Advances in Oncology Drug Discovery – Sachs Conference Talk by Roche pRED Head of Oncology DTA, William Pao

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A fascinating talk about cancer drug discovery was given by Dr. William Pao, Head of Oncology Discovery and Translational Area (DTA) at Roche Pharmaceutical Research and Early Development (pRED) during the Sachs 16th Annual Biotech in Europe Forum.

He started to explain what is cancer, for us to gain more insights:

  • cancer is a genetic disease: tumors can harbor over 400 somatic mutations
  • cancer is heterogenous: there are more than 200 types of cancers and a single patient tumor displays intra and inter-tumoral heterogeneity
  • cancer can metastasize: once spread, it is virually incurable. Metastatic cancer survival at 5 years is extremely low (between 4 and 28%)

Based on those considerations, treatment is becoming much more complex today with a blend of chemotherapy, targeted medicines and immunotherapies. A right combination could extend survival by several months.

But how to develop drugs with increased efficacy against the smart strategies used by the disease (such as tumor angiogenesis)? According to Dr. Pao, 3 elements are necessary:

  • understanding disease biology as well as druggable targets in the complexity of cancer molecular pathways
  • developing fit-for-purpose molecules allowing to create the right drug with the right format against the right target
  • personalizing healthcare with the administration of the right drug to the right patient at the right time

Beyond a better understanding of the disease, using more than a single strategy to target the cancer:

  • Host directed with cancer immunotherapy. This approach is particularly challenging as some patients do not respond to it (innate or acquired immune escape) and other patients may fully benefit with long term survival
  • Tumor directed with targeted medicines

External innovation, collaborations and partnerships, is fully leveraged in order for Roche to complement existing capabilities in the field (immunotherapy examples: CuraDev, Pieris, BluePrint; targeted medicines: Tensha, C4Therapeutics).

As a conclusion, Roche is well positioned to address the cancer challenges and, since the beginning of innovative cancer treatments, the company has always been perceived as the leader of the therapeutic area.

addressingcancerchallenge

Missing points in his talk were considerations of patient’s quality of life (extending life does not always go with good quality of life because of treatment’s side effects) and drug pricing (adding more and more drugs to the treatment cocktail costs a lot of financial resources, not only paid by the health insurance but also by the patient).

 

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DNA methyltransferase 1 has a role in the establishment and regulation of tissue-specific patterns of methylated cytosine residues. Aberrant methylation patterns are associated with certain human tumors. www.enzymlogic.com. Work done with the molecular visualization VMD program developed at the University of Illinois: www.ks.uiuc.edu/Research/vmd/

Alphabet (ex-Google) is the next big thing in Life sciences

A lot is currently written about the initiatives launched by Alphabet (Google) in the life sciences field.

I had the wonderful opportunity to have a look at the report written by the internet analyst, John Blackledge, from Cowen and I must say that he’s very smart at showing the huge potential of the life sciences at the core of Alphabet (Google).

This report is amazing as it allows you to have a better understanding of what’s happening now inside Alphabet (Google). I summarized the key points/quotes from the report below and I added other articles at the end of this blog post. Moreover, I will update it frequently as the news come in. This is a fascinating topic, I really hope Alphabet (Google) would be able to replicate the same success it has built with its search engine.

Key quotes & comments from the report:

  • Expansion into health care and related segments allows Google to leverage its core competencies in Internet communications technology, data structuring and analysis, and fundamental process reinvention.

 

  • Specific areas of focus in healthcare include:
    (1) the sequencing the human genome and the rise of precision medicine: despite the monumental significance of mapping the human genome and the implications for drug discovery, this was but one step in a long journey that continues to this day. Moreover, genes are but one factor in disease, and little is known about what role environment and lifestyle play.
    (2) the digitization of health data is exploding, with a virtually endless list of sources that can offer insight into clinical data, drug studies and more. As more data is digitized, there will be a profound impact on how patient care is administered, how therapies are researched, and how drugs are tested. EHRs (Electronic Health Records) are crucial but implementation is very challenging. Harmonization and data aggregation need to find their way. Wearables are another interesting topic in the digitization of health data. Social media and discussion boards as well as patients website are essential parts of the system that must be closely monitored as more and more patients use those communication channels in order to provide feedback and comments on treatments and daily struggles with healthcare providers.
    (3) the shift to value-based care, where payments are based on the value of care, is driving a change in how services are delivered and how much consumers engage in the process. From a provider perspective, doctors are incentivized to manage patients to the best possible health outcome at the lowest cost. From a patient perspective,
    consumers are being empowered to take a more active role in their own health care.
    These health care trends are being accommodated by technology advances in areas such as social, mobile, analytics and cloud computing, all areas of Google expertise.

