Novel drugs for depression – The Economist

depression

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/

Life Sciences Innovation Hotspot

LSIHS_April2016

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

 

 

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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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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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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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

 

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

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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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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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Global Oncology Trend Report 2015 – IMS Institute for Healthcare Informatics

NonSmallCellCarcinomaOfTheLung

A must-have in oncology market analysis

A new report has been published by IMS Institute for Healthcare Informatics about trends in oncology.

It is really informative (stats, charts, visuals…) and insightful. An outstanding overview of what are the main trends (biomarkers, complexity…), the challenges (financial burden, expenditures…) and the opportunities (improved survival, new approaches…) in oncology today.

Key points

biomarkers are more widely used in treatment selection, especially for tumor types with low survival rates and few treatment options;

– new complexity for doctors, payers and patients;

– rising expenditures dedicated to cancer care linked to earlier diagnosis, longer treatment duration as well as increased effectiveness of drug therapies;

USD 100 billion of cancer expenditures registered for 2014 (CAGR 2009-2014: 6.5%);

– increasing financial burden faced by patients (patient access varies widely by country);

– growth in the use of social media by patients in order to find support to cope with their disease;

rise in 5-year survival rates for major cancers;

– promising new approaches in immuno-oncology.

The report

Global Oncology Trend Report 2015: IIHI_Oncology_Trend_Report_2015

 

Image Source (Non Small Cell Carcinoma of the Lung)