A professional transformation is already under way

THE FUTURE OF THE PROFESSIONS: how technology will transform the work of human experts

The concept of professional job is changing. A new book tries to reflect the impact of technology on professions.This is a key statement:

We are on the brink of a period of fundamental and irreversible change in the way that the expertise of these specialists is made available in society. Technology will be the main driver of this change. And, in the long run, we will neither need nor want professionals to work in the way that they did in the twentieth century and before. There is growing evidence that a transformation is already under way.

Understanding the new scope and implications of such transformation requires an open mind and being aware. It will be a slow change, the authors say, but deep and irreversible towards the post-professional society.

In the long run, increasingly capable machines will transform the work of professionals, giving rise to new ways of sharing practical expertise in society. This is the central thesis of our book. We cannot commit to timeframes, in large part because the speed of change is not in our hands. But we are confident that the change will constitute an incremental transformation rather than an overnight revolution. In the language of the book, the shift itself can be characterized in many ways: as the industrialization and digitization of the professions; as the routinization and commoditization of professional work; as the disintermediation and demystification of professionals.

A much-needed start: soda tax

Soda Politics: Taking on Big Soda (and Winning)

Obesity is a top concern on public health. Personal and collective responsibilities are linked. The concrete issue is the following one: government may require manufacturers to release information to consumers (about calories, composition, etc.), but is there anything else that he can do?
Current strategies fall short to achieve the goals of obesity reduction. Nutritional labels are not enough, are taxes an option?. Some countries have already implemented taxes on fizzy drinks, fat or salty foods. There are complex technical issues to be considered. However, The Economist says that taxes on fizzy drinks seems to work as intended. If this is really so, then there is a much-needed reason to start in this way.
Marion Nestle in her latest book "Soda Politics" provides the hole list of arguments. Any regulator should read in detail the book, specially part IX on "Advocacy: Soda caps, taxes and more", and take into account her recommendation:

 Let me acknowledge immediately that advocacy to reduce soda intake faces special challenges that distinguish it from advocacy for reduction of alcohol, tobacco, or junk foods. Like these other industries, the soda industry sells relatively inexpensive products that are available in almost every corner of the globe. Like them, this industry is extremely wealthy. Also like the others, health is the industry’s Achilles’ heel. But in sharp contrast to companies selling junk food, alcohol, or tobacco, Coca-Cola and PepsiCo consistently rank among the most admired, respected, and honored companies in the world. Health and environmental advocates must recognize the power of this favorable public perception when encouraging others to resist it.

PS. A must read. Understanding 25 years of health policy in Catalonia, released in this journal: Referent. You'll find an article that I have written for the occasion.

Lab tests and biomarkers regulation: a pending topic

La regulació de proves diagnòstiques i biomarcadors: una assignatura pendent

Pere Ibern

Centre de Recerca en Economia i Salut. Universitat Pompeu Fabra. Barcelona.

ANNALS DE MEDICINA: VOLUM 98, NÚMERO 4, octubre / novembre / desembre 2015

http://www.acmcb.es/Portal/academia/Publicacions/Annals/Article/_eUM6Y0IFwOjYx8pCJ5Bx15ay5J1m7qc6wEtj4brzgoIIUpHpSJRmF06atHkGpIiX2pkBCC6HLMLzC5Sk3H_dIR1o3PfH30UE

Introducció

A partir de l’any 2010, a Europa va augmentar la preocupació per la seguretat i l’eficàcia dels subministraments mèdics i proves diagnòstiques. Les alertes relatives a la seguretat de les pròtesis mamàries PIP (Poly Implant Prothèse) van ser-ne el detonant. El Parlament europeu va iniciar una revisió de la regulació existent perquè la considerava desfasada i insatisfactòria per assolir els objectius que pretenia. L’any 2012 es van publicar les propostes de nova regulació, però malauradament el cicle electoral no va permetre la seva aprovació. Recentment, el mes de juny de 2015 s’ha publicat la nova proposta que conté múltiples esmenes i tracta d’arribar a un consens definitiu. Si tenim en compte que la regulació actual relativa a proves diagnòstiques és de 1998, i que es proposa un termini de 5 anys per a la seva aplicació, hauran passat efectivament més de vint anys sense adaptar-ne la regulació en un context d’innovació tecnològica accelerada.

La medicina estratificada

La regulació per a protegir la salut dels ciutadans hauria de seguir un procés paral·lel al canvi tecnològic i la innovació, però sabem que es produeixen retards notables en la presa de decisions públiques. Així, per exemple, en aquestes dues darreres dècades hem estat testimonis de l’eclosió de la medicina estratificada¹. Entenem per medicina estratificada aquella que millora els resultats de salut i la capacitat predictiva mitjançant la utilització de biomarcadors. Les condicions necessàries per desenvolupar la medicina estratificada són tres: un mecanisme biològic singular que aporti respostes diferencials dels pacients a la teràpia, unes opcions terapèutiques múltiples que ofereixin respostes heterogènies i un biomarcador clínic que relacioni les teràpies amb una subpoblació de pacients que probablement mostrarà una resposta diferencial. L’aparició de proves diagnòstiques complementàries (companion diagnostics), una de les modalitats de biomarcadors, permet l’estratificació de pacients i la selecció de medicaments i dosi; així augmenta l’eficàcia i es redueixen els efectes adversos.

L’Agència Europea del Medicament (EMA, sigla de l’anglès European Medicines Agency) havia autoritzat fins a 20 medicaments oncològics a principis de 2014 que inclouen un biomarcador farmacogenòmic (Tau1a 1)². L’aplicació acurada de la medicina estratificada té la singularitat de reduir la mida del mercat potencial de la medicina empírica, quan s’administra als pacients que presenten una característica determinada. Aquest fet té implicacions múltiples en relació al preu i el retorn de la investigació, qüestions que ara són de gran actualitat.

TAULA 1. Medicaments oncològics autoritzats per l’Agència Europea del Medicament a principis de 2014 que inclouen un biomarcador farmacogenòmic²

Abreviacions: UE: Unió Europea; DCI: Denominació comuna internacional; ACA: Assaigs clínics aleatoritzats.

La dificultat essencial rau en l’avaluació de les proves diagnòstiques complementàries en la mesura que presenten nous reptes desconeguts fins el moment³. Els tres àmbits on el regulador ha d’oferir resposta són: validesa analítica (la fiabilitat i precisió per detectar la variació genètica d’interès), validesa clínica (fiabilitat i precisió per detectar pacients amb la malaltia d’interès) i utilitat clínica (possibilitat que aporti una millora en la salut). Tradicionalment, l’enfoc de l’avaluació de les proves diagnòstiques ha estat en la validesa analítica; amb la medicina estratificada cal anar més enllà i relacionar-ho conjuntament amb l’opció terapèutica.