 

  • Alphabet invests in health in five different ways:
    (1) Google Life Sciences originated in Google[x], a research lab within Google that was funded by the company’s board of directors in January 2010 to pursue “moonshots”—audacious new projects that have a low probability of succeeding, but could be truly revolutionary if they do. The company views moonshots as critical in driving the true innovation required to affect revolutionary change and avoid the “incrementalism” or evolutionary change that tends to lead to corporate irrelevance over time. The Life Sciences team is responsible for such innovations as glucose monitoring smart contact lenses. With an expanding list of intellectual property, Life Sciences has begun to accelerate its collaborative efforts with third parties. The company is aggressively partnering with leading players in the health care space on a growing number of programs.
    (2) Calico’s mission is to harness advanced technologies to increase understanding of the biology that controls lifespan. Calico was originally conceived by Google Ventures President and General Partner, Bill Maris, who observed that most companies seek to find treatments for disease and associated symptoms, but that none address the root cause of disease and death. He wondered if studying the impact of aging on genetic material could lead to the discovery of drugs that could address many age-related diseases and significantly extend the human life span.
    (3) Google Ventures has provided seed, venture and growth stage funding to a host of companies in diverse fields, but its stated focus is machine learning and life science investing.
    (4) Google Capital was formed to invest in later-stage technology companies with a focus on emerging technology leaders and potential disruptors. Unlike the earlier stage companies in Google’s other investment vehicles, the later stage companies in Google Capital tend to be fairly common household names. Although the stated focus of Google Capital is on technology companies, the collision of technology and health care is blurring the lines of what a traditional “technology” company looks like.
    (5) Google Core: Over time, Google has invested in numerous health-related initiatives within its main corporate division. These have tended to be very closely linked to the company’s core businesses, such as Search. Earlier this year, the company announced that it will add health information that has been fact-checked by physicians directly to search results. The company is also talking to the FDA about using search query data to identify adverse drug reactions.

 

  • Google’s Health-Related Focus Areas: regardless of where they are housed within Google’s corporate structure, most of Google’s health-related endeavors share common characteristics.
    (1) Longevity
    (2) Genetics and Chronic Care
    (3) Diagnostics
    (4) Diabetes/Digital Health
    (5) Medical Devices
    (6) Telehealth/Digital Health
    (7) Wearables/Fitness

 

  • Google’s health endeavors fit with the company’s goals of “making the world’s information useful” and helping millions of people. Indeed, Google believes that many of the same principles, techniques and problem solving capabilities employed by its software developers can be applied to the massive inefficiencies that exist in health care to create transformational solutions and medical breakthroughs that help people live longer, healthier lives. Health care ambitions can be summarized as:
    (1) Analyze: Analytics to inform decision-making and provide business insight
    (2) Attract: Attract health care constituents to platforms and solutions that drive engagement
    (3) Aggregate: Aggregate data from disparate sources onto the Internet or GCP

 

Additional resources:

Alphabet to help researchers predict disease – Financial Times – April 2017

Google Life Sciences Exodus – STAT – March 2016

Verily, Google’s Health Gambit, Is Stacked With Scientists. Now It Needs to Build a Business – ReCode – December 2015

Google hires mental health expert to lead new life sciences unit – Financial Times – September 2015 (Subscription required)

Head of Mental Health Institute Leaving for Google Life Sciences – The New York Times – September 2015

Google Bets on Insurance Startup Oscar Health – WSJ – September 2015

Is Health Care Google’s Next Big Business After Search? This Investment Bank Thinks So – Re/Code – September 2015

Why Google Is Going All In On Diabetes – NPR – September 2015

Google’s health startup, AbbVie team up on drug research – Chicago-Sun Times – September 2015

Google Life Sciences Company Has New Deal, Official Nemesis in Diabetes – Re/Code – August 2015

Alphabet Breathes New Life, Resources Into Google’s Health Care Projects – iHealthBeat – August 2015

Google Health – Easy as ABC. Alphabet, Calico and the Aging of Humanity – What on Earth are they doing? – Digital Intervention – August 2015

 

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A Better Understanding of the US Drug Pricing Landscape and Financial Risks Associated – Health Affairs Blog

An excellent article posted on the Health Affairs blog takes a step back regarding US drug pricing and the financial risks associated with drug development well beyond the clinical trials stage.

