The Global Guide to Compassionate Use

Compassionate use or expanded access refer to the means for providing unapproved medications for treating patients with serious medical conditions when no other treatments are available. Governments around the world have created provisions for granting access to unlicensed or investigational drugs to patients who have exhausted all available treatment options or have no treatment alternatives in their country. These pathways are grouped under the labels of compassionate use programs (CUP), expanded access programs (EAP), early access Programs, or named patient programs (NPP). Every small and mid-sized biopharmaceutical company with an orphan treatment will consider launching a global early access program.  

The Global Guide to Compassionate Use Programs was developed over four years by industry, government, regulatory, operational and pharmacovigilance experts with decades of combined global experience. This online resource will help you navigate the complex and often confusing individual country regulations, guidance documents, submission requirements and applications – and clarify the roles and responsibilities of the key parties involved in initiating program requests including manufacturers and innovator-led programs.

Access to this comprehensive reference guide will save you valuable time by translating the regulations from various countries. This compendium includes over 60 of the most common countries, where there is a validated demand for compassionate use/expanded access programs, specific details on the various regulatory pathways that allow you to immediately access source regulation documents, including those dealing with pharmacovigilance and drug labelling requirements.

Problem Statement
Locating country-specific information on compassionate use takes too long - Biopharma companies spend months and even years collating information. 
Locating information on compassionate use costs too much money - Due to the intense labor of collating information on compassionate use programs, The collation of such information may cost biopharma upwards of $100,000 - $300,000. 
Compassionate Use information needs to be accurate and up to date  - Accuracy may be low and the documentation created by biopharma becomes easily and instantly outdated
Companies must be compliant with stipulated regulations or risk significant fines and oversight
Patients are waiting for life-saving medications. 

Since 2021, with minimal marketing efforts, the company has grown to attract 24+ licensees, including major companies like BeiGene, Sanofi, Daiichi, Ultragenyx, Alnylam, Kura Oncology, Amylyx, BioMarin, Sobie, and AbbVie and others are in the queue. Many are repeat customers.
 
There are over 900 biopharma companies with Phase 2+ oncology/neurology/rare disease portfolio  that can benefit from the subscription with direct and near-term impact on their portfolio. Additionally, Clinical Research Organizations, (N= 50), Ministries of Health (N= 200) and vendors can benefit substantially from subscription.  With judiciously designed marketing and CRM initiatives the potential for GCU is tremendously larger. Market revenue potential for licenses could reach $4.5M per year and as high as $9.5M per year with a doubling of licensing fees. 

Our GCU database has a listing of 400 + companies that have downloaded a country sample providing immediate outbound engagement with potential customers. 
A separately owned Medical Information resource (The Global Regulatory Framework for Medical Information in the Pharmaceutical Industry has attracted over 84 licenses since 2014 with 227 BioPharma contacts. 
 
Proof of concept is complete, and the company is poised to grow dramatically in 2024 and beyond. We are currently contemplating Letters of Intent to: 

·         Fully acquire the company - for synergies with acquirer

·         Invest capital in the company - to fuel growth 

Complete Acquisition
100% acquisition of the company

Valuation at 7x 2027 Projected Revenue 

Integration period with acquirer flexible to continue to grow revenue, deploy 2nd edition and marketing efforts

Capital Investment:
Update the GCU to a 2nd edition which includes additional countries and regulations specifically for Cell and Gene Therapies (CG&T)
Integration with AI tool for immediate updates
Launch Desktop App for scalability and potential synergies with other software platforms
Deploy robust marketing program
Operational and overhead expenses
 
I am the sole owner of this resource and currently work in the field of biotechnology. I do not have any full-time employees (FTEs) and rely on consultants for periodic updates. What started as a weekend project has evolved into a promising business opportunity.
 
Thank you for your time.