Actualment, els biomarcadors són considerats per la regulació europea de diagnòstic in vitro com de baix risc i, per tant, no calen dades d’eficàcia clínica o utilitat clínica per a la seva adopció. A la proposta de nova regulació hi ha un canvi de classificació i es consideren classe C, és a dir, que tenen un risc moderat per a la salut pública o un alt risc individual. En aquests casos, el procediment actual d’autocertificació ja no serà suficient; caldrà aportar informació precisa de validesa analítica i clínica i d’utilitat clínica a les entitats certificadores. El procés de coordinació amb l’EMA encara no està definit i aquesta és una mancança important. Les diferències entre la regulació europea i la dels Estats Units són notables. La Unió Europea ha optat per autoregulació i certificació en l’àmbit privat, amb empreses certificadores, mentre que als Estats Units hi ha regulació directa des de l’àmbit públic. Ara bé, la darrera proposta europea assenyala un nivell d’intervenció superior que implica major control directe sobre les entitats certificadores i sobre els requeriments d’informació. Es trobaria, per tant, a mig camí. Atesa la complexitat dels biomarcadors, la garantia d’aplicació d’un procés d’avaluació homogeni pot quedar en dubte quan no hi ha un organisme central que ho verifica, com és el cas de l’EMA per als medicaments. És llavors quan, a posteriori, les agències d’avaluació de tecnologies previsiblement acabaran tenint un paper clau en aquest àmbit.

L’experiència d’avaluació de tecnologies sanitàries per a les proves diagnòstiques complementàries és encara molt primerenca. L’aplicació de l’anàlisi cost-efectivitat a aquestes proves planteja reptes metodològics nous que han estat descrits per part de l’agència britànica NICE (National Institute for Health and Care Excellence)⁴. La diferència clau sorgeix si la prova diagnòstica s’ha desenvolupat en el marc de l’assaig clínic del medicament o no i, per tant, quin és el seu impacte per estratificar subpoblacions. En aquest sentit, la guia del NICE suggereix avaluar l’impacte diferencial d’una prova diagnòstica complementària en el cost-efectivitat del medicament. El programa d’avaluació de proves diagnòstiques dins el NICE ha anat prenent forma i publica els informes corresponents una vegada els medicament tenen l’autorització de comercialització.

Allò que les agències d’avaluació de tecnologies prendran com a punt de partida són uns biomarcadors que han estat aprovats per ser aplicats conjuntament amb un possible medicament segons la indicació establerta per l’EMA. Però cal tenir en compte que la definició dels punts de tall (cut off) de la prova diagnòstica i aquesta definició la duu a terme el fabricant. En la mesura que la sensibilitat i l’especificitat no siguin del 100%, hi ha proves diagnòstiques que mostraran falsos negatius i falsos positius. És llavors quan la definició del punt de tall té a veure amb la mida del mercat potencial per al medicament. Per exemple, si el punt de tall és alt això comportaria una elevada especificitat i pocs falsos positius i s’obtindrien els millors resultats clínics. Però, alhora, el fabricant obtindria menors ingressos degut al sistema de preus vigents, que no té en compte el valor en salut que aporten els medicaments. Hi ha, per tant, implicacions múltiples de la definició del punt de tall, tant per a pacients com per a empreses, regulador i finançador. Per ara, aquesta decisió recau en les empreses, però caldria avaluar-ne possibles alternatives i que les agències hi tinguessin un paper. Segons Trusheim i Berndt⁵, amb l’actual sistema de preus dels medicaments, l’estratègia preferida per fabricants i pacients seria la d’un punt de tall baix o mitjà —que aporta més tractaments a més pacients i també amb més falsos positius. Però això els portaria a una situació tipus “dilema del presoner”, on cadascú fent el millor per ell mateix acaba obtenint el pitjor resultat, lluny de l’eficiència òptima.

Hi ha almenys dues qüestions diferencials de caràcter regulador en la medicina estratificada: l’avaluació conjunta de medicament i prova diagnòstica complementària i la fixació del preu. Tant la US Food and Drug Administration (FDA) com l’EMA han publicat les seves guies d’avaluació sobre aquest tema, si bé la qüestió dels punts de tall comentada resta pendent de clarificació. De fet, planteja problemes pràctics perquè més enllà d’exigir que la prova diagnòstica s’inclogui a l’assaig clínic —que no passa sempre—, cal també establir diferents escenaris per calibrar millor l’impacte de la sensibilitat i l’especificitat en els resultats en salut.

En relació a les alternatives a la fixació de preus, en certa mesura, els acords de risc compartit serien una opció a tenir en compte si els indicadors fisiològics de la malaltia són clars i unívocs. Sabem que no sempre és així i aquest és el motiu pel qual no es poden dur a terme o que aquells que ho fan acabin essent imperfectes. Les decisions de prioritats terapèutiques de recerca i previsions de facturació a la indústria farmacèutica es fan amb caràcter global. Els preus acaben essent locals; cada país o cada finançador té els seus. L’opció pràctica a considerar és que si es mantenen els preus com a mecanisme i no hi ha o no són possibles contractes de risc compartit, les agències del medicament també haurien de tenir veu a l’hora de decidir el punt de tall que maximitza el valor en salut.

La medicina de precisió

Al llarg d’aquest article s’ha utilitzat el terme medicina estratificada. Recentment, el terme medicina de precisió ha estat objecte de gran ressò per la inversió multimilionària en recerca anunciada pel govern nord-americà. La definició que s’estableix és pròxima: són aquells tractament dirigits a les necessitats de pacients individuals a partir de característiques genètiques, epigenètiques i biomarcadors que els distingeixen d’altres pacients similars. Aquesta definició ens situa més enllà dels biomarcadors farmacogenòmics dels que hem parlat abans.

Ens trobem doncs en un moment de confluència d’aplicació de tecnologies a la medicina que obliga a estar atents a la seva adopció en funció del valor que aporten. La comprensió de la seva complexitat exigeix, a més a més, millor coneixement i formació per part dels diferents actors en el sistema de salut.  Entre ells destacaria també els organismes reguladors. Les proves diagnòstiques són una peça clau d’aquest desenvolupament tecnològic i, a data d’avui, la seva regulació encara és una assignatura pendent de resoldre.

REFERÈNCIES BIBLIOGRÀFIQUES

1. Trusheim MR, Berndt ER, Douglas FL. Stratified medicine: strategic and economic implications of combining drugs and clinical biomarkers. Nat Rev Drug Discov. 2007;6(4):287-93.
2. Pignatti F, Ehmann F, Hemmings R, Jonsson B, Nuebling M, Papaluca-Amati M, et al. Cancer drug development and the evolving regulatory framework for companion diagnostics in the European Union. Clin Cancer Res. 2014;20(6):1458-68.
3. Ibern P. The hole for genetic testing market entry. Bloc Econsalut, 25 de febrer de 2014. Consultable a:http://econsalut.blogspot.com/2014/02/the-hole-for-genetic-testing-market.html. Accés el 9 d’octubre de 2015.
4. Byron SK, Crabb N, George E, Marlow M, Newland A. The health technology assessment of companion diagnostics: experience of NICE. Clin Cancer Res. 2014;20(6):1469-76.
5. Trusheim MR, Berndt ER. An overview of the stratified economics of stratified medicine. Working Paper No. w21233. National Bureau of Economic Research; 2015.

