Life Sciences

More Precision
for Patients

Healthcare is about to enter a new paradigm. Under the heading “personalized medicine,” experts in diseases, genetics, pharmaceuticals, and IT are focusing on individualized treatments and medications in targeted ways. Insiders expect the new technologies to trigger enormous changes that will affect all stakeholders in the sector. Porsche Consulting The Magazine asked leading experts for their thoughts on these developments.

10/2022

Eight experts in healthcare, IT, genetics, and pharmaceuticals shed light on the future of medical treatment. Porsche Consulting/Lea Einbrodt
Opportunities for patients and companies
People who are ill yearn for treatments that target their individual condition. With the technologies currently available, however, treatments can be defined at most on the basis of semi-individualized processes. At the same time, it is increasingly clear that some medications do not work for everyone. While certain active substances are well tolerated by many patients, in others they can trigger drastic side effects.

Dr. Roman Hipp, Senior Partner and Global Head of Life Sciences Practice at the Porsche Consulting management consultancy, therefore sees major opportunities in the expansion of personalized treatment. “The volume of available medical data, which can be analyzed with artificial intelligence, is steadily rising. Added to this are the advances in genetics. Together, they are creating enormous potential, so that even more individualized treatments can soon become reality.”

The future of personalized medicine is already here

Dr. Roman HippDr. Roman Hipp
Senior Partner at Porsche Consulting

The key to greater suc­cess here is what is called “per­son­al­ized med­i­cine,” which essen­tial­ly means giv­ing patients the right dose of the right med­i­cine at the right time. That is not a dis­tant utopi­an dream, says Hipp. On the con­trary: “The future of this new and pre­cise form of med­i­cine is already here.”

Patients are benefiting today

One exam­ple can be seen in treat­ments for breast can­cer. Around 20 per­cent of patients with breast car­ci­no­mas are car­ri­ers of a strong­ly over­ex­pressed HER2 recep­tor. If this is con­firmed by genet­ic test­ing, per­son­al­ized treat­ment with Trastuzum­ab anti­bod­ies can be offered. The anti­bod­ies bind to HER2 recep­tors and there­by inhib­it the growth of can­cer­ous cells. The method is not effec­tive, how­ev­er, for patients who do not have an over­ex­pressed HER2 receptor.

This is one of many exam­ples already gov­erned by the prin­ci­ple of apply­ing more tar­get­ed forms of treat­ment to cer­tain groups of peo­ple. “We expect to be see­ing per­son­al­ized meth­ods applied on a wide scale as of 2030,” says Hipp, who heads the Life Sci­ences divi­sion at Porsche Con­sult­ing. “As we work up to this abil­i­ty, the health­care sys­tem as a whole will con­tin­ue to transform—in fact, we’re on the verge of a real breakthrough.”

Digitalization is driving development

A cru­cial dri­ver of this devel­op­ment is the increas­ing degree of dig­i­tal­iza­tion. Accord­ing to Hipp, the abil­i­ty to ana­lyze large vol­umes of data and infor­ma­tion will make it pos­si­ble to iden­ti­fy hereto­fore unknown caus­es of dis­ease. The use of smart­watch­es, smart­phones, and other wear­ables on an every­day basis is already gen­er­at­ing new and indi­vid­u­al­ized infor­ma­tion. “Real-world data are enabling sci­en­tists to devel­op even more effec­tive treat­ments and help­ing doc­tors make their diag­noses ever more precise.”

But that is just the begin­ning. While Ger­many is still debat­ing the ques­tion of dig­i­tal health records, many experts are already talk­ing about dig­i­tal twins. These are dig­i­tal copies of each and every indi­vid­ual, con­tain­ing infor­ma­tion about their bio­log­i­cal func­tions, gene expres­sion, and needs for pre­ven­tive care. As Hipp explains, “In the future we’ll be able to test poten­tial ther­a­pies on a dig­i­tal twin first in order to assess their effec­tive­ness as well as their side effects.”

New opportunities for companies

Per­son­al­ized med­i­cine is promis­ing not only for patients. It is also open­ing new hori­zons for com­pa­nies. “The impor­tance of per­son­al­ized med­i­cine will keep grow­ing for every­one involved in health­care, and ulti­mate­ly lead us to rethink what we mean by health,” says Hipp. “Before reach­ing that stage, busi­ness lead­ers, pol­i­cy­mak­ers, and soci­ety as a whole will have to mas­ter the chal­lenges that inevitably accom­pa­ny new developments.”

Three-phase model for success

In his work as a con­sul­tant and busi­ness expert, Hipp con­cen­trates on com­pa­nies seek­ing to seize new oppor­tu­ni­ties in a con­text of dra­mat­ic change. These com­pa­nies gen­er­al­ly need to think through three phas­es before they invest in per­son­al­ized med­i­cine as a busi­ness field (see info box). The first step con­sists of strate­gic deci­sions on the extent to which they want to add per­son­al­ized treat­ments such as cell and gene ther­a­pies to their port­fo­lios. “The main ques­tion here is whether they pos­sess the core exper­tise or can find part­ner com­pa­nies to enter this new market—because only then does it make sense to invest,” says the expert from
Porsche Con­sult­ing. If a com­pa­ny can answer this ques­tion with yes, the sec­ond phase con­sists of “align­ing process­es, orga­ni­za­tion­al struc­tures, and employ­ee abil­i­ties toward the tar­get. To put the strat­e­gy suc­cess­ful­ly into prac­tice, they will also have to effi­cient­ly man­age small-scale pro­duc­tion process­es and a more com­plex sup­ply chain.”


