Precision medicine: a deep breakthrough in life sciences paradigm

Bioscience - Lost in Translation? How precision medicine closes the innovation gap

It is not so easy to translate knowledge into practice, and this is the case of biosciences into clinical applications. However, recently this trend is accelerating and precision medicine is emerging. A new book gives us the highlights to understand precisely what's going on: Bioscience - Lost in Translation? How precision medicine closes the innovation gap.

Richard Barker (the author of 2030 - The future of medicine) says:

The classic definition of diseases has been in terms of the symptoms they cause and/ or where in the body they appear. This was the best that medicine could do when external observation of the patient was the only or primary means of diagnosing disease. The  powerful new tools of molecular biology are reinterpreting disease in terms of aberrant,
defective, or unbalanced molecular mechanisms at the cellular, organ, or organism level. Molecular level diagnosis becomes a real possibility. Such an approach brings effective therapy immediately closer. Molecular diagnostics can separate diseases with similar symptoms but different underlying causes— and often suggest a different starting point for intervention.

If this is so, what should we do?

The seven changes of mindset and of practice are:
1. Advance the molecular definition of disease and the application of systems biology. We need a more decisive move from a classic definition of diseases— in terms of the symptoms they cause and/ or where in the body they appear— to a definition in terms of aberrant, defective, or unbalanced molecular mechanisms at the cellular level. And we need to marry this with a recognition that singular target- based innovation rarely works: we need a systems biology approach.

2. Partner academia and industry in more collaborative, impact- oriented research. We need to extend the ‘open innovation’ approach in which academia and companies invest together and share IP. We need to define new pre- or non- competitive spaces, especially in work on disease mechanisms and disease models. And we need to provide for new types of links and incentives to break down the barriers between these two worlds. 

3. Move decisively to a more adaptive approach to development, trial and approval design. We need to build on successful experiments in more flexible trial design, development pathways, and regulatory appraisal to a globally accepted adaptive approach. This involves collaborative design of the evidence package needed to secure approval and reimbursement, and greater teamwork through the process. 

4. Create new reward and financing vehicles for leading edge innovation. We need to move from reward systems based purely on unit sales of products, irrespective of outcome, to rewarding innovators for positive outcomes, patient by patient. We also need to design financing mechanisms that bridge between cost- effectiveness and affordability. We must be able to accommodate high- cost precision therapies that offer cures and so generate long- term returns for the system.

5. Engineer tools and systems for faster and better innovation adoption and adherence. We need to move from reliance solely on promotion to doctors and passive patient participation to a disciplined approach to establishing new pathways of care. These will be based on modern behavioural science, clinical decision support, and other digital technologies.

6. Develop an infrastructure for real- world data- driven learning. We now have the opportunity to study in large populations how lifestyle and treatment choices lead
to outcomes, learning from every patient as if in a clinical trial. New analytical tools will empower this.

7. Bring patients into the mainstream of decision- making and engage them  hole heartedly throughout the process. It is time to move from a process and mindset in which patients are regarded as passive subjects for clinical trials and recipients of products and procedures. Their input and engagement needs to be sought along the whole innovation chain: on treatment benefits, acceptable risks, optimal clinical trial design, adherence support, and outcomes.

Highly recommended.

Drug pricing overhaul

 Pricing for Medicine Innovation: A Regulatory Approach to Support Drug Development and Patient Access

The message:

We introduce a regulatory approach we term “Pricing for Medicine Innovation” (PMI), which departs dramatically from the market-equilibrium assumptions of conventional (neoclassical) economics. The PMI approach recognizes the centrality of collective investments by government agencies and business firms in the productive capabilities that underpin the drug development process. PMI specifies the conditions under which, at the firm level, drug pricing can support both sustained investment in these capabilities and improved patient access. PMI can advance both of these objectives simultaneously by regulating not just the level of corporate profit but also its allocation to reinvestment in the drug development process. PMI suggests that although price caps are likely to improve drug affordability, there remain two potential issues with this pricing approach. Firstly, in an innovation system where a company’s sales revenue is the source of its finance for further drug development, price caps may deprive a firm of the means to invest in innovation. Secondly, even with adequate profits available for investment in innovation, a firm that is run to maximize shareholder value will tend to use those profits to fund distributions to shareholders rather than for investment in drug innovation. We argue that, if implemented properly, PMI could both improve the affordability of medicines and enhance the innovative performance of pharmaceutical companies.

