Ketamine – is it the new hope for depression?

Light At The End Of The Tunnel

After decades of lack of innovation in the depression field, maybe patients having tried several treatment options could see the light at the end of the tunnel.

Ketamine is a potent analgesic used in surgery. As the compound is highly soluble in lipids, it ensures a rapid onset of the effects leading to a quick relief of depression symptoms without the typical side effects of standard antidepressants like SSRI.

However, there are some health consequences of administering that drug in the long term:

  • CNS effects: as ketamine is considered to be a cerebral vasodilator that increases cerebral blood flow, it has anticonvulsant effects. However, its use could be limited as it has also unpleasant emergence reactions such as hallucinations, out-of-body experiences, and increased and distorted visual, tactile, and auditory sensitivity.
  • Cardiovascular effects: ketamine increases blood pressure, heart rate and cardiac output.
  • Respiratory effects: ketamine relaxes bronchial smooth muscles and may be helpful in patients with reactive airways and in the management of patients experiencing bronchoconstriction.

The cardiovascular effects could limit its used in depressed patients with cardiovascular conditions. Furthermore, we know that ketamine is safe when used for anesthesia but we have no idea about its long-term safety. For depression, it is given every few weeks for several months.

Another point worth to mention is the lack of reimbursement: it is not covered by any health insurance today and patients have to pay out of the pocket. Depending on the dose and the healthcare provider, it could range from USD 400 to 800 in USA.

Currently, late-stage studies are ongoing with compounds closed to ketamine developed by Johnson & Johnson and Allergan in order to fill this gap and provide patients with access to a safe and effective drug.

Thinking forward about mental health, we could maybe study psychoactive drugs more in-depth in order to discover whether they could be used in a controlled setting to ease some mental disorders.

Initiatives are launched to go into this direction. The future will tell…

 

Additional insights:

Ketamine Stirs Up Hope—and Controversy—as a Depression Drug – Wired – 2019

Ketamine Could Be the Key to Reversing America’s Rising Suicide Rate – Bloomberg Businessweek – 2019

Can we stop suicide? – NYT – 2018

Psilocybin – A long, strange trip – Because psilocybin research has been restricted, scientists actually don’t know a lot about how it does what it does; only recently has that started to change. To begin with, its chemical structure is similar to the neurotransmitter serotonin. Evidence from a 2012 study suggests that psilocybin “knocks out” serotonin receptors by occupying them, which “appears to allow information to travel more freely in the brain”; two areas in which it knocks out some activity are associated with self-awareness. – Quartz – 2018

Mind molding psychedelic drugs could treat depression, and other mental illnesses – The Conversation – 2018

The Effects of Cannabis Among Adults With Chronic Pain and an Overview of General Harms: A Systematic Review – Annals of Internal Medicine – 2017

Benefits and Harms of Plant-Based Cannabis for Posttraumatic Stress Disorder: A Systematic Review – Annals of Internal Medicine – 2017

The Ketamine Breakthrough for Suicidal Children – Scientific American – 2017

New Hope for Depression – TIME – 2017

The War on Drugs Halted Research Into the Potential Benefits of Psychedelics – Slate – 2017

Scientists and Silicon Valley want to prove psychoactive drugs are healthy – The Guardian – 2016

End the Ban on Psychoactive Drug Research – Scientific American – 2014

 

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Novel drugs for depression – The Economist

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The Economist took a deep plunge into mental health research for depression. The article is extremely interesting as it seems that the scientific community understands depression a bit more than before. Despite the lack of interest from the industry, old drugs like ketamine seem to be very useful in treating that disorder. In order to patent their invention, new drugs with the same benefits as ketamine are being developed by pharmaceuticals companies.

Even if we do not yet have a complete picture, we took a step in the right direction.

Article link

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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/

E-health Day – Internet of Me: Vision and Challenges

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I had the opportunity to attend the e-health day in Sierre (TechnoArk) on June 3rd 2016. The event was well organized around big players (Boston Scientific, Roche), showing their vision of the technology and its implementation in their own business model, and startups (L.I.F.E. Corporation, Karmagenes) unveiling their innovations in the field. Moreover, other stakeholders like the health insurance Groupe Mutuel and government-financed agency CIPRET presented their real-world experiences. The event was closed by a panel discussion on the relationships between eDoctors and ePatients.

Globally, the vision of health is: health care providers will be able to improve health outcomes by working with digital patients (the data collected by sensors will be integrated and analyze to provide personalized treatments and consequently better outcomes). Several projects are developed: prosthesis control, diabetes management, vital signs monitoring for elderly people…

Below I summarize the key takeaways from the most interesting talks (not all of them).