Inga Bergen: “We need a holistic take on health”

Katja Hentschel

Inga Bergen is an entre­pre­neur and expert on inno­va­tion and dig­i­tal­iza­tion in the health­care sec­tor. She built up the well­doo dig­i­tal health soft­ware ser­vice provider as its CEO, as well as the Magnosco start-up, which is advanc­ing skin can­cer diag­nos­tics with the help of laser sys­tems and arti­fi­cial intel­li­gence. In her Visionäre der Gesund­heit (“health­care vision­ar­ies”) pod­cast she dis­cuss­es cut­ting-edge top­ics with other experts in the field. Her new book of the same title will come out in the fall 2022.

Production in flux

Healthcare systems today operate under a “one size fits all” principle, says Inga Bergen, an entrepreneur and expert on healthcare innovation and digitalization. Treatment decisions are influenced at most by patient age and gender. “Hardly any attention is paid to individual needs, living conditions, biological features, or genetic makeup,” she says. But that is precisely where personalized medicine steps in. “It takes all the available individual factors into account, from social conditions to genetic materials, in order to guide people in health, support them during illness, and continuously refine relevant treatments.”

Testing treatments before trying them

If this ideal is to be achieved, how­ev­er, a new way of think­ing about health­care is need­ed. Bergen speaks about hav­ing to devel­op a “holis­tic take on health.” This approach, which focus­es on the patient and all of their data, sounds promis­ing. The hope is to gain a pre­cise pic­ture of the indi­vid­ual effects of any given med­ical inter­ven­tion, prefer­ably before try­ing it out. “This could be done by means of a dig­i­tal copy of that per­son, which reflects their biol­o­gy, genet­ic mate­r­i­al, and other spe­cif­ic fea­tures,” she says. She would there­fore sup­port the idea of cre­at­ing “dig­i­tal twins,” an aim also defined as vision­ary by Roman Hipp, the life sci­ences expert. Accord­ing to Bergen, dig­i­tal twins would be of major ben­e­fit to both patients and physi­cians. “We could check the effects of a treat­ment before actu­al­ly apply­ing it. After­ward, we could also deter­mine how it worked.”

The approach seems rev­o­lu­tion­ary. And Bergen insists that appear­ances here do not deceive. “Per­son­al­ized med­i­cine will fun­da­men­tal­ly change the entire health­care sec­tor,” she pre­dicts. The health­care indus­try could con­ceiv­ably devel­op into a “hyper­local­ized econ­o­my” to pro­vide patients with med­ical treat­ment tai­lored to their indi­vid­ual needs. Med­ica­tions would then be pro­duced direct­ly at the point of care, sim­i­lar to how CAR T‑cell ther­a­py is con­duct­ed in part today, or to take an entire­ly dif­fer­ent exam­ple, to how providers such as Amazon’s Pill­pack operate.

New incentives needed for change

These devel­op­ments are not a fore­gone con­clu­sion, how­ev­er. Health­care sys­tems need new and dif­fer­ent incen­tives to put visions such as these into prac­tice, explains Bergen. More­over, fur­ther invest­ment in inno­va­tions is need­ed to make per­son­al­ized and indi­vid­u­al­ized med­i­cine pos­si­ble in the first place. “Money alone is not enough, because the inno­va­tions are based in large part on data,” she says. “In order for us to know exact­ly what will help whom, the data have to be avail­able in dig­i­tal­ized and stan­dard­ized form.”

Open discussions about data

Bergen there­fore views cur­rent pub­lic debate on this topic as an obsta­cle to achiev­ing more pre­ci­sion-based forms of med­i­cine. “Media reports are often marked by skep­ti­cism as soon the ques­tion of health data comes up. The empha­sis here shouldn’t be on fuel­ing fear but rather on how shar­ing data can ben­e­fit patients.” The expert, who also runs a pod­cast on health­care, urges open dis­cus­sions and a new look at the topic. “When patients real­ize they’re get­ting some­thing from shar­ing their infor­ma­tion, they’re will­ing to do so. We need a pos­i­tive, future-ori­ent­ed nar­ra­tive in our soci­ety in order to clear the way for inno­va­tions in Germany.”


Dr. Bernd Boidol: “What was a death sentence 20 years ago is now a chronic condition”

Proxygen

Dr. Bernd Boidol became CEO of the Vien­na-based phar­ma­ceu­ti­cal start-up
Prox­y­gen in Sep­tem­ber 2020. He had pre­vi­ous­ly worked for Porsche Con­sult­ing as an expert in the med­ical tech­nol­o­gy and phar­ma­ceu­ti­cal sec­tors. Prox­y­gen is cre­at­ing a plat­form to sys­tem­at­i­cal­ly study what are known as mol­e­c­u­lar glue degraders. With their capac­i­ty to tar­get, “glue” to, and there­by degrade path­o­gen­ic pro­teins, these mol­e­cules could rev­o­lu­tion­ize drug devel­op­ment. In June 2022 the start-up entered a 500 mil­lion-euro part­ner­ship with the Ger­man phar­ma­ceu­ti­cal giant Merck to do fur­ther research on glue degraders.

What data can do

“The concept of personalized medicine is hard to define,” says Dr. Bernd Boidol, CEO of the Proxygen pharmaceutical start-up in Vienna. “If we’re considering it medical treatment tailored to individuals, the question then arises of whether it’s actually an entirely new approach.” In his view, when pathologists analyze a cell or tissue sample, that’s also essentially a personalized approach. A similar take would apply to morphological assessments. “When I look at a patient’s cells under the microscope and then administer an ‘individual’ medication, it’s no less personalized—and that’s been done for centuries.”

Developing and successfully applying precise treatments

Boidol there­fore prefers to talk about “pre­ci­sion med­i­cine.” For him that would mean the fol­low­ing approach: “The first step is to use the lat­est tech­nol­o­gy to gain an extreme­ly pre­cise under­stand­ing of the indi­vid­ual con­di­tion, and the sec­ond is to find and offer a very pre­cise treat­ment for exact­ly that con­di­tion.” His strong empha­sis on under­stand­ing the con­di­tion is based on very sound rea­son­ing. “The mol­e­c­u­lar caus­es of an ill­ness are often underestimated—but we can only devel­op and suc­cess­ful­ly apply a pre­ci­sion treat­ment if we have thor­ough­ly under­stood the condition.”

