La 30 august 2017, Novartis a anuntat ca FDA a aprobat primul terapie cu celule T cu receptor antimeric chimic (CAR-T): Kymriah (tisagenlecleucel). Terapia are un pret listat de 475.000 de dolari in prima indicatie, pentru leucemia acuta cu celule B recidive la pacientii cu varsta sub 25 de ani.

Numeroase povesti de presa au mentionat un „acord intre Novartis si CMS” prin care CMS va plati doar pentru pacientii cu rezultate reusite. CMS a emis un comunicat de presa care a fost redat incredibil de vag, iar apoi, aparent, scos de pe site-ul web al CMS.

     Update 13 septembrie: Congr. Doggett solicita CMS sa clarifice „Acordul;” Vezi aici. Ce s-a intamplat?

In comunicatul de presa al lui Novartis (aici), avem aceasta afirmatie:

De asemenea, colaborarea cu Centrele pentru Servicii Medicare si Medicaid 

Novartis a anuntat, de asemenea, o colaborare noua cu Centrele pentru Servicii Medicare si Medicaid din SUA (CMS), axata pe imbunatatirea eficientei cerintelor actuale de reglementare, pentru a oferi ingrijire bazata pe valoare si a asigura accesul pentru acest lucru populatie specifica de pacienti. Aceasta abordare este destinata sa includa preturi pe medicamente si sa sustina platile pentru un medicament, cum ar fi Kymriah pentru indicarea sa initiala, pe baza rezultatelor clinice obtinute, care ar elimina ineficientele din sistemul de sanatate. Alte abordari bazate pe valoare legate de indicatiile viitoare pentru terapiile cu celule Kymriah si CAR-T sunt in discutie. Mai mult, Novartis colaboreaza cu CMS pentru a pune la dispozitie o abordare bazata pe rezultate, care sa permita plata numai atunci cand TOATE pacientii pediatrici si adulti tineri raspund la Kymriah pana la sfarsitul primei luni. Indicatiile potentiale viitoare ar fi revizuite pentru cea mai relevanta abordare bazata pe rezultate. „Novartis a fost in fruntea preturilor bazate pe rezultate si este foarte incantat sa lucreze cu CMS la aceasta prima colaborare de acest fel cu o tehnologie care are potentialul de a transforma ingrijirea cancerului”, a spus Joseph Jimenez, CEO al Novartis . “Abia asteptamcontinuand sa lucreze cu CMS pentru a extinde potential aceasta abordare la alte produse si stari de boala. “

Observatiile lui Novartis au fost rezumate in surse secundare, cum ar fi Endpoints (aici), Fortune (aici) si mai pe larg intr-un articol de Agus & Goldman din Fortune (aici). De exemplu, acestia din urma, care sunt experti in stabilirea preturilor bazate pe valoare, scriu:

A existat un alt anunt de la o alta agentie guvernamentala, Centrele pentru Medicare si Serviciile Medicaid (CMS). Dar, de asemenea, ar putea avea un impact de anvergura asupra modului in care platim – si in cele din urma tratam – cancerul de tot felul. CMS ar acoperi pretul anuntat de Kymriah de 475.000 USD pentru tratament. Acest lucru a apucat titluri, dar pe termen lung nu se compara cu cealalta parte a anuntului de la CMS. Pentru prima data agentia a acceptat un plan de plata care depinde de faptul daca medicamentul produce de fapt un beneficiu pentru pacient. Novartis va primi plata a 475.000 de dolari numai daca pacientii raspund la medicament pana la sfarsitul primei luni de terapie.

Comunicatul de presa al CMS este mult mai vag 

I dug up the CMS announcement last week and found it amazingly vague.  It didn’t refer to any agreement.  It only stated vaguely that CMS was interesting in better pricing for drugs.   When I went back to the CMS website today, September 6, the press release had disappeared.  However, I contacted CMS and they described the problem as a web glitch.  By September 7, the unchanged press release was back up at CMS (here).

With today’s [approval of Kymriah]…the Centers for Medicare & Medicaid Services (CMS) is continuing to explore the development of payment models and arrangements…  CMS is working actively with all stakeholders, including state officials, on innovative payment arrangements. These arrangements may, for example, include outcome-based pricing for medicines…CMS will be issuing future guidance to explain how…  CMS is committed to further exploring the development of innovative pricing systems that reflect the value delivered to patients. Through the authority provided to the Center for Medicare and Medicaid Innovation (CMMI), CMS will aim to identify and alleviate regulatory barriers in Medicare and Medicaid as may be necessary to test payment and service delivery models that involve value-based payment arrangements.

CMS doesn’t say they’ve “

agreed to a payment plan

” (as stated by Agus & Goldman, but rather that CMS is “exploring” and “committed to further exploring” and “working actively” (is there another kind of working?)

What Could the CMS-Novartis Process Have Been?

  • Drug prices aren’t set willy-nilly by CMS, and not at the spur of the moment judgment of thoughtful administrators.  
  • Drug prices are set by law and regulation in both the hospital and freestanding settings (the “average sales price” or ASP lawmaking dating to 2003.)  
    • See Hays v Leavitt, 2009, halting CMS from implementing drug pricing differently than allowed by law just because CMS found a pricing method that made more sense than the law, in the view of the agency.
  • But can’t a company voluntarily agree to a different pricing scheme with CMS?   Without due process?  I’m not so sure.  What if CMS had determined that it only pays $50,000 for Kymriah, and only after proof one-year outcomes?  
    • A satisfied pharma would never complain, and the issue would never reach court.  But an unhappy pharma?  Wouldn’t a pharma then complain (and in court) that administrators have no such ad hoc powers to assign one-off coverage and prices by fiat?  
    • Put this way:  If CMS can make up any rule or policy it wants overnight, that you like and agree to, it can make any new rule overnight, that you don’t like at all. 
  • For Medicare, coverage decisions, by law, are called National Coverage Determinations or NCDs and follow a regulatory process laid out in law.  I don’t think CMS can ignore those laws at the drop of a hat when it seems like the right moment.  But here, CMS announced it was covering Kymriah out of the blue – a de novo agency coverage decision.
  • CMS senior staff don’t pay for drugs: The claims are paid by the eight regional contractors, following specific instructions from CMS.  How would you define everything in the Novartis agreement?  Those long explicit instructions to contractors would usually be transmitted publicly.  And they’d depend on existing rules and regulations.  
    • And by the way, pharma companies don’t bill CMS: Hospitals do.  
    • The hospital would get paid — or not paid — by CMS after it’s already bought the drug.   (Maybe Novartis could rebate each hospital upon proof that CMS had denied the hospital payment – but CMS payments or payment denials initially involve only the hospital that submitted the bill.)    
  • And Kymriah doesn’t currently involve Medicare at all, where CMS has more control by hiring and instructing its contractors.  Rather, Kymriah is for patients under age 25 who would have to be Medicaid (CHIP) patients to fall under CMS authority.  

While the first indication for Kymriah is for young Medicaid patients, later indications will follow quickly and they involve Medicare.  What if it’s a drug given for inpatients?  Then, it bumps up across DRG bundles with no extra payment for drugs administered.  Or bumps into the Oncology Care Model bundle.  And for inpatients, even the New Tech DRG add on payment by law only pays 50% of the added cost – not much good for a $475,000 drug.

With this background, try reading the Novartis quotations and CMS quotations again.  I see the goal, but I don’t see the process.

CMMI May Be the Missing Link, Because CMMI Can Do Anything

CMMI, which CMS now prefers to call the Innovation Center, was created by the ACA in 2010.  It has a budget of about a billion dollars a year and has established numerous small and large demonstration programs.

By law, CMMI can conduct Demonstration Programs under which it may waive any Medicare law for the purpose of the demonstration program.   This quickly led to concerns among conservatives that CMMI is bad law – that Congress can’t give the agency a large legal rulebook, and then, delegate to the same agency the authority to waive any or all of the laws that create the agency.   (See Grace-Marie Turner in Forbes last year, here; see also here).  (And Congress agreed with Marie-Turner, see House Ways & Means, May 2016, here.)

De exemplu, multe parti interesate s-au opus in 2016, cand CMMI a propus un set nou de politici pentru plata medicamentului Medicare, care sa prevada legile existente si la nivel national si cu durata nelimitata.

(De aici, titlul meu: CMMI poate face orice. Un program „demonstrativ” ar putea fi la nivel national si 100 de ani , deoarece Congresul nu a spus niciodata cat de mult poate fi sau cat de mare poate fi. Cel putin pana cand o instanta nu a trebuit sa inteleaga Autoritatea legala deschisa. Deoarece CMMI poate renunta la orice lege dintr-un program demonstrativ si un program demonstrativ poate fi de orice anvergura, se pare ca CMMI ar putea scadea varsta Medicare la 60 sau ar putea ridica la 70 sau poate limita la 85. Si majoritatea din creatiile politice ale CMMI sunt protejate de lege de controlul judecatoresc. *)

I think that’s why, vague as it was, CMS’s press release stated that “Through the authority provided to the Center for Medicare and Medicaid Innovation (CMMI), CMS will aim to identify and alleviate regulatory barriers in Medicare and Medicaid…”

So CMMI could produce a program, specific to Kymriah, and that applies to all 50 state Medicaid programs, and that specificies a “Kymriah demonstration program” payment process to be implemented immediately and nationwide.  But it would be a “demonstration program” that was abruptly and unilaterally created by executive branch burocrats, national in scope, exactly what Republicans went crazy over in 2016 when Obama was in charge.

A Less Gentle CMS, For Better and Worse?

Many of us who have worked on CMS policy issues for a decade have seen instances where CMS declined to make a policy change, because CMS wasn’t totally, completely sure it had enough authority, perhaps even under notice-and-comment rulemaking; because somebody of nerdy temperament buried in the Office of General Counsel of CMS had cold feet.

With the direction that Novartis’ announcement went in, CMS was appearing to act much faster and more dramatically than it had been able to, under its previous understanding of the limits and guardrails of its legal authority.  

_____

[*]

(d) Implementation [of CMMI].—

(1) Waiver authority.—The Secretary may waive such requirements of [CMS law:] titles XI and XVIII and of sections 1902(a)(1), 1902(a)(13), and 1903(m)(2)(A)(iii) as may be necessary solely for purposes of carrying out this section with respect to testing models described in subsection (b).

(2) Limitations on review.—There shall be no administrative or judicial review under section 1869, section 1878, or otherwise of—

(A) the selection of models for testing or expansion under this section;

(B) the selection of organizations, sites, or participants to test those models selected;

(C) the elements, parameters, scope, and duration of such models for testing or dissemination;

(D) determinations regarding budget neutrality under subsection (b)(3);

(E) the termination or modification of the design and implementation of a model under subsection (b)(3)(B); and

(F) determinations about expansion of the duration and scope of a model …

____

Inside Health Policy recently reported that CMS may be issuing a request for public comments on the operations and future goals of CMMI; here.   Updatge:  It’s out; September 20; here.

For an essay on CMMI by Manatt, September 12, 2017, here.

For a law journal article that includes a section on “unprecedented and unauthorized” CMMI overreach, here (PDF page 14ff).

ARTICOLE SIMILAREDE LA ACELAȘI AUTOR