Why healthcare providers merge?

Scaling Care: An analysis of the structural, social and symbolic dimensions of scale in healthcare

The specific answer to this question depends on the context. However, if you think about the Netherlands, then you'll find the reply in chapter 1 of this book and as a journal article.

Scale is used to achieve political and organizational goals. In these developments, policy makers, executives and other actors have high expectations of the relation between (changes in) scale and positive outcomes, like quality and efficiency of care. To meet the expectations, they ‘upscale’ and ‘downscale’ the organization and provision of care on both organizational and geographical scales. However, the outcomes of these policies and strategies turn out to be uncertain and contested.

The autors conducted a survey with healthcare managers and show their results:

Of the five categories of merger motives, healthcare executives most often mention the category related to healthcare provision (n=107; 69 per cent). This indicates that executives regard merger as an instrument to change the organization and delivery of healthcare services. By realising a broader/more specialized range of services or by providing services to new groups of patients, they seem to aim at attracting new patients and/or offer more or better services to their existing patients. Almost equally frequently mentioned is the category of motives related to strengthening the market or bargaining position.

Sounds obvious. Measurement according to opinions finally get that, opinions. That's why I'm concerned about Delphi questionnaires. The opposite approach is to get data and check the hypothesis. Have a look at this book: Quantitative Techniques for Competition and Antitrust Analysis and you'll understand why this quantitative approach is as relevant as difficult to implement.The final answer is still open for a new estimate and discussion.

PS. What's going on in UK NHS? Peter Roderick gives us some clues. (25 years of marketisation in a short article). More details, here.

How universal is universal coverage?

An analysis of perceived access to health care in Europe:How universal is universal coverage?

Two different realities are intertwined: healthcare access right and needs-based access. The first is widely acknowledged in European countries, the second depends on the specific measurement of geographic (and financial) barriers to healthcare providers.
An article in Health Policy sheds some light on the issue. And its results are compelling:

Despite clear commitments to move towards universal health coverage in Europe, our results suggest that there remains significant heterogeneity among individuals in terms of their perceptions of access to care across and within countries. Overall, we find that the poorest groups are still the most likely to feel they will be unable to accesscare if they need it. In some countries however, differences in the probabilities of perceiving access barriers between low and high-income individuals are relatively small. This insinuates that rationing mechanisms that affect all income groups, such as low quality care and long waiting times may serve as important barriers.

PS. There is no clear pattern between out of pocket expenditure as a percentage of total health expenditure, and the predicted probability of perceived inability to access care:

MABS in history of medicine

The Lock and Key of Medicine Monoclonal Antibodies and the Transformation of Healthcare

While reading FT this summer I came across an article quoting a unique book on history of monoclonal antibodies (MABS). Right now there are more than 30 drugs in the market based on hybridoma technology that was created in 1975.
The birth of MABS is explained with full details, how the creators finally didn't patented it and why, the difficulties for research in an unconnected world, etc... An exciting story that is worth reading. Right now, it would be completely different, commercialization of research and medicine has raised considerably.

That a British company spearheaded the first marketing of Mabs, a technology devised in a British laboratory by an émigré Argentinian scientist with his German colleague, highlights the international nature of biotechnology commercialization. Sera- Lab’s venture to sell Mabs took place in the midst of the excitement generated by the founding of Genentech in 1976. The emergence of Genentech, which had been set up
to market recombinant DNA products, galvanized numerous alliances among academics, entrepreneurs, and venture capitalists to launch new companies to commercialize biotechnology. Most of the early enterprises set up in the wake of Genentech’s birth were dedicated to exploiting recombinant DNA for the mass production of natural products such as interferon and insulin for drugs. But the early germination of the modern biotechnology industry did not rest solely on recombinant DNA. By the 1970s a number of pioneering companies were developing Mab products, including Sera- Lab and two startups: Hybritech in San Diego and Centocor in Philadelphia. Entrepreneurs who risked entry into the field had no guarantee of success and were entering totally uncharted
territory. Such individuals faced major fi nancial, personal, professional, and regulatory challenges as well as a great deal of hostility, pessimism, and litigation.

Milstein with Köhler at the time of their receiving the Nobel Prize in 1984 together with Nils Jerne.

Mabs have had their strongest therapeutic impact in the field of cancer. The first Mab to reach the market for cancer was edrecolomab (Panorex), which was granted German regulatory approval in 1995 for the treatment of postoperative colorectal cancer. Developed by Centocor in partnership with the Wistar Institute, it was withdrawn in 2001 because of its poor effi cacy in comparison with other drugs. Since 1997, however, the U.S. Food and Drug Administration (FDA) has approved twelve Mab drugs for cancer treatment, including rituximab (Rituxan), approved in 1998 for the treatment of non- Hodgkin’s lymphoma. By 2012 there were over 160 candidates in clinical trials for cancer, with seventy of them in phase III trials, the stage before a drug is submitted for regulatory approval.

Mabs have enabled the identification and characterization of cancerous tumors previously difficult to detect and diff erentiate from other tumors, thereby providing a better understanding of cancer. They have also opened a path to more personalized medical treatment. Trastuzumab (Herceptin), for example, was specifically developed to target HER2/neu, a protein overexpressed by tumors found in 25 percent of newly diagnosed breast- cancer patients

Alcohol and health

Alcohol consumption and cardiovascular disease, cancer, injury, admission to hospital, and mortality: a prospective cohort study

The relationship between alcohol intake and health is a complex one. A recent article in The Lancet tries to disentangle the causes and consequences:

The amount, type, and pattern of alcohol consumption can have differing associations with health outcomes. For example, low–moderate regular intake of alcohol is associated with reduced risk of myocardial infarction, whereas heavy episodic drinking is associated with sudden cardiac death.6 Risk of injury increases with extent of intoxication, whereas risk of cancer is related to the amount consumed over time.

In a study of 114.970 participants from 12 countries on the relationship between alcohol and its impact on certain diseases has allowed to understand that geographic location plays a relevant role, beyond the type of alcohol consumption. These are the results:




In summary, the study shows that current drinking is not associated with a net health benefit. We confirm that high intake is associated with increased risk of mortality, cancer, and injury. A serious alert for spirit drinkers and a key message for an updated public health strategy. Have a look at the UK example.

Disposable income trends

From: EU Statistics and book.

Healthy longevity

Health at a Glance 2015

Every year OECD updates health database. The latest one was released yesterday. Japan, Spain and Switzerland are at the top of life expectancy at birth with 83 years. However,  in healthy life expectancy at 65,Switzerland is in the 9th position, and Spain in the 13th. Why is this so?. The priority on quality of life should increase.

Physicians' quality: incentives and information

In the USA, the Affordable Care Act requires the federal government to post information about physician performance and quality of care on a public website. The recent experience of public and private initiatives has been reviewed in a Health Affairs Brief. If you want to know the implications and details on how to make information accessible for citizens, this is a key document to read.