Although Boidol may define the mat­ter in some­what tighter terms, he and many other experts agree on one thing: these new meth­ods have already led to enor­mous advances in oncol­o­gy in par­tic­u­lar. Chron­ic myeloid leukemia is one exam­ple. A spe­cif­ic genet­ic muta­tion is often a causal fac­tor in this type of leukemia. If clin­i­cal tests detect the muta­tion, a new type of med­ica­tion can be given that selec­tive­ly binds the genet­ic vari­a­tion and sup­press­es its function—with only min­i­mal side effects. As Boidol notes, “What was a death sen­tence twen­ty years ago is now a chron­ic dis­ease that can be treat­ed for ten or fif­teen years.”

Progress only possible with more data

Anoth­er point on which the Prox­y­gen CEO agrees with most experts is that rapid progress in pre­ci­sion med­i­cine can only be made if more data from patients become avail­able. “The data are there,” he says. “The prob­lem lies in the strict and there­fore lim­it­ing data pro­tec­tion reg­u­la­tions.” And in fact one might ask why hos­pi­tals can­not sell their data at least in anonymized form. After all, the data would be used to devel­op med­ica­tions that could help grave­ly ill indi­vid­u­als and there­by reduce suf­fer­ing. “I think that should be pos­si­ble,” he says. The key there­by is to ensure that “the focus is on what ben­e­fits patients.”

Leave the pricing to companies

Boidol does not cur­rent­ly see an acute need for addi­tion­al mea­sures specif­i­cal­ly designed to boost these new meth­ods to a break­through. As he explains, “Many things that would advance the phar­ma­ceu­ti­cal indus­try in gen­er­al would also be help­ful from a pre­ci­sion med­ical per­spec­tive.” In his view, progress would already be served by leav­ing the pric­ing of cer­tain med­ica­tions such as antibi­otics to com­pa­nies and refrain­ing from impos­ing price caps. “The rapid and suc­cess­ful devel­op­ment of the Covid vac­cines is a good exam­ple of the level of med­ical and com­pet­i­tive ben­e­fits that pol­i­cy can have.”


Dr. Dorothee Brakmann: “We’re going to stop diseases before they set in”

Alexandra Malinka

Dr. Dorothee Brak­mann is a mem­ber of the man­age­ment board of
Janssen Deutsch­land and direc­tor of its oncology/hematology divi­sion. Her career at Janssen Deutsch­land began back in 2008 as head of health pol­i­cy. She had pre­vi­ous­ly worked as a hos­pi­tal phar­ma­cist in the UK, for health­care fun­ders, and on busi­ness soft­ware solu­tions for phar­ma­cies. The Janssen phar­ma­ceu­ti­cal com­pa­ny is part of the John­son & John­son glob­al health­care group.

Incentives for innovation

When asked about her vision for the future of healthcare, Dr. Dorothee Brakmann admits that it can sometimes sound a little like science fiction. That doesn’t mean she is a dreamer. Brakmann is a member of the management board of the Neuss-based Janssen pharmaceutical company and has gained experience in nearly every part of the sector over her career, including at a hospital pharmacy, with healthcare funders, and in pharmaceutical research. As a pharmacist she knows what she’s talking about when she says, “In the future we’re going to stop diseases before they have a chance to set in—and we’re not all too far from that right now.”

Promis­ing inno­va­tions in the field of per­son­al­ized med­i­cine are expect­ed to make this pos­si­ble. “We have to get away from the ‘one pill fits all’ approach and adopt treat­ments tai­lored as close­ly as pos­si­ble to indi­vid­ual diag­noses and needs,” she says. “We have to move beyond the repair approach to sick people—which is more of an ill­ness than a health system—and estab­lish a health­care sys­tem wor­thy of the name.”

The right individual treatment at the right individual time

The good news is that the first steps have already been taken and we’ve entered the new world of pre­ci­sion med­i­cine. Cit­ing cur­rent infor­ma­tion from the Ger­man Asso­ci­a­tion of Research-Based Phar­ma­ceu­ti­cal Com­pa­nies (vfa), Brak­mann notes that 97 med­ica­tions autho­rized in Ger­many meet the cri­te­ria of tar­get­ed treat­ment, most of them in oncol­o­gy. Com­mon to all is a con­sid­er­a­tion of indi­vid­ual genet­ic char­ac­ter­is­tics. In other words, before one of these med­ica­tions is admin­is­tered, spe­cial­ists test the patient’s genet­ic mate­r­i­al. The bio­chem­i­cal and genet­ic pre­lim­i­nary assess­ments show which prepa­ra­tions will work well and which will prob­a­bly not. “This is a way to give the right sub­stances to the right patients at the right times,” she says.

Oncology is only the beginning

There’s a good rea­son for why this approach is being used espe­cial­ly in oncol­o­gy. “Can­cer is a dis­ease of the genes,” says Dorothee Brak­mann. It there­fore makes sense to look for rel­e­vant genet­ic fea­tures and exam­ine them before pre­scrib­ing treat­ment. Janssen is work­ing in this area and has devel­oped numer­ous active agents for var­i­ous blood, lung, prostate, and blad­der can­cers. Pre­ci­sion med­i­cine is not restrict­ed to treat­ing can­cer, how­ev­er. As Brak­mann explains, “Oncol­o­gy has made a start, and other areas are following.”

