A Disruptive Innovation to Fight and Cure Intractable Diseases

A Disruptive Innovation to Fight and Cure Intractable Diseases

Several important facets of health care often arrest general attention. These are also widely discussed, analyzed and argued vehemently – with each person or group trying to justify one’s own point of view. Among these, following 6 critical areas, broadly dominate the deliberations:

• Incredible advancement in the medical science driving health care,
• Infrastructure, facilitators and providers of health care,
• ‘Wolves of health care in sheep’s clothing’, as described by many
• Large populations facing inadequate availability and access to health care,
• The need for Universal Health Care (UHC)
• Public investments, policies and regulations governing health care.

In this article, I shall focus only on the first area – incredible recent advancement in the medical science driving health care, especially the very recent developments on a disruptive innovation called ‘Gene Therapy’.

Gene Therapy:

As some would know, one of the latest developments in the pharma world, relates to marketing approval in the United States and Europe of ‘Gene Therapy’ – a disruptive innovation in the medical science.

This technique of treatment using genes to manage, cure or prevent many intractable diseases are fast gaining ground globally, including India – at a slower pace, though. As I said, in America, the first gene therapy has already obtained the approval of the US-FDA in August 2017, closely followed by the second in October 2017, with the third waiting in the wings. In the European Union (EU), the first gene therapy was approved in 2012, but faced some commercial issues that I shall discuss later in this article.

During approval of the first gene therapy in the United States (US), the FDA Commissioner Scott Gottlieb reportedly said, this new frontier in medical innovation has the ability to reprogram a patient’s own cells to attack a deadly disease, such as cancer, creating an inflection point to treat, and even cure many intractable illnesses.

According to an October 10, 2017 publication of the U.S. National Library of Medicine, gene therapy may allow doctors to treat a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. Extensive research is ongoing, adopting several approaches to this treatment, including:

• Replacing a mutated gene that causes disease with a healthy copy of the gene.
• Inactivating, or “knocking out,” a mutated gene that is functioning improperly.
• Introducing a new gene into the body to help fight a disease.

Thus, gene therapy is fast emerging as a promising treatment for a number of life-threatening diseases, including inherited disorders, some types of cancer, and certain tough to treat viral infections. That said, the technique being risky, is still under study to make it safer the patients. Currently, it is being tested only for diseases that have no other cures.

The first approval of gene therapy in the United States:

On August 30, 2017, US-FDA took a historic decision with its approval for the first ever gene therapy in America – meeting an unmet need in its true sense, and thus creating a major milestone in medical science. US-FDA approved this treatment for certain pediatric and young adult patients with a form of Acute Lymphoblastic Leukemia (ALL) – resistant to standard treatment, or which often relapses. The overall remission rate within three months of this treatment was found 83 percent in clinical trials.

This path-breaking therapy (tisagenlecleucel) is named Kymriah, and is made by Novartis. Nevertheless, it is worth noting that the treatment was developed by a group headed by Carl H. June  at the University of Pennsylvania and licensed to Novartis.

A customized treatment:

The US-FDA approval letter to Novartis says, “Kymriah is a genetically modified autologous T-cell immunotherapy. Each dose of Kymriah is a customized treatment created using an individual patient’s own T-cells, a type of white blood cell known as a lymphocyte. The patient’s T-cells are collected and sent to a manufacturing center where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukemia cells that have a specific antigen (CD19) on the surface. Once the cells are modified, they are infused back into the patient to kill the cancer cells.”

Nevertheless, Kymriah can cause life-threatening side effects, such as dangerous drops in blood pressure. This has prompted US-FDA to caution that hospitals and doctors should be specially trained and certified to administer this therapy, and require stocking of drugs to control severe reactions, if and when required.

The price tag is jaw dropping:

As  reported by New York Times (NYT), Kymriah will be given to patients just once and must be made individually for each, costing US$ 475,000. Novartis reportedly has said, if a patient does not respond within the first month after treatment, there will be no charge. The company also said it would provide financial help to families who were uninsured or underinsured. This is indeed a commendable gesture.

