Rethinking drug regulation and health risk mitigation

FDA in the Twenty-First Century. The Challenges of Regulating Drugs and New Technologies

A new book on FDA addresses the  "perennial and new problems and the improvements the agency can make to better serve the public good.". The book would deserve a detailed critique, however, let me skip directly to chapter 25: Device-ive Maneuvers FDA’s Risk Assessment of Bifurcated Direct-to-Consumer Genetic Testing.
In this blog I've written about the same topic. My position is clear: stop direct-to consumer testing. I have explained the rationale here. And what the book says, it is exactly the same:

Three steps are necessary to ensure the safety and effectiveness of DTC
genomic information. First, the underlying data must be analytically  valid—that is, the genomic data sequence must be accurate and precise. Second, the information must be clinically valid—the findings must be causally associated with clinical outcomes. And third, the risks of disclosing the genomic information must be minimized. FDA’s ability to effectively regulate genomic information hinges upon the approach taken to each of these challenges

Meanwhile, in Europe, nobody cares about it. It's a great shame.

 

 

La Lídia Pujol canta l'imne d'Occitània, "Se canta" (apelat tanben "Aqueres montanhes" en la Val d'Aran), dins lo programa de TV3 (Television de Catalonha) 

Learning occitan

Comunicat del 20 de genièr de 2016 del President de l’IEO sul tractament de las lengas regionalas per l’estat francés.
ASSASSINAR SEI LENGAS, ANSIN VÒU LA FRANÇA ?
L’Institut d’Estudis Occitans (IEO) denóncia aut e fòrt que l’Assemblada Nacionala ague fach rebuta a la proposicion de lèi de Paul Molac, relativa a l’Ensenhament immersiu dei lengas regionalas e a sa promocion dins l’espaci public e audiovisuau.
Per memòria, aquesta denegada vèn après : la promessa vana dau candidat Hollande sus la ratificacion de la Carta europenca dei Lengas Regionalas ò Minoritàrias (ambé lo debat mancat a l’Assemblada Nacionala), lo rapòrt Filippetti de julhet 2013 e sei 42 prepausicions passadas per malhas, tornat mai en octòbre de 2015 l’engatjament dau president de la Republica per la Carta, rebutat per la drecha senatoriala e que dire de l’ensenhament dei lengas regionalas assecat…
Convendretz que i a de qué se pausar la question.
Sorda que mai se pòt pas a la demanda populària, la França, es que vòu pas finalament assassinar sei lengas regionalas per de bòn ?
A la veritat, aquesta decision fa chifrar, notadament per son escart d’ambé la demanda sociala, e discredita lo foncionament democratic.
D’escondons, vesèm s’organizar l’eliminacion metodica de nòstrei lengas de l’espaci public republican.
Amb aqueste refús, la França demòra un còp de mai dins lo rodolet dei país reborsiers que donan ges d’estatut a sei lengas. Lo pluralisme linguïstic, pasmens, es una aisina de coësion indispensabla per la Nacion e un element de sa credibilitat internacionala.
Acceptar la postura dau Govèrn actuau de la França que mespresa de lengas que parlan sei ciutadans a milierats se pòt pas mai !
L’IEO demanda per aquò au Govèrn d’iniciativas nòvas que laissan oblidar lei còps que s’es mancat.
Coma lo digueriam encara en octòbre 2015 a Montpelhier, volèm una lèi !
Volèm una lèi que done un estatut vertadier ai lengas regionalas e garantisse sei locutors de tota exclusion e discriminacion.


Pèire Brechet
President de l’IEO

 

Inducing methylation

Nutrition, Exercise and Epigenetics: Ageing Interventions

Epigenetics refers to an inheritable but reversible phenomenon that changes gene expression without altering the underlying DNA sequence. Thus, it is a change in phenotype without a change in genotype. The field of epigenetics is quickly growing especially because environmental and lifestyle factors can epigenetically interact with genes and determine an individual’s susceptibility to disease. Interestingly, aging is associated with substantial changes in epigenetic phenomena. Aging induces global DNA hypomethylation and gene-specific DNA hypermethylation due to the altered expression of DNA methyltransferases (DNMTs).

The evidence of the impact of epigenetics on aging is growing. And nutrition plays a key role on epigenetics through the life course. Thus, there are crucial reasons to focus on nutrition early in life.