Near­ly all the fields of research at Janssen take a per­son­al­ized approach—“with all the atten­dant prob­lems,” observes Brak­mann. One chal­lenge has to do with the con­sid­er­ably small­er patient cohorts. Par­tic­i­pants are select­ed on the basis of genet­ic fea­tures need­ed for clin­i­cal stud­ies focus­ing on ever more spe­cif­ic sub­groups of a par­tic­u­lar dis­ease. Anoth­er chal­lenge con­cerns the high costs required to devel­op med­ica­tions for only a few patients, albeit with tar­get­ed effects. “Health­care sys­tems do not com­mand end­less resources,” says Brak­mann. “We, too, are well aware of this.”

The health system’s magic triangle

The phar­ma­ceu­ti­cal expert there­fore draws a “fine line” between three deter­min­ing fac­tors in a health sys­tem: patient access to med­ica­tion, afford­abil­i­ty, and incen­tives for research com­pa­nies to pur­sue inno­va­tions. “We need to main­tain a good level of dia­logue among every­one involved,” she says, while not­ing that’s eas­i­er said than done. “When you focus your efforts on one fac­tor in this magic tri­an­gle, you always affect the other two as well.”

While Dorothee Brak­mann attests to the good work done by reg­u­la­to­ry author­i­ties, she sees room for improve­ment in the reim­burse­ment system—including in Ger­many. “There are far too many old-school mod­els in oper­a­tion here,” she says with ref­er­ence to the exten­sive ran­dom­ized clin­i­cal stud­ies that fun­ders pre­fer. “They’re the gold stan­dard of course,” she con­cedes. “But if you’re devel­op­ing med­ica­tions for very small groups of patients, it’s very dif­fi­cult to set up that type of com­pre­hen­sive study.” To enable pre­ci­sion med­i­cine to take a quan­tum leap for­ward, adjust­ments are need­ed to the sys­tem itself—over and above the release of anonymized health data that Brak­mann and many other experts are call­ing for.

Preventing disease with the aid of biomarkers

If we devote our efforts to the research part of the health­care system’s magic tri­an­gle, a clear road stretch­es on into the future—namely a future in which we stop dis­eases before they have a chance to set in. At Janssen this approach is called “dis­ease inter­cep­tion.” It func­tions sim­i­lar­ly to that for per­son­al­ized can­cer treat­ments: by iden­ti­fy­ing bio­mark­ers such as spe­cif­ic gene muta­tions that make cer­tain dis­eases more like­ly. “If you detect this type of pre­dic­tor, there’s an espe­cial­ly high like­li­hood that the dis­ease will be found in that indi­vid­ual even before any symp­toms appear. You can there­fore take coun­ter­mea­sures at an early point in time to pre­vent it from break­ing out.”

The more bio­mark­ers can be detect­ed with the help of AI-sup­port­ed analy­ses and the more anonymized patient data are made avail­able, the eas­i­er it will be for com­pa­nies to devel­op bet­ter treat­ments. Brak­mann is con­vinced we won’t have to wait too much longer. “The first treat­ments could come onto the mar­ket in four or five years,” she says. Which sounds like sci­ence fic­tion might in fact become reality.


Professor Christoph U. Herborn: “We’re already offering personalized therapies”

Porsche Consulting/Andreas Laible

Pro­fes­sor Christoph U. Her­born became CEO of Bergman Clin­ics in Ger­many in August 2022. Head­quar­tered in Naar­den in the Nether­lands, Bergman Clin­ics is build­ing an inter­na­tion­al plat­form for high­ly spe­cial­ized, plannable med­ical care and seeks to gen­er­ate fur­ther growth espe­cial­ly in Ger­many with the help of Her­born. Pre­vi­ous­ly the med­ical direc­tor and one of five man­ag­ing direc­tors for the
Askle­pios Group, which runs around 170 facil­i­ties in 14 Ger­man states, Her­born gave the inter­view for this arti­cle in his for­mer capac­i­ty as man­ag­ing direc­tor of Asklepios.

Applications in practice

Medicine of the future is often associated with new and precise approaches to treatment. In some places, however, the future appears to have already arrived. “At our clinics, and others as well, personalized treatment and the corresponding diagnostic processes are already on offer,” says Professor Christian U. Herborn.

Trained as a radi­ol­o­gist, he became CEO of Bergman Clin­ics in Ger­many in August 2022 but gave the inter­view for this arti­cle while still the med­ical direc­tor and one of five man­ag­ing direc­tors for the Askle­pios Group. The diag­nos­tic process­es he refers to are part of treat­ment strate­gies based on mol­e­c­u­lar genet­ic test­ing for tumors. And they are clear­ly lead­ing to suc­cess­ful results at Askle­pios. “By com­bin­ing con­ven­tion­al chemother­a­py with new anti­body ther­a­pies we’re able to arrest tumor growth or even reduce the size of pri­ma­ry tumors,” reports Herborn.

Tumor board decides on individual cases

Although many patients are still unfa­mil­iar with this type of treat­ment, they only rarely have reser­va­tions about it, says Her­born. “The vast major­i­ty are open to the new meth­ods.” Their con­fi­dence is rein­forced by the fact that Askle­pios does not assign them to one depart­ment alone. “Can­cer med­i­cine at our clin­ics is an inter­dis­ci­pli­nary affair.” The heart of the mul­ti­dis­ci­pli­nary team is the “tumor board,” con­sist­ing of experts from oncol­o­gy, radi­ol­o­gy, pathol­o­gy, radio­ther­a­py, and surgery. “They con­fer to make the best treat­ment deci­sions for each indi­vid­ual case.”

Step one consists of funding

When the expert com­mit­tee selects an indi­vid­u­al­ized treat­ment, the first ques­tion for the for­mer Askle­pios man­ag­ing direc­tor has to do with fund­ing. “Per­son­al­ized treat­ments are often very cost­ly and gen­er­al­ly not includ­ed in stan­dard med­ical cov­er­age,” he notes. He there­fore either had to either nego­ti­ate with the insur­ance com­pa­nies or pur­sue the treat­ment as part of a study. “In such cases the phar­ma­ceu­ti­cal indus­try assumes the costs.”