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BOSTON SCIENTIFIC – MEDTECH : WHICH BUSINESS MODEL FOR THE E-PATIENT ?
By Frédéric Briguet, EU Digital Health Engagement

  • The goal of the presentation was to provide clues on which business model is the most appropriate for digital health companies.
  • Medical technology companies create and develop products for patients but they really have to be aware of their ecosystem and the influences that will drive patients’ behavior.
    Body sensors brought revolutionary tools to life. They are wireless, responsive, use mobile devices and allow data analysis to be performed. However, what is the real impact on health and disease management? Many companies have sensors supported by solid hardware, cloud data collection and a dashboard for mobile phone.
  • Despite the evolution of technology, we are still lacking clinical studies and proofs. In addition, the user dropout rate is pretty high (after 6 to 8 months, users abandon the sensor(s) and the app). Needless to say that, on top of all the previous disadvantages, the amount of data generated is extremely heavy and it is difficult to extract the most relevant indicators to analyze them.
    However, the first digital health products helped open new perspectives and the potential of connections between all the stakeholders. Empowering and engaging healthcare providers is also one of the key benefits of the first digital health initiatives. Beyond those elements, what’s crucial to ensure adoption and reimbursement is to demonstrate the cost savings the technology could bring to the current health care settings. Doctors also have to support it and be convinced of the use and utility for their own patients.
  • All in all, the future of healthcare is expected to improve outcomes, reduce hospital readmission rate and control costs while maintaining care access.
  • The experience of the speaker allowed him to say which business models where the most appropriate to survive and thrive in that new field. He established 4 directions (that can be combined):
    1. The patient is not a consumer. Generally speaking, he is not really willing to know that he’s ill. His main focus is to live. Family and friends are the most concerned about the patient’s health and wellbeing. Creating and developing tools that could ease the burden for the supportive people around the patient is generally well received and adopted.
    2. Understand the business ecosystem. Knowing where to position the company is fundamental to avoid being stuck in a no man’s land. Focusing on lifestyle, coaching or care pathways is different and requirements increase massively for the care pathway segment.
    3. The population is ageing and increasing. The health care system will have to support an additional financial burden with the passing of the years because we know that the majority of the costs is generated toward the end of life. Hospitals are paid today according to their own efficiency (shorter hospital stays as well as improved outcomes will generate higher payments from heath insurances). It is the OPM principle (Other People Money) meaning that the patient (who consumes) is not the payer. It is then crucial to find new solutions to reduce the costs.
    4. Understanding the disease is more than fundamental. Compliance and adherence management and control in order to avoid hospital readmissions is one of the main issues of the whole healthcare system. Beyond that point, enhancing and improving the patient’s experience as well as the quality of care could well trigger new motivations for the patient to be compliant to his treatment.
  • Go beyond sensors-mobile-cloud-dashbord to include blockchain technology, augmented reality, internet of things… + any relevant technology or innovation that can bring value to the system. This value has to be demonstrated and proved as viable for the whole system.
  • The technology has to be integrated in the patient’s experience, nearly invisible, but not less complex.

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PATIENTS LIKE ME – THE POWER OF WE
By Chris Fidyk, Business Development Director Europe

  • Accelerating research and development of new treatment but also allowing patients to support each other and exchange life experience with a disease is the main goal of PatientsLikeMe. That network is maybe the smallest social network but the larger medical registry with more than 500’000 patients. Patientslikeme provides tools for patients to put their disease into context.
  • Today, there is a lot of momentum about patient centricity. It becomes more mainstream. Patients owe other patients their own experience (drugs, symptoms,…). Empowering people to express themselves about their journey in the disease. Then, when all the stories are aggregated, meanings and trends can be extracted.
  • It is also possible to see all people taking the same drug, its perceived effectiveness as well as some conversation analytics allowing to understand which symptoms are the most talked about, the treatment awareness, the barriers to access in addition to the reasons behind their treatment failure or cessation or continuation.
  • Data (experience and discussions) stay online and available even when the patient dies to enrich other patients’ lives. Regular video postings on Patientslikeme Youtube channel show patients sharing their own experience with the website and how it helps them cope with their disease.

