The next 1 0 years A regulatory view Presented by: Jordi Llinares - PowerPoint PPT Presentation

Nov 27, 2023 •92 likes •197 views

The next 1 0 years A regulatory view Presented by: Jordi Llinares Head of Orphan Medicines An agency of the European Union Some headlines I hope I never see (and I am not playing with fear politics) EU bureaucrats kill patients hope

The next 1 0 years A regulatory view Presented by: Jordi Llinares Head of Orphan Medicines An agency of the European Union
Some headlines… I hope I never see (and I am not playing with fear politics) EU bureaucrats kill patient’s hope Greedy industry push health system to the limit National budgets insensitive to rarity National budgets insensitive to rarity Patient ’ s Icarus flight Presentation title (to edit, click View > Header and Footer) 2
The future -It is on us, depends on our actions today -Individual responsibility - Aim shared by different stakeholders Even though we have different short term objectives Responsibility to build on something that already works Different stakeholders to play different roles - keep pushing, but safely Presentation title (to edit, click View > Header and Footer) 3
Regulator’s responsibilities / opportunities ( I ) a. Keep balance between development chances and data required (at designation and authorisation) b. Wise management of challenges -Sub setting policy (consistency and communication) -Biological advances and application for designation c. Transparency - Better communication on designation and SB – collaboration with Member states and HTA bodies Presentation title (to edit, click View > Header and Footer) 4
Regulator’s responsibilities / opportunities ( I I ) d. Further assistance / incentives - Pre designation assistance (?) e. Advice to EC : full implementation of the COMP role (research and policy) f. Monitoring orphan drugs: -Development -After marketing: registers, shortages of supply Presentation title (to edit, click View > Header and Footer) 5
The future as I see it (I) - Debate about regulation (not future anymore) - Need for in depth assessment - Legal advice (amendment?; to open all or part of text?) - What needs change - Risk assessment -Increased transparency - Procedural / outcome - Development (clinical trials) Presentation title (to edit, click View > Header and Footer) 6
The future as I see it (II) - Increased international collaboration - Development - Post marketing issues - Consolidation of EU structure on rare diseases - Will strengthen rare diseases position - Opportunity to fight back misconceptions Presentation title (to edit, click View > Header and Footer) 7
The people / resources We will need dedicated people (like you) Ideas and challenges Continuous support to Agency and fee reduction policy Collaboration, discussion and more collaboration Presentation title (to edit, click View > Header and Footer) 8
And a big thanks to all those at the Agency that made this possible… . Agnieszka Cinzia Patrick Stiina Agnes Eric Marco Francesco Melanie Federica Spiros Teresa Bo Channa Florence Marisa Sandra Johanna Monica Sarah Frida Thomas and Paolo Cathrin Juan more … Driss Stelios Presentation title (to edit, click View > Header and Footer) 9

Recommend

D r o u g h 33.6 -45.5 33.6 -45.5 11 years 5.4 years 11 years 5.4 years years t

D r o u g h 33.6 -45.5 33.6 -45.5 11 years 5.4 years 11 years 5.4 years years t years Figure 2. Precipitation anomalies during 11 years of the dust bowl (1930-1940) Station Set-up Montana Mesonet January 2018 4 36 ranchers

533 views • 36 slides

CLASS OF 2021 Four years of English Three years of social studies Three years of

CLASS OF 2021 Four years of English Three years of social studies Three years of science Three years of mathematics Two years of a world language Four years of physical education Five credits of visual/performing arts

1.04k views • 72 slides

CLASS OF 2023 Four years of English Three years of social studies Three years of

CLASS OF 2023 Four years of English Three years of social studies Three years of science Three years of mathematics Two years of a world language Four years of physical education Five credits of visual/performing arts

915 views • 67 slides

CLASS OF 2020 Four years of English Three years of social studies Three years of

CLASS OF 2020 Four years of English Three years of social studies Three years of science Three years of mathematics Two years of a world language Four years of physical education Five credits of visual/performing arts

1.25k views • 88 slides

Who Cares About the Impact on Performance Memory Hierarchy? Suppose a processor executes at

8/20/08 Technology Trends Computer System Architecture Capacity Speed (latency) Logic: 2x in 3 years 2x in 3 years DRAM: 4x in 3 years 2x in 10 years Memory Part I Disk: 4x in 3 years 2x in 10 years DRAM Chalermek

290 views • 5 slides

Nature Education Kickoff 2018 Years of Service Recognition Heidi Brown 10 years Lois Peterson 10

Nature Education Kickoff 2018 Years of Service Recognition Heidi Brown 10 years Lois Peterson 10 years Tom Taber 10 years Gary Keeth 15 years Betty Murray 15 years Bob Walker 15 years Jackie James 25 years Carol Matthews 25 years Program

396 views • 22 slides

Who Am I 10 years factory planing 4 years live sience 3 years research assistent 12

Now You Got a Search Engine MICES 14. June 2017 Peter Rieger Who Am I 10 years factory planing 4 years live sience 3 years research assistent 12 years search Dropped 3 courses of study Freelancer for 12 years CEO for 8 years

487 views • 20 slides

2 Years of Real World FP at REA @KenScambler Scala Developer at Me 14 years 5 years 5 years

2 Years of Real World FP at REA @KenScambler Scala Developer at Me 14 years 5 years 5 years when possible when bored when forced - 3 teams @ - 17 codebases - 43K LOC Jul 13 Jan 14 Jul 14 Jan 15 Why Functional Programming?

1.95k views • 160 slides

ALL ABOUT ME JULIE EDWARDS 25 years in Marketing/PR 8 years in NPO Management 4 years

ALL ABOUT ME JULIE EDWARDS 25 years in Marketing/PR 8 years in NPO Management 4 years as Executive Director Mom to rescues Roxie & Charlie Vegetarian home chef Lover of naps, folk art and happy hours Show

132 views • 11 slides

Floral Design Committee Kickoff 2018 Years of Service Recognition Irene Kane - 10 years Joan

Floral Design Committee Kickoff 2018 Years of Service Recognition Irene Kane - 10 years Joan Upton -10 years Michelle Bosschart -16 years Debbie Kundrat - 20 years Georgine Premo - 20 years Marian Vanden Bosch - 20 years Meeting Agenda 1)

112 views • 10 slides

Migrate Python from 2.X to 3.X WHO AM I? C++ & PYTHON DEVELOPER 6,5 years in 20,5 years in

Migrate Python from 2.X to 3.X WHO AM I? C++ & PYTHON DEVELOPER 6,5 years in 20,5 years in CAD&Numeric C++ simulations PHILIPPE BOULANGER 3 years in 19,5 years in embedded Python programming 11,5 years in finance

879 views • 51 slides

www.agilegurgaon.com Who am I? 1. 20+ years of experience , 18+ in IT and 2 years in

www.agilegurgaon.com Who am I? 1. 20+ years of experience , 18+ in IT and 2 years in manufacturing 2. 6 years of experience in Agile Transformation 3. Clients worked - HP Product Division, Symantec, SKF, Healthways and Philips 4. Currently

643 views • 21 slides

Art Committee Kickoff 2018 Years of Service Recognition Debra Symons - 10 years Pathamar Park -

Art Committee Kickoff 2018 Years of Service Recognition Debra Symons - 10 years Pathamar Park - 11 years Elizabeth Holman - 15 years Marian Vanden Bosch - 20 years Lillian Balliet - 39 years Meeting Agenda 1) Jane Fernald & Judy Webster,

303 views • 12 slides

Hounslow Highways Hounslow Highways 25 years and beyond First 5 Years 25 years and beyond

Hounslow Highways Hounslow Highways 25 years and beyond First 5 Years 25 years and beyond www.hounslowhighways.org Hounslow PFI Update July 2017 INTRODUCTION What is included in the PFI? Core Investment Period Progress