The professor’s empha­sis on fund­ing might raise some doubts. Does that mean only patients who are pri­vate­ly insured will receive these effec­tive but expen­sive treat­ments? Might the rise of pre­ci­sion med­i­cine even lead to two diver­gent class­es of med­ical care? Her­born, how­ev­er, sees no rea­son to fear that the inno­v­a­tive treat­ments will acquire an elit­ist char­ac­ter. “Every­thing that’s avail­able and can be offered as part of good med­ical prac­tice is in fact on offer. Noth­ing needs to be democ­ra­tized or made any more acces­si­ble than it already is,” he declares.

New hope for cardiovascular patients

With the new forms of treat­ment already offered at clin­ics on a reg­u­lar basis, 
Her­born
 is look­ing fur­ther into the future. “Over the next ten or fif­teen years I expect the new per­son­al­ized forms of treat­ment to be applied not only in oncol­o­gy but also to other dis­eases such as car­di­o­log­i­cal and cir­cu­la­to­ry con­di­tions.” Their treat­ment cur­rent­ly focus­es on risk fac­tors from unhealthy lifestyles that lead to high blood sugar or lipid lev­els. “A good way to sup­ple­ment this approach would be to deter­mine the ideal pre­ven­tive mea­sures on the basis of car­dio­vas­cu­lar pro­files.”
 


Professor Ariel Dora Stern: “Personalized medicine will become the new standard”

Jan Pauls

Ariel Dora Stern is Asso­ciate Pro­fes­sor of Busi­ness Admin­is­tra­tion at Har­vard Busi­ness School in Boston, a fac­ul­ty mem­ber of the Har­vard-MIT Cen­ter for Reg­u­la­to­ry Sci­ence, and holds a guest pro­fes­sor­ship at the Dig­i­tal Health Cen­ter of the Hasso Plat­tner Insti­tute in Pots­dam. She stud­ies the health­care sec­tor with a focus on inno­va­tion man­age­ment, dig­i­tal health, and health eco­nom­ics in gen­er­al. She serves on the sci­en­tif­ic advi­so­ry board of the Ger­man Soci­ety for dig­i­tal Med­i­cine (Deutsche Gesellschaft für Dig­i­tale Medi­zin) and advis­es start-ups in the health­care sec­tor. Before enter­ing acad­e­mia she worked as an econ­o­mist on Wall Street, at the Fed­er­al Reserve Bank in New York, and at the Ger­man Insti­tute for Eco­nom­ic Research (DIW).

New approaches to study design

The idea of tailoring treatments to individual needs is not new. Indeed, for centuries medical specialists have tried to address patients’ specific problems and have made decisions about medication dosing and treatments on the basis of factors like age, gender, and physical constitution. Professor Ariel Dora Stern therefore notes that it’s difficult to delineate what is meant by personalized medicine.

“Gen­er­al­ly speak­ing, how­ev­er, we’re now devel­op­ing treat­ments that are con­sid­er­ably more tar­get­ed and pre­cise than has ever been pos­si­ble before.” These recent devel­op­ments are what ulti­mate­ly make the dif­fer­ence for the Har­vard pro­fes­sor. “Over the short or long term, we can assume that per­son­al­ized med­i­cine will become the stan­dard treatment.”

That being said, per­son­al­iza­tion will not pro­ceed at the same pace for all dis­eases. Stern expects the near future to bring advances for can­cer in par­tic­u­lar, but also more tar­get­ed approach­es for oph­thal­mo­log­i­cal and hema­to­log­i­cal dis­or­ders. “Patients will enjoy sub­stan­tial ben­e­fits in these set­tings where their med­ica­tions will be increas­ing­ly tai­lored to their respec­tive genet­ic make­up or the muta­tions from their spe­cif­ic type of cancer.”

For researchers, outdated approval processes present an obstacle

There are a num­ber of hur­dles to clear, how­ev­er, before many per­son­al­ized treat­ments can achieve break­throughs and become wide­ly avail­able. Ariel Dora Stern speaks of an “insti­tu­tion­al mis­match” between FDA and EMA reg­u­la­to­ry frame­work con­di­tions on the one hand and research require­ments on the other. She advo­cates for what she calls “reg­u­la­to­ry inno­va­tion,” not­ing that “Our cur­rent med­ical prod­uct reg­u­la­tions are a prod­uct of the pre­vi­ous cen­tu­ry. They sim­ply no longer reflect the needs of the next gen­er­a­tion of prod­ucts or cut­ting edge prac­tices in ther­a­peu­tic research and devel­op­ment.” A good exam­ple is the need to set up plat­form stud­ies instead of tra­di­tion­al clin­i­cal tri­als. “Plat­form stud­ies focus on the over­all clin­i­cal pic­ture,” she explains. “They let us divide groups of patients into dif­fer­ent arms of the study based on bio­mark­ers and to test dif­fer­ent med­ica­tions at the same time. Because estab­lished reg­u­la­to­ry approach­es were not designed with such stud­ies in mind, get­ting them off the ground has been a con­sid­er­able challenge—despite the fact that they offer clear advan­tages for both patients and clin­i­cal investigators.”

Ariel Dora Stern doesn’t try to down­play the con­sid­er­able time and fund­ing required to set up a plat­form study. But she also says the advan­tages far out­weigh the dis­ad­van­tages. “This type of trail isn’t over after you’ve con­duct­ed the first series of tests—the plat­form struc­ture means that mean­ing­ful research can con­tin­ue even after spe­cif­ic clin­i­cal ques­tions have been answered. This means you can keep research­ing that dis­ease in a sus­tain­able way.” In addi­tion, dif­fer­ent sta­tis­ti­cal mod­els are applied that facil­i­tate dynam­ic analy­ses and there­by yield faster and more effi­cient results in a sta­tis­ti­cal­ly rig­or­ous manner.

