--- title: "Pediatric Molecular Target List: Evolution & Impact of Orphan Drug Programs" description: "Pediatric Molecular Target List: How It Evolved and How It Could Impact Your Orphan Drug Program — The Bracken Group" --- [![logo](https://www.thebrackengroup.com/hubfs/TBG/Images/logo.svg) ](https://www.thebrackengroup.com/) - Our Services & Expertise - Consulting - [Consulting Overview](https://www.thebrackengroup.com/expertise-overview) - [Clinical Trial Strategy & Operations](https://www.thebrackengroup.com/clinical-trial-strategy) - [Medical Imaging Strategy & Operations](https://www.thebrackengroup.com/medical-imaging-clinical-trials) - [Radiopharmaceutical Strategy & Operations](https://www.thebrackengroup.com/radiopharmaceuticals) - [Mergers & Acquisitions Advisory](https://www.thebrackengroup.com/mergers-and-acquisitions-due-diligence) - [Musculoskeletal Services](https://www.thebrackengroup.com/musculoskeletal-services) - [Neuroscience Expertise](https://www.thebrackengroup.com/neuroscience) - [Functional Service Provider](https://www.thebrackengroup.com/functional-service-provider) - [Consultant Publications](https://www.thebrackengroup.com/publications) - [Regulatory Affairs](https://www.thebrackengroup.com/regulatory) - [FDA Meeting Request Calculator](https://www.thebrackengroup.com/fda-meeting-calculator) - [Creative Digital Marketing](https://www.thebrackengroup.com/creative-digital-marketing-strategy) - [Data & Analytics](https://www.thebrackengroup.com/custom-analytics) - [Bracken CRM Add-On](https://www.thebrackengroup.com/bracken-crm) - [Bracken CRM Add-On Demos](https://www.thebrackengroup.com/analytics-demos) - [Our Team](https://www.thebrackengroup.com/team) - [Our Values](https://www.thebrackengroup.com/our-values) - Our Media - [Events & Conferences](https://www.thebrackengroup.com/upcoming-events) - [Case Study Library](https://www.thebrackengroup.com/case-studies) - [Blog](https://www.thebrackengroup.com/blog) - [Infographics](https://www.thebrackengroup.com/infographics) - [Interactive Digital Tools](https://www.thebrackengroup.com/interactive-digital-tools) - [FDA Meeting Request Calculator](https://www.thebrackengroup.com/fda-meeting-calculator) - [Thought Leadership](https://www.thebrackengroup.com/thought-leadership) - [Radiopharma Roundtables](https://www.thebrackengroup.com/radiopharma-roundtable-series) - [Fractals: Our Podcast](https://www.thebrackengroup.com/fractals-podcast) - [The Leaflet: Our Newsletter](https://www.thebrackengroup.com/the-leaflet-newsletter) - [Contact Us](https://www.thebrackengroup.com/contact) ## Pediatric Molecular Target List: How It Evolved and How It Could Impact Your Orphan Drug Program Clinical trials are expensive. In 2016, the estimated median cost of a Phase III trial was $19 million, and this number continues to rise. So, if a manufacturer can avoid having to conduct a clinical trial—because it seems unaligned with the product’s targeted patient population, or unlikely to give a reasonable return on investment—they will explore every option to do so. Unfortunately, while that makes great business sense, it does nothing to advance medical care for patient subsets such as pediatrics. In 1994, the FDA attempted to improve treatment of pediatric patients by enacting the Pediatric Labeling Rule, and in 2002 published the Best Pharmaceuticals for Children Act (BPCA). BPCA required manufacturers to assess existing data and determine whether it was sufficient to support the inclusion of pediatric use information in product labeling. Since the Act did not require manufacturers to actually conduct pediatric studies, it resulted in little progress.   ![Pediatric Molecular Target List How It Evolved and How It Could Impact Your Orphan Drug Program](https://www.thebrackengroup.com/hs-fs/hubfs/Pediatric%20Molecular%20Target%20List%20How%20It%20Evolved%20and%20How%20It%20Could%20Impact%20Your%20Orphan%20Drug%20Program.png?width=278&height=360&name=Pediatric%20Molecular%20Target%20List%20How%20It%20Evolved%20and%20How%20It%20Could%20Impact%20Your%20Orphan%20Drug%20Program.png) ##### **Learn more in the full version of this whitepaper by downloading your free copy. ** ##### **Written by Lisa D. Cooper, PhD, RAC. ** ## Complete the form to download your copy. ![logo](https://www.thebrackengroup.com/hubfs/TBG/Images/logo.svg) ###### [CONTACT](https://www.thebrackengroup.com/resources/pediatric-molecular-target-list-how-it-evolved-and-how-it-could-impact-your-orphan-drug-program#) The Bracken Group 12 Penns Trail Newtown, PA 18940 - [ + 1 215 648 1208 ](tel:+12156481208) - [ info@thebrackengroup.com ](mailto:info@thebrackengroup.com) ###### [SUBSCRIBE TO OUR NEWSLETTER: THE LEAFLET](https://www.thebrackengroup.com/the-leaflet-newsletter) ###### [SCHEDULE A CALL](https://meetings.hubspot.com/allyson-pauly) - [ LINKEDIN ](https://www.linkedin.com/company/the-bracken-group) - Services - [C-suite](https://www.thebrackengroup.com/functional-service-provider) - [Regulatory](https://www.thebrackengroup.com/regulatory) - [Creative Digital Marketing](https://www.thebrackengroup.com/creative-digital-marketing-strategy) - [Custom Analytics](https://www.thebrackengroup.com/custom-analytics) - [Consulting](https://www.thebrackengroup.com/expertise-overview) - Solutions - [Bracken CRM](https://www.thebrackengroup.com/bracken-crm) - Media - [Blog](https://www.thebrackengroup.com/blog) - [Case Studies](https://www.thebrackengroup.com/case-studies) - [Resources](https://www.thebrackengroup.com/en/resources-landing) - [The Leaflet](https://www.thebrackengroup.com/the-leaflet-newsletter) - Ecosystem - [Our Team & Story](https://www.thebrackengroup.com/team) - [Careers](https://www.thebrackengroup.com/career) - [Contact](https://www.thebrackengroup.com/contact) ![](https://www.thebrackengroup.com/hubfs/The%20Bracken%20Group/TBG%20Logo.png) ###### It's almost like getting free consulting... Subscribe to our monthly hand-crafted newsletter for the latest insights on clinical trial trends and the life science industry. [Subscribe Now  ![](https://www.thebrackengroup.com/hubfs/The%20Bracken%20Group/Combined%20Shape.png)](https://www.thebrackengroup.com/newsletter) 12 Penns Trail, Newtown, PA 18940 [+1 215 648 1208](tell:+12156481208) © 2026 the bracken group - [Privacy Policy](https://thebrackengroup-4628766.hs-sites.com/privacy-policy) - EXPERTISE - [Overview](https://www.thebrackengroup.com/expertise-overview) - [Virtual C-Suite](https://www.thebrackengroup.com/functional-service-provider) - [Due Diligence Support](https://www.thebrackengroup.com/mergers-and-acquisitions-due-diligence) - [Medical Imaging in Clinical Trials](https://www.thebrackengroup.com/medical-imaging-clinical-trials) - [Regulatory Strategy & Implementation](https://www.thebrackengroup.com/regulatory) - [Clinical Trial Strategy](https://www.thebrackengroup.com/clinical-trial-strategy) - RESOURCES - [Blog](https://www.thebrackengroup.com/blog) - [Case Studies](https://www.thebrackengroup.com/case-studies) - [Newsletter](https://www.thebrackengroup.com/newsletter) - ABOUT US - [The Bracken Team](https://www.thebrackengroup.com/team) - SUPPORT - [Contact Us](https://www.thebrackengroup.com/contact) - [Schedule a Call](https://www.thebrackengroup.com/contact) - [Terms of Service](https://www.thebrackengroup.com/privacy-policy) 12 Penns Trail, Newtown, PA 18940 [+1 215 648 1208](tell:+12156481208) © 2026 the bracken group - [Privacy Policy](https://thebrackengroup-4628766.hs-sites.com/privacy-policy) [![The Bracken Group](https://www.thebrackengroup.com/hubfs/TBG/Images/logo.svg) ](https://www.thebrackengroup.com) Consulting, creative/digital marketing, and data analytics for life science and digital health companies. #### Services - [Consulting](https://www.thebrackengroup.com/expertise-overview) - [Regulatory Affairs](https://www.thebrackengroup.com/regulatory) - [Fractional C-Suite](https://www.thebrackengroup.com/functional-service-provider) - [Creative / Digital Marketing](https://www.thebrackengroup.com/creative-digital-marketing-strategy) - [Data & Analytics](https://www.thebrackengroup.com/custom-analytics) #### Explore - [Our Team](https://www.thebrackengroup.com/team) - [Fractals: Our Podcast](https://www.thebrackengroup.com/fractals-podcast) - [Case Studies](https://www.thebrackengroup.com/case-studies) - [Publications](https://www.thebrackengroup.com/publications) #### Connect The Bracken Group 12 Penns Trail, Newtown, PA 18940 [+1 215 648 1208](tel:+12156481208) [Contact us →](https://www.thebrackengroup.com/contact) [LinkedIn ](https://www.linkedin.com/company/thebrackengroup/) © 2026 The Bracken Group dba Bracken Data, Inc. · All rights reserved. [Privacy Policy](https://thebrackengroup-4628766.hs-sites.com/privacy-policy)