Journey  towards  FOP  treatment

Stéphanie  HoffmannVice-­‐President,  General  Manager  EUMEA  

CORD  Rare  Disease  Day  conference,  Vancouver,  30-­‐31  March  2017

The  Clementia  team  is  dedicate,  experienced,  and  focused

July  23,  2014 2

Our  goal  is  to  ensure  that  every  FOP  patient  who  needs  palovarotene  has  access  to  it

u Parallel  development  tracks  to  accomplish  our  goal:

1. Palovarotene clinical  development  program  for  FOP  aiming  at  obtaining  relevant  regulatory  approvals

2. Development  of  robust  commercial  infrastructure  capable  of  supplying  palovarotene  throughout  the  world  which  meets  the  various  national  requirements

3. Collaborate  with  the  FOP  community  to  ensure  access  to  palovarotenefor  all  FOP  patients  in  medical  need  

3-­‐-­‐CONFIDENTIAL-­‐-­‐

FOP  Narrative  Project:  Objectives  &  Methodology  

Objectives  u Understand  where/when  FOP  patients  intersect  local  healthsystems  u Anticipate  if/how  healthsystems  would  adapt  to  innovations  in  FOP  treatment

u 2-­‐4  year  project  in  which  learnings  from  each  stage  could  be  integrated  into  the  next

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Project  design  and  materials

Interviews  with  patients

Interviews  with  FOP  savvy  physicians

Surveys  with  FOP  naïve  physicians  

FOP  Narrative  Project:  Timeline

u 71 affected  individuals  from  14  countries  have  been  interviewed§ Recruitment  via  the  national  patient  organization  leaders  

u 39 ‘FOP  savvy’  physicians  from  15  countries  have  been  interviewed  § Recruitment  via  the  national  patient  organization  leaders,  via  FOP  medical  experts  and  at  international  medical  conferences  

u 305 US  physicians  ‘naive  to  FOP’  have  been  surveyed

1.  Patient  Interviews  

3.    Naïve  Physician  Research  (US)

2015

2.    FOP  Physician  Interviews  

2016 2017 2018

FOP  Narrative  Project:  General  Observations

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BIRTH

Confirmatory  Dx  

Path  to  Dx  

Long-­‐term  Mgmt

FOP  Tx

Ø Delayed  by  multiple  referrals;  risk  of  iatrogenic  harm

Ø Starts  with  primary  care;  referrals  influenced  by  presentation;  many  specialties  involved

Ø Accelerated  by  educated  parents

Ø Primarily  performed  in  university  hospitals  by  a  pluridisciplinary  team,  with  a  key  role  of  the  geneticist

Ø Genotyping  is  not  common  practice  in  all  countries;  technology  not  widely  available  &  cost  barrier

Ø Lack  of  local awareness  &  expertiseØ Most  patients  in  contact  with  F.  Kaplan/PennØ Challenging  transition  pediatric  to  adult  careØ Bone  specialists  &  geneticists  most  likely  

managing  physicians  – in  tandem  with  GP’sØ Overall  good  coverage  for  health  services;  

country/  regional  differences  with  regards  to  supportive  tool  coverage

Ø High  interest  of  medical  community  in  developing  FOP  Tx

Ø Bone  specialists  &  geneticists  most  likely  prescribers

Ø Prescription  authorization  most  likely  limited  to  selected  experts  or  specialties  in  EU  &  ROW

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FOP  Narrative  Project:  Conclusions

u This  project  is  confirming  our  hypothesis§ Health  systems  tend  to  be  indifferent  about  ultra-­‐rare  diseases  until  an  innovation  in  treatment

u This  project  is  informing  our  planning§ Identifies  key  challenges  &  opportunities  along  the  diagnosis  and  treatment  pathway  of  FOP  patients  around  the  world

§ Guides  us  in  defining  priority  planning  initiatives:  - FOP  awareness  &  education,  - Support  of  FOP  healthcare  infrastructure  development

u We  feel  responsible  and  committed  to  facilitate  access  § In  close  collaboration  with  medical  &  patient  communities

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Creative  Solutions  in  Market  Access  -­‐ 2016

THANK  YOU!

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