Greater trust among competitors

Reg­u­la­tions are not the only fac­tors hold­ing up these stud­ies. The orga­ni­za­tions that con­duct plat­form stud­ies are designed as open sys­tems. In other words, they aren’t just a sin­gle enter­prise but instead can con­sist of mul­ti­ple par­tic­i­pants. “This type of setup is com­pli­cat­ed, of course, when you have com­peti­tors work­ing togeth­er,” observes Stern, who also cur­rent­ly holds a guest pro­fes­sor­ship at the Dig­i­tal Health Cen­ter of the Hasso Plat­tner Insti­tute in Pots­dam. Her solu­tion: “Start­ing a non-prof­it foun­da­tion or iden­ti­fy­ing anoth­er trust­ed, inde­pen­dent third party to coor­di­nate and run the study has proved to be a good way of build­ing trust. We have seen this, for exam­ple, in the work of the Glob­al Coali­tion for Adap­tive Research (GCAR) in the Unit­ed States.” Once a plat­form study is estab­lished, its infra­struc­ture makes the entry require­ments lower than those for launch­ing a tra­di­tion­al clin­i­cal trial. “This allows small­er biotech and phar­ma­ceu­ti­cal com­pa­nies study dis­eases and pop­u­la­tions that they may not oth­er­wise have inves­ti­gat­ed, and also cre­ates incen­tives to devel­op treat­ments for rarer diseases.”

Healthcare systems determine drug access

Whether and when peo­ple can ben­e­fit from per­son­al­ized med­i­cine will depend on more than the research itself. Stern notes that although the new treat­ments are often very effec­tive, they can also be very expen­sive. “Each country’s health­care sys­tem will there­fore play a key role in pro­vid­ing access to these treat­ments.” Cur­rent approach­es to fund­ing the cor­re­spond­ing med­ica­tions in the USA can look baf­fling to Ger­man eyes. “Pay­ment mod­els there range from ‘drug mort­gages’ (where expen­sive treat­ments are paid off over time) to hav­ing the amount of reim­burse­ment depend on the suc­cess of the treat­ment.” Europe will cer­tain­ly be tak­ing a dif­fer­ent approach here, although inno­v­a­tive pay­ment mod­els are already being piloted.


Dr. Yacine Hadjiat: “We’re translating new technical capabilities into tangible health solutions”

Biogen

Dr. Yacine Had­ji­at is Glob­al Head of Dig­i­tal Health Solu­tions at Bio­gen Dig­i­tal Health (BDH), hav­ing pre­vi­ous­ly worked for lead­ing life sci­ence com­pa­nies as well as start-ups and gov­ern­ment agen­cies in the E.U., U.S., and Asia. Cre­at­ed in 2021,
Bio­gen Dig­i­tal Health is a glob­al unit of Bio­gen, ded­i­cat­ed to pio­neer­ing per­son­al­ized and dig­i­tal med­i­cine in neuroscience.

New apps for peace of mind 

“The right treatment for the right patient at the right moment—that is what personalized medicine means for me,” says Dr. Yacine Hadjiat. As Global Head of Digital Solutions at Biogen Health (BDH), Hadjiat primarily focuses on technical applications: AI-powered imaging solutions, smartphones, smartwatches, and other wearables equipped with sensors gather data to enable treatment to be tailored precisely to the individual patient. Motion, speech, writing and reading habits—all these things function as digital biomarkers.”

For Had­ji­at, these dig­i­tal bio­mark­ers pro­vide rev­o­lu­tion­ary data when it comes to track­ing the pro­gres­sion of mul­ti­ple scle­ro­sis and other neu­ro­mus­cu­lar dis­eases. With Cleo and Konec­tom, he has already launched solu­tions that make patients’ lives eas­i­er using pre­cise­ly these tech­nolo­gies. Cleo, for exam­ple, pro­vides mul­ti­ple scle­ro­sis patients with daily sup­port, infor­ma­tion, tips, symp­tom track­ing, reminders, and other cus­tomized pro­grams. The down­load data shows how high­ly patients value this app: as of 2022, Cleo has been down­loaded over 600,000 times world­wide. The Konec­tom plat­form, in con­trast, focus­es on the improve­ment of neu­ro­log­i­cal dis­ease mea­sure­ment. It uses smart­phones and wear­able sen­sors to enable a much high­er res­o­lu­tion in the way we track, mon­i­tor, and mea­sure neu­ro­log­i­cal diseases.
Konec­tom takes into account the patient’s key neu­ro­log­i­cal func­tions, from cog­ni­tion to fine and gross motor skills.

The goal of Had­ji­at and his team at BDH: to use tech­nol­o­gy to address patient needs in a goal-ori­ent­ed man­ner and drive the change toward a more patient-cen­tered and bet­ter care. Dig­i­tal bio­mark­ers play a major role in this endeav­or: they not only allow peo­ple to track and bet­ter man­age their own dis­ease but also enable their neu­rol­o­gists to mon­i­tor progress more accu­rate­ly and with high­er res­o­lu­tion from afar, all thanks to technology.

Using apps to predict clinical conditions

Cen­tral to such solu­tions is smart­phone sen­sor tech­nol­o­gy, which helps to con­tin­u­ous­ly gath­er rel­e­vant health data. If crit­i­cal pat­terns emerge, neu­rol­o­gists are alert­ed and can help their patients at an early stage if nec­es­sary. Accord­ing to Had­ji­at, the poten­tial of the new tech­nol­o­gy thus aris­es from obser­va­tion of con­di­tions that progress slow­ly and the pos­si­bil­i­ties of this tech­nol­o­gy to pre­dict clin­i­cal con­di­tions before they occur.