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ROCHE DIABETES CARE – EHEALTH: THE FUTURE WE CREATE TODAY
By Horst Merkle, Diabetes Management Solutions Director

  • You can only do something with data when you can access to it.
  • Infrastructure is the foundation for innovation and creativity” – as quoted by a speaker at the Connected Health Conference.
  • How to meet the future? The lack of healthcare staff, the increase in chronic diseases and the rising healthcare costs are the challenges. How to manage them: accountability and empowerment for the patient and the consumer. Mastering your own health with less health and care services.
  • Today, technology-driven health is messy. The solutions have to be easy to use and secure for the patient.
  • The Personal Connected Health Alliance (PCHA) is at the forefront of health and wellness in today’s society, driving advancements in mobile and communications technologies, and the growing use of new devices, health trackers and apps by consumers and healthcare providers.
  • PCHA brings together the critical elements needed to ensure that these technologies are user-friendly, secure and can easily collect, display and relay personal health data. In PCHA’s vision for healthcare, consumers can use readily available technologies to access their personal health data, receive targeted health and wellness education, consult with healthcare providers and gain support from friends and family to improve their health.
  • PCHA focused on engaging consumers with their health via personalized health solutions designed for user-friendly connectivity (interoperability) that meet their lifestyle needs.
  • Business models are the main obstacles for interoperability to work.
  • Accu-Check Connect System from Roche provides an integrated meter, an app, and online tools for better diabetes management. The glucometer can share data in the cloud with the healthcare provider.

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WITHINGS – FROM QUANTIFIED SELF TO PREVENTIVE MEDICINE
By Alexis Normand, Health Development Director

  • The main goal of Withings is to sell connected products to the general public. Those products can be bought in supermarkets and will help the consumer monitor vital signs such as weight, blood pressure,… Without being a doctor, the consumer can create a dashboard for his health. Changes in health are driven by him and, due to the fact that he generates data, he will be at the center of the data collection and analysis.
  • Those tools could also be used to enhance corporate wellness and engage employees through gamification. Employers will offer a connected bracelet and will organize a competition. Employers are however inherently screening employees for health issues and can analyze aggregated data to discover trends. Data around workout and physical activity are enriched by environmental and lifestyle inputs but also by stress management and absenteeism information.
  • In the e-health field, we are in the prevention area and also on new territories like personal health dashboard and employer focus on employees’ health.
  • E-health could be widely applied together with EHRs (Electronic Health Records). Linking EHRs to outcomes allow hospitals to monitor their performance to get more money from payers as they will limit the expenses for the system as a whole.
  • Withings also builds an open health data platform with the implementation of national observatories aggregating data from all the users. Those platforms will support research on connected devices with scientific publication and could stimulate partnerships and collaborations with other data sources.

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GROUPE MUTUEL – IGNILIFE – DAILY HEALTH COACH
By Stéphane Andematten, Marketing Leader

  • Real world business case from concept to realization in partnership with the startup Ignilife.
  • Today more than 165’000 health apps are available with 40% dedicated to medical uses and 60% to wellness.
    Regarding Switzerland, few initiatives have been developed to date. Groupe Mutuel would like to be the forerunner and not a follower in the field.
  • Ignilife is a French startup with a subsidiary in Switzerland. It is the perfect combination of entrepreneurship, medical skills and user experience. Ignilife has a previous experience with Malakoff Médéric, the leader for private health insurance in France.
  • Ignilife is a e-coaching platform based on people. It covers physical and emotional wellbeing. A first auto-evaluation is performed by the user in order to have a broad overview. An assessment is then released by the system with risk factors and advices to manage and lower their impact. Support and follow-up is provided as a selection of programs the user can choose from. He can also connect his devices.
    Close to 300 video and audio plays are available. Each time a contest or challenge is won, it is input in the platform to show the progress and evolution.
  • It is essential to develop a rich, engaging and fun platform to keep the user motivated. The goals set are reasonable and not out of reach. There is a social media component where users can exchange experience and tips. More functionalities will be developed in the next future (health at work, back health, burnout prevention,…)
    Data protection is well managed, as all the data are stored in Switzerland on independent servers.
  • Groupe Mutuel pretends it does not use data collected by user but only on an aggregated basis. The rationale behind the implementation of such a project is the focus on prevention. It will help to keep health expenses at an acceptable level in the longer term.
  • Some stats:
    25’000 Ignilife users (out of more than 1’100’000 insured people)
    Mobile users connect much more than desktop users (2x)
    91% did their auto-evaluation
    61% engaged in a coaching program
    47% changed their habits

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MANAGE MY DATA OR BE MANAGED BY MY DATA
By Stéphane Koch, Expert in Digital Experience

  • People like to measure their own weight, the calories they burnt, their heart rate,…
    There is a real value added in using quantified self.
    However, coherence is not always part of the measures and can distort statistics extracted out of the data. Sensors sometimes lose connection with the app leaving gaps or errors in measures. Improving apps with coherence system would be a big step further or even allowing the user to correct the data himself.
  • Quantified self generally has a positive impact on the user’s wellbeing but it can also generate stress if the progress takes time to emerge or if it stops. Knowing the scope of the technology as well as his body are fundamental.
  • The website DMD (in French) allows the evaluation of digital tools and the sharing of everyone’s user experience.