• By 2019 doctors who treat Medicare beneficiaries must choose between two options—enroll in a program called the merit-based incentive payment system or sign up to be part of an alternative payment model.
• The merit-based incentive payment system will adapt and combine multiple programs —PQRS, the EHR meaningful-use program, and the value-based payment modifier initiative—into one.
• Doctors opting for the merit-based incentive payment system must report quality-of-care measures to CMS. How is to be determined, but CMS likely will use an upgraded PQRS reporting system.
• Physicians will be scored on four components of care: quality (30 percent); resource
use (30 percent); meaningful use of EHRs (25 percent); and practice improvement  activities (15 percent).
• Physicians choosing the alternative payment model path would have to be part of an integrated health system, join an ACO.

Sounds interesting. Incentives and information altogether, a hint for other health insurance markets on what to do about it.

Owners and managers in state owned firms

Governance issues are at the center of current debates in public and private firms. Unfortunately, too often as a cosmetic perspective. The roots of the problem are left for another day. Let me give an example. Recently, a hospital selected its CEO by a unique procedure: asking the physicians about the right candidate (at least this is what has been published in the press).
Selecting the right leadership for a firm is the most relevant issue for any board of directors. However, in this case, they will have only one candidate coming from a proposal of professional employees of the firm. The board will confirm their option, for sure, otherwise it will boost a huge crisis.
The worst approach to selecting leadership is to confound participation with governance. In state owned firms, the governance belongs to elected politicians (of course they delegate to appropriate professionals selected for the board). The ownership is public, and citizens are represented through politicians. That's it.
In this unique case, politicians or their delegates have been supplanted by professional employees. This is a biased way to understand governance functions in state owned firms, and somebody should review it in depth before this approach is converted into a trend for other hospitals. Here you'll find the roadmap.

The Theranos contretemps as a serious scandal

Last Thursday WSJ released a long article on Theranos clinical lab. In this blog you may check my February and July posts on this firm under the title: A closely guarded secret. As you may imagine, such a title was not coincidental. There were some clues that justified it, something unusual was happening. And WSJ has contributed to shed light on the issue. All the details in it. Basically, the summary is that analytic validity and clinical validity is under compromise. This is an exemple:



If you want to read a first person account, you'll find it here and here. Some additional articles: Wired, New Yorker, Clinical Chemistry and Laboratory Medicine (CCLM), Forbes, NYT, WP,...
This is not only a contretemps, it is a serious scandal and a huge problem to credibility for this start-up.
From Wired:

Theranos got a lot of traction by tapping into the frustration—both from consumers and the medical community—that diagnostic testing is too painful, too slow, and too expensive. “Their problem is they tried to do it with existing diagnostic instrumentation, instead of innovating new diagnostic instrumentation,”

Theranos is a black box that has touted results rather than process. “The ability of the lab medicine community to police and correct itself depends on that flow of information,” says Master. Instead, Theranos’ research was internal, and rather than submit their work to peer review the company cited their FDA approvals as evidence that the technology worked.

At least in the USA there is a regulator, the FDA, lab regulation in Europe was enacted in 1998, completely outdated under a third party scheme, not a direct public regulator. Therefore, there is a pressing motive to speed up new and different rules in Europe. Microfluidics and nanotechnologies are calling for and urgent overhaul.


 PS. An statement from WSJ:

In 2005, Ms. Holmes hired Ian Gibbons, a British biochemist who had researched systems to handle and process tiny quantities of fluids. His collaboration with other Theranos scientists produced 23 patents, according to records filed with the U.S. Patent and Trademark Office. Ms. Holmes is listed as a co-inventor on 19 of the patents.

The patents show how Ms. Holmes’s original idea morphed into the company’s business model. But progress was slow. Dr. Gibbons “told me nothing was working,” says his widow, Rochelle. In May 2013, Dr. Gibbons committed suicide. Theranos’s Ms. King says the scientist “was frequently absent from work in the last years of his life, due to health and other problems.” Theranos disputes the claim that its technology was failing.

Precision medicine in an outdated regulation

Specially dedicated to european politicians, this was my op-ed in Diario Médico last week:

La medicina de precisión en una regulación obsoleta

El retraso regulatorio europeo en pruebas diagnósticas y suministros médicos es descomunal

El avance tecnológico coge por sorpresa a gobiernos y parlamentos. El retraso en actualizar la regulación a las nuevas realidades frena a su vez la difusión de las innovaciones e introduce incertidumbres sobre la eficacia y seguridad. Desde hace más de una década sabemos que el impacto en la asistencia sanitaria de los avances en el genoma humano tendría lugar algún día inexorablemente. Sin embargo,a fecha de hoy, estamos a la espera de su regulación.
En 2005 un periódico anunció que llegaba la medicina personalizada pero muy pronto nos dimos cuenta que todo era más complicado de lo que algunos proclamaban. En 2011, Science decía en portada: “Lo mejor está por venir”. A la secuenciación del genoma humano le faltaba todavía mucho trayecto para que se concretara en impacto en la salud poblacional. Y en el mismo año se publicó el informe del National Research Council estadounidense: “Toward Precision Medicine”. En él se mostraba el impacto del conocimiento biomédico en la clasificación de la enfermedad, describía el reto de la redefinición de la enfermedad a partir de la biología molecular, a la vez que popularizaba el nuevo término: Medicina de Precisión.
En marzo de 2012, Cell publica un artículo crucial donde se relata la aparición molecular de una enfermedad. Un profesor de Stanford relata la evolución de su perfil ómico personal, analiza qué ocurre durante dos años, cómo surge la enfermedad y cuál es la reacción molecular del cuerpo. Explica cómo descubre el riesgo de diabetes y cuando se confirma, qué hace a partir de aquel momento.
Del titular del periódico de hace una década nos acercamos, ahora sí, a la medicina de precisión, intensiva en datos. Las inversiones públicas en iniciativas de investigación son tan solo una parte del todo. Lo que llamábamos “medicina estratificada”, la que une pruebas diagnósticas complementarias (companion diagnostics) para determinar el tratamiento farmacológico, ya está entre nosotros. Ahora es un ejemplo más de medicina de precisión. Y resulta que a fecha de hoy, en Europa tenemos una regulación vigente de 1998 (!). Desde 2012 circula un borrador para actualizar la directiva sin éxito. Recientemente, el pasado 23 de septiembre, ha surgido un nuevo borrador que la Comisión quiere elevar al Parlamento para su aprobación. Veremos qué sucede en esta ocasión.
El texto considera las pruebas diagnósticas complementarias esenciales para definir aquellos pacientes que son candidatos a un tratamiento específico con un medicamento, a través de la determinación cuantitativa o cualitativa de marcadores específicos que identifican los sujetos con mayor riesgo de desarrollar reacción adversa al medicamento o la identificación de los pacientes en la población para los que el producto terapéutico se ha estudiado de manera adecuada, y es seguro y efectivo. La forma como se evalúa la seguridad y eficacia queda al margen de los procesos equivalentes en los medicamentos y se mantienen las entidades certificadoras, si bien con cierto control adicional. Y la pregunta es la de siempre: ‘Quis custodiet ipsos custodes?’, ¿quien vigila al vigilante?. Este “cierto” control gubernamental será suficiente? O quizás cada estado lo resolverá a su propio estilo?.
El sistema europeo basado en la autoregulación, en entidades certificadoras privadas, queda lejos del sistema norteamericano donde las pruebas diagnósticas complementarias se regulan públicamente por la FDA. De esta forma, un medicamento que requiere una prueba diagnóstica queda regulado en Europa en dos ámbitos distintos, con requisitos de evaluación desiguales. En definitiva, estamos a las puertas de una regulación que es incapaz de tener en consideración que la validez y utilidad clínica en la medicina de precisión debe ser objeto de regulación pública directa.
El retraso regulatorio europeo en pruebas diagnósticas y suministros médicos es descomunal. A fecha de hoy se sigue un proceso para acceder al mercado que requeriría una revisión profunda de su impacto en términos de validez y utilidad clínica. La credibilidad de la evaluación se confirma siempre que existan criterios objetivos, fiables y homogéneos. Los mecanismos actuales de certificación son insuficientes para los objetivos que se esperan de una regulación avanzada. Del mismo modo que el escándalo de los implantes mamarios PIP recordaron la falta de garantía y control en suministros médicos, las pruebas diagnósticas requieren una regulación fiable que garantice seguridad y calidad, al mismo tiempo que aporte información sobre su efectividad. No hay opción para más demora. La nueva propuesta de regulación de pruebas diagnósticas necesita una mejora inmediata o, de otro modo, se convertirá en obsoleta a corto plazo.

 

Mapfre Foundation. Barcelona exhibition.

Disentangling ageing and health

World report on Ageing and Health

Although it may surprise, there is no clear pattern to indicate that elderly people  always utilise more health services. When one looks at the aggregated indicators, one may reach this conclusion, however taking into account the realities and individual characteristics, there are many remaining factors to explain the situation. This fact is evidenced by the last WHO report on aging and health. (P.90).
Even considering the increased morbidity at older ages, the burden of chronic diseases is diverse in terms of cost. Thus, few users and its distribution in the phase prior to death, it is the main cause of the high cost of chronic disease. Proximity to death and corresponding therapeutic effort confound the ageing factor in increasing health costs.
Furthermore, it has been shown that the compression of morbidity continues to advance. That is, the occurrence of chronic diseases is delayed gradually, the phenomenon of "survival of the fittest" is confirmed. The impact or impairment of chronic disease in human physiology for the very old is attenuated .
It is for these reasons that the report is worth, a new look to a reality that is already upon us and we need to give appropriate response.

A cause and consequence of progress (again)

Today I would like to suggest you check my previous posts on Angus Deaton, the economics Nobel prize winner. And if you have more time, a close reading of his book is required: The Great Escape: Health, Wealth, and the Origins of Inequality.
Angus Deaton deserves the prize -NYT dixit-. John Cassidy writes an excellent op-ed (The New Yorker) with the title: Angus Deaton: A Skeptical Optimist Wins the Economics Nobel. Nothing to add.

Using behavioral insights for policy

Social and Behavioral Sciences Team 2015 Annual Report

Some weeks ago, an executive order by President Obama boosted the application of behavioral insights to policy.

To more fully realize the benefits of behavioral insights and deliver better results at a lower cost for the American people, the Federal Government should design its policies and programs to reflect our best understanding of how people engage with, participate in, use, and respond to those policies and programs. By improving the effectiveness and efficiency of Government, behavioral science insights can support a range of national priorities, including helping workers to find better jobs; enabling Americans to lead longer, healthier lives; improving access to educational opportunities and support for success in school; and accelerating the transition to a low-carbon economy.

Sounds interesting. It poses a tweak on the current approaches to policy design. Cass Sunstein says in NYT:

 When government programs fail, it is often because public officials are clueless about how human beings think and act. Federal, state and local governments make it far too hard for small businesses, developers, farmers, veterans and poor people to get permits, licenses, training and economic assistance.

 Behavioral research shows that efforts at simplification, or slight variations in wording, can make all the difference.

The UK and now the USA are introducing this new way to define policies (and health policy). Let's keep an eye on its application and performance. What are we doing in this respect? Why are we waiting to introduce something similar?


 President Barack Obama speaks with members of the Social and Behavioral Sciences Team in the Oval Office in January 2015.

Cost-effectiveness of public health interventions

The case for investing in public health

The evidence shows that a wide range of preventive  approaches are cost-effective, including interventions that address the environmental and social determinants of health, build resilience and promote healthy behaviours, as well as vaccination and screening. The evidence in this report shows that prevention is cost-effective in both the short and longer term. In addition, investing in públic health generates cost-effective health outcomes and can contribute to wider sustainability, with economic, social and environmental benefits.

Cost-effectiveness studies  are usually focused towards treatments. This report shows some examples related to public health. Unfortunately,  this is not so common. Up to now my reference on this tòpic was this article. Now I'm adding this report by WHO Euro. And the question remains: if these interventions are so cost-effective, why are we waiting for their implementation?

It is recognized that a comprehensive strategy needs to include a combination of population and targeted individual preventive approaches, but it should be noted that, on average, individual-level approaches were found to cost five times more than interventions at the population level (WHO, 2011a). In general, evidence also shows that investing in upstream population-based prevention is more effective at reducing Health inequalities than more downstream prevention (Orton et al., 2011). Meanwhile, the National Institute for Health and Care Excellence in the United Kingdom found thatmany public health interventions were a lot more cost-effective than clinical interventions (using cost per QALY), and many were even cost-saving (Kelly, 2012).

The healthcare funding conundrum (once again) (2)

Fiscal Sustainability of Health Systems. Bridging Health and Finance Perspectives

Sustainability defined by OECD (p.25):

The OECD defines fiscal sustainability as the ability of a government to maintain public finances at a credible and serviceable position over the long term (OECD, 2013). Fiscal sustainability implies governments are able to maintain policies and expenditure into the future, without major adjustments and excessive debt burdens for future generations. The term refers to overall government spending, revenues, assets and liabilities that reflect past commitments and adapt to future trends such as socio-economic trends and environmental factors.

For the health sector, fiscal sustainability is perhaps best understood as a constraint that needs to be respected, rather than an objective in itself (Thomson et al., 2009). This implies that how governments achieve fiscal sustainability matters, rather than it  becoming a simple cost-cutting exercise.

Of course!. Sometimes OECD writes obvious statements. There is a budget constraint! Perhaps someone has forgotten it.
I understand that in some countries the budget is not credible, because its health basket is more generous and prices are higher compared to the available resources. Is  this a reason to complain about cutbacks?. If there is a budget, the role of any politician is to comply with the budgetary constraint, they must prioritise. Health care is not an excuse to surpass the budget and responsible citizens and parties should acknowledge it.

