2023全球医药研发现状分析-52页-WN6.pdf

《2023全球医药研发现状分析-52页-WN6.pdf》由会员分享，可在线阅读，更多相关《2023全球医药研发现状分析-52页-WN6.pdf（52页珍藏版）》请在淘文阁 - 分享文档赚钱的网站上搜索。

1、Pharma R&D Annual Review 2023by Ian Lloyd,Senior Director,Pharmaprojects at CitelineWhite PaperApril 2023Reimagining the futureone chapter at a timeWelcome to Pharmaprojects 2023 review of trends in pharmaceutical R&D.For over 30 years now,Ive been taking an annual look at the evolution of pharma R&

2、D,and in this article,Ill examine the state of play at the start of 2023.Well assess industry trends by examining the pipeline by company,therapeutic area,disease,target,and drug type,using data from primarily Pharmaprojects,part of the Citeline suite of products,which has been tracking global drug

3、development since 1980.This report will be followed up by our annual supplement reviewing the New Active Substance launches for the year just passed.But here,well tell the story of pharma R&D as it is today:an epic tale,with a rich cast of characters and a global scope,and no doubt a number of unexp

4、ected plot twists.Hopefully,it will be a real page-turner!Regular readers of this report(which has been running since 1993,so is presented here in its 31st edition)will know that in recent years,Ive threaded a different theme through each edition,to highlight points,to draw analogies,and to add a li

5、ttle character into what could otherwise be a rather lengthy narrative through a potentially dreary parade of statistics,charts,and tables.Themes selected so far have included astronomy,movies,the natural world,music,food and drink,science fiction,and,last year,travel.This year,I decided to make a f

6、ew nods to the world of literature and books to help illustrate my points.Storytelling is as old as humanity itself,and fiction has always been used as a proxy to help us to understand the real world.The best literature enlightens,thrills,amuses,and engages the reader.Im certainly no Shakespeare,but

7、 I hope that my words will throw some light on the current state of the pharmaceutical industry,in the same way that a great novel does on the human condition.So,what will be my opening lines to this years magnum opus?A memorable set of bon mots to pique the interest of the reader is important.Vario

8、us online polls pick different books as having the best attention-grabbing first sentences,but a number come up regularly in top 10s,so there is some degree of consensus of what constitutes a classic first sentence construction.Among those repeatedly cited are:“It is a truth universally acknowledged

9、,that a single man in possession of a good fortune,must be in want of a wife”Jane Austen,Pride and Prejudice(1813);“It was a bright cold day in April,and the clocks were striking thirteen”George Orwell,1984(1949);“If you really want to hear about it,the first thing youll probably want to know is whe

10、re I was born,and what my lousy childhood was like,and how my parents were occupied and all before they had me,and all that David Copperfield kind of cr*p,but I dont feel like going into it,if you want to know the truth”JD Salinger,The Catcher in the Rye(1951),and“All this happened,more or less”Kurt

11、 Vonnegut,Slaughterhouse-Five(1969).But perhaps one of the most famous opening lines to a novel in history which best describes the experience of pharma over the past few years can be borrowed from Charles Dickens seminal 1859 opus,A Tale of Two Cities:“It was the best of times,it was the worst of t

12、imes,it was the age of wisdom,it was the age of foolishness,it was the epoch of belief,it was the epoch of incredulity,it was the season of Light,it was the season of Darkness,it was the spring of hope,it was the winter of despair.”Of course,its the COVID-19 pandemic that Im thinking of here.A globa

13、lly cataclysmic event,it threw the entire world into turmoil,and placed the pharmaceutical industry firmly at the center of the action.Suddenly,drug R&D was supposed to ride to a heroic rescue,but at the same time,a whole set of new barriers were being thrown in the way of its delivering the much-de

14、sired happy ending.Clinical trials had to be paused,lab workers found themselves marooned at home,and the whole script for drug development had to be rewritten.Introductioncontinued on next page.Introduction ContinuedBut,as any good author will tell you,without jeopardy,theres no tension and thus no

15、 story.What has followed,during a time when many of us felt like we were living through a dystopian drama worthy of Margaret Atwood,was an almost Homeric quest for a solution to an almost unprecedented crisis.With the stoicism worthy of Thomas Hardys Gabriel Oak(Far from the Madding Crowd,1874),the

16、industry hunkered down and just got on with it.Almost unbelievably,the golden fleece of a vaccine was found in an incredibly short timespan its discovery turned out to be more a novella than the expected War and Peace-like weighty tome by Leo Tolstoy(1869)like weighty tome allowing the world to retu