Digital health solutions are on track to become the status quo

Yacine Had­ji­at is cer­tain that dig­i­tal health appli­ca­tions will soon be part of the med­ical stan­dard. But sim­ply trans­fer­ring solu­tions from the offline to the online world is not enough, he says. After all, he notes, dig­i­tal health and tech­nol­o­gy offer a much greater promise: “Through dig­i­tal health, for the first time, we have the pos­si­bil­i­ty of trans­lat­ing new tech­ni­cal capa­bil­i­ties into tan­gi­ble health solutions—that’s some­thing com­plete­ly new.” Had­ji­at notes that he will have reached his goal when the dig­i­tal health hype sub­sides, and dig­i­tal solu­tions and appli­ca­tions have become an inte­gral com­po­nent for med­ical stan­dards of care. “Or, to put it anoth­er way, when dig­i­tal health is the sta­tus quo.”


Susanne Baars: “Personalized treatments should be available to everyone on the planet”

Erik van't Woud

Susanne Baars is senior glob­al thought lead­er­ship man­ag­er at Siemens Health­i­neers in Erlan­gen, and respon­si­ble for expand­ing pre­ci­sion med­i­cine. She also directs SocialGe­nomics, a start-up she found­ed in 2018 to help patients by means of an arti­fi­cial intel­li­gence-dri­ven net­work that extracts infor­ma­tion about their con­di­tion and tumors to dig­i­tal­ly con­nect them with oth­ers in a sim­i­lar con­di­tion; share best prac­tices to find per­son­al­ized treat­ments; and col­lect real-world data to improve out­comes. 

Solving global problems 

Susanne Baars is a woman with a mission. Her goal is nothing less than to make information and personalized medicine available and affordable for every person on the planet. In addition to calling for this at high-level events including TED talks, she has launched her own genomics start-up to help patients with cancer and rare conditions to find the right treatments for their individual needs. In early 2021 she also became senior global thought leadership manager for expanding precision medicine at Siemens Healthineers.

Per­son­al­ized med­i­cine is a pas­sion for Baars. In her view, it’s nei­ther a niche nor an add-on to estab­lished meth­ods of treat­ment, but rather the essence of med­ical care. It already helps many patients today, and can offer more effec­tive and less ardu­ous forms of treat­ment to those diag­nosed with can­cer and rare con­di­tions. What may sound like a break­through to many peo­ple is just the begin­ning for Baars. “We’re talk­ing about a rev­o­lu­tion here,” she says. “The tremen­dous increase in com­pu­ta­tion­al capac­i­ties and glob­al con­nec­tiv­i­ty is what will real­ly unleash the full power of per­son­al­ized medicine.”

Precision medicine fueled by pattern recognition

Pre­dic­tive bioin­for­mat­ics and machine-learn­ing mod­els make it pos­si­ble to gath­er mil­lions of data points from the real world. These can be patient data, clin­i­cal data, lab­o­ra­to­ry tests, genomics or imag­ing. Com­pil­ing these data is just the first step. “Data alone can’t save a sin­gle life,” notes Baars. True med­ical progress, she adds, lies in ana­lyz­ing and learn­ing from these com­pre­hen­sive data points, detect­ing pat­terns, and mak­ing action­able insights acces­si­ble for physi­cians and patients inde­pen­dent of their geo­graph­ic loca­tion. No two can­cer patients are the same. So, no two treat­ments should be either.

The aim is not only to pro­vide more tar­get­ed treat­ments, but to redesign the health­care sys­tem to make it more intel­li­gent. “Togeth­er with Var­i­an, the lead­ing can­cer-care com­pa­ny that we acquired last year, we’re lever­ag­ing logic and arti­fi­cial intel­li­gence to build a com­pre­hen­sive oncol­o­gy ecosys­tem that trans­forms work­flows.” For exam­ple, the path from diag­no­sis to treat­ments can be accel­er­at­ed from weeks to hours. For patients, hos­pi­tal providers, and soci­ety this makes a huge dif­fer­ence. In Baars’s view, we need to move beyond the cur­rent sys­tem of “sick care” and final­ly attain actu­al “health care.” As she puts it: “We have the tech­nolo­gies and the exper­tise, but if patients don’t have access to solu­tions, then it’s miss­ing its pur­pose. The data we gath­er in the course of per­son­al­ized med­ical care will enable the shift towards early detec­tion and pre­vent peo­ple from devel­op­ing these con­di­tions in the first place.”

Per­son­al­ized med­i­cine is not mere­ly a mat­ter of refin­ing a few parts of the old sys­tem. The vision inspir­ing Baars is one of transformation—organizing life­sav­ing infor­ma­tion and mak­ing it acces­si­ble for every­one to give more patients access to per­son­al­ized treat­ments, when and where they need them.

Early and highly precise diagnoses lower medical costs

Accord­ing to Baars, this new approach will reduce ill­ness and suffering—while also sav­ing a great deal of money. For exam­ple, many cases of can­cer today are not diag­nosed until they have reached the most advanced stage or have already spread. Suit­able treat­ments at this point are not only dif­fi­cult but also like­ly to need mul­ti­ple dif­fer­ent inter­ven­tions, increas­ing side effects for patients. This means longer hos­pi­tal stays for patients and also high­er expens­es for health­care sys­tems. “If we can diag­nose can­cer at an early stage, we can real­ly cure patients and it doesn’t cost as much,” she explains.

In order to reach patients early on, patient-sup­port­ing tools such as dig­i­tal twins and multi-dis­ci­pli­nary oncol­o­gy plat­forms will play an ever greater role. A patient dig­i­tal twin inte­grates clin­i­cal data from dis­parate sources—and turns them into a vir­tu­al rep­re­sen­ta­tion of the patient that can be used to free up physi­cians’ time and run sim­u­la­tions to reveal future health sce­nar­ios. When algo­rithms detect crit­i­cal pat­terns, the cor­re­spond­ing tools will not only alert peo­ple but also tell them where in the health­care sys­tem to go for a check-up. “Com­bin­ing a dig­i­tal twin, AI tech­nol­o­gy, and per­son­al­ized care enables us to achieve the best pos­si­ble out­comes for patients,” says Baars. This is based on the patho­log­i­cal and mol­e­c­u­lar specifics of their tumors, imag­ing char­ac­ter­is­tics, treat­ment his­to­ry, and evi­dence-based treat­ment options in dis­cus­sions with physi­cians and providers. “The chal­lenge here is to redesign our sys­tems around patients,” she says.

