timeline question

Dear all,

Sorry about length of this post. The first 2/3 is just copied and pasted from Wiki though (so can probably be skipped by most of you). Think I may have got myself a little over-excited about the potential timeline for drug availability (assuming all goes well of course). Was hoping someone more knowledgeable than me – which is just about everyone – could clarify or correct me.

My initial belief was that orphan drug designation allowed for quicker approvals. However, from one of the Govt’s main source of info – Wikipedia – I understand that the advantages are more w.r.t longer market exclusivity etc rather than time to market.

Orphan drugs generally follow the same regulatory development path as any other pharmaceutical product, in which testing focuses on pharmacokinetics and pharmacodynamics,dosing, stability, safety and efficacy. However, some statistical burdens are lessened in an effort to maintain development momentum. For example, orphan drug regulations generally acknowledge the fact that it may not be possible to test 1,000 patients in a phase III clinical trial, as fewer than that number may be afflicted with the disease in question. http://en.wikipedia.org/wiki/Orphan_drug

While orphan drug status seemed to be what got most people excited, to my reading (only of Wikipedia) Fast Drug designation seemed more exciting as it allowed -
• More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
• More frequent written correspondence from FDA about such things as the design of the proposed clinical trials
• Eligibility for FDA Accelerated Approval, i.e., approval on an effect on a surrogate, or substitute endpoint reasonably likely to predict clinical benefit
• Rolling Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed. NDA review usually does not begin until the drug company has submitted the entire application to the FDA
• Dispute resolution if the drug company is not satisfied with an FDA decision not to grant Fast Track status. http://en.wikipedia.org/wiki/FDA_Fast_Track_Development_Program

Drugs with accelerated approval can initially be tested in clinical trials that use a surrogate endpoint, or something that is thought to predict clinical benefit. Surrogate endpoints typically require less time …….. Drugs approved under the FDA Accelerated Approval Program still need to be tested in clinical trials using endpoints that demonstrate clinical benefit, and those trials are known as Phase 4 confirmatory trials. (wiki again).


The question -

At present we have a Retts Phase 2 going, with top line results 2nd half 2014. Fragile X a year later. Both above designations taken together, is it fair to say we may still need a Phase 3 trial, albeit with some lesser statistical burdens? If so, drugs will still be several years away.
Fast Track allows different endpoints to be used. Does anyone know if this can simply be Phase 2 trials already undertaken??????? When is it going to be known what the requirements will be (and therefore the time likely required)? Thanks for any input.

All the best,

KMS