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CIPRET – 7000 SWISS PEOPLE STOPPED SMOKING THANK TO A FACEBOOK PROGRAM
By Alexandre Dubuis, PhD, CIPRET Valais leader

  • CIPRET is a center for the prevention of tobacco use, recognized of public interest by authorities.
    It launched early 2016 a program to help people stop smoking via Facebook.
  • A real human adventure started and succeeded. The initiative was in 2 phases: recruitment and program on the same platform. It was completely free for the users. They just had to like the page, say they are interested and accept that all the posts written by CIPRET were the first they saw when they opened their Facebook app.
  • 3 pillars of the program:
    – Daily advices (personalized and not always linked to tobacco consumption)
    – Group support (tips and experience sharing)
    – Physical desire to smoke (will only last between 3 and 5 minutes => tips given to avoid relapse).
  • Professional support has been organized at 3 levels : the first one, community managers answer simple questions ; the second one, prevention experts take specific questions ; the third level, medical practioners take care of medical questions.
  • Some stats:
    1’500 posts created
    Support was on call during the whole week from 6am to 11pm
    13’000 messages have been answered in the first weeks
  • Key strengths: no moral scolding, always up-to-date, focus on workouts, nutrition, Sunday evening chats, real meeting groups.

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L.I.F.E – EXPRESS YOUR TRUTH THROUGH YOUR ACCURATE DATA
By Dario Ossola, PhD, Algorithm R&D Coordinator

  • L.I.F.E stands for Live – Inspire – Free – Entertain
  • The project started at the core of The Ark and focused on predictive models for tiredness, exhaustion and strain with the development of a garment.
    That garment is equipped with sensors to monitor vital signs (it is medically accurate and there is no need of a smartphone). It allows total freedom.
    It can be the third platform of communication (with the first 2 being computers and smartphones).
  • The medical accuracy is fundamental and it can be used in medical practice but also in sports. That garment has the same results as the invasive methods to analyze vital signs. It allows real life measurements and expansive communication (data omnipresence, diverse data analysis levels).
  • Two web sites: http://x10x.com/ (for women) and http://x10y.com/ (for men)

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ISYPEM2 – PERSONALIZATION OF TREATMENT DOAGE THANK TO MEDICAL DATABASES
By Séverine Petitprez, Scientific associate, Clinical pharmacology CHUV and Alevtina Dubovitskaya, Research assistant, HES-SO Valais

  • Software for personalized treatment dosage.
    Unique treatment dosage works very well for simple drugs like paracetamol. Unfortunately, for oncology or chronic diseases drugs, which are much more complex molecules, it doesn’t work as well. It can lead to toxicity, side effects or no effect at all.
  • Therapeutic follow-up normally starts with a blood sample, pharmacology experts interact and guide the doctor in order to personalize the treatment.
  • A new software (EzeCHieL) do exactly the same but in a much faster and more practical way. The software can create the patient’s drug metabolism curve based on the EHR (Electronic Health Record) and medical databases. Some genetic characteristics or co-morbidities can lead to changes in drug blood concentration.
  • Interoperability (web interface) as well as confidentiality and data security are guaranteed (pseudonymisation, anonymisation).

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KARMAGENES – BE A GAME CHANGER MEET YOURSELF
By Kyriakos Kokkoris, CEO

  • Karmagenes is a game combining gene profiling and psychological analysis. Integrating genetics with psychology for improved personal development.
  • What defines who you are: what you are (DNA) and where you live (environment & perception).
  • Genetics meet psychology.
  • Human centric approach
  • Could be a network of personalities and connect locally.
  • Personal guide for career development, personal motivation, and physical as well as emotional well-being.

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PANEL DISCUSSION – E-DOCTORS AND E-PATIENTS, AN EMERGING RELATIONSHIP
Moderated by Sébastien Mabillard, Swiss Digital Health coordinator with 5 paticipants: Dr. Jean-Gabriel Jeannot; Dr. Pietro Scalfaro; Pierre-Mikael Legris; Christine Bienvenu; David-Zacharie Issom.

  • The market is not yet mature, a lot of opportunities are waiting to be seized.
    The patient is at the center of all motivations.
  • Despite new technologies, there is a lot to do to reach the patient. Few success stories (the CIPRET is however an excellent example).
  • What about doctors’ digital education? Patients are really driving the trend today; they stimulate doctors to be up to date and they push technology adoption.
    Patients are very often looking for information online. Doctors should be prepared and help patients to use the right web sites in order to find appropriate and correct information. Doctors should also contribute and provide content on website to populate them with correct information.
  • All that information help to start interesting conversations. Information exchange should also be facilitated. The patient could be educated to provide the appropriate amount of information to avoid overload.
  • Patient should take part and be part of medical research. The patient is the least used resource in health care. With patients’ associations, precious information is stored and exchanged. That data can be analyzed.
  • The social component is extremely important for patients. Several of them like to share their experience and find support online. They also feel useful to provide information for other that have been diagnosed recently.
  • By giving access to medical and health information, communication and interactions with doctors will be easier and improved.

Life Sciences Innovation Hotspot

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An event has been organized in Geneva (Campus Biotech) in order to give an overview of funding opportunities in R&D in Switzerland. European as well as Swiss opportunities were explained, each time with insightful success stories and business cases.

That event combined both useful information together with relevant stories of companies having experienced the process, sometimes time-consuming but clearly worth the efforts.

Horizon 2020 was an extremely important topic during the event as Switzerland is now in a partial association framework with EC (Fact Sheet on the Status of Switzerland in Horizon 2020).

Below I summarized the key points and “take home messages” from the event in a MindMap format (much better than taking notes and reading them afterwards).

The event was organized by Inartis and BioAlps.

LifeSciencesInnovationHotspot_April2016.png

 

 

The Nobel Prize in Medicine 2015 is the recognition that nature plays a role in our pharmacopeia

Anopheles_stephensi

The Nobel Prize in Physiology or Medicine 2015 has been awarded for two major discoveries tackling severe illnesses. William C. Campbell and Satoshi Ōmura were rewarded for their discoveries concerning a novel therapy against infections caused by roundworm parasites and Youyou Tu for her discoveries concerning a novel therapy against Malaria.”

Collectively, the prize praises drug discoveries from nature that transformed the treatment of two of the world’s most devastating parasitic diseases.

327 scientists have been nominated for the 2015 Nobel Prize in Physiology or Medicine. Among them, 57 individuals were nominated for the first time.

All Nobel Laureates in Physiology or Medicine

 

Additional articles

Anti-parasite drugs sweep Nobel prize in medicine 2015 – Nature – October 2015

2015 Nobel Prize In Medicine Awarded For Discovery Of Malaria, River Blindness Drugs From Nature – Forbes – October 2015

Nobel Prize in Medicine Awarded to 3 Scientists for Parasite-Fighting Therapies – The New York Times – October 2015

Nobel prize in medicine goes to pioneers in parasitic diseases – as it happened – The Guardian – October 2015

The 2015 Nobel prizes: Physiology or medicine – The Economist – October 2015

Nobel Prize in Medicine honors treatments for malaria and parasitic diseases – The Verge – October 2015

 

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Open innovation – Is the pharmaceutical industry ready?

Open innovation is a fascinating topic. According to Dr. Henry Chesbrough, who is the co-founder of the Open Innovation Community, “Informally, open innovation is the idea that companies should make greater use of external ideas and technologies in their own business, and allow unused internal ideas to flow out to others for use in their business. It is the antithesis of a closed innovation process which relies on internal R&D and deep vertical integration.”

The traditional business model of pharma (based on blockbuster multi-billion sales generating products) has shown its weaknesses and is today doomed to fail. The industry must reinvent itself and open innovation is thought as one tool among others to help reconnect with R&D productivity and profitability.

What could be the advantages of open innovation for pharma?

  • go beyond the closed model of innovation. Collaborate and try to find the best companies for each project instead of the companies focusing on the same targets with differentiated successes…
  • focus on what you do best. The cost of R&D will thus not be duplicated and will be dramatically lower. It will benefit the industry as a whole.
  • concentrate on specific markets and sell the IP for the other ones.
  • open your R&D to other industries and take a look at them too as cross-fertilization could lead to fantastic and awesome ideas.
  • expand your network to exchange ideas and challenge your own ones.
  • use crowdsourcing when you are stuck. Somebody in the world has maybe already encountered such an issue or the wisedom from the crowds could help you.

Companies like AstraZeneca and Eli Lilly have already started their own transformation by opening up to the external world. They are not alone and the transformation is on track…

As a conclusion, I propose you to watch an pretty old but excellent TED Talk on the subject, not dedicated to the pharmaceutical industry. He talks about the role of consumers and end users but also about the fact that creativity is both collaborative and interactive. The pharma industry absolutely needs (an it has already started to do it!) to think out of the box. It has definitely to look toward other industries to learn from them. Another point highlighted by the speaker is the fatal error of looking for incremental innovation instead of striking/disruptive innovation because of the perceived risk. We know that big companies are far more risk averse that smaller ones. Attitude has to change.

Additional resources:

Measuring Open Innovation_Creativity_Innovation_Management_2015 – A brilliant article on how to measure open innovation

Open Innovation in Pharmaceutical Industry: A case study of Eli Lilly – Master of Sciences Thesis by Borja Hernandez Raja & Priyadarsini Sambandan – 2015

Managing innovation in Pharma – PriceWaterhouseCoopers – 2014

Restructuring The Pharma Industry Mizuho 2014 – On page 30, the open innovation model is compared to the traditional model. This report brings to light which company leads the way when it comes to open innovation.

Connecting Knowledge: How big pharmaceutical companies invest in Open Innovation? – Futuribles – 2014

Models for open innovation in the pharmaceutical industry – Drug Discovery Today – 2013

Pharmaceutical Innovation Hits the Wall: How Open Innovation Can Help – Forbes – 2011

JNJ_Open Innovation An Imperative for the Pharma Industry_BIF_Spring_2010 – An excellent presentation by the head of external research at JNJ

Is open innovation the way forward for big pharma? – Nature Reviews Drug Discovery – 2010 (subscription required)

Future of OI – R&D Management – 2010

Pharma 2020: Challenging business models – PriceWaterhouseCoopers – 2009

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The world’s first true “smart drug” enhances cognition and is deemed safe by health experts

Serendipity or “making discoveries, by accidents and sagacity, of things which we were not in quest of” is again one of the best source for innovation in the drug industry.
This is the story of a sleep disorder pill (modafinil or Provigil) that has been deemed the first pure cognitive enhancer.

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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Failure to publish the results of all clinical trials is skewing medical science – The Economist

Truth

A recent article published in The Economist highlights the fact that more than half of all the clinical trials are never published.

In any situation, if we only know half of the truth, distorsions ocurr and could lead to inappropriate and harmful decisions. A US law was passed in 2007 to encourage pharmaceutical companies to register them on a website and give follow-up on the results.

But this is not always precisely executed. After the legal maximum of a year was up, the percentage of clinical trials which had had their results published:

– 17% of those paid for by industry;

– 8% of those sponsored by the National Institutes of Health;

– 6% of those paid for by other government agencies/academic institutions.

The quantity of missing trials is huge and hiding poor results is not what we expect from scientists.

It has to change as hidden or missing data could lead to the use of inappropriate drugs in some patients. There is hope: the website Alltrials (launched by Ben Goldacre) and the charity behind this initiative are leading the movement toward more transparency. The charity “Sense About Science is working on an index, to be published later this year, that will rate pharma firms according to the extent of their commitment to publish all trials”.

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Why Are We Failing To Fund Mental Health Research?

Insightful post on the reasons of mental health underfunding.

Snackable Science

Everyone is affected by mental illness in some capacity, either directly or through those that we know. Around a quarter of the population experience a mental health condition each year and this high prevalence has considerable repercussions, both socially and economically. Indeed, many would be surprised to hear that on top of obvious human suffering, mental illness is estimated to cost the UK an eye-watering £105 billion a year once healthcare expenses and lost productivity have been taken into account.

With mental illness disrupting the lives of so many and harming the economy to such an extent, I was genuinely surprised to hear of the funding gap that exists in mental health research. Compared to other diseases that place a similar burden on society, publicly funded research into mental health is disproportionately low. Cancer research provides some of the strongest evidence of this effect, receiving around 20% of total UK research expenditure, almost four times more than the amount invested…

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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

 

A word about the gene-editing technology, called Crispr-Cas9 – WSJ

DNA_strand

There has been a lot of buzz going around about the Crispr-Cas9 gene-editing technology

But what it is? and what impact could it have on drug development, innovation and new treatments?

What it is?

A fascinating short video created by the McGovern Institute for Brain Research at MIT explains the key characteristics of the technology.

Why is it so exciting?

A Wall Street Journal article reveals what this technology could allow regarding the development of potential new treatments and why it is so exciting.

This new technology will enable the “editing” of defective genes and their replacement by healthy (or normal) genes. Pharmaceutical companies are just starting to explore how to use it as drug discovery would probably be revolutionized. Diseases like cancer, cystic fibrosis,… caused by a mutated gene will eventually find a solution.

Another key advantage is the ease of use. Unless other technologies, Crispr-Cas9 gene-editing technology is much easier to handle than prior methods.

Is it already used?

Yes, it is selected to create genetically engineered research animals for the time being but there is more to come…

Is it dangerous?

It could be, if it is not used correctly and ethically. In April 2015, Chinese scientists used this method to manipulate the DNA of human embryos. This raised fears that it could be used to tune babies up before birth. Manipulating life has to be ethically monitored in order to protect life.

In another article that was then retracted, it seems that Crispr technology caused nearly 2,000 unexpected mutations — 10 times what was previously observed (in two mice that it cured of blindness). It is not the “death” of the technology but it proved that we don’t know yet how it really works.

