How to Work with your Statistician

Transcription

1 How to Work with your Statistician Fang-Shu Ou Assistant Professor of Biostatistics Mayo Cinic Aiance Fa Group Meeting November 2, 2017

2 Outine Why you need a statistician What statisticians do How to work with your statistician Concusion

3 Why Do You Need a Statistician? In cinica research, we use treatment effect observed in patients treated on a tria (sampe) and generaize it to a patients (popuation) with those conditions Concusions drawn from trias assume patients on the tria are representative sampe of the popuation based on statistica inference hypothesis testing, estimation

4 Why Do You Need a Statistician? Statistica tests are vaid under ony certain assumptions and statisticians are trained to understand these assumptions They can hep ensure Study is designed in a way that Satisfies the appropriate assumptions Minimizes bias and confounding factors Appropriate anaysis is panned at tria competion

5 Why Do You Need a Statistician? For secondary anaysis The statistician who works on the origina tria knows the data best Provide insight toward whether certain anaysis is feasibe based on the avaiabe data

6 What Can a Statistician Do? Design stage Provide scientific input Transate cinica questions into statistica hypotheses Participate in tria design - not just sampe size cacuations (more on this ater) Formuate an anaysis pan Provide input in data coection pan CRFs Database

7 What Can a Statistician Do? During tria Safety monitoring DSMB report Interim anaysis End of tria Anayze data Provide anaysis report Participate in abstract/manuscript writing

8 What Can a Statistician Do? Secondary anaysis Transate cinica questions into statistica hypotheses Coaborate on proposa writing for use of NCTN cinica tria biospecimens (power cacuation, anaysis pan, and beyond) Anayze data Provide anaysis report Participate in abstract/manuscript writing

9 Utimate Goa Hep design and conduct feasibe, vaid, and successfu* trias and secondary anayses *successfu positive

10 How to Work with your Statistician

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12 Contact your statistician as eary as possibe Most statisticians are invoved in mutipe projects In addition to methodoogy research, teaching, and service Assigned to mutipe committees Interactive and iterative process (more on this ater) Provide sufficient time to pan the study

13 What to expect at the initia meeting? Provide background information Disease we want to understand the science behind the study What is known about disease or treatment What does statistician want to know? Objectives of study (your hypothesis) Outcomes/endpoints to assess objectives How are they measured At what time point

14 What to expect at the initia meeting? Your initia pan (iterative) Eigibiity Accrua rate/duration Sampe size imitations if exists Timeine Funding not appicabe for Aiance-funded studies

15 What to expect at the initia meeting? For secondary anaysis Bring your preiminary hypothesis Bring a few pubished manuscripts (for scientific background) Be prepared to expain the science

16 Tria Eements Affecting Study Design Background Objectives/Endpoints Patient eigibiity Protoco intervention(s) Foow-up schedue Phase Number of arms Treatment assignment Binding Interim anaysis Sampe size Tria duration

17 How Background Affects Study Design Purpose Summarizes previous work Justifies the new tria Provides Outcome estimates (historica contro) Treatment effect Design eements affected (a) Phase Number of arms Randomization/binding Interim anaysis Sampe size Tria duration

18 How Objectives/Endpoints Affect Study Design Purpose - Defines objectives/endpoints Provides Type of response Response time point Design eement affected: Phase Number of arms Randomization Sampe size (methodoogy) Tria duration (timing of response)

19 How Eigibiity Affects Study Design Purpose Sets criteria for patients to be incuded in tria Provides Type of patients incuded in tria Disease, stage, moecuar profie Age, performance status etc Design eement affected Sampe size Outcome estimate (historica contro) Adherence, withdrawa, drop-out Enroment rate

20 Interactive and Iterative Process Stating research aims (objectives) Primary? Secondary(ies)? Determining outcomes (endpoints) Toxicity? PFS? Choosing experimenta design Singe arm? Randomized? Deveoping anaysis pan Descriptive statistics? T-test? Surviva anaysis? Sampe size justification

21 Hypothesis is the Driver for Secondary Anaysis Hypothesis defines your Popuation Outcome Variabe of interest The reationship between outcome and variabe of interest We defined hypothesis is the key to successfu secondary anaysis project

22 Concusion Contact your statistician eary and often A good pan is the key to success Stay engaged and be interactive

23 Thank You!