The healthcare funding conundrum (once again)

Fiscal Sustainability of Health Systems. Bridging Health and Finance Perspectives

Forget economic forecasts, most of them have failed many times. You don't need to be precise about the size of the GDP devoted to health in 2030, it will definitely be more expenses than now. The OECD tries in a new report to review these forecasts and to spread fear in the near future. I think that our societies before the great recession, were able to manage to some extent fiscal deficits. Now it is different, the size of the deficit for future generations is unacceptable.
The report reviews former approaches to supply side and demand side measures for cost-containment. A well known story on the macro-prescription for governments.  My view is not reflected in the document. I have highlighted many times the importance of organizational change, the micro-perspective, i.e. the changes in the structure of incentives and coordination mechanisms in the health system.
This is the most challenging effort for any government, because organizational change and prioritisation represents an attempt to modify the current status quo. Governments are affraid of it, but in my opinion somebody has to handle this conundrum and tell  the population exactly that unless we change current health care organizations, taxes will not be sufficient to pay the bill. Then two options will be open to us: out of pocket or no access. This is the message that this report is unable to explain clearly and now it's time for somebody to disseminate it.

PS. Read the Fiscal Sustainability of Health Systems: Policy Brief 

Measuring population health

Morbilidad, utilización de recursos y costes sanitarios en la comarca del Baix Empordà

In this blog I've explained many times that if I had to pick one health system as a benchmark I would say that it is  Serveis Sanitaris Integrats del Baix Empordà (SSIBE). This is my choice. I've been contributing over the last decade to understand what has happened to the morbidity, utilization and costs of this population.
Now, you have all this research effort in a PhD Dissertation by J. M. Inoriza, a must read. I strongly recommend you to have a look at it. Congratulations to all contributors.

Health policy: what works?

Successes and Failures of Health Policy in Europe. Four decades of divergent trends and converging challenges

Three questions to answer:

• Do differences in rates of disease reflect differences in related policies?
• What would a country gain if it implemented the policies of the best performing country?
• Which social, economic and political factors influence a country's success in health policy?

And the (partial) answer is in chapter 14 of the book:

Gains in health since 1970 for the causes analysed in this chapter have clearly been enormous. While not all of these declines can be attributed to health policies, part of the decline in all these causes can  be, as shown in the previous chapters. For example, some of the declines in lung cancer and  ischaemic heart disease can be attributed to tobacco control; some of the declines in external cause mortality among children to injury prevention, and some of the declines in death rate from RTIs to road safety measures, and so on. It is impossible to estimate the specific contribution of preventive health policies to these declines, but even if these accounted for only half or a quarter of the cause-specifi c declines, the successes would be immense. At the same time, not all countries have been  equally successful in bringing down mortality from these preventable causes, as shown again by the calculations.

The book was released in 2013 with data from 5 years before, an update would be necessary. Anyway, a close reading is required.

The modeling approach to health decisions

Applying Modeling to Improve Health and Economic Policy Decisions in the Americas: The Case of Noncommunicable Diseases

On the role of modeling in health, this OECD report says:

In the health sector, the purpose of modeling is to structure evidence on clinical and economic outcomes in a form that can inform decisions about clinical practices and health care resource allocations. Models synthesize evidence on health consequences and costs from many different sources, including clinical trials, observational studies, insurance claim databases, case registries, public health statistics, and preference surveys. A model’s logical, mathematical framework permits the integration of facts and values and links these data to outcomes that are of interest to health care decisionmakers

I agree. The report shows some examples of aggregated modeling without panel microdata. If you compare this approach with David Eddy one, Archimedes models, it is exactly the opposite. And my opinion is in favour of Archimedes, as you can check in my previous posts. The most useful approach to modeling is the one that allows improve decisions at micro level, physician and patient.

PS. An innovative approach with practical implications in SSIBE, Palamós.

 Bonnard exhibition in Madrid.

The size of income inequality (2)

Source: World Economic Forum: Inclusive Growth and Development Report 2015

Epigenetics contribution to clarify disease mechanisms

Epigenetics at the Crossroads of Genes and the Environment

You may find an updated definition of epigenetics in this JAMA article:

 Epigenetics refers to information transmitted during cell division other than the DNA sequence per se, and it is the language that distinguishes stem cells from
somatic cells, one organ from another, and even identical twins from each other. Examples include (1) DNA methylation, a covalent modification of the nucleotide cytosine, that is copied during cell division at CpG dinucleotides by the maintenance enzyme DNA methyltransferase I; (2) posttranslational modifications of nucleosome proteins about which the DNA double helix is wrapped; and (3) the density of  nucleosomes and higher-order packaging of chromatin within the nucleus, including its relationship to the nuclear lamina.

If this is so, why is the message of predictive genetics so widespread?. I've insisted on this issue before.

 The field of epigenetics and epigenetic epidemiology have much to do to improve measurement of epigenetic marks, inform natural variation in such marks, and the biological and population level relationships between genes, environment, and epigenetics. This is an important emerging area as it holds promise for better risk prediction in precision medicine as well as for clarification of disease mechanisms among the existing opaque landscape only partially informed by traditional genetic and environmental studies to date.

 A short and relevant article that provides hints for further reading.

PS. Epigenetic phenomena, from Nature.

Ownership and access to medical data

Unpatients—why patients should own their medical data

Eric Topol says in Nature Biotechnology:

Today, in the United States, health data live in a plethora of places, from electronic health record (EHR) systems, insurance claims databases, siloed personal health apps, research and clinical trial databases, imaging files and lots of paper. Although seemingly everywhere, any true semblance of an overarching organization or standardization of medical data are lacking, whether at the individual or societal level

His proposal is straightfoward: the ownership of the clinical record is of the patient. This situation is completely different in our country. We have public centralised repositories and the patient is the owner. There is still a lack of coordination and many things to solve, however the basics are covered in the publicly funded System, that's not the case in the private sector.

In contrast to the legal and technical difficulty an individual faces to obtain all his or her own medical data is the relative ease with which hackers have managed to breach ~100 million patient records in the first half of 2015

And his proposal:

 We must begin talking about creating a health data resource in a much broader and more universal context, controlled by the individuals who supply the data. This is a unique moment where we may be able to provide for personal control and, at the same time, create a global knowledge medical resource.

Sounds interesting, though methodology is crucial for success.


PS. Hacking electronic records:

The timeline for electronic medical data hacks in the United States of over 1 million individuals

Cardiovascular disease and diabetes: progress and prospects

OECD Health Policy Studies Cardiovascular Disease and Diabetes

A recent OECD report highlights the huge improvement on cardiovascular disease and at the same time explains the policies and strategies needed for the near future. Treatment improvement accounts for 40% in the decrease of mortality, change in risk factors 50%, while 10% is unexplained. This means that public policy is critical for success. Nowadays it is still the first disease to tackle. On diabetes, the report explains that 85 million europeans are currently suffering from the disease. Therefore, it remains a severe issue.
I suggest you take a close look at the report.

Healthy life expectancy: the key indicator

Global, regional, and national disability-adjusted life years (DALYs) for 306 diseases and injuries and healthy life expectancy (HALE) for 188 countries, 1990–2013: quantifying the epidemiological transition

In former posts I have advocated that healthy life expectancy should be used as an aggregated indicator of outcomes of health determinants. Unfortunately although it is imperfect, it is the best we have, and today you can check the latest estimates in a salient article in The Lancet.

Countries with highest healthy life expectancy, both sexes, 2013

1	Japan
2	Singapore
3	Andorra
4	Iceland
5	Cyprus
6	Israel
7	France
8	Italy
9	South Korea
10	Canada

Spain has disappeared from the former top 10. I have always said that ordinal rankings have flaws, however, comparisons across time are worth taking into account.
Internally in Spain it is difficult to reverse this trend. If you consider geographic variations of the indicator you can see a range from 55 years up to 65 (p.25) .Therefore there is too much noise when you focus on the state as the unit of analysis. Forget the results and the ranking.
The only situation that is unique in the ranking is Andorra, it always appear at the top and it is a small state. That's remarkable and merits close analysis.

Henri Matisse, Promenades intérieures

Exposition du 12 juin au 4 octobre 2015

Musée Matisse

Beware of healthcare providers consolidation

The Potential Hazards of Hospital Consolidation Implications for Quality, Access, and Price

The key message:

  With the current most substantial consolidation of health care in US history, the concerning implications of the trend of hospital consolidation on quality, access, and price must be carefully considered. However, unlike banks that became too big to fail, 85% of US hospitals pay no taxes because they are designated as nonprofit organizations serving a public good. Hospitals can set prices that are ultimately passed on to others in the form of escalating insurance deductibles and taxes.

The alternative:

 The good work of integrated hospitals should continue to create networks of coordinated care, while at the same time, physicians and patients should insist that hospitals compete on transparent prices and quality outcomes. Achieving this goal is an important prerequisite to a functional health care system.

Tackling obesity: the toolbox

Patchy progress on obesity prevention: emerging examples, entrenched barriers, and new thinking

World Cancer Research Fund International NOURISHING framework 
Food policy framework for healthy diets and the prevention of obesity and diet-related non-communicable diseases. 

 Key message:

The problem of obesity must be reframed to acknowledge on one hand that individuals bear some personal responsibility for their health, but that, on the other hand, environmental factors exploit biological, psychological, social, and economic vulnerabilities that promote overconsumption of unhealthy foods. A vicious cycle is created in which the preference and demand for unhealthy products are not only shaped by the environment, but lead to environmental changes that further encourage consumption of unhealthy foods. This cycle makes it difficult for people to act in their own long-term self-interest, but it can be broken with regulatory actions from governments and joint efforts from industry and civil society to create healthier food systems.

European health regulation on lab tests, the final round?

Medical devices: Council getting ready for talks with EP

Last June 15th, there was a small but significant step towards the final agreement on Medical devices and in vitro diagnostics regulation in Europe. The need for reform has been widely requested but the lack of political consensus and the low priority given to the issue has delayed its approval in many ocasions. It seems that now is the right opportunity, however if finally passes, it will be applied on 2020!!!. It really sounds weird that it would take 5 years to be fully developed.
Anyway, if you want to have a look at the details check here and here. Right now, the lobbies are not on vacation, they are fighting against some provisions that limit their current freedom of market access. Pay attention to final result, just to check who wins and who loses, and the current state of power balance between society and the lobbies.

 



NESBITT, Lowell. Dos ponts a Nova York, 1975

Exhibition at Espai Carmen Thyssen. San Feliu de Guixols

The size of income inequality

 

Income declared to tax authorities, not actual income. Selected statements:

• 3% of citizens account for 30% of tax collection!
• In 5 years, plutocrats have decreased by 50%!
• In 5 years, average work income has plummeted 3,6% and taxes jumped to 9,5%!

My congratulations, tax authorities!

 

 



The price of cancer drugs, where is the limit?

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Stratified medicine: defining the size of the market

AN OVERVIEW OF THE STRATIFIED ECONOMICS OF STRATIFIED MEDICINE

Trusheim and Berndt provide an excellent overview of the economics of stratified medicine. I have reviewed the topic before and specifically in this post. An ideal companion diagnostic perfectly identifies and distinguishes treatment responders from those who will not.Unfortunately, in practice no diagnostic performs ideally. All diagnostics experience some level of error. In the case of a companion diagnostic, some patients will receive false positive results, scores indicating they will respond, but will not when treated. Other patients will receive false negative results, scores indicating they will not respond, but if treated they would.
So what? The key issue is about the setting of the cut-off that will define the size of the market:

Under competition, three essentially identical drugs may receive dramatically different labels, incremental cost-effectiveness ratio (ICER) justified pricing, and market positioning depending on their stratification approach. It appears superior to use an imperfect biomarker to none at all. It is less obvious whether patients, payers and firms prefer the same cut-off values for the companion diagnostic, or even whether each stakeholder a priori prefers the high, low or perhaps some other CDx cut-off value.
The competing development teams may face a version of the game theory ‘prisoner’s dilemma’ in which the optimal result for patients and all firms would be to select a low or mid companion diagnostic cut-off value but the advantages of a potentially differentiating high efficacy claim may drive developers to select a high cut-off value. If all choose this approach, overall value may be reduced with many patients excluded from treatment. But the potential advantage of a higher cut-off value may prove too alluring, or the fear of a competitor selecting one, may drive all to do so. Each situation will depend on the specific facts of the indication, therapeutic, companion diagnostic and competitors

The implications for regulating and financing of companion diagnostics are immediate. In Europe current legislation is as old as of 1998, the year that Herceptin entered into the market and the begining of stratified medicine. New regulation is still pending and there are  no perspectives about a unified approach to lab tests assessment. European regulator is still on vacation.

Lab on a chip concept, the future of diagnostics?

Future vision: next generation lab on chip concept from imec on Vimeo.
Imec develops the next generation of “lab on a chip” concepts. The idea is that such a disposable chip could be loaded with a sample of blood, saliva or urine and then quickly analyzed using a smartphone, tablet or computer, making diagnostic testing faster and easier for applications such as disease monitoring and management, disease surveillance, rural health care and clinical trials.

The market for health insurance price comparison

Internet has provided multiple option for price comparison through website aggregators. On health insurance you can find several alternatives. Is this market competitive? Who wins and who losses?. This is the question asked in a recent article in The Economist:

Consumers should celebrate that; the firms’ losses are their gains. But there is a catch. Comparison sites, whether for insurance or something else, introduce a new layer of costs, including their own splashy advertising campaigns. In theory, competition in the market for comparison sites ought to keep those costs down. But in a recent paper, David Ronayne of Warwick University argues that consumers often lose out from comparison sites. They earn a commission for each shopper who uses them to buy insurance. That referral cost is incorporated into the price the consumer ends up paying. If the increased costs outweigh the saving the comparison enables, consumers end up worse off.