17、rn rapidly to some kind of normality,and the pharma industry to emerge as a Gandalf-ish wizard leading the people to peace and prosperity.At the time of writing,in January 2023,it feels,in the West at least,as though this dark episode in our lives has largely drawn to a close.However,with China unex

18、pectedly and suddenly abandoning its much-vaunted but ultimately disastrous zero-COVID policy,the story has clearly not quite finished its final act.It is expected that China will now undergo the biggest waves of infection which the world has yet seen.Quite apart from the devastation likely to be wr

19、eaked domestically,the consequences for the world at large remain to be seen.Theres nothing that the coronavirus likes more than a huge cast of supporting characters in which to ferment the potential curveball of a new variant.As serialized novels often state,To be continuedBut it appears that the p

20、harmaceutical industry is emerging from this tumultuous episode stronger and emboldened,and,while maintaining an independent critical eye,to misquote Shakespeares Julius Caesar(1599):“Friends,Romans,countrymen:We are here(largely)to praise pharma,not to bury it.”Trends in the pharmaceutical industry

21、 can be as opaque to the onlooker as that famously unreadable novel,James Joyces Ulysses(1922),so my aim here is to unravel all the competing and conflicting plot strands threading through the story of pharmaceutical drug development as it stands at the start of 2023.Think of me as the literary crit

22、ic adding companion notes and explainers to the main text of the tale.Im hoping that my text will serve as footnotes or an explanatory commentary which will help you untangle the story of R&D today,its characters and its nuances.In many ways,each drug undergoes a journey like any literary character.

23、We are introduced to each new molecule at the start of the story during its preclinical development,where we find out about its defining qualities,its strengths and weaknesses,and its potential role in the emerging story.As we go through the clinical stages of development,some drugs move forward,the

24、ir narrative enhanced by positive outcomes,whereas some stumble and fall on Hard Times(Charles Dickens,1854),and other characters are completely killed off.As the will-they-wont-they plotline reaches its denouement,the dramatic tension climaxes as we wait to see whether the powers-that-be will give

25、us that longed-for happy ending,or seal the drugs fate to a more downbeat final act.If Alls Well That Ends Well(William Shakespeare,1623),the pharma companies responsible for our molecules success will soon be embarking on a sequel.So,settle down in your favorite chair,pour yourself a mug or a glass

26、 of whatever is your chosen tipple,and lets open the pages on the story of 2023.Guiding you through the cast of characters and the inevitable twisty-turny plot,Ill be your narrator.If youre sitting comfortably,Ill begin.“The beginning is always today”Mary Shelley,Short Stories Vol.II(2014)ContentsCh

27、apter One An Introduction:Total Pipeline Size 05 Pharmas story has a bigger word count than everChapter Two Setting the Scene:The 2023 Pipeline by Phase 07 More words,but does that mean longer,less-digestible sentences?Chapter Three Dramatis Personae:Top Companies 10 Main characters take a smaller r

28、ole in our cast of thousandsChapter Four The Motivations Propelling Events:Top Therapies 21 Cancer still the industrys main call to armsChapter Five Deus Ex Machina:Mechanisms and Targets 37 Immuno-oncology leading subtle shifts in most popular proteins to hitChapter Six Modus Operandi:Types of Pipe

29、line Drugs 43 To biotechnology or not to biotechnology that is the questionEpilogue:Where Next for the Pharma Saga?47 Could global events send the industry off topic?About the Author 514April 2023 Copyright 2023 Pharma Intelligence UK Limited,a Citeline company.5April 2023 Copyright 2023 Pharma Inte

30、lligence UK Limited,a Citeline company.As a forward to this years tome,lets set the scene with an overview of the scope of our story something akin to the maps of Middle Earth often presented in the frontispieces of the novels of J.R.R.Tolkien by looking at the total number of drugs currently in the

31、 R&D“pipeline.”All of the analyses in this report will be focusing on this set of drugs,so its worth starting off with a definition of what we exactly mean by the term“pipeline.”Here,we are counting all drugs in development by pharmaceutical companies,from those at the preclinical stage,through the

32、various stages of clinical testing and regulatory approval,and up to and including launch.Launched drugs are still counted,but only if they are still in development for additional indications or markets.Drugs whose development has been terminated,or is complete,are not included.All data were collect

33、ed on 34 of January 2023.With the past few volumes of the drug development saga so full of incident and intrigue,now is perhaps the time to take stock and get some perspective on where our world is and its direction of travel.Figure 1 certainly shows that this episode has once more increased its wor

