Challenge
A small company sought to register a new treatment with a previously unvalidated surrogate endpoint. Regulatory precedence from the Regulatory Agency had indicated their reluctance to consider this biomarker as a potential surrogate endpoint in closely related, but different, diseases.
Solution
PharmaLogic Consulting developed a multi-pronged strategy with the client to: validate the surrogate endpoint (statistical analysis), engage Key Opinion Leaders to champion the case, and engage with the Agency as an industry participant in a multi-disciplinary project on the disease in question.
Result
Successful End-of-Phase 2 meeting in which the Agency agreed to the use of the surrogate endpoint in the pivotal registration trial.
Challenge
A biotech company had received a GCP inspection report for an on-going early phase clinical trial in an immediately life-threatening condition. The report listed several critical findings and over 30 major findings to be addressed in order for the study to be permitted to continue.
Solution
PharmaLogic Consulting took the lead in preparing the responses to the report with the Sponsor, their contract research organization, the clinical investigator and their staff, and hospital pharmacy.
Result
A comprehensive response document was submitted in response to the GCP inspection report, addressing all issues, and including action plans to remediate the deficiencies. The study was allowed to continue to completion.
Challenge
A company conducting a Phase I study in a rare disease patient population observed adverse effects which appeared to be related to the cumulative exposure to the investigational medicinal product over time. The sponsor looked into their data and determined that they should administer a lower dose but thought they would have to terminate their current study and start again at a later date.
Solution
PharmaLogic Consulting guided the Sponsor on the use of an urgent safety amendment in order to lower the dose for patients currently enrolled in the study, and engaged the concerned regulatory agencies to confirm the acceptability of the urgent safety amendment for patients actively receiving treatment, plus a subsequent substantial amendment to lower the dose for continued enrollment into the study.
Result
The clinical trial did not need to be halted and there was a minimal disruption to patient recruitment and study timelines.
Challenge
A small company had previously sought orphan drug designation in Europe but was not successful. They assumed that it would not be possible to apply again.
Solution
PharmaLogic Consulting, with the client and Key Opinion Leaders, reviewed the current status of the disease, and most importantly current medical practice as it pertained to the diagnosis of patients. A new strategy for collecting prevalence data with developed in collaboration with PharmaLogic Consulting and executed by a specialist consultant.
Result
A successful orphan drug designation application, in which EMA congratulated the Sponsor on the quality of prevalence data provided.
Challenge
A very small biotech company had applied unsuccessfully for SME (Small and Medium Enterprise) status within the EU due its ownership structure.
Solution
PharmaLogic Consulting reviewed the case, the details of the company’s ownership structure and engage the SME office at EMA in a discussion regarding the interpretation of the pertinent legislation and the specifics of the company’s ownership structure.
Result
The case was sent to the EU Commission for a decision and SME status was granted to the company.