Case study – small virtual EU-naïve sponsor based in California

10^th December 2025

We were approached by a “virtual” pharma, who had just completed a small first-in-human study in the US for a rare disease.  The outcome was positive so the company was interested in doing a phase 2a open label study in the US and the EU, with the aim of extending to a phase2b/3 type design if successful.

This company had no prior experience of working in the European Union. They engaged a small US based CRO who were helpful in the phase 1 study, but who lacked EU experience, so delays had started to arise when they contact us for advice. The company also complained about poor responsiveness to basic questions from the CRO in question.

Regintel was able to quickly move through the basic issues that were holding them back, which were:

• Registration as an SME

• Registration of the substance

• Registration of a Research Product Indicator

• Orphan Drug Designation application

• Scientific advice at zero cost thanks to incentives

• Subsequently, once advice was received work continued with the

• Insertion of XEVMPD EV code, acting as Responsible Person for Eudravigilance

• Acted as Legal Representative under the Clinical Trial Regulation and support for the CTIS CTA submission process

• Acted as Data Protection Representative advising on data protection issues in the EU including

  • Policies

  • SOPs

  • Technical and Organisational Measures for compliance

  • Data protection training

  • Data flow analysis / implementation of Standard Contractual Clauses for export of data

  • Contracts with sites and vendors

  • Review of ICFs and Protocol

  • Record of Processing Activities and Data protection Impact Assessment

  • Dealing with RPIs

    
    

Because a Data Protection Officer was not required in the jurisdictions where the study was undertaken, this service was not needed, but was eventually engaged at the start of their follow-up study, which included Germany. RegIntel was able to provide this service as well through our retained DPO.

Because Regintel has extensive experience of these processes and can act very quickly, the initial delays were caught up and the results of the Phase 2a study were delivered on time.

Stage EU	Date	Stage USA
Initial contact	Feb 2022	IND preparation
SME	March 2022
Substance	March 2022
RPI	March 2022
RPEV/XEVMPD insertion	May 2022
ODD	July 2022	IND submission
Completion of compliance documentation	September 2022	FDA approval
Scientific advice	September 2022
Finalisation of submission documentation	October 2022	FDA study start
CTIS submission	November 2022
Approval	January 2022	FDA study completion
Study completed	March 2024
Study report	June 2024	FDA report

Overall project was completed on time and RegIntel’s final fees over the 29 months was 12% less than the initial budget.