34、d count,as the overall pipeline size has again hit a new high,with 21,292 drugs in development.This represents a 5.89%expansion of 2022s number,which is admittedly a lower growth rate than that seen last year of 8.22%.However,it is up from 2021s 4.76%,and,crucially,is not lagging far behind the 6.90

35、%which is the five-year average growth rate.So,perhaps it is a more stable and sustainable increase as pharma settles down after the dramas of the past three years.Chapter One An Introduction:Total Pipeline SizePharmas story has a bigger word count than everFIGURE 1:Total R&D pipeline size,by year,2

36、00123Source:Pharmaprojects,January 202320015,00010,00015,00020,00025,000200220032004200520062007200820092010201120122013201420152016201720182019202020212022DRUG COUNT5,9956,1986,4166,9947,3607,4067,7379,6059,7379,71310,45210,47911,30712,30013,71814,87215,26716,18117,73718,5829,21720,109202321,292YEA

37、R6April 2023 Copyright 2023 Pharma Intelligence UK Limited,a Citeline company.This means that there are 1,183 more drugs in development than there were this time last year,compared to the 1,527-drug rise seen last year,and the 845-drug uplift reported the year before.In total,5,082 new drugs were ad

38、ded to the Pharmaprojects database during 2022 considerably fewer than the 6,343 added during 2021.But there was definitely something of a“COVID-bounce”contribution to that years figure,as last years number was still in excess of the 2020 number of 4,730.As our continuous reviews of companies pipeli

39、nes get ever more up to date,it certainly feels like adding around 5,000 new drugs each year is a truer representation of the number of new candidates advanced into development in a 12-month period.But,as noted earlier,the size of our subject material is not just governed by additions,but also by wh

40、at has been edited out.To end up with a net surplus of 1,183 drugs,having added 5,082 drugs,must mean that 3,899 candidates exited(pursued by a bear?)the R&D pipeline.During 2022,282 drugs were confirmed as being discontinued(up from 2021s 221),while a further 3,804 were moved out of the“Active”data

41、set as a result of being marked as“No development reported”(down from 4,658 the previous year note that the figures dont exactly sum as drugs can move in and out of development in a single year).Overall,there was somewhat less churn seen during 2022,although the rate of flux is still pretty remarkab

42、le.Of our new protagonists,where are their primary allegiances?Over 40%of newly identified drug candidates 40.7%to be precise are targeted against at least one form of cancer.This is up from the previous years figure of 38.8%,leaving neurologicals,with 13.5%,a distant second.Interestingly,19.5%of ne

43、w candidates are focused on one or more rare disease a significant rise on the 2021 figure of 17.5%.In terms of which companies contributed the most to the increased word count,its fascinating to note that for the first time it was a Chinese company,Jiangsu Hengrui Pharmaceutical,leading the pack he

44、re.The more traditional contributors Pfizer and Roche followed next.As regards to the location of development of 2022s batch of neophyte drugs,the US is still far out in front with 1,840 candidates,but China is coming up on the rails with 1,457.Of course,well be examining the pipeline as a whole by

45、all of the above measures in much more detail later.So,this years book of R&D is even more of a door-stopping magnum opus than ever.As always,we caveat the excitement at a bigger-than-ever pipeline with the warning that the vast majority of these drugs are still prelaunch,so are costs with risks att

46、ached rather than successful revenue-generators curing patients.2021 once again was a record-beater in terms of the number of New Active Substances(NASs)successfully debuting onto the market,with 97 far outstripping the previous record of 82 seen in 2020.Can 2022 come close to keeping up with this p

47、henomenal output?Its possible:so far,we are reporting around 60 NASs for 2022,about the same number we saw at the same point last year.As we further refine our data towards the spring publication of our annual NAS Supplement,it will certainly be interesting to see if 2022 can get within touching dis

48、tance of 2021.After all,a bigger novel is only better if it satisfies the reader more.“I believe there is a theory that men and women emerge finer and stronger after suffering,and that to advance in this or any world we must endure ordeal by fire”Daphne du Maurier,Rebecca(1938)Chapter One An Introdu

49、ction:Total Pipeline SizePharmas story has a bigger word count than ever7April 2023 Copyright 2023 Pharma Intelligence UK Limited,a Citeline company.Pharmaceutical R&D,like great literature,has a beginning,a middle,and an end.This classic three-act structure is often cited as the overarching templat

50、e for any work of fiction,and has famously been adopted by the movie industry.Of course,most books are really more complex than that.Similarly,arguably,drug development has three major“acts”:preclinical,clinical,and regulatory,which can be broken down into smaller sections the phases of development