Key quotes from the article:

  • It may be helpful for the policy discussion to think of a drug’s value as the clinical performance and patient outcomes, while the price reflects both the value and the growing uncertainty around in-market risks of market consolidation and restricted access, branded therapeutic competition, mandatory discounts, and restrictive coverage policy.
  • As competition heats up, each sector and each entity strives to reduce input costs and maintain or improve prices — and consolidation can be an important tool to accomplish these goals. Specific to biopharma, consolidation strengthens payers’ and providers’ ability to press for drug discounts that are contractual, proprietary, and confidential.
  • Net lifetime revenues of new biopharma therapies declined from profitability in the late 1990s to slightly negative profitability by the end of the first decade of 21st century.
  • In addition to cross-sector market competition through payer and provider consolidation, there is growing intra-sectorial competition among generics, biosimilars, and branded therapeutic alternatives.
  • We should not underestimate the potential effect of mandatory price discounts on drug launch prices.
  • In-market risks for biopharma are very significant today. Because of rapid changes in the market environment, revenue expectations established when the decision is made to proceed with product development can be very different than actual revenue several years later when a product is launched.

 

Additional Resources

When Is a Virtual Business Model Suitable for Biopharmaceutical Companies? – BioProcess International – 2015

Understanding the pharmaceutical value chain – IMS Institute for Healthcare Informatics – 2014

Innovative Business Models in the Pharmaceutical Industry: A Case on Exploiting Value Networks to Stay Competitive – International Journal of Engineering Business Management – 2014

The Real Cost of “High-Priced” Drugs – Harvard Business Review – 2014

Rapid growth in biopharma: Challenges and opportunities – McKinsey – 2014

 

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Google Willingness to Help Life Sciences – Wired

Google, or Alphabet, wants biomedical research and life sciences to be more than just side projects.

Several years ago, its efforts under way in that field were probably not considered seriously by the industrial stakeholders like Big Pharma and biotech companies.

Today it is different after several investments commited lately.

Discover more in the Wired article

 

Additional Resources

4 of the biggest healthcare challenges Google is tackling – HealthcareDIVE – August 2015

Here’s why Google Ventures invests so much money in life-science companies – Business Insider – May 2015

Andrew Conrad – Google Life Sciences – The 25 most influential people in biopharma in 2015 – FierceBiotech – May 2015

Google Continues To Build Upon Its Life Sciences Ecosystem – Forbes – September 2014

Meet the Google X Life Sciences Team – WSJ – July 2014

 

 

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Allergan & Teva – Two sides of one table

Deals

Allergan (former Actavis) is restructuring its operations with the divestment of its generics business to Teva for approximately USD 41 billion in cash and stock ($33.75 billion in cash and Teva shares valued at $6.75 billion, giving it a 10% stake in Teva).

But it’s not done. According to an interview with the Financial Times, the CEO, Brent Saunders, is eager to sign another mega deal with the proceeds from the sale mentioned above. Allergan can then be put on the list of serial deal makers.

On the other side of the table, Teva is about to join the club of the biggest pharmaceutical companies. According to a Business Insider UK article, “Allergan’s generic business is generally seen as a better fit than Teva’s previous target Mylan because it will improve Teva’s distribution channels and because Allergan is strong in so-called biosimilar drugs.”

In the generics market, Teva will stay one step ahead of Novartis Sandoz division (estimated proforma 2014 sales of USD 15.7 billion for Teva-Allergan vs. USD 8.5 billion for Novartis Sandoz division).

In this context, Teva will probably drop its pursuit of rival Mylan, which in turn will be able to focus on buying Perrigo.

Additional links:

Allergan signals appetite for new mega deal after $41bn disposal – Financial Times (Subscription required)

A $40.5 billion deal with Allergan will make Teva one of the biggest drug companies on the planet – Business Insider

Teva to Buy Allergan Generics for $40.5 Billion – WSJ

The Last-Minute Phone Call That Spurred Teva-Allergan Deal – Bloomberg

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With Big Data & Digital Health – New Collaborations are Emerging in the Pharma Industry – PWC

TeamWork

A newly published report by PWC reveals novel collaboration models for improved value of medicines

“Driven by empowered consumers and connected technology, the New Health Economy is shifting business incentives from volume to value with a focus on health outcomes beyond the clinic.”

Big data together with EHR (electronic health records) and wearables drive patient empowerment. Today, more and more, people have their say in treatment choices. Actually, health benefits and prices are crucial criteria for decision, especially as patients face today higher out-of-pocket expenses than ever before. We all well know that expensive treatments are financially disastrous for patients as mentioned in one of my previous posts.

