Special Situations are problems or questions that occur outside of the ordinary day-to-day work. In the life science industry, the Special Situation often arises from the unique technology, research or regulatory requirements of the client's product. A Special Situation can occur in any phase of the drug or device development cycle, from pre-clinical research to regulatory or reimbursement filing.
Below are 3 cases of Special Situations from our Clients.
(All 3 cases are anonymized to hide the Client's identity)
What are Special Situations?
Background: The client has filed for reimbursement in a European country for one of their recently approved cancer drugs.
Problem: During the HTA by the government agency, several critical questions were asked regarding the statistical methodology used in the value dossier. The government agency was also asking for additional analyses.
Solution: We addressed the agency's concerns in 2 ways: by further motivating the choice of methods based on NICE guidelines and by performing custom-made simulations to assess the impact of modelling assumptions. After reviewing our analyses, no further analyses were required by the agency.
Case 1: Big Pharma Client
Company Market Cap: $100B+
Background: The Client has recently compiled the results from their first phase 3 clinical trial, with a positive and significant outcome.
Problem: Though significant, the Client now wants to evaluate if the results are clinically meaningful.
Solution: Our solution was to compare the efficacy of the drug from the Client's trial to the efficacy of all other approved treatments for the same indication. 5-10 approved treatments already existed. We compared the results from the Client's trial to the results of existing treatments by performing a systematic literature search and meta-analysis. We identified 40-80 clinical trials that studied the effect of the approved drugs, and where data could be extracted from scientific publications. With this data, we performed a meta-analysis, and were able to assess if the Client's results were clinically meaningful.
Case 2: Clinical stage Biotech company
Company Market Cap: $100m-$1B
Background: The Client is developing a drug for a disease which is uncommon, but the exact incidence and prevalence of the disease is unknown.
Problem: The client wants to know if the disease is rare enough to qualify for an Orphan Designation.
Solution: We performed a non-systematic literature search to assess the incidence of the disease. We were able to find sources which indicated that the disease was not rare enough to qualify for an Orphan Designation.
Case 3: Clinical stage Biotech company
Company Market Cap: $10m-$100m
Do you have a Special Situation that you want to discuss with us?
If you have a Special Situation in your organization, describe it in the the form below and we will contact you ASAP!