Replies to post #4192 on Amarin Corp Plc (AMRN)

[kaylawa]

sts66, Written Requests come from the FDA. A company can ask for a request but it's the FDA's decision on whether to issue said request.

In fairness, the "obtain peds waiver request" was just one of several criteria for the "bonus" and was weighted low.

Q2. Can an applicant ask FDA to issue a Written Request for pediatric studies?

A2. Yes. The guidance to industry describes a process in which a sponsor of a new drug or the holder of an application for a product on the list may make a submission to FDA in which it makes a detailed proposal for a Written Request. Where an applicant has more than one product containing the active moiety, the proposal should address each indication that has applicability to pediatric patients and should propose studies in all appropriate age groups. FDA encourages applicants to make such proposals. FDA will review the submitted proposal and MAY issue a Written Request to the applicant based on the applicant's proposal.

Q3. Where should proposals for Written Requests from applicants be sent?

A3. Proposals for Written Requests should be sent to each of the review divisions with regulatory responsibility for any of the applicant's products that contains the same active moiety cited in the Written Request.

Q4. Does a sponsor HAVE to conduct pediatric studies if it receives a Written Request from FDA?

A4. No. A company is not required to do the studies requested by FDA in a Written Request if it chooses not to do so. If, however, the applicant decides to conduct studies that differ from those described in the Written Request, the applicant will not qualify for pediatric exclusivity upon submission of the reports thereof. Applicants who choose to deviate from the studies described in the Written Request and who are interested in obtaining pediatric exclusivity should contact the review division and seek to amend the Written Request prior to the conduct of the studies. The applicant must receive an amended Written Request prior to submission of the study reports to NDA.

Q5. Can an applicant obtain exclusivity by submitting an analysis of the literature in response to a Written Request?

A5. Simply compiling the literature on pediatric use of a drug in a given indication will not qualify an applicant for pediatric exclusivity. The generous incentives were made available to stimulate performance of studies necessary to provide useful information on drug use in children.

Q6. Can the applicant for any approved product qualify for pediatric exclusivity?

A6. No. Pediatric exclusivity is an ADD-ON to existing marketing exclusivity or patent protection. In general, products with no patent life or exclusivity remaining cannot qualify. Under certain conditions, however, pediatric exclusivity may be granted to a product without remaining exclusivity IF the supplemental application itself qualifies for a new exclusivity period under the Drug Price Competition and Patent Term Restoration Act (Waxman-Hatch Amendments).

For example, an application to extend an approved adult indication to the pediatric population for a product with no patent life or exclusivity remaining could obtain pediatric exclusivity IF new clinical studies of safety and efficacy are required for approval. In that case, the pediatric supplement would earn 3 years of marketing exclusivity under the 1984 amendments, to which the additional 6 month pediatric exclusivity would be added. A somewhat different situation would be where dosing information was needed in children under 6 years of age that could be based on PK studies. An oral solution dosage form exists but has no marketing exclusivity remaining. However, the firm has a controlled release product containing the same active moiety that is still protected by a 3 year exclusivity period. The controlled release product would not be appropriate for this age group because of the dose delivered. In this situation, PK studies conducted to label the oral solution could be the basis of a 6 month extension of the exclusivity for the controlled release product, provided the terms of the Written Request are met.