Currently, increased focus is put on drug cost effectiveness as we switch progressively from a fee-for-service to an outcome-based world. Every treatment should be precisely calculated in order to be sure that it is optimized for all stakeholders from all points of view. In this context, already last year, PWC highlighted the beginning of a new system: the New Health Economy, where outcomes and quality are rewarded (instead of volume). It is the continuum of what has been started by Michael E. Porter several years ago.

All these changes have essential meanings. Therefore business models need to adapt to current trends: digital is unavoidable; purchaser perspective is necessary; patients need to become partners; regulatory changes have to be anticipated.

Adapting business models is fantastic but not sufficient. Novel collaborations are needed to optimize them for long-term success. All the stakeholders have to be integrated and blended by the biopharma companies: government agencies, insurers (payers as a whole), new entrants, consumers. Beyond collaborations, a consensus on the value of new medicines should be agreed on. Additionally, consumer and patient health information should be leveraged to improved personalization and precision of treatments.

In one word, several changes will flow the industry and challenges will pave the way to success. But it will become much more exciting to develop drugs in this context than ever before!

Report: PWC-21st-century-pharmaceutical-collaboration-July2015

 

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Partnering with patients in the development and lifecycle of medicines – TIRS

Infusion

A wave of new articles on patient-centric drug development flooded the news during the last weeks

It is not a new concept. A task force was started back in 2003 by ISPOR on this topic and several thinkers proposed this vision even before.

One of the more comprehensive article on this topic is the Call for Action written by several experts in the field both in the industry and academia as well as people coming from patient associations, together for the development of a master framework for systematic patient involvement.

Several stakeholders are committed to the success of a specific medicine: researchers, developers, industrials, regulators, insurers, doctors,… but very often they all forget about the main and the most important among them: the patient. He or she is the one taking the drug, benefiting from care or even cure in some cases but also suffering from side effects.

At each stage of development, involving and engaging the patient will bring advantages (pages 8 and 9 of the article gave great figures about them):

– Early research: better prioritization, improved resource allocation, clinical trial protocols reflecting patient needs, superior recruitment rates.

– At launch and beyond: more appropriate benefit-risk assessment, focus on drugs of value to patients, improved treatment adherence.

A part from e-patients blogs, dedicated societies emerge in order to make coordination effort toward common goals like the Society for Participatory Medicine: “Participatory Medicine is a model of cooperative health care that seeks to achieve active involvement by patients, professionals, caregivers, and others across the continuum of care on all issues related to an individual’s health. Participatory medicine is an ethical approach to care that also holds promise to improve outcomes, reduce medical errors, increase patient satisfaction and improve the cost of care.”

In conclusion: “It is essential that all stakeholders participate to drive adoption and implementation of the Framework and to ensure that patients and their needs are Embedded at the heart of medicines development and lifecycle management.”

 

You probably remember the fascinating talk given by e-patient Dave in 2011 during TEDxMaastricht. Dave deBronkart said as an introduction that patients need to be much more involved in order to drive better healthcare as well as superior patient outcomes. The future is now!

 

Additional material

What’s Next for Patient-Focused Drug Development? FDA Announces Final PFDD Meetings, and BIO Recommends Broader Use of the Benefit-Risk Framework – 2015

Partnering with patients in the development and lifecycle of medicine – 2015 – Therapeutic Innovation & Regulatory Science

Integrating the patient perspective in the assessment of benefits and risks of medicines – ISPOR 2014 Workshop

Finding the patient in the drug development process – F. Lewis-Hall (Pfizer) – ISPOR 2013

Toward Patient-Centered Drug Development in Oncology – 2013 – NEJM

 

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Can Finance Cure Cancer? Andrew W. Lo Talk at the University of Geneva – June 29th 2015

I had the chance to attend this refreshing talk about what finance could do to help drug development. My summary of the talk is shown below.

CanFinanceCureCancerAWLGenevaJune2015

 

The Rise of the Empowered Health Care Consumer – Deloitte Dbriefs Health Sciences Series

Deloitte-Debrief-EmpoweredConsumer-June2015

An excellent webcast took place yesterday.

The key points are above on the MindMap I did. The presentation is available here: Deloitte_Dbriefs_Empowered_Healthcare_Consumer_Jun2015

The main take-home message: companies really should include the new health care consumer as a stakeholder. The patient has now more power than ever. He/she is more informed, more connected, more commited but also more demanding.

