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EFPIA Pipeline Review 2021 Update Project Report February 2021 Disclaimer • Information contained within this document is based on publicly available information and Primary Market Research • Findings and recommendations are based on the views of IQVIA, and do not necessarily represent those of EFPIA • Please note: the research to inform this report was conducted between August and Nov 2020 © 2020. All rights reserved. IQVIA® is a registered trademark of IQVIA Inc. in the United States, the European Union, and various other countries. This document contains a pipeline overview, deep dives of the eight areas, as well as considerations for innovation access Hyperlinks for Content Navigation Pipeline overview Benefit of Innovation From Innovation to Access 1 IQVIA_EFPIA Pipeline Review 2021 - Full Report Hyperlinks to detailed content allow for easy reference and navigation of the document Slides Slides numbers numbers Pipeline overview 8-22 4. Adapting Financing Models……………………………………... 194-201 5. Developing infrastructure to support care delivery……..……202-209 Benefits of Innovation …………………………………………. 23-24 6. Optimising patient management/treatment strategies………..210-216 1. NSCLC Checkpoint Inhibitor Combinations…………………..... 25-39 7. Enabling data science and technology partnerships………….217-232 2. Disease Modifying Therapies for Alzheimer’s Disease…… …40-58 8. Horizon scanning and stakeholder dialogue………………….…233-235 3. Gene Therapies ….… 59-70 4. Cell Therapies (including CAR-Ts) 71-86 5. PPAR / FXR Agonists for NASH…………………………………… 87-102 Appendix…………………………………………………………........236-318 6. Remyelinating CNS Therapies …………… …103-120 Pipeline product details 7. mRNA vaccines for Glioblastoma ………………………………121-130 Glossary 8. Curative therapies for HBV and HIV 131-154 Project methodology Pipeline overview details From Innovation to Access…………… . .155-163 (intro) Contents page 164 1. Updating regulatory guidance & procedures 165-173 2. RWE to address payer uncertainty……………………………...... 174-180 3. Valuing and rewarding innovation……………………………...… 181-193 2 IQVIA_EFPIA Pipeline Review 2021 - Full Report Contents + Pipeline Overview + Benefit of Innovation + From Innovation to Access 3 2020 has witnessed the EMA approval of 55 new active substances, mainly in infectious diseases, immunology and haematology Cardiovascular disease Immunology Oncology / Onco-haematology Haematology Conditional approval, exceptional circumstances, and/or Dermatology Infectious disease Ophtalmology Other accelerated approval, n = 11 Gastrointestinal Neurology Rare diseases Orphan designated products, n = 12 Oncology & onco-haematology 1 2 darolutamide (prostatic neoplasms) 2 Rare diseases entrectinib (non small cell lung cancer) 2 onasemnogene abeparvovec (SMA) larotrectinib sulfate (abdominal neoplasms) 12 2 osilodrostat phosphate (Cushing’s trametinib (melanoma) syndrome) isatuximab (MM) 3 polatuzumab vedotin (B-cell lymphoma) gilteritinib fumarate (AML) 2020 Immunology belantamab mafodotin (multiple myeloma) to date1 5 glycopyrronium bromide (asthma) glasdegib maleate (AML) NAS = indacaterol (asthma) luspatercept (Myelodysplastic syndrome) 55* indacaterol acetate (asthma) avapritinib (GI stromal tumours) budesonide / formoterol (asthma) acalabrutinib (CLL) 10 indacaterol, glycopyrronium bromide, melphalan (multiple onco-haem indications) 8 mometasone (asthma) Infectious diseases ozanimod hydrochloride (MS) relebactam (Gram negative bacterial infections) ivacaftor /tezacaftor / elexacaftor (cystic imipenem monohydrate (Gram negative bacterial 8 fibrosis) infections) Vaxchora, vaccine (Cholera) upadacitinib (rheumatoid arthritis) Zabdeno, vaccine (Ebola) Pretomanid (Tuberculosis) filgotinib (rheumatoid arthritis) Mvabea, vaccine (Ebola) MenQuadfi, vaccine (Meningococcal Meningitis) Ervebo, vaccine (Ebola, hemorrhagic fever) Obiltoxaximab (Anthrax) Note: (1) As of 11.2020, (*) innovative products only excludes biosimilars, generics, influenza