Studies, published in Nature Medicine, revealed that edited cells might cause cancer.

What are the technical hurdles to overcome before creating a drug treatment based on this technology?

Today we do not know how to deliver a drug that could target the right DNA in the right cells. Inactivated viruses or nanoparticles could lead be used as potential carriers.

What’s next?

Overcoming drug delivery hurdles, perfecting the technology, translating it into drugs able to cure genetic disorders will be a fantastic improvement in health sciences. It will bring hope to many patients suffering from these diseases without any cure today.

As the technology is very promising, scientists and universities are currently fighting on the legal battlefield to know who will be the owner of the patents and who will make money from the technology and its application. More details

Additional Articles

As it moves out of the lab, CRISPR brings tough questions into the clinic – STAT – October 2018

A flawed study shows how little we understand CRISPR’s effects – Wired – April 2018

Biotechnology: At the heart of gene edits in human embryos – Nature – August 2017

Redesigning Life: How Genome Editing Will Transform the World – London School of Economics Book Review – September 2016

Beyond CRISPR: A guide to the many other ways to edit a genome – Nature – August 2016

Do CRISPR enthusiasts have their head in the sand about the safety of gene editing? – STAT – July 2016

Federal panel approves first test of CRISPR editing in humans – Washington Post – June 2016

Riding The Gene Editing Wave: Reflections On CRISPR/Cas9’s Impressive Trajectory – LifeSciVC – May 2016

Scientists solve CRISPR’s “Energizer bunny” problem – STAT – April 2016

CRISP biotech Intellia strikes licensing deal with Regeneron, readies for IPO – BioPharmaDive – April 2016

Bill Gates, together with other well-known investors, recently invested in Editas Medicine as part of CRISP-Cas9 frenzy.

Medical specialists urge more debate on gene-editing technology – Reuters – September 2015

CRISPR player Intellia looks IPO-ready after $70M round – FierceBiotech – September 2015

Editing humanity – The Economist – August 2015

Eight Things You Might Not Have Known Until Editas Filed Its S-1 – Xconomy – January 2016

Historic CRISPR Patent Fight Primed To Become Head-To-Head Battle – Xconomy – January 2016

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Scientists want to make sure medical breakthroughs aren’t just for white men

Precision medicine could reduce the gender gap in clinical trials and genetic databases. This will lead to better care and improved outcomes for women and minorities.

2015 Design of the Year – Human Organ on Microchip – Financial Times

Innovation

Accelerating the path to market for drugs is the dream of many scientists. Reality is closer than you think…

A Financial Times article (subscription required) highlights this breathtaking innovation that could revolutionize the life sciences world.

“These new organs on chips will enable scientists and researchers to mimic responses to drugs and treatments in human tissue without recourse to animal or human testing”.

It will enable researchers and pharmaceutical companies to spend less time and money in animal models as well as in human clinical trials. It will also accelerate the development and allow patients to be provided with the needed drugs sooner than ever before.

Human organ on a microchip has won the 2015 Design Museum’s Design of the Year contest. Thought and developed by a cross-disciplinary team of researchers led by D. Ingber and D. Dongeun Huh at Harvard University’s Wyss Institute, this chip is able to mimick several different types of organs. By using it, it will also be possible to see whether a patient would respond or not to treatments. Personalized medicine will be more precise and more targeted. Additionally, multiple chips can be connected to play the role of the human body.

Have a overview of the technology in their 2-minute video presentation:

 

Go deeper into this amazing technology and its potential application with the TEDx Boston talk by Geraldine Hamilton:

 

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The Vaccine for Type-1 Diabetes Is Moving Forward

Fascinating use of the BCG vaccine – it could bring hope for Type 1 diabetes patients in reversing their disease. Looking forward to seeing the results in 5 years! A story to follow!

TIME

A promising vaccine that has the potential to reverse the symptoms of type I diabetes—an autoimmune disease often diagnosed in childhood—is heading on to a phase II trial, which will test the vaccine on humans with the chronic disease.

The vaccine, called bacillus Calmette-Guérin (BCG) has succeed in reversing type 1 diabetes in a trial among mice and in a phase I trial in 103 humans. The new trial, which the researchers announced on Sunday at the Scientific Sessions of the American Diabetes Association, will last for five years and will test the effect of the vaccine on people with type 1 diabetes among adults between ages 18 to 60. The vaccine may be able to improve the disease in people who have small but detectable levels of insulin coming from their pancreas. Lead researcher Dr. Denise Faustman, director of immunobiology at Massachusetts General Hospital (MGH), estimates that about one…

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8 emerging technologies that could revolutionize the life sciences – Medium

DNA_1

Google Ventures managing partner’s fascinating view on the next wave of innovations in life sciences

Bill Marris is a key influencer to follow in the life sciences industry, but only on Google+… On Twitter, it’s better to follow Google Ventures. Anyway… There is no need to present him.