Medtronic – The Digital Pancreas – The Future of Diabetes Management? – Fierce Medical Devices

pancreas

A truly interesting article of the transformation of Medtronic Diabetes business

2015 could be a year to remember as the start of a new era in improved diabetes management thank to digital tools.

Diabetes management at Medtronic & deals history

It started several years ago but back in May 2001, a transforming event took place: Medtronic bought Minimed as well as an affiliated company for USD 3.8 billion. Looking only at Minimed, Medtronic paid USD 3.28 billion for USD 400 million in sales (8.2x 2001 sales). Despite its price, quoted as high by some investors and analysts at that time, the deal rationale was pretty compelling as it allowed Medtronic to enter the diabetes management arena. MiniMed offered a beachhead into the field of technological management of diabetes, one of the fastest-growing chronic diseases in the world and one that affects an estimated several millions of people. The devices produced by MiniMed help patients manage their insulin needs and monitor glucose levels.

Since 2001, several deals and partnerships paved the way up to where Medtronic is today. After having a look at my deals database, I can say that some of them clearly stand out:

November 2004: agreement with Novo Nordisk on prefilled insulin cartridges;

– August 2007: co-promotion & co-marketing with Lifescan and Bayer of blood glucose meters (in USA together with Lifescan and outside USA with Bayer extended in 2011). These devices had wireless data transmission to insulin pums;

May 2009: strategic marketing collaboration with Eli Lilly on patient education and disease management;

June 2009: acquisition of PreciSense, a medical device company developing CGM (continuous glucose monitoring) technology. A step forward for “closed loop” systems dedicated to insulin delivery;

January 2013: research collaboration with GI Dynamics on EndoBarrier;

August 2013: acquisition of Cardiocom, a developer and provider of integrated telehealth and patient services for the management of chronic diseases;

June 2014: global strategic alliance with Sanofi, aimed at improving patient experience and outcomes for people with diabetes around the world. The priority is the development of drug-device combinations and delivery of care management services to improve adherence as well as simplify insulin treatment.

Medtronic has built its Diabetes franchise over the last several years and is still fully committed to be the leader in this field. This pledge could lead to the achievement of the digital pancreas, a fully autonomous device (closed loop system) monitoring blood glucose continuously and adjusting insulin doses as perfectly as the biological pancreas without human interaction. Some researchers already have prototypes, studies are ongoing, universities and hospitals are teaming up. The field is really at its boiling point!

Deals and partnerships in 2015

In this FierceMedicalDevices article, we can see Medtronic advancing its franchise by investing with determination and dedication in new technologies.

Many partnerships have been signed in 2015: Diabeter (a diabetes clinic and research center dedicated to providing comprehensive and individualized care for children and young adults with diabetes), DreaMed (artificial pancreas technology for integration into future Medtronic insulin pumps). Beyond healthcare companies, Medtronic is also expanding its network into consumer electronics with Samsung (integration of mobile and wearable devices to improve disease management with an Android mobile app) and diabetes data with a startup called Glooko. It also partnered with IBM Watson Health for next-generation disease management solutions.

All these deals are clearly accelerating innovation at Medtronic but in a patient-centric fashion as in every deal we can see the benefits for them and the management of their disease. What could be the next step? A deal with Apple in order to nearly fully cover all the mobile OS in the world.

For my conclusion I will quote Medtronic CFO, Gary Ellis: “We’re focused on transforming our diabetes group from a market-leading pump and sensor company into a holistic diabetes management company focused on making a real difference in outcomes and costs,” summed up Medtronic CFO Gary Ellis on the most recent quarterly conference call.

 

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Mergers & Acquisitions: what are the current key trends for the pharmaceutical industry? – KP

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2014 was spectacular and fascinating with a lot of deals… What’s next?

Tremendous amount of reports have been produced (some of them, a selection from the bests, are at the bottom of this post). Rumors are sometimes saying it’s the end on Monday, it’s just the beginning on Tuesday and sky is the limit on Wednesday…

Internet serendipity is great! Indeed, I came across a very interesting website offering free reports on the pharmaceutical industry: Kurmann Partners. Just have a look! They are M&A consultants for several industries (I do not work for or is paid by them).

They put online another useful tool which will be a good starting point to look at M&A multiples: a visualization tool offering several types of charts to monitor the M&A activity over the years from different standpoints and for private companies: Multiples. Below is an example of a chart you could find.

KP_RevenueMultiples_EBITDAmargins

2015 – What happened up to now and what’s ahead?