Here is where Baars’s argu­ments come full cir­cle. “I envi­sion a world with glob­al­ly con­nect­ed per­son­al­ized health path­ways designed to drive effi­cien­cies and deliv­er world-class treat­ment along the can­cer con­tin­u­um to increase impact where it mat­ters most: help­ing patients,” she says. The huge vol­umes of data need­ed to help patients with tai­lored treat­ments can only be gath­ered by holis­ti­cal­ly con­nect­ing patients, physi­cians, gov­ern­ments, and indus­try in a true team effort through dig­i­tal solu­tions on an inter­na­tion­al scale, she believes. In short: the bet­ter we col­lab­o­rate and the greater num­ber of peo­ple who ben­e­fit from pre­ci­sion treat­ments, the more things will improve for every­one. This, then, is also a pow­er­ful impe­tus for coun­tries with advanced health­care sys­tems to make pre­ci­sion med­i­cine acces­si­ble to every­one on the planet.


Opportunities and risks for companies

Conclusion

The breakthrough changes sketched by experts clearly indicate that personalized medicine is bringing a veritable paradigm shift in healthcare. “That creates opportunities if companies can adapt their strategies accordingly at an early point in time, but also brings risks for those companies—as well as societies—that miss out on these developments,” notes Hipp, the consultant.

New balance between data protection and innovative power

One of the key take­aways from the inter­views with experts is that med­ical infor­ma­tion and dig­i­tal­iza­tion will both play con­sid­er­ably greater roles in the con­text of per­son­al­ized med­i­cine. “We rec­om­mend that phar­ma­ceu­ti­cal and med­ical tech­nol­o­gy com­pa­nies expand their data strate­gies and invest in rel­e­vant new prod­ucts and ser­vices,” says Hipp. If this new mar­ket is to offer oppor­tu­ni­ties for devel­op­ment, trans­mis­sion of at least (semi-) anonymized data will have to be stream­lined. “It’s cru­cial to have secure data man­age­ment that also enables inno­v­a­tive break­throughs,” says Hipp. The aim here has to be a “new bal­ance” that fos­ters research and inno­va­tion to pro­mote human well-being while also pro­vid­ing the high­est degree of data pri­va­cy. As he notes, “This might not be a con­vinc­ing approach for absolute­ly every­one, but we shouldn’t over­look the fact that some inno­v­a­tive health start-ups have already left Ger­many to pur­sue research and health­care inno­va­tions in other parts of the world.”

Companies need to adapt to decentralized logistics

The new meth­ods will bring fun­da­men­tal changes to the phar­ma­ceu­ti­cal indus­try. Med­ica­tions are cur­rent­ly pro­duced in large num­bers for broad groups of patients. By con­trast, pre­ci­sion med­ical approach­es require the man­u­fac­ture of indi­vid­u­al­ized med­ica­tions that address diag­nos­tic details or even inte­grate patients’ cell mate­r­i­al into the pro­duc­tion process­es. “These indi­vid­u­al­ized means of pro­duc­tion, which in some cases will sure­ly be decen­tral­ized, will place new demands on logis­tics and tech­ni­cal sys­tems that com­pa­nies will have to meet,” says Hipp. In his view, strate­gies from the auto­mo­tive industry—such as the “smart fac­to­ry” principle—can serve as guides for phar­ma­ceu­ti­cal companies.

It is by no means clear which com­pa­nies will be able to take the lead in the new field of per­son­al­ized med­i­cine. Patients will be the win­ners in any case, accord­ing to Roman Hipp. “They can expect faster and more pre­cise diag­nos­tics and more promis­ing treat­ments, includ­ing the prospect of actu­al cures.”

The three-phase model for success


If a company wants to succeed in the new market for personalized medicine, it has to evaluate the opportunities, assess the risks, and strategically plan each step. Porsche Consulting has developed a three-phase model that makes this possible.
1. Strategy: The first step consists of deciding whether the company can in fact define a market for itself in personalized medicine and where it could then expect that market to develop. “Investments only make sense if a company brings its own core areas of expertise and can apply them,” says Dr. Roman Hipp, Senior Partner at Porsche Consulting. If it does have core expertise, this might need to be reinforced with the help of additional partners, “in order to safeguard investments and increase the chances for success by consolidating know-how and synergies.” When a partner is found the type of collaboration needs to be defined, which might mean a joint venture, strategic alliance, or shared research project. Additional strategic questions concern the market entry strategy, including which regions the company can be successful in and what its go-to-market approach is, namely how its treatments will reach patients in concrete terms and what its scaling on the market will look like. 2. Operating model: This phase consists of aligning processes, organizational structures, and employee abilities toward the target. In other words, can the company use its existing operating model to put its strategy into practice? “Many companies develop promising strategies but fail when it comes to implementation. What’s needed here are the right processes, structures, and above all the employee skills,” says Hipp. 3. Operations: All the processes that will enable the company to provide its personalized medical products and services should be systematically analyzed in advance. Because batch sizes will generally be considerably smaller, questions arise such as whether the conditions will even allow for efficient production of personalized medications. “A major additional challenge lies in recruiting a sufficient number of very highly trained employees,” says Hipp. Another question is the extent to which digitalization can improve production processes. And a further challenge has to do with the supply chain, which is structured completely differently for personalized medicine than for conventional pharmaceutical production. “Here it’s necessary to define suitable and practice-based processes in advance of any investment—not only at one’s own company but also in collaboration with partners such as hospitals.”
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