456 views • 22 slides

BUILDING ON LIBBITCOIN L I S B O N 2 0 1 8 Libbitcoin Developer (5 years) E R I C VO S K U

BUILDING ON LIBBITCOIN L I S B O N 2 0 1 8 Libbitcoin Developer (5 years) E R I C VO S K U I L Entrepreneur (20 years) eric@voskuil.org Investor/Advisor (12 years) https://github.com/evoskuil Microsoft Architect (3 years)

479 views • 17 slides

Years Down Years To Go A WALK A W A L K A R O U N D Q U E E N AROUND E L I Z

Years Down Years To Go A WALK A W A L K A R O U N D Q U E E N AROUND E L I Z A B E T H O L Y M P I C P A R K QUEEN ELIZABETH OLYMPIC PARK LEYTON Clapton Park Forest Gate Homerton Maryland HA HACK HA

424 views • 15 slides

A Community Where You Belong Laying The Foundation For The Next 100 Years Our First 100 Years

A Community Where You Belong Laying The Foundation For The Next 100 Years Our First 100 Years The Indiana Masonic Home was established to care for widows and orphaned children of Masons in 1915. Through the years, the community grew and demand

402 views • 25 slides

Page 1 Example: Branch Stall Impact Example: Calculating CPI bottom up Run benchmark and collect

Review, #1 CS252 Technology is changing rapidly: Graduate Computer Architecture Capacity Speed Lecture 2 Logic 2x in 3 years 2x in 3 years DRAM 4x in 3 years 2x in 10 years Review of Instruction Sets, Pipelines, and Disk 4x

496 views • 13 slides

WELCOME CLASS OF 2024 8 TH GRADE OPEN HOUSE MINIMUM GRADUATION REQUIREMENTS Four years of

WELCOME CLASS OF 2024 8 TH GRADE OPEN HOUSE MINIMUM GRADUATION REQUIREMENTS Four years of English Three years of social studies Three years of science Three years of mathematics Two years of a world language Four years of

869 views • 66 slides

Jacobs Life: In Egypt 17 Years In Assyria (Laban) 20 Years In Canaan 110 Years Life

And Jacob lived in the land of Egypt seventeen years. So the length of Jacobs life was one hundred and forty-seven years. Gen 47:28 Jacobs Life: In Egypt 17 Years In Assyria (Laban) 20 Years In Canaan 110 Years Life of

350 views • 34 slides

Training in the UK All trainees now have 5-6 years undergraduate; 2 years postgraduate prior

3/7/2015 Training in the UK All trainees now have 5-6 years undergraduate; 2 years postgraduate prior to full registration Surgical training then 6-8 years (+/- 1-3 years Training and practice of breast MD/PhD research) surgery:

275 views • 6 slides

F ROM THE C ARTEL TO A F IRST -T IME U SER The Challenges of Drug Prosecution in an Opioid

F ROM THE C ARTEL TO A F IRST -T IME U SER The Challenges of Drug Prosecution in an Opioid Epidemic INDIANA CRIMINAL CODE - 2014 LEVEL MINIMUM ADVISORY MAXIMUM Murder 45 years 55 years 65 years Level 1 20 years 30 years 40 years

841 views • 55 slides

Data Clustering: Data Clustering: 50 Years Beyond K means 50 Years Beyond K means 50 Years

Data Clustering: Data Clustering: 50 Years Beyond K means 50 Years Beyond K means 50 Years Beyond K-means 50 Years Beyond K-means Anil K. Jain Department of Computer Science Michigan State University King-Sun Fu King-Sun Fu King-Sun

1.03k views • 39 slides

The Hundred Years War Target Describe the causes of the 100 years war, the advantages and

The Hundred Years War Target Describe the causes of the 100 years war, the advantages and disadvantages, and the 3 phases of the war. Long Term Causes English wanted to regain lost lands from the French Economic rivalry (Flanders

617 views • 16 slides