GRAP Opportunity – Post-Marketing Phase IV Studies of Type 1 Diabetes: Science or Marketing? (Kaplan)
Opportunity ID: 01-Kaplan
Project Title: Post-marketing Phase IV Studies of Type 1 diabetes: Science or marketing?
Faculty Mentor: Warren Kaplan wak@bu.edu
Description Statement:
Post-marketing research has received little scientific scrutiny. Current procedures to license a drug represent a compromise between the need to make potentially valuable new drugs available quickly and the need to assure an acceptable level of safety. This means that relatively few patients have been exposed to a given agent by the time it is licensed. The drug in question may then require further investigation in special patient subgroups, in special situations, or for other indications that might give useful pointers to its future use. Such studies may result in an application for a change in the prescribing information or “label.” Phase IV studies, defined by ClinicalTrials.gov as “post-marketing studies [which] delineate additional information including the drug’s risks, benefits and optimal use,” are intended to fulfil this function.
A further and more recent development has been the observational study, defined as “a study …without recourse to experimental intervention.” From the regulatory standpoint, pharmacovigilance is the main justification for observational studies. The US Food and Drug Administration has advised that “postmarketing safety data collection and risk assessment based on observational data are critical for evaluating and characterising a product’s risk profile.” Drug companies often seek added value from post-marketing studies. This may (for example) be obtained by investigations designed to show clinical effectiveness as well as safety. Since such studies typically introduce the new drug to many clinical investigators in different environments, they also serve a tacitly acknowledged marketing function. Post-marketing studies thus represent a fine balance between the marketing advantage sought by the companies and the safety data sought by the regulators.
Insulin is a very well-studied protein and the only therapeutic treatment for Type 1 diabetes so one might infer that post-marketing studies would not be necessary as insulin has been in the clinic for many decades. It would be interesting to see what kinds of post-marketing studies are being performed for this relatively rare condition and for this single medicine. Are they primarily pharmacovigilance studies? Studies on devices? Studies on other, non-insulin medicines?
The research consists of:
- A ‘datamining” analysis of clinicaltrials.gov, the registry and results database of public and private supported clinical studies of humans around the world.
- An analysis of the relevant descriptive information in the post-marketing clinical trial population for Type 1 diabetes.
Scope of Work:
This work is intended to be a preliminary descriptive analysis of the post-marketing clinical trials conducted for Type 1 diabetes around the world. Insulin is the only known therapeutic agent to treat this condition.
We would hypothesize that post-marketing studies for Type 1 diabetes would be primarily NOT related to pharmacovigilance, i.e., adverse events, we suspect such post-marketing studies are related more to devices that deliver insulin and use of other medicines besides insulin. We would expect that the number of participants in these trials is low, given the relative rarity of Type 1 diabetes- which also raises some questions about study intent. Factors to be analysed are: type of sponsor (industry, university), location of trial (country) number of participants, purpose of trial, outcomes, type of study (interventional/observational).
We may extend this analysis ‘backwards” into Phase 3 trials to see if there are differences between these clinical trial phases.
Minimum skills required/desired:
Good to excellent familiarity with SAS or “r” or SPSS or STATA and Excel®
Time / Date Expectations :
From January 2016-June 2016: We would expect no more than 2-3 hours per week
Additional Material Requested: None
Number of Positions: One (1) to Two (2)
Logistics & Support:
