Amerika sunkiai mokės už itin brangias genų terapijas

  "Buteliukas yra žinomas, skystis viduje gali būti vanduo, bet kaina yra šiek tiek neįprastesnė. Zolgensma, genų terapija stuburo ir raumenų atrofijai gydyti, kainuoja 2,1 mln. dolerių.

 

     Tai vienas iš naujos kartos itin brangių vaistų. Beta talasemijos ir hemofilijos – dviejų kraujo ligų – gydymas kainuoja atitinkamai 2,8 mln. ir 3,5 mln. dolerių. Jų kainas gali aplenkti genų terapija nuo pjautuvo pavidalo ląstelių ligos, kuri, kaip tikimasi, bus patvirtinta šiais metais, ir viena nuo Diušeno raumenų distrofijos, kuri gali būti patvirtinta bet kurią dieną.

 

     Daugelyje vidutines ir mažas pajamas gaunančių šalių tokios terapijos nebus įmanoma taikyti. Ji taip pat sukels bėdų turtingiausiems, net, Amerikai. Farmacijos įmonės pažymi, kad vaistų kūrimas yra brangus, daugiausia retiems sutrikimams gydyti, ir gali būti naudingas visam gyvenimui. Vyriausybės ir draudikai turi nuspręsti, ar vaistai yra verti dabartinėmis kainomis, ir, jei ne, bandyti juos sumažinti. Sveikatos priežiūros ekspertai svarsto, ar šis procesas laikui bėgant gali priversti pakeisti Amerikos valstijų apmokėjimą už vaistus.

 

     Vertex, viena iš pjautuvinių ląstelių ligų gydymo firmų, teigia, kad dabartinis labiausiai nukentėjusių pacientų gydymas gali kainuoti 4–6 mln. dolerių per visą gyvenimą. Tačiau Klinikinės ir ekonominės apžvalgos institutas, ekspertų grupė, apskaičiavo, kad įmonės naujasis vaistas būtų ekonomiškai efektyvus tik už mažiau nei 2 mln. Dar neaišku, kiek ilgai išliks vaisto nauda.

 

     Pjautuvinių ląstelių liga, kuri gali sukelti didelį skausmą, insultą, rimtas infekcijas ir plaučių sutrikimus, yra ypatinga vyriausybių ir draudikų problema, nes ji yra gana dažna. Amerikoje nuo jos kenčia 100 000 žmonių, daugelis jų yra apdrausti pagal Medicaid – oficialią neturtingųjų sveikatos priežiūros programą. Michaelas Kleinrockas iš analitikos įmonės „iqvia“ žmogaus duomenų mokslo instituto tikisi, kad „Medicaid“ turės teikti pirmenybę pacientams, kai bus patvirtinti vaistai, nes ji negalės sau leisti mokėti už visus, kurie tuo pačiu metu gali gauti naudos.

 

     Vidutinės trukmės laikotarpiu gali prireikti pakeisti požiūrį. Francisas Collinsas iš Nacionalinių sveikatos institutų, finansuojančių medicininius tyrimus, teigia, kad vyriausybė pripažįsta, kad reikės „ypatingo kūrybinio mąstymo, kad būtų galima gauti [šių vaistų]“. Oficialios agentūros cms Inovacijų centro ataskaitoje siūloma pereiti prie sistemos, kurioje vyriausybė derasi valstijos Medicaid vardu, tikėdamasi panaudoti federalinę jėgą geresniems sandoriams laimėti. Nors detalės dar neišspręstos, tikimasi, kad mokėjimai gali būti susieti su vaistų veiksmingumu, kaip jau daroma Didžiojoje Britanijoje, Prancūzijoje ir Vokietijoje.

 

     Su sunkumais susidurs ir privatūs draudikai. Daugelis savo politikoje taikė tiesiogines išimtis arba apribojimus gauti genų terapiją. Kadangi draudikai turi didelę klientų kaitą, jie gali neturėti naudos iš gydymo sutaupytų lėšų, kurios kaupsis visą gyvenimą. Kalbama apie perdraudimo programas ir rizikos sutelkimą, tačiau pažangos padaryta nedidelė.

 

     Kai kurie teigia, kad, laikui bėgant, išlaidos mažės. Zandy Forbes, „Meiragtx“ vadovė, teigia, kad jos įmonė kuria Parkinsono ligos genų terapiją, kuri bus konkurencinga su esamais gydymo būdais. Siekdama šio tikslo, bendrovė nusprendė sutelkti visą plėtrą ir gamybą viduje, kad radikaliai sumažintų išlaidas. Istorija rodo, kad farmacijos prekių kainos gali nukristi. 1998–2009 m. gamybos patobulinimai leido 50 kartų sumažinti monokloninių antikūnų prekių kainą. Dabar jie reguliariai naudojami medicinoje.

 

     Yra ir kitas variantas, kad laimėjimai nueis perniek. Kai kurios valstijos nenorėjo mokėti kainos, reikalingos hepatitui c, virusinei ligai, panaikinti, nepaisant to, kad yra antivirusinių gydymo priemonių, kurios vienam pacientui kainuoja apie 20 000 dolerių, sako dr. Collinsas; dėl to gydomiesiems kyla įvairių kliūčių. Būtų nepaprastas švaistymas, jei tas pats nutiktų su nauja genų terapijos banga. [1]

 

·  ·  · 1.  "America will struggle to pay for ultra-expensive gene therapies." The Economist, 3 June 2023, p. NA.

America will struggle to pay for ultra-expensive gene therapies.

"The vial is familiar, the liquid inside could be water—but the price tag is a little more unusual. A shot of Zolgensma, a gene therapy for spinal-muscular atrophy, comes to $2.1m. 

It is one of a new generation of ultra-expensive medicines. Treatments for beta-thalassemia and haemophilia, two blood disorders, cost $2.8m and $3.5m, respectively. Their prices may be overtaken by gene therapies for sickle-cell disease expected to be approved this year, and one for Duchenne muscular dystrophy, which could be approved any day now.

Such therapies will be beyond the means of many middle- and low-income countries. They will also cause trouble in the richest, not least America. Pharmaceutical firms point out the drugs are expensive to develop, mostly for rare disorders and may offer benefits that last a lifetime. Governments and insurers must decide if the medicines are worth it at current prices and, if not, try to negotiate them down. Health-care experts wonder if this process could, in time, force sweeping changes in how American states pay for medication.

Vertex, one of the firms working on sickle-cell-disease therapies, argues that current treatment for the worst-affected patients can cost $4m-6m over the course of a lifetime. Yet the Institute for Clinical and Economic Review, a think-tank, calculates that the firm's new medicine would only be cost-effective at a shade under $2m a patient, both because the initial cost would earn a return if put to other uses and because there is uncertainty over how long the benefits of the drug will last.

Sickle-cell disease, which can lead to extreme pain, strokes, serious infections and lung difficulties, is a particular problem for governments and insurers, since it is relatively common. In America there are 100,000 people who suffer from it, and many are covered by Medicaid, an official health-care scheme for the poor. Michael Kleinrock of the iqvia Institute for Human Data Science, an analytics firm, expects that Medicaid will have to prioritise patients when the drugs are approved, as it will be unable to afford to pay for everyone who might benefit at the same time.

In the medium term, a change of approach may be necessary. Francis Collins of the National Institutes of Health, which funds medical research, says there is recognition in government that there will have to be "special creative thoughtfulness to make access [to these medicines] happen". A report by the cms Innovation Centre, an official agency, suggests a move to a system in which the government negotiates on behalf of state Medicaid outfits, in the hope of using federal heft to win better deals. Although the details are yet to be worked out, the hope is payments can be linked to drug performance, as already happens in Britain, France and Germany.

Private insurers will face difficulties, too. Many have imposed outright exclusions or restriction on access to gene therapies in their policies. As insurers have a high turnover of customers, they may not benefit from the savings of a cure, which will accrue over a lifetime. There is talk of reinsurance programmes and risk pooling, but little progress has been made.