 David Hockney

Rewardable pharmaceutical innovation

A Review of Current Approaches to Defining and Valuing Innovation in Health Technology Assessment

Key messages, 

Although, formerly, denoting a new drug as innovative was based on the drug having received patent protection or being a new molecular entity, this criterion is no longer sufficient from the perspective of many stakeholders. Rather, it became widely accepted that the central criterion to identify a drug as truly innovative should be the drug’s benefit or usefulness. With usefulness being a relative quality though, innovation is often defined by the particular view of what is deemed important or valuable. 

On the one hand, there is the notion that the value of innovation derives exclusively from the effect that the innovative drug has on the therapeutic benefit. What matters most for patients is whether a drug is the best choice to achieve treatment goals. It is this basic principle that guides most of the work on how to define rewardable innovation in the context of drug value assessments. The main argument is that, assuming a limited budget for healthcare expenditures, valuing anything beyond this therapeutic benefit in pricing and reimbursement decisions would reduce population health by displacing more cost-effective therapies. Furthermore, as a consequence, pharmaceutical companies might be incentivized to increasingly invest in R&D of slightly modified but basically similar products as opposed to drugs with a potentially larger positive therapeutic impact on patients.

On the other hand, it is argued that there might also be other, less obvious benefits from innovation in a wider sense. First, there may be benefits related to health or well-being that are not captured in measures of health outcomes typically used in clinical trials. A novel drug with less side effects or one that allows a more convenient treatment, for example, oral versus intravenous administration, may have the same effect on the therapeutic outcome but still improve the subjective well-being of the patients.

 

Tuscany, man made landscape

Are we paying too much for pharmaceutical innovation?

Economics of the Pharmaceutical Industry

The question of whether innovation is too high or too low is a first-order—perhaps the first- order—policy question in the economics of the pharmaceutical industry. Yet, economists have not produced a definitive answer. At best, we have suggestive analyses that point to the low rate of surplus capture by innovators, and modeled estimates suggesting that the social return to innovation is positive on the margin. While helpful, these findings each require a speculative leap in order to draw an inference about the social efficiency of innovation. Much rides on the answer to this question. If innovation is indeed excessive, then public and private payers are wasting money paying excessively high rewards to firms producing innovations. In contrast, if innovation is too low, lives are being lost prematurely due to a lack of medical progress.

This is the "answer" that Lackdawalla gives in an interesting article in Journal of Economic Literature (more details inside). If you want a review in 50 pages of the main issues surrounding the industry, this is the article you have to read.

Courtauld Impressionists exhibition at the National Gallery
Video

Repensar la innovació en medicaments

 Rethinking Innovation in Drugs: A Pathway to Health for All

En un article recent la Mariana Mazucatto insisteix en el seu argument. És prou conegut i alhora encara convé insistir més. Cal reconèixer el paper del finançament públic a la recerca i innovació en nous medicaments. L'exemple de Moderna i la vacuna de la Covid és prou clar i ja l'he explicat anteriorment. Moderna va rebre 1000 milions $ del govern en ajudes per la recerca i alhora va comprar 1500 milions $ en vacunes per a 100 milions de dosis. El total dedicat pel govern nordamericà a la tecnologia de vacunes mRNA va ser de 31.900 milions $.

Els missatges:

• It is essential to recognize that health innovation emerges from collective intelligence
• It is imperative to bolster financial commitments to medical research and development, viewing this as a strategic long-term investment rather than a short-term expenditure, and to protect existing budgets
• It is crucial to leverage procurement mechanisms to shape market opportunities that align with public health needs  

Malgrat que el finançament és imprescindible, cal una nova governança de la innovació. Unes noves prioritats de recerca i una revisió del problema de les patents. Una proposta que ja s'ha fet moltes vegades però que ningú gosa encapçalar. En altres ocasions ja ho he explicat. Malgrat que les regles del mercat són transnacionals, els governs actuen a l'àmbit nacional. S'han creat organismes multilaterals per garantir el comerç mundial però no per a la regulació necessària de la política sanitària. La OMS en aquest sentit i a l'àmbit dels medicaments afegeix poc.

La Mariana Mazucatto resumeix en aquest article la seva posició sobre la innovació al sector farmacèutic. Ho ha dit en altres llocs, però insisteix que cal una nova governança de la innovació. Bàsicament el missatge és que el govern no pot restar passiu com ara. Reprodueixo el paràgraf clau:

While funding is essential, it alone is not the solution. Government should adopt a mission-oriented approach to drug innovation, setting bold goals related to public health that serve to catalyze innovation and investment — goals that prioritize improved patient outcomes, reduction in disease prevalence, and access equity.Achieving such bold goals would necessitate a reform of intellectual property rights. Moreover, it would require a shift in how collaborations between the public and private sectors are structured to recognize that innovation results from a collective effort, valuing contributions from both public and private entities. And it would require governments to foster collaboration across different ministries, thereby avoiding the compartmentalized governance of health. The excessive tendency of governments to outsource key operations has unfortunately weakened these capacities.

Monet, ara a Orsay

 

Drug pricing 101 (2)

The New Drug Reimbursement Game. A Regulator’s Guide to Playing and Winning

In my former post I was backing a complete review of current drug pricing regulation. Any official that has to perform this task needs some fresh ideas and knowledge and this is precisely what a new book provides. In The New Drug Reimbursement Game by Brita A.K. Pekarsky you'll find the economic foundations for a new drug pricing regulation.
The basic argument:

Higher prices today mean increased economic rent for the pharmaceutical industry (Pharma) otherwise firms would not lobby for them. It is in Pharma’s interest to protect and seek these economic rents. Whether higher prices and more R&D today increase future health remains an empirical question. If higher prices also mean a higher net present value of the population’s health, then it is in the institution’s interest to increase prices. Given the institution’s objectives, the most effective strategy Pharma can use to protect these rents is “the Threat”: lowering prices is against the interest of health funders because it will reduce a population’s future health.

Therefore,

 The regulator’s challenge is to answer the following question:
• “How should rational institutions respond to the Threat?” (A rational response is one that is consistent with a given institution’s stated objective function, whatever this may be.)
This introduction places this research question in the context of current evidence and research, by addressing the following three questions.
• Is it plausible that the Threat exists and that it influences the price of new drugs?
• Is there rigorous empirical evidence that suggests that lower drug prices will result in reduced future health?
• Are economists in agreement as to the value of a decision threshold for new  drugs that accommodates characteristics of the health budget such as allocative and technical inefficiency?

And the conclusion:

When the Institution buys this new drug, it buys the health effects from this drug and the health benefits from future innovation. This is not the case with other health programmes. Therefore, unless the Institution pays a premium for the health effects from the new drug, the population will be worse off because innovation will be suboptimal and the future drug will not be produced.

Unfortunately, when you get to the end you'll miss any consideration about what innovation means. If you look at recent patterns of effectiveness of new drugs, you'll see that the value of innovation is under scrutiny, and most drugs would not pass the test. In my opinion, the regulator has to send signals about the value of health improvement in certain diseases and pharmaceutical companies should focus R+D on such fields and avoid others.
Therefore, a new companion to this book should be written. This is only a regulator's guide to play, but not to win. Take it only as a starting point.

PS. Just FYI, you'll not find the term "Budget impact analysis"  in the book, a close term Programme budgeting marginal analysis PBMA is what you'll find. It is suggested a price effectiveness analysis as a previous requirement for any PBMA, otherwise it makes no sense. This approach seems quite different to  yesterday's news.

Thomas Piketty speech at UPF Oct 2014, it starts at min 8:10. My post, last May.

COVID-19 innovation response

 The COVID-19 Innovation System

EIT Community COVID-19 Response

 The COVID-19 innovation system represents a departure from business as usual. Considering the remarkable progress to date, especially on vaccine development, this raises the question of whether this model is useful only for crisis times, or whether biomedical innovation policy in “normal” times might productively incorporate some elements of the COVID-19 model as well.

The largest funding response has been from the US government. Roughly $14–$15 billion of the $4 trillion allocated to the COVID-19 response was for R&D for vaccines and treatments.8,9 Though this increased US federally funded biomedical R&D by about one-third (compared to previous NIH funding; see “NIH Data” in the online appendix),10 it is small compared with the potential value of these interventions for ameliorating or preventing the disease and securing a return to normalcy

 

 

 

Creative construction or how big companies innovate

 Creative Construction. The DNA of Sustained Innovation

The conventional wisdom is that only disruptive, nimble startups can innovate; once a business gets bigger and more complex, corporate arteriosclerosis sets in. Over the last three decades, I’ve researched innovation and have had extraordinary on-the-ground experience with big companies and fast-growing ones that have moved beyond the startup stage. The culmination of that work is “Creative Construction,” which yields a new perspective about how bigger companies can leverage scale for competitive advantage in innovation.

You can check in this book how innovation is misunderstood quite often, and how to create a culture, strategy and organizational system that embraces it.

 

Mazzucato as a supplier of a flattering narrative for politicians (2)

Mission Economy. A Moonshot Guide to Changing Capitalism

My former post on a recent book by Mazzucato was based on a comment by McCloskey. Now, she has published a new one, and the best comment has been made by John Kay, clear message, I don't have anything to add.

Ever since 1969, people have asked themselves why if humans can land on the moon, can’t they solve pressing problems here on Earth, such as poverty, dementia and climate change. Mariana Mazzucato offers an answer: if only governments would apply the mission-driven methods of the Apollo project, they could.

Mission Economy, the new book from the high-profile economist noted for her advocacy of a more active state, contains many screenshots of the whiteboards beloved of brainstorming meetings, each with an ambitious goal at the top: secure the future of mobility, clean oceans, defeat cancer; below is a jumble of boxes and circles linked by multidirectional arrows.

We need a “solutions based economy”, driven and co-ordinated by more powerful governments engaged in every stage of the process of innovation.

But Apollo was a success because the objective was specific and limited; the basic science was well understood, even if many subsidiary technological developments were needed to make the mission feasible; and the political commitment to the project was sufficiently strong to make budget overruns almost irrelevant. Centrally directed missions have sometimes succeeded when these conditions are in place; Apollo was a response to the Soviet Union’s pioneering launch of a human into space, and the greatest achievement of the USSR was the mobilisation of resources to defeat Nazi Germany.

Nixon’s war on cancer, explicitly modelled on the Apollo programme, was a failure because cancer is not a single illness and too little was then — or now — understood about the science of cell mutation. Mao’s Great Leap Forward, a vain bid to create an industrial society within five years, proved to be one of the greatest economic and humanitarian disasters in human history. At least 30m people died.

Democratic societies have more checks and balances to protect them from visionary leaders driven by missions and enthused by moonshots, but the characteristics which made the Great Leap Forward a catastrophe are nevertheless still evident in attenuated version.

With political direction of innovation we regularly encounter grandiosity of ambition and scale; the belief that strength of commitment overcomes practical problems; an absence of honest feedback; the suppression of sceptical comment and marginalisation of sceptical commentators. All these were seen in Britain’s experience with Concorde, the Channel Tunnel and the AGR nuclear reactor programme, some of the worst commercial projects in history. More recently, there is the £12bn wasted on the NHS computerisation programme — a project that Mazzucato mentions, though only to blame private contractors for their failure to deliver on the political imperative.

On a smaller scale, Britain has suffered in the last year from the delays resulting from Public Health England’s insistence on central control of the coronavirus testing programme and the predictable fiasco of the attempt to sideline the expertise of Apple and Google in order to develop a uniquely advanced NHS test and trace app. And in September there was prime minister Boris Johnson’s “operation moonshot”, designed to control the coronavirus by testing 10m people daily in early 2021.

In contrast to these failures, the rapid development of vaccines is, at least provisionally, a success story. That development is not the product of visionary central direction but is the result of a competitive process with many different teams around the world attempting to be among the first across the finishing line.

Their work has drawn on a combination of existing academic science with the expertise in development and testing and the manufacturing and logistics capabilities of the global pharmaceutical industry. The role of government, appropriately, has primarily been in funding basic research and assuring that there will be a rewarding market for successful products.

Mazzucato lists “twenty things we wouldn’t have without space travel”. Athletic shoes, CAT scanners, home insulation, baby formula, artificial limbs. Yes, really. But beyond the ridiculous headline, we see the reality of productive innovation: a decentralised process in which developers draw on and help create the collective intelligence that leads to constant incremental improvement in so many fields — including better running shoes.

When historians of technology review the past 50 years, they may conclude that Neil Armstrong exaggerated when he announced “one giant leap for mankind”. The “new frontier” of the late 1960s turned out to be, not space, but information technology. And the development of IT was characterised by a striking absence of centralised vision and direction.

No moonshots; but piecemeal innovation through disciplined pluralism in which temporary winners were almost always displaced as they failed to anticipate the next step of the journey. Do you remember Digital Equipment, Word Perfect, Wang Laboratories, CompuServe, Netscape, AOL, BlackBerry? Each once a leader, now forgotten. Even Apple suffered more than one near-death experience, Microsoft failed to anticipate mobile computing or the cloud, IBM was swept out of the industry it had created.