It is clear that epigenetic alterations caused by aging may provide a milieu that can develop age-associated diseases such as cancer, cardiovascular diseases, neurocognitive diseases and metabolic diseases. Nutrition is one of the most important environmental factors that can modify epigenetic phenomena. Therefore, one might speculate that nutrition may delay the age-associated epigenetic change and possibly reverse the aberrant epigenetic phenomena that can cause age-associated diseases. Indeed, many nutrients and bioactive food components, which can affect one-carbon metabolism that can regulate methylation of DNA and histone or directly inhibit epigenetic modifying enzymes, are showing promising results in delaying the aging process and preventing age-associated diseases through epigenetic mechanisms.

And beyond nutrition, there is exercise. This is what this book explains and it shows the foundations for better health. If it's "only" an issue of regulating methylation...where are the incentives?

Economic perspectives on public health

Promoting Health, Preventing Disease:The Economic Case

The rethoric behind disease prevention and Health promotion is well known. However, in practice, more needs to be done. A new book by WHO Euro reflects the economic case for such policies

Health promotion and disease prevention have a major role to play in Health policy worldwide, yet they are underused, partly because evidence to suport a strong case for action is difficult to gather. Aimed at a broad audience of policymakers, practitioners and academics, this book is designed to provide an economic perspective on the challenges to better health promotion and chronic disease prevention.

Four diferent considerations:

• The costs of inaction: What are the economic consequences of not taking action to promote and protect the health of the population?
• The costs of action: What would it cost to intervene by providing a promotion or preventive measure?
• The cost-effectiveness of action: What is the balance between what it costs to intervene and what would be achieved in terms of better outcomes – e.g. emotional well-being, physical health, improved quality of life, educational performance?
• The levers for change: What economic incentives can encourage more use of those interventions that are thought to be cost-effective and less use of those interventions which are not?

This book will be a key reference for public health policies, the background and state of the art, pros and cons of each policy, are clearly detailed. Highly recommended.

The quid pro quo at the heart of the international health regulations

Disease Diplomacy. International Norms and Global Health Security

The implementation of international health regulations has its pluses and minuses. Some countries show reluctance to accept a new landscape of global health security. All these issues are reflected in an interesting recent book.

We have seen dramatic changes in the past decade. The engagement of high-level political and security communities with an area that had previously been primarily treated as a technical concern has been a major cause of that change, but it has also meant that the new system is taking time to settle. Prior to 2005, the IHR had not been substantially revised since 1969.

International norms are by their very nature collective ideas, and they rely on that collectivity—the notion of states as forming a cohesive international society—in order to function effectively. When a number of states cannot meet the IHR core capacity requirements and cannot attract the help they need to do so, the entire ethos of the global health security regime is undermined. This, for us, is the challenge facing norm leaders: how to maintain the regime’s political purchase when the security discourse used to establish it is increasingly met with antipathy post-H1N1 and with a lack of financial support to institutionalize the necessary capacities in the domestic structures of the poorest states. Thus, one of the key lessons we draw from the norm-building process examined in this book is that recognizing appropriate behavioral standards and “wanting to do the right thing” are not the same as having the ability to conform to those standards.

A disease-producing organism

Disease Selection. The Way Disease Changed the World


Understanding human life is a great undertaking. After all these years the origins of our cells are not so clear. But let me quote a recent book and its suggested approach:

Evolutionary biologists have looked for some time for a suitable prokaryotic cell that when engulfed by another would form the nucleus of the nascent eukaryotic cell, but none has been identified that matches all the required criteria. However, Luis Villarreal, working with viruses, has come to the astounding conclusion that the primitive cell nucleus could have originated from a complex virus. The vaccinia virus, for example, seems to have all the same mechanisms that are required by a eukaryotic cell nucleus. The virus that formed the nucleus brought with it all the basic genes – thought to number about 324 – that are necessary to form the cell.
It requires a little time, and perhaps rereading of what has just been said, to realize that every cell in our bodies has a nucleus that was derived from a virus. We are the result of a very early disease process!

So not only is the nucleus of our cells derived from a virus but the mitochondria are from a parasitic bacterium. There can be no closer link between us and disease-producing organisms.