The trend seems here to stay as there is still a need to replenish pipelines and build a competitive advantage in specific therapeutic areas. I suppose that some domains will attract more buyers than others like immuno-oncology (a very hot topic today), antibiotics (as the governements are trying to incentivize companies). More generally, all therapies that could stand out from the crowd by being innovative (and not “me-too“-improved version of already existing drugs) will attract opportunity hunters.

 

Additional resources:

2015 Global life sciences Outlook – 2015 – Deloitte – Starting on page 8, you have a overview of the deals that happened in 2014 and some thoughts on what could happen next

Royalty Rates and Deal Making Survey – 2015 – IMS – This short report gives a very good overview of a large survey (that can be bought) realized by IMS on royalty rates and deals during the first months of 2015. Access it here: 2015_RoyaltyRateDealmakingSurveyOverview

Global M&A report – Pharma/Biotech 2015 – 2015 – IMAP

Firepower fireworks drive record M&A in 2014. What’s ahead for 2015? – 2015 – Ernst & Young

HBM Pharma/Biotech M&A Report 2014 – 2015 – HBM Bioventures – It is a very interesting overview with a perfect coverage of the topic, from an global overview to some detailled analysis of the deals done by public as well as private companies. It is a MUST-HAVE.

 

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Defining Digital Medicine – Nature Biotechnology

Notes from the AppsWorld Europe 2013 panel "The Internet of Things Revolution - Functional, Usable, Wearable" with Tamara Roukaerts, Saverio Romeo, Paul Lee, Ben Moir and Mike Barlow.

Notes from the AppsWorld Europe 2013 panel “The Internet of Things Revolution – Functional, Usable, Wearable” with Tamara Roukaerts, Saverio Romeo, Paul Lee, Ben Moir and Mike Barlow.

Healthcare transformation ahead

In this excellent article from Nature Biotechnology written by people at PureTech, we have the chance to get an overview of this new exciting field: Digital Medicine.

This convergence of technology and health will lead to several transformations: disease management, research, clinical trials, medical practice,… A new era is beginning!

However, opportunities are doubled by challenges. We need to tackle the lasts to profit from the firsts.

Definition

Why do we use digital medicine instead of digital health? According to the authors, digital health is too broad as it also includes apps and products not medically validated but simply focused to enhance people’s wellness and wellbeing.

Digital medicine is defined by “technology and products that are undergoing rigorous clinical validation and/or that ultimately will have a direct impact on diagnosing, preventing, monitoring or treating a disease, condition or syndrome.” (quote from the article p. 457)

Digital medicine themes

1. Continuous and remote monitoring. A tool to detect disease earlier leading to lower healthcare costs.

2. Digital phenotype. A additional layer of information enabling the construction of more accurate disease models in order to better understand them.

3. Remote disease management. Chronic disease management will be nearly effortless and the lack of data gap will allow doctors to improve their medical follow-up of patients.

4. The connected patient. The empowered patient. The engaged and sharing patient. Communities of patients.

5. Interpreting the data torrent. The challenge of integrating large and heterogenous datasets could be solved one day with powerful algorithms and machine Learning.

6. Security and privacy. The obtention of the patient’s consent is crucial and should be much simpler than today.

7. Opportunities and challenges. Increased scientific evidence but reimbursement issues, new field of research based on wearables, changes to patient-doctor interactions, integration of patient-specific data by the doctor.

Conclusion

Digital medicine is unavoidable and doctors will have to integrate this trend in order to optimize their relationships with their patients by empowering them and maintaining a two-way discussion toward a unique goal: better patient outcomes.

Nature Biotechnology Article (free)

More on Digital Medicine

Have a look at my posts on this topic.

Why Digital Health Has Not (Yet) Transformed Pharmaceutical Drug Development – 2015 – Forbes

Rock Health Founder On The Future Of Digital Medicine – 2015 – TechCrunch (Video)

Top 20 Technologies that Will Change our Lives: Next Up – Digital Medicine – 2014 – Forbes

How Digital Medicine Will Soon Save Your Life – 2014 – WSJ

Medicine goes digital – 2009 – The Economist

Digital Medicine – Implication for Healthcare Leaders – 2003 – Healthfutures.net (a nice document from 2003, projecting the reader in 2013… with futuristic visions sometimes…)

What is digital medicine? – 2002 – D. W. Shaffer (the first article on the subject)

 

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How can we improve patient access to experimental drugs? – WSJ

Ambulance

New approaches are needed for terminally-ill patients

In this article published in the Wall Street Journal by J. D. Rockoff a very important issue is raised: how can we give some hope to terminally-ill patients that could benefit from unregistered experimental drugs?