vaccines, indication expansions, and non-human products; Abbreviations: Spinal Muscular Atrophy (SMA), Acute Promyelocytic Leukemia (APL), Myeloid Leukaemia (AML), Chronic Lymphocytic Leukaemia, Multiple Sclerosis (MS) – link to glossary; Source: EMA European public assessment reports IQVIA_EFPIA Pipeline Review 2021 - Full Report 4 The number of new active substances approved by EMA this year increased by ~80% as compared to 2019 Cardiovascular disease Immunology Oncology Haematology Dermatology Infectious disease Ophtalmology Endocrynology Gastrointestinal Neurology Rare diseases Other 1 2 2 5 2 7 Across TAs: 12 2 3 • 7 orphan designations 3 (vs 7 in 2020 to 2020 2019 date) 1 to date1 5 NAS = 30 +80% NAS = 55 • 12 conditional 1 approvals / exceptional 3 1 10 circumstances, and/or accelerated 2 8 approvals (vs 9 in 3 2020 to date) 2 2 8 For comparison, 30 new active substances were approved by EMA in 2019, mainly for haematology, infectious diseases and cancer. This indicates that the COVID-19 pandemic has not negatively impacted the approval process for innovations coming to the market so far. Note: (1) As of 21.09.2020; Abbreviations: New active substance (NAS) - link to glossary; Source: EMA European public assessment reports; Human medicines highlights 2019 IQVIA_EFPIA Pipeline Review 2021 - Full Report 5 The volume of initiated clinical trials has increased year on year since 2015 with oncology having the most extensive pipeline Full pipeline – number of trials started in 2015-November 2020 Pipeline summary – key therapeutic areas2 [# trials started in 2020] Phase 1 TAs with share < 3% Oncology Phase 2 Infectious Disease Phase 3 Neurology +5% TBD1 5,500 5,228 5,369 Hematology* 4,903 5,000 4,850 4,789 25% Respiratory 4,441 1,271 4,500 1,305 Dermatology 1,283 1,227 1,173 4,000 Endocrinology 1,288 Cardiovascular 3,500 1,829 4% Psychiatry 3,000 1,872 1,708 1,651 1,783 Ophthalmology 2,500 4% 1,546 Gastrointestinal 2,000 4% Rheumatology 1,500 18% Women’s Health / Sexual Health 2,269 1,000 1,912 1,972 2,051 6% Allergy/Immunology 1,607 1,833 500 Hepatology 8% Nephrology 0 10% 2015 2016 2017 2018 2019 Nov 2020 Other (*) Incl. Onco-haematology The volume of pipeline activity has continued to increase over the last years. Assuming that in 2020 the average number of clinical trials launched per month will remain as in the period of Jan 2020 – Nov 2020, total number of clinical trials started in 2020 will be similar to 2019 (-3%). Source: Clarivate Analytics Cortellis, Aug 2020 (TA’s share) and Nov 2020 (total number of trials); Phase II includes Phases I/II, II, IIa, IIb. Phase III includes Phase II/III and III. Terminated trials were excluded from the analysis. Trials not industry sponsored and device trials were excluded; (1) Total number of trials started in 2020 to be defined – final number will be available in the beginning of 2021; (2) Data from August 2020 IQVIA_EFPIA Pipeline Review 2021 - Full Report 6 However, if we exclude trials related to COVID-19, the clinical activity in other areas has decreased as compared to 2019 Full pipeline – number of trials started in 2015-November 20201 Change in the number of trials excluding COVID-19 studies Other • When we exclude the clinical trials related to COVID-19, constituting COVID-19 trials ~15% of the clinical pipeline in 2020, we observe that the total number of studies launched in other therapeutic areas has decreased as 5,500 5,228 5,369 compared to 2019 4,903 4,850 5,000 4,789 • The average number of trials launched per month in 2020 (excluding 4,441 4,500 695 (15%) COVID-19 trials) is ~372, whereas the average monthly number in 4,000 2019 was ~447 (17% decrease) -17% 3,500 • Average monthly number is used in comparison due to unavailability of 3,000 full-year data for 2020 2,500 4,094 2,000 (85%) 1,500 1,000 500 0 2015 2016 2017 2018 2019 Nov 2020 The final effect of COVID-19 on the clinical development in other therapeutic areas is still to be confirmed – full 2020 data expected early 