Technology, organization and the health system

The digital revolution: eight technologies that will change health and care

A short article by the King's Fund describes key technologies for the future. Really, the future is now:
1. The smartphone
2. At-home or portable diagnostics
3. Smart or implantable drug delivery mechanisms
4. Digital therapeutics
5. Genome sequencing
6. Machine learning
7. Blockchain
8. The connected community
As usual, we need to split technologies according to its value and social willingness to pay. Easier said than done. That's why I would suggest a close look at the report on Better value in NHS. Technology innovation without organizational change to take advantage of its value is extremely costly.
This is the agenda for NHS transformation:

Fragmented regulators in globalized markets

Food and Drug Regulation in an Era of Globalized Markets

The complexities of a globalized world have its impact on food and drug regulation. The options for a collaborative space between different agencies are huge, though the interest is low. Its an issue of power and fear, everybody knows that cooperating would be better, but a lack of commitment is the final result. This is not only an issue for health, of course, but I would like to highlight the fact that this should be the first issue of concern by health politicians worlwide. Meanwhile, you can read this book, though it is partial and limited but shows the current situation.

Public health for the world

Readings in Global Health Essential reviews from the New England Journal of Medicine

The book starts with this statement:

“Global Health” is a relatively recent construct, largely replacing and extending concepts embedded in prior terms such as “Tropical Medicine” and “International Health.” We have adopted a working definition of Global Health as “Public Health for the world”. In this view, everyone in the world is the relevant population, and Global Health seeks to prevent and treat the diseases that compromise good health anywhere in the world. A brief review of the origin and evolution of the concept of Global Health may cast light on some of the institutions and impulses still intrinsic to the study of Public Health for the world.

That's it. A selections of 18 articles from NEJM (plus intro and conclusions) is presented  in the book in order to understand the current change of paradigm:

1. Harvey V. Fineberg and David J. Hunter: A Global View of Health-An Unfolding Series
Part I: Global Disease Patterns and Predictions
2. Christopher J.L. Murray and Alan D. Lopez: Measuring the Global Burden of Disease
3. Anthony J. McMichael: Globalization, Climate Change, and Human Health
Part II: Infectious Diseases
4. Anthony S. Fauci and David M. Morens: The Perpetual Challenge of Infectious Diseases
5. Peter Piot and Thomas C. Quinn: Response to the AIDS Pandemic-A Global Health Model
6. Harvey V. Fineberg: Pandemic Preparedness and Response-Lessons from the H1N1 Influenza of 2009
7. Rupa Kanapathipillai, Armand G Sprecher, and Lindsey R Baden: Ebola Virus Disease: Past and Present
8. Gary J. Nabel: Designing Tomorrow's Vaccines
9. Donald R. Hopkins: Disease Eradication
Part III: Non-Communicable Diseases
10. Majid Ezzati and Elio Riboli: Behavioral and Dietary Risk Factors for Noncommunicable Diseases
11. Prabhat Jha and Richard Peto: Global Effects of Smoking, of Quitting, and of Taxing Tobacco
12. David J. Hunter and K. Srinath Reddy: Noncommunicable Diseases
13. Anne E. Becker and Arthur Kleinman: Mental Health and the Global Agenda
14. Robyn Norton and Olive Kobusingye: Injuries
Part IV: Health System Responses
15. Zulfiqar A. Bhutta and Robert E. Black: Global Maternal, Newborn, and Child Health-So Near and Yet So Far
16. Anne Mills: Health Care Systems in Low- and Middle-Income Countries
17. Jennifer Leaning and Debarati Guha-Sapir: Natural Disasters, Armed Conflict, and Public Health
Part V: Global Institutional Responses
18. Julio Frenk and Suerie Moon: Governance Challenges in Global Health
19. Lawrence O. Gostin and Devi Sridhar: Global Health Law
20. Nigel Crisp and Lincoln Chen: Global Supply of Health Professionals
21. David J. Hunter and Harvey V. Fineberg: Convergence to Common Purpose in Global Health

The book is specially appropriate for any health professional and politician. Clearly written and concrete messages, allows the reader to undestand that public health requires a deeper global coordination.

Health economics of genomics

Genomics and Society: Ethical, Legal, Cultural and Socioeconomic Implications

 Current advances in genomics require an update for health economists. A first reading that  I would recommend is the chapter by James Buchanan titled "Health economics perspectives of genomics" that has been published in a recent book. James is a health economist that works in Oxford University, and his blog is a must read on the topic.

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: