PRE-APPROVAL INFORMATION EXCHANGE (PIE) IN THE UNITED STATES. TRENDS FOLLOWING THE PASSAGE OF THE PIE ACT: WHERE ARE WE NOW?
Author(s)
Anna De Boer, PhD1, Don Smith, PhD2;
1Thermo Fisher Scientific, Waltham, MA, USA, 2Thermo Fisher Scientific, Senior Scientific Director, Wakefield, MA, USA
1Thermo Fisher Scientific, Waltham, MA, USA, 2Thermo Fisher Scientific, Senior Scientific Director, Wakefield, MA, USA
OBJECTIVES: To explore trends in PIE since the passage of the PIE Act (2023-2025) and compare them to 2017-2022 trends when PIE was shared based on guidance from the FDA.
METHODS: A targeted literature review was conducted in PubMed, Embase, and the grey literature between January 2023 and December 2025 using keywords related to PIE (e.g., pre-approval, information). Studies reporting outcomes related to PIE were included.
RESULTS: All 14 sources included covered payer/healthcare decision maker (HCDM) perspectives. HCDMs were especially interested in receiving PIE for rare diseases, cell and gene therapies, oncology products, or expedited FDA approval pathways. HCDM ratings of the most important information manufacturers should share for PIE included place of product in therapy (68-100%), pricing (30-88%), proposed indication (52-82%), clinical trial results (39-94%), anticipated timeline for FDA approval (35-82%), and preliminary product information (17-50%). Broader topics, like disease overview and treatment landscape, were rated as important to include for cell and gene therapies and products for rare diseases. When exploring changes before vs after the passage of the PIE Act using an earlier literature review (2017-2022), some similarities and some differences were identified. HCDMs preferred to receive PIE 4-12 months prior to anticipated FDA approval both before and after the PIE Act. Moreover, the proportion of HCDMs receiving an unsolicited pre-approval dossier from a manufacturer appeared to be similar before and after the PIE Act (50-60%). However, up to two-thirds of HCDMs noticed a gap in available PIE information (47-67%) in surveys after the PIE Act, although surveys from before the PIE Act suggested that this gap was closing.
CONCLUSIONS: While HCDMs have preferences for more tailored approaches to PIE, areas to better align with manufacturers were still noted. Future studies covering the manufacturers’ perspective would be useful to show different perspectives on PIE.
METHODS: A targeted literature review was conducted in PubMed, Embase, and the grey literature between January 2023 and December 2025 using keywords related to PIE (e.g., pre-approval, information). Studies reporting outcomes related to PIE were included.
RESULTS: All 14 sources included covered payer/healthcare decision maker (HCDM) perspectives. HCDMs were especially interested in receiving PIE for rare diseases, cell and gene therapies, oncology products, or expedited FDA approval pathways. HCDM ratings of the most important information manufacturers should share for PIE included place of product in therapy (68-100%), pricing (30-88%), proposed indication (52-82%), clinical trial results (39-94%), anticipated timeline for FDA approval (35-82%), and preliminary product information (17-50%). Broader topics, like disease overview and treatment landscape, were rated as important to include for cell and gene therapies and products for rare diseases. When exploring changes before vs after the passage of the PIE Act using an earlier literature review (2017-2022), some similarities and some differences were identified. HCDMs preferred to receive PIE 4-12 months prior to anticipated FDA approval both before and after the PIE Act. Moreover, the proportion of HCDMs receiving an unsolicited pre-approval dossier from a manufacturer appeared to be similar before and after the PIE Act (50-60%). However, up to two-thirds of HCDMs noticed a gap in available PIE information (47-67%) in surveys after the PIE Act, although surveys from before the PIE Act suggested that this gap was closing.
CONCLUSIONS: While HCDMs have preferences for more tailored approaches to PIE, areas to better align with manufacturers were still noted. Future studies covering the manufacturers’ perspective would be useful to show different perspectives on PIE.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HPR76
Topic
Health Policy & Regulatory
Topic Subcategory
Insurance Systems & National Health Care, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, STA: Multiple/Other Specialized Treatments