Orphan drugs AMR

Should we talk about orphan drug designation for AMR antibiotics?

In Market Access by Freya Smale

With a little over 3 months to go until the inaugural World Anti-Microbial Resistance Congress, we’ve been busy putting the finishing touches to the conference agenda. During our research, we’ve been posing the question: should orphan drug designations apply to antibiotics?

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On the one hand, ODD provides incentives to encourage AMR antibiotic development. Between 1935 and 1968, there were over 15 new classes of antibiotics developed, since 2000, there have only been 3. We all know why this is the case: antibiotic development is not only risky, it also does not have a viable ROI. In response, the US government provided the GAIN Act which waived fees and market exclusivity. Federal funding awarded through NIAID and BARDA are also available to alleviate the high cost of development, making antibiotic development lucrative and attractive to pharma and biotech. Private funding agencies such as Wellcome Trust, IMI and ARLG have also come up with plans to provide relief. But is this enough?

Could ODD be the answer? Is this a sustainable approach to antibiotic development?

Certainly the Orphan Drug market has seen a number of success stories recently, highlighting the advantages of this regulatory path. However, can antibiotics really be designated Orphan Drugs? The FDA says not. Right now, the instances of resistant infections are relatively low, but there is no guarantee this will remain that case.

What is evident is that the debate is healthy!

Here is a snapshot of the conversations we’ve been having with our World Anti-Microbial Resistance Congress speakers to find out more about the Orphan Drug Designation debate. We’ll be continuing the discussion on October 20, live in Washington D.C.

Mark Jones, Basilea Pharmaceutica, Ltd.

In the US if an ODD is obtained the Sponsor qualifies for seven-year FDA-administered market Orphan Drug Exclusivity (ODE), “tax credits of up to 50% of R&D costs, R&D grants, waived PDUFA fees, protocol assistance.” These are certainly of great interest to Pharma. In EU ODD is much more difficult to achieve and the only real advantage that it brings is to make a potential difference in pricing and reimbursement discussions which can lead to higher prices.I would certainly think that bit is another ‘pull incentive’ that would be interesting to discuss at this meeting if you can find a good speaker.

Ian Friedland, Achaogen

This has come up on a number of occasions so I absolutely think it’s worthwhile. I’ve heard the arguments from the anti-infective division of FDA why orphan drug does not apply to AMR but I think we should continue to debate this.FDA has publicly stated that AMR is not suitable for orphan drug and their guidance on pathways for AMR does not include orphan drug pathway, but I don’t know if anyone has formally applied for orphan drug. Certainly no-one has gotten the designation.The anti-infective division of FDA has shared criteria under which orphan drug might be suitable but these criteria are different and more stringent than what is applied in other divisions for a novel antibiotic.

Eszter Nagy, Arsanis

I am convinced it is an attractive regulatory path for some of the novel, pathogen specific approaches. Companies may not consider it appealing due to the notions that orphan drug status equals small market. However, the recent successes of orphan drugs with high price in other therapeutic areas are good examples of attractive market potentials.It would be helpful to highlight the advantages of this regulatory path at the conference.

Mike Dudley, The Medicines Company

Orphan drug has been kicked around for anti-infective but not received much traction in the US. Given that there are other efforts to address drugs for “limited patient populations”, I would certainly be interested in hearing more on this perspective.  Certainly, orphan drug designation has been granted for some pathogen/drug combinations in the EU; for example the beta-lactam antibiotic temocillin has orphan drug designation for Burkholderia cepacia infections in cystic fibrosis. Similarly, a number of inhaled antibiotics for management of chronic pulmonary infections due to Pseudomonas aeruginosa have had orphan drug designation in the US.  But these programs have often been designed to give exclusivity for “older drugs” to support development, whereas for novel drugs, this is less of an issue.

Join us in Washington on October 20, 2015 for a unique panel debate
Do orphan drug designations apply to antibiotics developed to treat “serious and life threatening infectious diseases” caused by “antibiotic-resistant bacteria? What about to diagnostics?

Edward Cox,
 Director, Office of Antimicrobial Products, FDA (TBC)
Kevin Outterson,
 Professor of Health Law, Bioethics & Human Rights, Boston University
Barrett Thornhill,
 Executive Director, Antimicrobial Innovation Alliance

Don’t miss the inaugual World AntiMicrobial Resistance Congress USA 2015. Book today by visiting the website >

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