The roller coaster of Duchenne has been in high gear this week with BioMarin’s announcement to halt trials and one day later, Acceleron’s press release about receiving Fast Track Status. I don’t know about you, but in a certain way, it sounds like Fast Track Status should be a high speed train, capable of 220 mph. The reality is that the Fast Track Status train is the same train, with better fuel (expedited review) and more tech support (FDA interaction).

I am pretty sure, everyone may have a different definition of Fast Track Status and thought maybe it would help to clarify what this means in reality.

In 1983, Congress passed the Orphan Drug Act.

Orphan Status:
  • Drug (or biologic) for an orphan indication.
  • Definition of rare disease– affects less than 200,000 people in the US
  • High unmet medical need
  • 7 years of market exclusivity
  • Tax credit of up to 50% for qualified expenses for research support to support approval of the Orphan drug
  • Exemption from several kinds of user fees
  • Guidance from FDA staff to sponsors

The Division of Orphan Products is responsible for:
  1. granting Orphan Status
  2. is able to provide small grants for sponsors (based on application/review)

Fast Track Status – the purpose of Fast Track Status (accelerated approval and priority review) is to speed reviews and provide more extensive guidance to sponsors about the nature of the evidence that will be required in order to support approval.

  • Includes a ‘rolling review’ by FDA, where companies submit modules of an NDA (new drug application) for a rolling review which allows more frequent consultation and dialogue with FDA on issues related to the application.
  • Fast Track may include ‘accelerated approval’ which allows the use of surrogate (secondary) endpoints likely to predict clinical benefit. The FDA would likely require post-approval studies in order to collect additional evidence about safety and benefit.
  • Fast Track applications may also qualify for ‘priority review’ which means FDA sets a goal of completing reviews within 6 months compared to the standard process of 10 months.

There is one more mechanism you might hear about to facilitate review – Special Protocol Assessment – which allows the FDA to provide expedited assessment of the adequacy or appropriateness of specific clinical trial protocols and to reach an agreement with sponsors about the design and size of a certain trial to support efficacy (benefit) claims. Normally, this is ONLY available at the end of a Phase II trial.

Bottom line: Acceleron has not gotten approval to start the trials in the US yet…Fast Track Status does not mean IND Approval.

This is all very difficult to take in and most of the time, seems pretty confusing. Often patients/families see the designation of Fast Track as some sort of vehicle that may deliver the drug quicker. In essence, because of the expedited review, guidance from FDA is intended for that purpose. But for those of us watching and waiting, heart in hand, it does not answer the ‘when’ question. It is strictly related to the FDA process.

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Comment by Dana Edwards on August 15, 2010 at 9:10pm
Thank you Pat, for ALWAYS taking the time to explain and break all this down for us. It is so hard to understand all this but you put in a way we all can understand. It's not what we want to hear (we want it's gonna be here tomorrow and it's great) but at least it's always the truth. Please keep explaining we need you. Dana

Comment by Pat Furlong on August 8, 2010 at 8:50am
Good morning, I do not claim to know all that is going on in terms of harmonization efforts. You already know FDA has developed a new position for rare disease (anne pariser). One of her tasks is 'harmonization'. Eurodis, NORD and Genetic Alliance are all involved in moving this forward as are some of the groups in DC - Kakkis (cure the process), Jacoby (rare project) and Crowley's caucus. In addition, the IOM report due out in Fall will have additional recommendations which may ultilmately result in legislation. I cannot predict the next 5 years, wish I could. These are ongoing discussions and certainly in the right direction, and are actively being worked on. I'm meeting with some folks this week and if there is any concrete news, I will pass it along.
Comment by Ofelia Marin on August 8, 2010 at 8:36am
What exactly is this harmonization you are talking about? Do EMA and FDA actually meet to work on this or it is what we (DMD, FA, other rare diesease communities) would like to see happen in the future? What is the chance this will happen in the next 5 years? I am assuming this was discussed for a while now, not necessarily in our community where no drugs were/are close to approval, but maybe in other diseases where drugs are actually approved only in US or EU. Was there any progress made at the FDA, EMA level (and by that I mean do they have people actively working on this at the moment) in the direction we would like?

Comment by Pat Furlong on August 7, 2010 at 10:39am
Whoa, as you know sponsors and fda communication are confidential and fda does not make generalizations.

Here's what we know. Fast track is the FDA's file cabinet that says -review within 6 mo. The dialogue and rolling submission must include everything for review. So, of course, whatever happened in the Phase I (healthy volunteer) will be available and whatever happens in the dose escalation trial will become part of the submission. Within 6 mo. (so, that means Jan-Feb) we will hear more. Given the fact that the Acceleron Team is committed to the DMD community and great with communications, I suspect we will receive updates along the way.
And Harmonization efforts are going on across countries. You have heard a lot about EMA (used to be EMEA) and US efforts, specifically in DMD, but there are other efforts going on as well in other diseases (such as friedreich's ataxia ) that you may not be aware of. As you might imagine, we are not the only community with these sorts of issues/concerns.

and as you know, some companies partner (for example Prosensa/GSK; PTC/Genzyme, etc) based on experience, expertise and financial capacity to develop trials and seek approvals in other countries.
Comment by Mindy Cameron on August 6, 2010 at 11:49pm
Most of the talk that I've heard about harmonization between the FDA and other world regulatory agencies deals with Europe. Are Canadian regulators present at talks between FDA and EMA, for instance? Does fast track designation ever mean that trial results from other countries might be looked on more favorably? I'm referring to the phase 1 (or 2) study of ACE 031 in post menupausal women being considered by the FDA even though it was done in Canada.
Comment by Ofelia Marin on August 5, 2010 at 11:19pm
Have they filed the IND application or have any plans to file before the end of this year?

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