He is a visionary for new technologies, new business models and he’s very smart when it comes to spot the next breakthrough innovation in life sciences. In this context, he wrote a captivating article on the 8 emerging technologies that could revolutionize the life sciences.

I propose a summary below but I agree it is a pure pleasure to read the entire article, so do it!

Many of the technologies that will transform healthcare already exist but have not yet been expoited at their full potential. Here is the list:

1. Artificial intelligence in healthcare – Machine learning and computer power will help drive better diagnosis and improved data analysis in order to support medical decision-making.

2. Understanding the brain – Decoding the brain is the key to find a cure for brain diseases as well as spinal cord injuries. With recent discoveries, we are at the start of a new era! Check here too!

3. Reinventing antibiotics – Widespread use as well as misuse are at the origins of the emergence of resistant bacteria. We have to really focus on this emerging threat by limiting the use of antibiotics and promoting research of new molecules. Have a look here for more!

4. Battling cancer – Amazing progress has been done over the last years and it is not finished yet! There is more to come. Additionally a real innovation will also be to make these treatments affordable for every patient.

5. Genetic repair – CRISPR, the gene-editing technique, allows the replacement and/or repair of any genetic mutation. Wow!

6. Understanding the microbiome – The 2.5kg of bacteria living on us are a treasure trove! The microbiome is an organ in itself, responsible for our health and wellbeing as well as our diseases and bad moods…

7. Organ generation – 3D cell culture and 3D bioprinting could be the solution to our ageing bodies… Spare parts…!

8. Stem cells – How to maintain the regenerative power of stem cells into adulthood?

All these fields are promising and could give hope to thousands of people in the world. More technology into healthcare for increased value is unavoidable. Let’s hope health and quality of life won’t become unaffordable.

 

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The $2.6 Billion Pill — What’s behind this number – NEJM

SpendInvest

Scrutinizing the numbers

Experts are putting into question the number released as well as the methodology used by researchers at the Tufts Center for the Study of Drug Development. Actually the Tufts announced last November it had calculated the cost of developing a new drug: USD 2.6 billion.

Jerry Avorn is not the first person suspicious about this figure. Several years ago in BioSocieties, the journal of the London School of Economics and Political Science, Donald Light and Rebecca Warburton dissected the high costs of drug development in an article: “Demythologizing the high costs of pharmaceutical research“.

Along the same line of thought, Jerry Avorn is making some thought-provoking points about the Tufts number in a NEJM Perspective article. I summarized them below:

limited analysis to “10 unnamed drug makers provided on 106 unnamed investigational compounds that they had self-originated.”

transparency issue: “numbers on which the analysis is based are not available for transparent review.”

capital costs were not updated to take into account the current interest rates: “capital costs were assessed at 10.6% per year, compounded — despite the fact that bonds issued by drug companies often pay only 1 to 5%.”

lack of consideration for “large public subsidies provided to pharmaceutical companies in the form of research-and-development tax credits or substantial payments received from the federal government for pediatric clinical activities.”

no acknowledgment for “drug-development costs borne by the public for the large number of medications that are based on external research that elucidated the disease mechanisms they address.”

opportunism by industry players as “some of the most important recent new medications were not developed by large drug manufacturers but were acquired through purchase of the biotech firms that discovered them. These, in turn, are often spinoffs based on the discoveries of NIH-funded university research laboratories.”

Beyond these methodological issues, there are two interesting points taken from the Tufts analysis:

1. The time to get the regulatory green light has been shortened allowing drugs to be quickly available for marketing (theoretically because market access hurdles remain).

2. “The most expensive weakest link points to the limits of companies’ ability to efficiently choose compounds for development and to identify adverse effects or limited efficacy earlier in the development process”. There is a clear need for better productivity processes and improved decision-making at the core of Big Pharma R&D centers.

Conclusion

Jerry Avorn is asking for more transparency and accuracy in the determination of the costs linked to the development of a new drug as it will lead to a better understanding on what are the key drivers and what could be done to support the most useful R&D efforts.

This will definitely help the society to contain healthcare costs and give patients a real value added as well as improved outcomes.

 

NEJM Article (free)

Biosocieties Article (free)

 

More resources on Drug Development Costs

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

The Ugly Cost Of Developing New Drugs — Can We Make It Prettier? – 2014 – Forbes

Does it really cost $2.6 billion to develop a new drug? – 2014 – Washington Post

The Cost Of Creating A New Drug Now $5 Billion, Pushing Big Pharma To Change – 2013 – Forbes

 

 

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