts and the drafting of manuscripts respectively. However, I am responsible for every word that is written, I am whom you meet by Zoom and I am who you trust your success to.

 I am not a lawyer, so I don’t charge hourly. We use project-based pricing with an initial deposit due at contract signing and the remaining balance due upon submission to the agency. We do not work on contingency but we also refuse all jobs that can’t win. This judgment is based on data availability and validity at the time of our initial call. In the rare event that an application is returned by an agency with anything other than the award of the designation sought, our contract provides for a free resubmission. As I said earlier, we work with clients who share our commitment to success in developing drugs for patients with rare diseases.