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The C ollabora+ve T rajectory A nalysis P roject (cTAP) Explaining varia,on in Duchenne disease progression to accelerate drug development
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The Collabora!ve Trajectory Analysis Project (cTAP)

Feb 14, 2017

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Page 1: The Collabora!ve Trajectory Analysis Project (cTAP)

The  Collabora+ve  Trajectory  Analysis  Project            (cTAP)  

       

Explaining  varia,on  in  Duchenne  disease  progression    to  accelerate  drug  development  

Page 2: The Collabora!ve Trajectory Analysis Project (cTAP)

Years  of  age  

Meters  walked  

Individual  trajectories    of  six-­‐minute  walk  distance  

Data  from  Imaging  DMD  

Page 3: The Collabora!ve Trajectory Analysis Project (cTAP)

Years  of  age  

Meters  walked  

Individual  trajectories    of  six-­‐minute  walk  distance  

Data  from  Imaging  DMD  

Page 4: The Collabora!ve Trajectory Analysis Project (cTAP)

Years  of  age  

Meters  walked  

Individual  trajectories    of  six-­‐minute  walk  distance  

Data  from  Imaging  DMD  

Page 5: The Collabora!ve Trajectory Analysis Project (cTAP)

Two  kinds  of  phenotypic  varia+on    –  and    why  we    care  

•  Larger,  longer  trials  •  Greater  risk  of  not  proving  

efficacy    

•  Disconnect  between  individual  experiences  and  research  on  average  outcomes  

•  Smaller,  shorter  trials  

•  Greater  confidence  in  trial  design    

•  Pathway  for  conAnually  improving  individualized  care  

Unexplained  varia+on  (we  don’t  know  what  causes  it)    

Explained  varia+on  (we  do  know  what  causes  it)    

Page 6: The Collabora!ve Trajectory Analysis Project (cTAP)

“Ideal”  number  of    boys    in  a  hypothe+cal  efficacy  trial  

Unexplained  variaAon  in  six  minute  walk  distance  (meters)  

350  

200  

89  24   6  

0  

100  

200  

300  

400  

100   75   50   25   10  

Unexplained  varia+on  (we  don’t  know  what  causes  it)    

Explained  varia+on  (we  do  know  what  causes  it)    

Page 7: The Collabora!ve Trajectory Analysis Project (cTAP)

How  can  we  explain  more  varia+on?  

Analyze  natural  history  data  

Discover  biomarkers  

Learn  from  trials  /  registries  

Page 8: The Collabora!ve Trajectory Analysis Project (cTAP)

cTAP  

How  can  we  explain  more  varia+on?  

Analyze  natural  history  data  

Discover  biomarkers  

Learn  from  trials  /  registries  

Page 9: The Collabora!ve Trajectory Analysis Project (cTAP)

The  Collabora+ve  Trajectory  Analysis  Project            (cTAP)  

Mission  

•  Explain  phenotypic  variaAon  in  DMD  

•  Translate  into  tools  for  trial  design/analysis  

•  Make  insights  available  to  everyone  in  drug  development  

•  Deliver  near-­‐term  impact  

 

 The  benefit:  accelerate  effec,ve  treatments  to  pa,ents  

Page 10: The Collabora!ve Trajectory Analysis Project (cTAP)

cTAP  at  year  1  who  we  are  and  what  we’ve  done  

Clinical  experts  and  registries  

•  Mercuri;  Telethon  -­‐  96  boys  •  Goemans;  UZ  Leuven  -­‐  65  boys  •  Muntoni;  Northstar  UK  -­‐  >  500  boys      …  Wong  (CincinnaA)    …  Vandenborne  (Imaging  DMD)  

Drug    developers   •  BioMarin  (Prosensa)  •  Pfizer  •  PTC  TherapeuAcs  •  Sarepta  •  Shire    …  Others  in  process  

Pa+ent  advocates   •  Cure  Duchenne  •  PPMD  •  Parents  

Analy+cs,  collabora+on  leadership  

•  Analysis  Group  •  Susan  Ward  

Page 11: The Collabora!ve Trajectory Analysis Project (cTAP)

Collabora+on:  central  to  cTAP  success  

Drug                    Developers  

Pa+ent  advocates  

Analy+cs  &  collabora+on  leadership  

Clinical  experts  

Shared  funding          The  right  ques6ons  

Clinical  insight  Pa6ent  data  

Seed,  engage,    establish,  connect,    

promote,  encourage,  advise  Data  protec6on  Analy6cal  exper6se  Dedicated  execu6on  team  

Page 12: The Collabora!ve Trajectory Analysis Project (cTAP)

The  Collabora+ve  Trajectory  Analysis  Project            (cTAP)  

Mission  

•  Explain  phenotypic  variaAon  in  DMD  

•  Translate  into  tools  for  trial  design/analysis  

•  Make  insights  available  to  everyone  in  drug  development  

•  Deliver  near-­‐term  impact  

 

 The  benefit:  accelerate  effec,ve  treatments  to  pa,ents