Biopharmaceuticals: New Hope for Patients

Metachro­mat­ic leukody­s­tro­phy (MLD) is a rare, genet­ic meta­bol­ic dis­or­der whose diag­no­sis used to be tan­ta­mount to a death sen­tence. A mutat­ed gene leads to the pro­gres­sive destruc­tion of the insu­lat­ing layer around nerve fibers in the brain, which means they can no longer trans­mit sig­nals. Affect­ed indi­vid­u­als grad­u­al­ly lose motor skills such as the abil­i­ty to move, talk, speak, and swal­low. In the absence of treat­ment, life expectan­cy is low. 

How­ev­er, a treat­ment — or more pre­cise­ly, a gene ther­a­py — for this dis­ease has recent­ly been devel­oped. A patient’s stem cells are removed and altered in a lab to give them a func­tion­al, intact copy of the defec­tive gene. The stem cells are then infused back into the body, where they can repro­duce in bone mar­row. The results are astound­ing. So too are the costs, cur­rent­ly with a price tag of 3.91 mil­lion euros for a sin­gle treat­ment, as report­ed by Orchard Pharmaceuticals. 

This exam­ple high­lights the inno­va­tion that bio­phar­ma­ceu­ti­cals can bring, as well as the cost chal­lenges. Tra­di­tion­al phar­ma­ceu­ti­cals are often effec­tive for large num­bers of peo­ple and, if their patents have expired and gener­ics come to mar­ket, often cost very lit­tle to relieve a headache or sup­press a cough, for instance. Bio­phar­ma­ceu­ti­cals, on the other hand, are noted for their tar­get­ed abil­i­ty to treat or elim­i­nate what are often rare con­di­tions. Exam­ples of these devel­op­ments include bis­pe­cif­ic anti­bod­ies for can­cer immunother­a­py, using the body’s own immune cells to com­bat tumors, and cel­lu­lar scis­sors to repair defec­tive genes such as the above-men­tioned cause of MLD. In each case, the strength lies in its more per­son­al­ized approach. Each patient receives treat­ment that is tai­lored pre­cise­ly to his or her con­di­tion. Esti­mates by Porsche Con­sult­ing sug­gest that the mar­ket for bio­phar­ma­ceu­ti­cals will near­ly dou­ble by 2030. 

From big phar­ma to start-ups, every­one wants a piece of the pie. But there are enor­mous hur­dles on the road to suc­cess in the bio­phar­ma­ceu­ti­cal busi­ness. Man­u­fac­tur­ing process­es are more com­plex than those for tra­di­tion­al chem­i­cal agents. And the new treat­ments are already fac­ing a series of set­backs in their development. 

An analy­sis by Porsche Con­sult­ing clear­ly shows that of 1,000 mol­e­cules con­sid­ered promis­ing can­di­dates for new med­ica­tions, only around half of them sur­vive the first phase of clin­i­cal stud­ies. All the oth­ers are reject­ed, usu­al­ly because of major side effects. In the sec­ond phase, which tests the dosage and effi­cien­cy of active sub­stances, the cohort loses around anoth­er three-quar­ters of its mem­bers. And in the third phase, with tri­als on thou­sands of peo­ple, anoth­er 40 per­cent of the can­di­dates do not pass muster. Ulti­mate­ly only around ten of the ini­tial 1,000 agents will make it through the approval process, ready for use as ther­a­pies. This often takes a decade or more, and can devour bil­lions of euros. 

These fig­ures are not spe­cif­ic to bio­phar­ma­ceu­ti­cals, but also reflect devel­op­ment chal­lenges faced by drug com­pa­nies that make med­ica­tions based on chem­i­cal mol­e­cules. But they indi­cate the mag­ni­tude of devel­op­ment costs in the sec­tor. How can the process­es become cheap­er and faster? One pos­si­bil­i­ty is to make greater use of arti­fi­cial intel­li­gence (AI). An analy­sis from the jour­nal Drug Dis­cov­ery Today has shown that 80 to 90 per­cent of can­di­dates select­ed by AI make it through the first phase of clin­i­cal stud­ies. That is an enor­mous increase over the num­ber select­ed “man­u­al­ly” by research per­son­nel. Tar­get­ed invest­ments in AI there­fore make sense for phar­ma­ceu­ti­cal com­pa­nies. Anoth­er pos­si­bil­i­ty lies in the phase before human tri­als and con­sists of replac­ing the oblig­a­tory, albeit error-prone, tests on ani­mals with organ-on-a-chip exper­i­ments. For this, human cells are cul­ti­vat­ed in a lab to make small amounts of tis­sue suit­able for drug tests — an approach that will depend on favor­able reg­u­la­to­ry frame­works in the future. 

It is also impor­tant to entrust the devel­op­ment of new med­ica­tions to diverse teams. Not only R&D spe­cial­ists should be involved right from the start but also rep­re­sen­ta­tives from man­u­fac­tur­ing, mar­ket­ing, reg­u­la­to­ry affairs, and qual­i­ty man­age­ment. The com­bined per­spec­tive helps to save time and costs because it ensures that only the most promis­ing can­di­dates are admit­ted to clin­i­cal stud­ies in the first place. 

Look­ing at this from a glob­al per­spec­tive, there is poten­tial for speed­ing up the pace of these process­es. Inter­na­tion­al com­pa­nies often reach phase 3 or beyond in advanced bio­log­ics, while Ger­man play­ers are still in phase 1 or 2. 

That being said, Ger­man com­pa­nies can learn from other com­pa­nies’ best prac­tices — par­tic­u­lar­ly of U.S.-based enter­pris­es — and quick­ly catch up. For this, they need to place a high­er pri­or­i­ty on inno­v­a­tive tech­nolo­gies in their R&D depart­ments, ide­al­ly in mar­ket sec­tors where they are already com­pet­i­tive. Strate­gic out­sourc­ing of indi­vid­ual activ­i­ties can fur­ther accel­er­ate progress.

Every larg­er com­pa­ny also has to explore whether it wish­es to pur­chase exter­nal inno­va­tions and inte­grate them into its exist­ing orga­ni­za­tion­al model, for exam­ple in the form of col­lab­o­ra­tive projects with spin-offs from uni­ver­si­ties or research insti­tu­tions, or whether it should build up the req­ui­site exper­tise inter­nal­ly. The experts at Porsche Con­sult­ing can pro­vide valu­able assis­tance in select­ing the right targets,and estab­lish­ing new value pools. 

Inno­va­tions are also need­ed in financ­ing. If a treat­ment costs mil­lions of euros, it will not be avail­able to every­one — it is sim­ply too expen­sive. And the solu­tion? We need to reassess remu­ner­a­tion mod­els and break free of tra­di­tion. Cur­rent­ly, the sales vol­ume of a drug is what is reward­ed. Anoth­er pos­si­bil­i­ty would be to com­pen­sate pure­ly on the basis of out­come — and pay pro­duc­ers not for every drug sold but for every treat­ment that succeeds. 

With­out a doubt, patients will ben­e­fit from bio­phar­ma­ceu­ti­cal inno­va­tions. And the faster these med­ica­tions can go onto the mar­ket and the more favor­able their prices, the more human lives they will be able to save.