Mazzucato has correctly emphasised the contribution of state funded basic research to Silicon Valley, but thank goodness the development was in the hands of Steve Jobs, Travis Kalanick and Elon Musk rather than a committee in the department of commerce.

No one has, or could have, the knowledge of present or future required to create or implement successfully the strategies that Mazzucato recommends. Take her modern signature example — Germany’s Energiewende, or energy transition to renewables. You will not learn from Mission Economy that this highly political, much publicised and wildly expensive project has brought about significantly smaller reductions in carbon emissions than Britain’s quiet, economically and socially beneficial substitution of gas for coal.

The failure of the Energiewende illustrates the dangers of moonshots and the mission economy. As talk of a “Green New Deal” becomes more frequent on both sides of the Atlantic, the prospect of more large, costly and ineffectual visionary projects grows.

Politicians readily fall in love with such proposals, and Mazzucato is not shy in reminding us how anxious they are to engage with her in discussing them. But the vision that propelled China’s economic development was not Mao’s Great Leap Forward or Cultural Revolution, but Deng’s “it doesn’t matter whether a cat is black or white if it catches mice”. It is more rewarding and effective to build better mousetraps than to shoot for a mice-free world.

John Kay is an economist, author and fellow of St John’s College, Oxford

 

Risky business

The productivity crisis in pharmaceutical R&D

L'evolució dels processos actuals d'innovació farmacèutica queden ben explicats a Nature. En Pammoli et al. ho descriuen i analitzen en un article clar. Selecciono aquest paràgraf:

The rate and direction of pharmaceutical innovation will continue to be affected by the interplay between patterns of technological change and market regulation. In our view, the value and cost of innovation should be assessed not only from a static efficiency perspective, but also from a dynamic one. From a static efficiency perspective, when two projects with the same potential market value but different POS values are compared, it is rational to drop the riskier project. However, such a
perspective fails to take into account the dynamic effects of competition among different organizations on market value and the risk of R&D projects. First, if all the organizations choose to invest in therapeutic areas in which the POS is high, those markets will experience fierce price competition, also due to the incentives designed by regulators and institutional payers through reimbursement and pricing schemes. If payers do not recognize any premium for incremental innovation, it is rational for investors to aim to achieve market exclusivity in difficult areas, which are characterized by a low POS, rather than being forced to compete on price in a low-risk but highly crowded market. Second, the benchmark to measure the degree of risk is endogenous: at the aggregate level, the more firms invest in high-risk markets, the lower is the risk premium each of them has to pay to investors. Taken together, these two effects have pushed pharmaceutical companies to focus on high-risk, high-potential areas of activity.

El que no expliquen és si aquestes àrees d'elevat potencial (valor de mercat), són també pels problemes de salut més rellevants (valor de salut). Aquesta qüestió acostuma a quedar pendent.

Ill-prepared for the arrival of new medicines

Pharmaceutical Innovation and Access to Medicines

While in the last years the number of new drugs in the market has been limited, this trend has changed and countries may expect larger bills in the next future. The OECD report explains the main challenges of pharmaceutical innovation and says:

Despite a slowdown in growth in the 2000s, pharmaceutical spending has nevertheless increased sharply in some therapeutic areas, such as oncology and certain rare diseases where many new medicines target small population groups and command high prices. While these may well address unmet needs, they often have prices that may not be justified by the health benefits they confer.

Countries may be ill-prepared for the arrival of novel medicines targeting wide  population groups. In 2013, the first of a new class of very effective but expensive
drugs known as direct-acting anti-virals (DAAs) for hepatitis C created a shock due to the potential budget impact of treating all infected people. Many countries initially restricted access to the most severely affected patients, creating frustration among patients and clinicians alike. Although subsequent entries of alternative products have created competition on prices and allowed payers to expand eligibility to treatment, the initial shock highlighted the lack of readiness of payers for such events.

In some countries, sudden, large price increases for off-patent medicines have made important treatments unaffordable for patients.

Finally, innovation is lacking in certain areas of high-unmet need, such as new antimicrobials, non-vascular dementia, and some rare diseases.

The report summarises different proposals and measures that would be helpful for a government that cares about citizens' welfare. Unfortunately, this is not our case.

Exhibition at Foto Collectanea

On ecosystems

 ECOSYSTEM EDGE.Sustaining Competitiveness in the Face of Disruption

Selected statements from the book:

Each of the four ways in which the development of an ecosystem can unlock new value—through new product bundles, new customer solutions, new platform economies, and spawning new industries—shares a common characteristic: they require a process by which new customer value is discovered. The new value is not just assembled or delivered from existing elements by following a predetermined blueprint; it has to be identified. Business ecosystems come into their own by facilitating the process of discovery.

Discovering new sources of value requires the three key capabilities that ecosystems excel at: a huge potential for rapid, joint learning and innovation; the ability to harness the capabilities of diverse players and channel them toward a common goal through the leadership of an enlightened company; and the flexibility for continuous reconfiguration in the face of an uncertain, fast-changing environment.

Given these demands, what can you do as an ecosystem leader to catalyze and promote the discovery of new value through your ecosystem strategy?

The rise of ecosystems will also rewrite the rules of competition and strategy as we have come to know them, encapsulated in the seminal work of Michael Porter. The classic cost-leadership strategy based on growing the volume of products and services a company produces to reap economies of scale that drive down costs below competitors, will be replaced. In a world of competing ecosystems, cost advantage will come from aggregating the scale of your entire partner network to spread the fixed costs of investments in everything from design and innovation through to production and distribution. Your ability to reap network economies will become decisive.

As competition between ecosystems grows, rewriting the rules of competition in the process, the demands on ecosystem leaders are only set to increase. The pressure to discover new sources of value for customers and to craft unique and attractive offerings will intensify. Partners will evaluate whether joining an ecosystem can deliver benefits, as well as how one ecosystem’s value proposition compares with that of another network. A potential ecosystem leader’s value proposition to partners will need to be top-notch. In the competition between ecosystems, the speed with which a leader kick-starts a virtuous spiral of cooperation and the rate at which it can scale its ecosystem are both critical. Those that get ahead because of network economies will surge forward in what could easily become a winner-take-all game. However, to sustain the ecosystem leader’s competitive position, the amount of learning and innovation generated by the ecosystem, and its ability to capture that learning, will be critical. Ecosystems that are less productive and efficient will be unable to compete. Mastering the strategies and capabilities we described earlier will become even more essential tomorrow than they are today.

This is the only book that finally provides some clear conceptualisation on ecosystems., as far as I know. 

La innovació a l'assistència sanitària

The Business of Healthcare Innovation 

El llibre "The Business of Healthcare Innovation," editat per Lawton Robert Burns, ofereix una anàlisi exhaustiva de la innovació en el sector sanitari.  El llibre se centra en els models de negoci i les tendències dels sectors tecnològics de la indústria sanitària, considerats els principals motors d'innovació en l'atenció mèdica. 

El llibre examina en profunditat diversos sectors clau de la indústria sanitària, com ara:

Indústria Farmacèutica:  S'analitza la investigació i desenvolupament de nous fàrmacs, els processos de comercialització, els reptes de la regulació i la dinàmica del mercat. 

Biotecnologia:  S'exploren les tecnologies emergents en biotecnologia, com l'edició genòmica, l'ARN d'interferència i les teràpies cel·lulars, i el seu impacte en el desenvolupament de nous tractaments.

Dispositius Mèdics: S'aborda l'evolució dels dispositius mèdics, els motors de creixement del sector, els reptes que afronten les empreses, com la pressió sobre els preus i la regulació complexa, i les innovacions en electrònica, ciència dels materials i la combinació de fàrmacs i dispositius.

Tecnologies de la Informació:  S'analitza el paper de les tecnologies de la informació en la millora de l'atenció al pacient, la gestió de la informació sanitària i l'eficiència del sistema sanitari. 

Al llarg del llibre, s'aborden diversos temes clau relacionats amb la innovació en el sector sanitari:

*   La importància de la innovació per afrontar els reptes de costos i qualitat en l'atenció sanitària.

*   La complexitat de les cadenes de valor sanitàries i el seu impacte en la innovació.

*   Els reptes que afronten les empreses del sector sanitari per innovar, com els elevats costos de R+D, les regulacions complexes i la demanda de solucions assequibles.

*   Les tendències d'innovació en els diferents sectors de la indústria sanitària, com la fusió de la biotecnologia i la indústria farmacèutica, i el desenvolupament de noves tecnologies disruptives.

*   Els models de finançament de la innovació, com el capital risc, les fusions i adquisicions i els pagaments condicionats.

"The Business of Healthcare Innovation" ofereix una visió completa i actualitzada de la innovació en el sector sanitari.  El llibre destaca la importància de la innovació per millorar l'atenció mèdica, però també subratlla els reptes i les complexitats que comporta. La comprensió d'aquests aspectes és crucial per impulsar la innovació de manera efectiva i garantir que els seus beneficis arribin a tots els pacients.

Creative destruction or how to save capitalism from the capitalists

The Power of Creative Destruction. Economic Upheaval and the Wealth of Nations

This book is an invitation to a journey: a journey through economic history, more specifically a journey to explore the enigmas of economic growth through the lens of creative destruction.

Creative destruction is the process by which new innovations continually emerge and render existing technologies obsolete, new firms continually arrive to compete with existing firms, and new jobs and activities arise and replace existing jobs and activities. Creative destruction is the driving force of capitalism, ensuring its perpetual renewal and reproduction, but at the same time generating risks and upheaval that must be managed and regulated.

This is precisely what this book explains. And behind the driving force of capitalism, you'll find ideas, as Deirdre McCloskey dixit. 

From Shumpeter:

The first idea is that innovation and the diffusion of knowledge are at the heart of the growth process.

The second idea is that innovation relies on incentives and protection of property rights

The third idea is creative destruction: new innovations render former innovationsobsolete. In other words, growth by creative destruction sets the stage for apermanent conflict between the old and the new: it is the story of all incumbent firms, all the conglomerates, that perpetually attempt to block or delay the entryof new competitors in their sectors.

Creative destruction thus creates a dilemma or a contradiction at the very heart of the growth process. On the one hand, rents are necessary to reward innovation and thereby motivate innovators; on the other hand, yesterday’s innovators must not use their rents to impede new innovations. As we mentioned above, Schumpeter’s answer to this dilemma was that capitalism was condemned to fail precisely because it was impossible to prevent incumbent firms from obstructing new innovations. Our response is that it is indeed possible to overcome this contradiction, in other words to regulate capitalism or, to take the title of Raghuram Rajan’s and Luigi Zingales’ 2004 book, to “save capitalism from the capitalists.”

PS. Shelling on the strategy of conflict.

La innovació dinàmica als medicaments

 Achieving dynamic efficiency in pharmaceutical innovation: Identifying the optimal share of value and payments required

En diferents ocasions s'ha argumentat que l'avaluació del cost-efectivitat dels medicaments té en consideració tant sols una visió estàtica. La hipòtesi és que els fabricants posen un preu basat en un llindar de QALY acceptable d'acord amb el període de protecció de patent. En canvi s'oblida el valor post-patent, i que la quantitat i qualitat d'innovació que aporta en el temps no es considera. Jo no puc contrastar aquesta hipòtesi. El que si sé és que un article recent ha revisat la qüestió i diu:

The share of value that would maximise long-term population health depends on how the quantity and quality of innovation responds to payment. Using evidence of the response of innovation to payment, the optimal share of value of new pharmaceuticals to offer to manufacturers is roughly 20% (range: 6%–51%). Reanalysis of a sample of NICE technology appraisals suggests that, in most cases, the share of value offered to manufacturers and the price premium paid by the English NHS were too high. In the UK, application of optimal shareswould offer considerable benefits under both a public health objective and a broader view of social welfare. We illustrate how an optimal share of value can be delivered through a range of payment mechanisms including indirect price regulation via the use of different approval norms by an HTA body.

Per tant el valor transferit als fabricants és superior al desitjable al NHS. I la taula clau, allò que es paga i el que s'hauria de pagar segons els autors:

 

Els autors avisen de la simplificació d'alguns supòsits i de com els seus resultats difereixen d'altres aportacions. Tot plegat és una mostra més que és un camp fèrtil per a la recerca, i obert a nous horitzons i respostes.

Addiction goods and innovation failure

When Innovation Goes Wrong: Technological Regress and the Opioid Epidemic 

The medical use of opioids to treat pain will always involve costs and benefits, and the optimal level of opioid prescription is unlikely to be zero. The mistake that  doctors and prescribers made in recent decades was to assume overoptimistically that a time release system would render opioids non-addictive. Thousands of years of experience with the fruits of the poppy should have taught that opioids have never been safe and probably never will be. The larger message of the opioid epidemic is that technological innovation can go badly wrong when consumers, professionals, and regulators underestimate the downsides of new innovations and firms take advantage of this error. Typically, consumers can experiment with a new product and reject the duds, but with addiction, experimentation can have permanent  consequences.

A must read, by Cutler et al.

 Robert Doisneau

A plea for public patents on COVID prevention and treatment

 Funding of Pharmaceutical Innovation During and After the COVID-19 Pandemic

Extensive public investments also are being made in therapeutics. The 2 most prominent monoclonal antibodies (by Regeneron and Lilly) have come to market with substantial governmental support for product commercialization. Both products derive from therapeutic research platforms established with governmental support before the COVID-19 pandemic, but product commercialization and manufacturing received major additional investments in 2020. Separately, the National Institutes of Health (NIH) Rapid Acceleration of Diagnostics program has committed $1.5 billion to supporting development of diagnostic tests related to COVID-19. The specifics of the federal contracts largely remain confidential.

Why do they remain confidential? 

The lesson of the COVID-19 experience is that, when innovation in the life sciences is imperative, the traditional reliance on pharmaceutical industry prices and profits is jettisoned in favor of governmental grants and procurement. Sustained public funding for product development and commercialization will permit the sustained financing of innovation, a renewed attention to major public health needs, and the global position of the US pharmaceutical industry.

If there is public funding, why there aren't public patents? 

La innovació disruptiva

The Disruptive Innovation Set 

El llibre "The Disruptive Innovation Set" reuneix dos llibres del difunt Clayton M. Christensen, un expert en innovació i gestió: "The Innovator's Dilemma" i "The Innovator's Solution". Les fonts proporcionades inclouen excerpts del llibre, centrant-se principalment en "The Innovator's Dilemma".

"The Innovator's Dilemma" explora per què empreses exitoses, amb bons gestors, fracassen davant de canvis tecnològics disruptius. Utilitza la indústria de les unitats de disc dur com a cas d'estudi, demostrant com les empreses líders van ser superades repetidament per nous entrants que van oferir tecnologies inicialment inferiors, però que van evolucionar per satisfer les necessitats del mercat.

Conceptes clau de "The Innovator's Dilemma":

Tecnologia disruptiva: Una tecnologia que inicialment té un rendiment inferior al que exigeixen els clients principals, però que millora amb el temps i eventualment supera les tecnologies establertes.

Xarxa de valor: El context en què una empresa identifica i respon a les necessitats dels clients, resol problemes, adquireix inputs, reacciona als competidors i busca beneficis. Les xarxes de valor determinen com les empreses perceben el valor d'una nova tecnologia i com assignen recursos.

Inèrcia organitzativa: La tendència de les empreses establertes a centrar-se en les necessitats dels seus clients actuals i a ignorar les tecnologies disruptives que podrien amenaçar el seu negoci principal.

"The Innovator's Solution" aprofundeix en com les empreses poden superar el dilema de l'innovador i aconseguir un creixement sostenible en un entorn disruptiu. Es basa en la investigació de "The Innovator's Dilemma" i proporciona un marc pràctic per als gestors.

Conceptes clau de "The Innovator's Solution":

Innovacions de mercat nou: Tecnologies que creen nous mercats adreçant-se a clients que abans no tenien accés al producte o servei.

Innovacions de gamma baixa: Tecnologies que s'adrecen al segment més baix del mercat, oferint productes o serveis més simples i assequibles que les solucions existents.

Gestió del cicle de vida del producte: La comprensió de com evoluciona el rendiment dels productes en relació amb les demandes del mercat ajuda a les empreses a anticipar les interrupcions i a prendre decisions estratègiques.

En resum, "The Disruptive Innovation Set" proporciona un marc per comprendre i gestionar la innovació en un entorn en constant canvi. Els seus conceptes clau ofereixen eines valuoses per als gestors que busquen impulsar el creixement sostenible i evitar ser superats per la disrupció.

Value-based pricing: a controversial narrative

 Rethinking value in health innovation: from mystifications towards prescriptions

From the abstract:

Debates over value in health innovation in the U.S. and Europe have
become increasingly dominated by “value-based pricing”. We examine
this prevailing narrative and its weaknesses and then present an
alternative framework for rethinking value in health. Drawing on
scholarship from the political economy of innovation, we argue that
value in health must be considered in terms of both value creation
as a collective process amongst public and private actors, as well as
value extraction that occurs due to financialization. In building this
alternative framework, we pose three questions that present areas
for further research and public policy change

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