Johnson & Johnson has found a way with the set-up of an independent panel which will review requests from seriously ill patients. A committee of doctors, bioethicists and patient representatives will be organized and managed by the New York University School of Medicine in order to give a quick and relevant answer to all these requests.

The current law prohibits companies to give out unapproved drugs but the FDA has a “compassionate use” exception where companies can give medicines to patients with serious conditions and who are not participating in clinical trials. Over 1’800 requests reached the FDA in 2014.

Expanded-Access-Requests-Granted-by-FDA

From the article: “Amrit Ray, chief medical officer of J&J’s Janssen pharmaceuticals unit, said “the setup should be easier for patients because the company is establishing a single website and toll-free hotline for requests, rather than the current patchwork of entry points across the company and its drug trials”. Dr. Ray expects the committee could issue recommendations within days for urgent requests.”

WSJ Article

 

More on compassionate use and programs:

FDA Expanded Access Program

Compassionate use of medicinal products in Europe: current status and perspectives

Compassionate use of medicines in the European Union

Navigating the landscape of compassionate use (how to manage expanded access from an industry point of view)

Expanded access programmes: patient interests versus clinical trial integrity – 2015 – The Lancet Oncology (subscription required)

From 100 Hours to 1: FDA Dramatically Simplifies its Compassionate Use Process – 2015 – RAPS

 

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Pharma’s getting savvier about social media – Fierce Pharma Marketing

SocialMedia

European companies seem more advanced in using social media to interact with key stakeholders

An extremely interesting report from Ogilvy Healthworld about the strategic use of social media by pharma companies has been published recently. It is possible to access the key points on SlideShare. Just 5 companies out of 14 are getting patients, doctors and the media interested, delivering relevant info, inspiring actual conversations.

Some aggregated trends spotted by Ogilvy:

– 1.3 million Facebook followers

– Average number of pharma tweets per week has gone up by 530% since 2013

– Number of Twitter followers has tripled to 790,000

Who are the best socially positioned companies?

1. Boehringer Ingelheim

2. Bayer

3. Novartis

Patricia Alves from Boehringer quote: “The conversation is already out there. People are talking about you, whether you’re active or not. Social media gives you the opportunity to engage in that conversation, to give your position and your statement, and maybe then hopefully change the opinion of one person or two.”

Some tips from Ogilvy in order to improve the social fingertip of a pharma company:

– Be Brave

– Get Personal

– Move Fast

– Back Yourself Up (from a regulatory point of view)

– Give Guidance

– Inform and Educate

Fierce Pharma Marketing Article

More resources:

Healthcare Marketers Trend Report 2014 – Ogilvy

Engaging patients through social media – 2014 – IMS Institute for Healthcare Informatics

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How to determine R&D’s productivity – McKinsey

Test tubesMcKinsey experts led by Eric Hannon, worked on a unique formula to help innovative companies value the productivity of R&D activities

They explained the issues in trying to estimate the performance of R&D. Some ratios like R&D in % of sales as well as the % of new products launched over the last two years can be used but unfortunately they are not capturing the reality.

They worked on this challenge, designed a unique formula and tested it in real corporate life.

The approach

R&D outcomes = multiplying a project’s total gross contribution by its rate of maturation and then dividing the result by the project’s R&D cost.

The advantages

1. A single metric

2. Goal to measure what R&D contributes within the sphere of what R&D can actually influence

3. By measuring productivity both at the project level and across the entire R&D organization (through simple aggregation), it endeavors to speak to the whole company

The ratio & its key components

McKinseyRDRatio

Total gross contribution is the product’s economic value to customers. It is best calculated, I think, with a NPV.

Achieved product maturity is how close it is to verifying and validating its technical and commercial requirements. The implication is that companies must be able to assess, in real time, how close their R&D projects are to full maturity (simply looks at critical dimensions (such as cost, functionality, and quality) during each of the quality gates a project passes through in its development).

For pharma companies, I think that the numerator of the formula could be the rNPV for a specific project (or the aggregation for all the projects of the company). Have a look at my lectures on valuation if you would like to find out more about rNPV.

Consumed R&D costs are easy to find.

Management impact

This formula will help the management in several fields:

1. Setting direction

2. Improving teams

3. Making objective décisions

4. Driving change

McKinsey Article

Other resources:

Is R&D earning its investment? – 2010 – Deloitte

Measuring the return from pharmaceutical innovation – 2014 – Deloitte

Pop That Bubble: 5 Reasons Biotech Needs A Different Metaphor – Xconomy

SoapBubbleAlex Lash said that, this time, there will be a “soft landing” in biotech instead of a bubble burst… We’ll see…

“There are signs the sector is strong enough to make it a gentle correction”.

Let’s discuss his arguments.

Resilience

External pressures that would trigger a trend reversal is not present and, despite some adverse events (Sovaldi price questioning, overvaluation statement by the Federal Reserve, ExpressScript exclusive move with AbbVie in HCV), in 2014, the sector is stronger than ever.

My comment: that’s right. The biotech sector supported a lot of strain but it is maybe not over and there will still be investigations about drug pricing rationale (especially those molecules not bringing enough value for the patient and the society). Why paying for drugs that will only extend your life and not saving it?

Cancer Immunotherapy

Several immuno-oncology projects have already passed several important tests. Big drug makers are firing all cylinders by putting together big internal teams. Three products have been approved since 2011. Among them Yervoy (ipilimumab) and Keytruda (pembrolizumab).

My comment: competition is tough and the field is crowded. Some experts are doubtful about the commercial potential of this class of drugs despite very high prices. Let’s take the example of Yervoy. Yervoy dosing is 3mg/kg every 3 weeks. The average body weight for a patient in US is 80.7kg and 70.8kg in Europe leading to respective dosages of 242mg and 212mg. After a little calculation we reach prices per cycle of 21 days of USD 31’370 for the US patient and EUR 19’850 on average for a EU5 patient… with a median overall survival of 10 months. So for the whole treatment (for a patient treated during 10 months): USD 313’700 in USA and EUR 198’500 in 5EU. Is it reasonable?

Even oncologists are complaining about cancer drug prices. A growing number of patients have difficulty affording treatment and, if nothing is done, people will not be cured with the latest technology and commercial revenues as well as profitability for drug companies will not meet expectations, not covering all the R&D expenses incurred during the previous years.

More about cancer immunotherapy

The Crossovers

A different type of investor according to Alex Lash: “crossover investors—hedge funds and other public funds looking for an ownership foothold and a pole position for the IPO”. They tend to be specialists, not generalists.

My comment: according to Alex Lash, bubble fears come essentially from generalists, biotech investors that do not understand fully the technology and its potential applications. It may be the case, we will see in the future how these small companies develop their business models.

More about crossover investors

Venture is now different

The landscape is different with more selection made by investors and more maturity shown by companies. All this contributed to a healthier investment environment.

My comment: I totally agree with him, especially because as of today even big pharma have a dedicated VC arm to finance promising small companies.

Less regulatory risk

Today approval is not as difficult to obtain as before. Drugs are coming to market faster with Fast Track and Breakthrough Therapy Status. Incentives to drive development for unmet needs have been implemented such as the GAIN Act. The regulatory sky is much less cloudy…

My comment: that’s true but the challenge is next door as approval is just an official green light and it doesn’t mean that you will get reimbursed and adopted on the market. All the work has still to be done after approval (or even before as market access is prepared earlier now). 

Xconomy Article

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Divestments: a fundamental tool in growth strategies

A lot of opportunities will come from divested assets. It will probably reshape the lifescience industry as it will force companies to optimize their portfolio. It will be crucial for acquirers to carefully choose the right assets that have the best fit from a value creation standpoint.

New Business Models for Antibiotics. What Can We Learn from Other Industries? – OHE

New solutions could emerge from incentivization models applied in other industries

ChangeThe Office of Health Economics held a workshop last year with companies that presented their business and incentivization models: BAE Systems (defence), Allianz (insurance), Barclays Bank (finance), EDF Energy (energy), Dun & Bradstreet (corporate information) and Knowledge Unlatched (academic publishing). These models were then explored further for their applicability to antibiotics R&D.

Key points & recommendations from the workshop:

– There is a need for consolidated and focused research of antibiotic R&D. “There is a need for a an entity that pools resources, science and compounds; one that is sustainable and independent with focus on the research and early development of antibiotics in line with predefined public health-need profiles. This entity should find a way to open up the science to all working in this area.”

– The creation of a global funding vehicle putting together the resources from multiple companies, academic institutions and public bodies, in order to fund the appropriate research, early development and good stewardship of antibiotics.

– The implementation of new commercial business model for antibiotics delinked from price/volume.

Find out more: New Business Models for Antibiotics

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