And the proposal:

How can you ensure the market for price comparison is competitive? Asking consumers to check multiple websites defeats the point of using them. One solution is to have only one site, but regulate it as a public utility. Alternatively, the government could run the site itself—much as the American government now runs comparison websites for health insurance under Obamacare.

I think that this is the right approach, why not apply it here?

Regulating sugar sweetened beverages

Searching for Public Health Law’s Sweet Spot: The Regulation of Sugar-Sweetened Beverages

Nowadays, obesity prevention lies at the heart on any public health policy. If sugar sweetened beverages contribute decisively to obesity, then something should be done. What?. A recent PLOS article explains the options:

The main regulatory approaches are taxes, restrictions on the availability of SSBs in schools, restrictions on advertising and marketing, labeling requirements, and government procurement and benefits standards.

On taxes:

Savvy regulatory design has tremendous potential. For example, there is growing evidence that taxes that are more salient to consumers, such as those included in a good’s posted price (rather than being levied at the register), are more likely to influence purchasing behavior

On public procurement, a practical suggestion for immediate application:

Restrictions on which beverages may be purchased using government funds are a less visible form of regulation, but one with potential to change the consumption patterns of large numbers of people. Outside public schools, these standards are most germane in two areas: procurement standards for public institutions (e.g., government agencies, hospitals, and prisons) and restrictions on what recipients of government benefits for the indigent may buy with those funds.
The UK’s Government Buying Standards prohibit central government bodies from  procuring SSBs larger than 330 ml and encourage the wider public sector to follow the guidelines. Massachusetts  and many US counties and cities have adopted nutrition standards for government contracts, but most apply to a limited set of institutions, such as childcare facilities or youth centers.

PS. My former posts on the same topic.
PS. Article: The impact of sugar sweetened beverages on depression risk in adults.
PS. Report: Scenarios of Macro-economic Development for Catalonia on Horizon 2030
PS. Report: FBBVA Essential Public Services.
PS. Report: Chronicle of a premeditated offensive.

Efficiency in drug patents buyout

Panning for gold: sourcing pharmaceutical innovation

Once upon a time there were pharmaceutical firms that invested mostly in internal R+D departments. Long time ago, the door was opened to contracting out, buying patents and licensing. The most recent step is to the acquisition of firms with promising molecules.
A short article in Nature sheds some light about the efficiency of recent mergers and acquisitions. And the summary is:

Our analysis suggests that most companies have a considerable opportunity to get better at deploying capital and resources efficiently when sourcing innovation externally. In our experience, we have found that the best performers develop robust forecasts for the key assets, are fiscally disciplined, and set up their innovation-sourcing teams and transaction capabilities to ensure that the right internal expertise is brought to bear and to ensure smooth hand‑offs through the life cycle of a deal.

The success lies within, the internal expertise is crucial. Have a look at the figure and you'll notice that the most efficient (defined as the commercial impact of the products acquired relative to capital deployed in M&As) is Roche. This is not by chance.

PS. Unfortunately, the study doesn't reflects any consideration to value in health or lobbying efforts in drug prices.
PS. FT's summer books 2015 

A closely guarded secret (2)

Direct Access Testing is the next battle for a new market for lab testing. This is at least what Theranos considers and has been lobbying for. Last December in The New Yorker there was an explanation about the firm and its goals. Afterwards JAMA added some caveats on the secretive way of conducting business and I wrote a post on that. Now The Economist has published an article with the details of the current situation about their business model.
Selling tests directly to the patient is a controversial issue. As in most of prescriptions, patients don't know enough to prescribe for themselves. However, how much is enough?. Arizona is starting to liberalise such prescriptions after Theranos successful lobbying efforts. Professional societies reflect in a position paper their perspectives on the issue.
My view is very straightforward: avoiding commercialism in health care. Under insurance coverage, prescriptions should be required after being cleared by regulators. Without insurance coverage, recreational tests have also to be licensed by regulatory authorities under a disclosure process that has not been the Theranos case. Nowadays, it still remains a secret. Selected tests could be accepted without prescription according to its implications on Health and information accuracy.

Beware of competition in healthcare

Competition among Health Care Providers – Investigating Policy Options in the European Union

Let's imagine an official in European Commission. They are in favour of more competition in health care and asks an expert committee to assess the issue. This is the concrete answer in selected statements from the report:

First, and foremost, introducing or increasing competition in the provision of health care services is a delicate policy exercise. The conditions for success and risks for failure need to be carefully assessed. In the right context, introducing competition may help to meet some health system objectives, although it is unlikely to contribute simultaneously and positively to all.

Neither economic theory nor empirical evidence support the conclusion that competition should be promoted in all health services

Neither competition nor strict reliance on government regulation will solve all health system problems. Attempts to avoid or correct market failure can result in government failure and vice versa.

Provider competition can contribute to improving value in health service delivery, but details about where, when and how to introduce competition are critical. Competition in health care provision will not solve all health system problems and may have adverse effects.

Competition is unlikely to achieve improvement in all aspect of health system performance at the same time. It will not solve all the trade-offs policy makers face between different, sometimes conflicting, health system objectives.

Competition can at the same time increase the number of services provided and billed, creating uncertainty in relation to overall health care costs. That is, the introduction of competition may well result in increased costs and add to fiscal pressures. Increased costs may, or may not, be justified by additional health benefits to the population (or some parts of the population).

As competition is an instrument, sound policy evaluation studies are needed to assess and judge its effects. Such empirical studies are currently rare and even absent in some countries.

The introduction of competition has uncertain effects on equity of access to health care, as it is conditional on the effects above and on the heterogeneity of patients. Empirical work has found that the introduction of competition among hospitals, in the UK, produced little or no result in equity terms. This limited evidence does not allow for general presumptions about the effects of competition on equity of access to health care.

Now it is crystal clear. The official has had a precise answer that it is exactly the opposite they were expecting. I've said the same in this blog several times. Take care.

Implications of real world data

Breaking New Ground with RWE

Some decades ago evidence based medicine was the key issue in understanding effectiveness in health care and drugs. Right now the new term is Real World Evidence. You'll not find it in the wikipedia, instead I would suggest you look at this IMS report:

RWE is drawn from robust anonymous patient-level data using sound scientific and commercial analytics. It is not about amassing "Big Data" so much as performing targeted analyses on ever expanding healthcare datasets.
While RWE is known to complement data from Randomized Clinical Trials (RCTs), its real potential is in moving decisions away from perceptions and broad extrapolations to the actual facts about patient journeys and outcomes. With innovations in data and technology, RWE is replacing other information sources such as non-behavioral primary market research (PMR), standard market reports, consumption/market data purchases, observational studies, and even selected spending on RCTs.

Its impact could be large if drug prescription decisions and pricing takes into account the outcomes from the "patient journey" as they say. This is a new paradigm with uncertain spillovers. The true evidence based medicine with the power of big data. We must be aware of it and follow it closely.

PS. How to introduce innovative medicines?. Have a look at this workshop.