Some argue that costs will come down over time. Zandy Forbes, chief executive of Meiragtx, says that her firm is working on a gene therapy for Parkinson's disease that will be competitive with existing treatments. To achieve this, the company has decided to bring all its development and manufacturing in house in order to radically reduce costs. History demonstrates that drops in the price of pharmaceutical goods are possible. Between 1998 and 2009 manufacturing improvements brought about a 50-fold reduction in the cost of goods of monoclonal antibodies. They are now routinely used in medicine.

There is another option, which is that breakthroughs go to waste. Some states have been unwilling to pay the price needed to eliminate Hepatitis c, a viral disease, despite the availability of antiviral therapies that cost around $20,000 per patient, says Dr Collins; this results in all kinds of obstacles being put up for those receiving treatment. It would be an extraordinary waste were the same to happen with the new wave of gene therapies.” [1]

·  ·  · 1.  "America will struggle to pay for ultra-expensive gene therapies." The Economist, 3 June 2023, p. NA.

 

Why legal writing is so awful.

 

""THE FIRST thing we do, let's kill all the lawyers," is one of Shakespeare's most memorable lines. You would struggle to find such a line in the writings of lawyers themselves—and not just because they would, presumably, disagree. 

Though some judges are sophisticated stylists, most legal language is fussy, tangled and incapable of producing anything so pithy. (This is no doubt one reason so many people want to kill all the lawyers.) But do lawyers write that way to impress, to bewilder—or perhaps because they must?

In a study published in Proceedings of the National Academy of Sciences, Eric Martínez and his colleagues from the Massachusetts Institute of Technology and the University of Edinburgh tried to find out. Contracts written in "legalese", as well as simplified versions conveying identical concepts, were shown to American lawyers and laypeople. It turns out that lawyers struggle with, and dislike, legal language almost as much as their clients.

Legalese is heavy on "centre-embedding", sentences in which related words are separated by a long insertion, as in "It is understood by artist and company that comprehensive liability insurance, protecting against any claim or demand up to $300,000, including attorney's fees, related to company's actions under this venue agreement, shall be purchased and maintained throughout the agreement by company." This puts a heavy strain on the brain's working memory. The word "insurance" must be held in the mind while some 20 other words go by before its attendant verb phrase "shall be purchased" arrives.

Another baleful feature of legal writing is jargon: uncommon words like hereinbefore, mala fides and lessor. These mean little more than above, bad faith and landlord. Even if most lawyers and many laypeople know the jargon, the words require more effort to recall than everyday ones.

Given the almost universal disdain for legal language, the obvious question is why it persists. Mr Martínez and his colleagues examined several hypotheses. One was "the curse of knowledge". This is the idea that many learned people do not know how to write for those less informed than themselves. But the researchers found that the lawyers struggle with legal language too. They found the content of the legalese contracts harder to understand and remember. So did laypeople, of course, but they remembered the simple contracts as well as the lawyers did the complex ones; they understood them almost as well, too.

A more cynical idea was the "it's just business" hypothesis. This holds that lawyers are intentionally opaque so as to gull clients into paying more for their supposed expertise. But that did not fit the data either, for the lawyers believed their clients would be more likely to sign the simplified contracts than the standard ones.

Perhaps legalese is a form of "in-group signalling"—behaviour used to signal belonging to a group, such as religious iconography or flag-waving at sports events, and aimed at fellow lawyers rather than clients? But the lawyers in the test group said they would be more likely to hire the writers of the simplified contracts than the authors of the traditional gobbledygook.

The most common defence of legalese is the need for precision, says Mr Martínez (who trained as a lawyer before switching to cognitive science). Legal language, in this view, is too important to leave to the imprecisions of ordinary style. But this argument was refuted too: the lawyers who read the simplified contracts rated them just as enforceable as the complex ones.

The researchers were left with a simple conclusion, which they call the "copy-and-paste hypothesis". Lawyers imitate what previous lawyers have done. After all, a good deal of rote legal work (such as drawing up contracts) can be copied in large chunks from one document to another.

Whatever the reason, changing behaviour will be hard. Experts in legal writing have called for clearer prose for decades. But the plague of legalese persists. Perhaps evidence from outside the profession will help change things—especially if it is written in plain language.” [1]

·  ·  ·1.  "Why legal writing is so awful." The Economist, 3 June 2023, p. NA.