From the rare pediatric disease priority review voucher fda guidance: (Sorry about the numbers)
Q5. What does “Does not seek approval for an adult indication in the original rare pediatric disease product application” mean? 249 250 251 An applicant cannot receive a rare pediatric disease priority review voucher if the application 252 seeks approval for an adult indication in the original rare pediatric disease product application. 253 We interpret this criterion to mean that, to preserve voucher eligibility, the applicant cannot seek 254 approval for a different adult indication (i.e., for a different disease/condition) in the original rare 255 pediatric disease application. If the applicant seeks approval for use by pediatric and adult 256 populations with the rare pediatric disease, the applicant will still be eligible for a voucher if the 257 approved use includes pediatric use, as described in Questions 3 and 4. If the applicant obtains 258 approval for use only in an adult population with the rare pediatric disease, the applicant is 259 ineligible for a voucher. 260 261 Thus, under this interpretation, an applicant can preserve voucher eligibility even if the applicant 262 seeks approval for use by adults in addition to pediatric patients with the rare pediatric disease. 263 One reason we are interpreting the statute in this way is to avoid incentivizing sponsors to 264 exclude adults affected by the rare pediatric disease from clinical trials or to exclude adult data 265 from the subsequent marketing application solely for the sake of voucher eligibility, when such 266 exclusions may not be scientifically or ethically acceptable for the reasons described below. 267 268 Clinical Trial Design – Clinical Trials for a Potential Rare Pediatric Disease Product May Need 269 to Include Individuals Over 18 Years of Age for Scientific or Ethical Reasons: Clinical trials for 270 rare diseases and conditions are challenging because, among other factors, the small patient 271 populations limit the opportunities for study and verification of results. Because such clinical 272 trials are likely to be small and at risk of being underpowered, FDA expects that rare disease 273 clinical development programs will attempt to include all patients with the rare disease or 274 condition that are available for study and who could reasonably be expected to benefit from the 275 intervention, regardless of the age of the patient (where feasible and appropriate based on the 276 disease/condition and expected effects of intervention).24 Indeed, studies using novel therapies 277 should generally be conducted in young adults (18 to 21 years of age) prior to exposing 24 See,e.g.,ICH andFDAGuidance,“E11Clinical InvestigationofMedicinalProductsinthePediatricPopulation,” SectionII.A,availableat http://www.fda.gov/downloads/RegulatoryInformation/Guidances/ucm129477.pdf. 8
Contains Nonbinding Recommendations Draft – Not for Implementation 278 adolescents and younger pediatric patients; for children to be included in early phase 279 investigations, there must be a prospect of direct benefit for an individual child to be studied in a 280 clinical trial in which more than a minor increase over minimal risk is presented by an 281 intervention or procedure.25 For all of these reasons, it may not be scientifically or ethically 282 appropriate to exclude those over 18 years of age from a clinical trial evaluating a potential rare 283 pediatric disease product. 284 285 Data to Include in a Marketing Application – Available Adult Safety and Effectiveness Data 286 Must be Included in the Application: If clinical safety and effectiveness data are available in an 287 adult population (i.e., individuals over 18 years) at the time of the submission of an original 288 application for a potential rare pediatric disease product, these data must be included in the 289 application for FDA’s review. 26 In many cases, if there is a population over 18 years of age with 290 the rare pediatric disease that could benefit from the product and for whom there are available 291 data to support the evaluation of the safety and effectiveness of the product, labeling for such a 292 population should be sought in the original product application. 293 294 As noted, seeking approval for use in both adults and pediatric patients with the rare pediatric 295 disease will not affect voucher eligibility. However, we remind applicants seeking a voucher 296 that – whether or not they seek approval for use in an adult population – we expect them to 297 submit data adequate for labeling the drug for use by the full range of affected pediatric patients 298 (see response to Question 4). 299 300 Note that after a sponsor has been awarded a rare pediatric disease priority review voucher for 301 approval of a drug, the sponsor can develop the same drug for additional indications, including a 302 different adult indication, without losing the voucher.
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