2021. Source: Clarivate Analytics Cortellis, Nov 2020; Phase II includes Phases I/II, II, IIa, IIb. Phase III includes Phase II/III and III. Terminated trials were excluded from the analysis. Trials not industry sponsored and device trials were excluded; (1) Total number of trials started in 2020 to be defined – final number will be available in the beginning of 2021 IQVIA_EFPIA Pipeline Review 2021 - Full Report 7 The impact of COVID-19 on trials geographic split is not visible to date; increasing share of Asia in clinical development is observed Full pipeline – trials started in 2010-2020 per region1 European pipeline – trails started in 2010-2020 per sub-region1 Northern America* Japan Africa and Middle East Eastern Europe** Southern Europe X% Share of clinical trials run in Asia Oceania Northern Europe Western Europe the UK in the total number of trials in Europe1 Europe LatAm 29% 17% 17% 17% 19% 20% 19% 17% 18% 19% 17% 15% 28% 27% 28% 31% 32% 29% 30% 32% 24% 32% 28% 27% 24% 31% 31% 23% 20% 23% 21% 29% 16% 19% 22% 21% 15% 14% 14% 14% 15% 22% 23% 20% 22% 23% 16% 22% 23% 25% 24% 23% 21% 31% 33% 30% 27% 29% 29% 27% 29% 31% 27% 21% 21% 21% 19% 19% 20% 29% 20% 21% 21% 20% 19% 25% 6% 5% 6% 6% 6% 6% 5% 5% 5% 20% 5% 7% 8% 8% 8% 7% 7% 7% 7% 6% 4% 8% 36% 34% 33% 32% 34% 32% 29% 30% 30% 30% 7% 5% 4% 5% 4% 6% 5% 6% 6% 6% 5% 4% 30% 6% 6% 6% 7% 7% 7% 6% 7% 7% 7% 7% 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 Nov 2010 2011 2012 2013 2014 2015 2016 2017 2018 2019 Nov 2020 2020 The geographic distribution of clinical trial location has not changed significantly compared to previous year, which indicates that the COVID-19 pandemic has not forced pharmaceutical companies to move their development activities. The long-term trend observed is the increasing share of clinical trials conducted in Asia (mainly China, South Korea), which grew from 14% in the years 2011-2013 to 24% in 2020. Source: Clarivate Analytics Cortellis, Nov 2020; Phase II includes Phases I/II, II, IIa, IIb. Phase III includes Phase II/III and III. Terminated trials were excluded from the analysis.
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  • Classification Decisions Taken by the Harmonized System Committee from the 47Th to 60Th Sessions (2011

    Classification Decisions Taken by the Harmonized System Committee from the 47Th to 60Th Sessions (2011

    CLASSIFICATION DECISIONS TAKEN BY THE HARMONIZED SYSTEM COMMITTEE FROM THE 47TH TO 60TH SESSIONS (2011 - 2018) WORLD CUSTOMS ORGANIZATION Rue du Marché 30 B-1210 Brussels Belgium November 2011 Copyright © 2011 World Customs Organization. All rights reserved. Requests and inquiries concerning translation, reproduction and adaptation rights should be addressed to [email protected]. D/2011/0448/25 The following list contains the classification decisions (other than those subject to a reservation) taken by the Harmonized System Committee ( 47th Session – March 2011) on specific products, together with their related Harmonized System code numbers and, in certain cases, the classification rationale. Advice Parties seeking to import or export merchandise covered by a decision are advised to verify the implementation of the decision by the importing or exporting country, as the case may be. HS codes Classification No Product description Classification considered rationale 1. Preparation, in the form of a powder, consisting of 92 % sugar, 6 % 2106.90 GRIs 1 and 6 black currant powder, anticaking agent, citric acid and black currant flavouring, put up for retail sale in 32-gram sachets, intended to be consumed as a beverage after mixing with hot water. 2. Vanutide cridificar (INN List 100). 3002.20 3. Certain INN products. Chapters 28, 29 (See “INN List 101” at the end of this publication.) and 30 4. Certain INN products. Chapters 13, 29 (See “INN List 102” at the end of this publication.) and 30 5. Certain INN products. Chapters 28, 29, (See “INN List 103” at the end of this publication.) 30, 35 and 39 6. Re-classification of INN products.