The second USFDA approval for gene therapy:

Just about a week ago, on October 18, 2017, US-FDA approved Yescarta (axicabtagene ciloleucel) of Kite Pharma Inc. – a Gilead company. This is gene therapy is to treat adult patients with certain types of large B-cell lymphoma who have not responded to or who have relapsed after at least two other kinds of treatment.…Continue reading…

‘Digiceuticals’: A Force Multiplier to Contain Chronic Diseases

‘Digiceuticals’: A Force Multiplier to Contain Chronic Diseases

There is a growing need for more effective prevention, treatment or management of many serious Noninfectious Chronic Diseases (NCDs), with greater safety and at a lesser cost. As a major step in this direction, ‘digiceuticals’ or ‘digital therapeutics’ are now drawing heightened interest from the medical and scientific communities.

‘Digiceuticals’ are basically custom made digital software working as drugs. These are presented mostly in the form of user-friendly smartphone apps for various disease conditions –  both as stand-alone therapy, or to augment other treatment processes for better outcomes.

Increasing usage of digital technology enabled therapy for a gamut NCDs, prompts me to discuss in this article the relevance, scope, promise, and of course, the rationale behind the same. Its importance swells manifold when the risks of lifelong health hazards that such chronic disorders may pose are factored-in, alongside their overall socioeconomic impact.

Chronic diseases – the invisible global epidemic:

Chronic diseases, such as heart disease, stroke, chronic respiratory disorders and diabetes, including cancer, are by far the leading causes of death and disability worldwide. The World Health Organization (W.H.O) also reiterates that this invisible epidemic is spreading out globally – across every region and pervading all socioeconomic classes.

The mortality, morbidity and disability attributed to chronic diseases, as estimated by the W.H.O, is expected to rise to 73 percent of all deaths and 60 percent of the global burden of disease by 2020. Interestingly, 79 percent of the deaths attributed to chronic disorders occur in the developing countries, such as India.

Can chronic diseases be prevented?

W.H.O also emphasizes that four of the most prominent chronic disorders, namely cardiovascular (CVD), cancer, Chronic Obstructive Pulmonary Diseases (COPD), and Type 2 diabetes – are linked by common and preventable biological risk factors, notably high blood pressure, high blood cholesterol and overweight. Most importantly, these relate to major behavioral risk factors, like unhealthy diet, physical inactivity and tobacco use, among several others.

Awaits a radical change – from “Suppliers Push” to “Consumer Pull”:

In the above perspective, a series of well-integrated action related to several human behavioral factors, could help prevent many NCDs, effectively. A fundamental change in mindset of all concerned is critical to avert the merciless onslaught of this epidemic. This calls for a radical change in the existing process of addressing these disorders – from “Suppliers Push” to “Consumer Pull.”

The January 2017 White Paper titled, “Human-Centric Health: Behavior Change and the Prevention of Non- Communicable Diseases”, published by the World Economic Forum (WEF), drives home this point succinctly, as follows:

“Decreasing the burden of NCDs will require a transformation through which the threat of disease is recognized and addressed. The transformation should move us away from the present state of ‘supplier push’, which emphasizes expensive, capital-intensive, hospital-centric interventions that have so far produced disappointing results, to a system that relies on ‘consumer pull’. A consumer-focused system would recognize the principles of behavioral economics to encourage and enable people to adopt healthier behavior across all aspects of their lives. Individuals would be supported in this effort by a network of critical stakeholders ranging from government to private enterprise, from healthcare providers to payers, from the technology developers to local communities.”

NCDs are triggered by behavioral pattern and lifestyle:

Picking up the thread from here, I shall deliberate now, how the state of the art digital technology-developers are playing a stellar role in this space, with greater precision and more cost-effective way. This is because, many NCDs are triggered by consistent behavioral and lifestyle pattern of an individual. Consequently, continuously monitoring of desired changes in individual behavior, are expected to gradually become the first-line treatment to effectively address these conditions. Several published studies indicate that the process has started rolling, aided by smartphone based sophisticated digital tools – in many cases even without any expensive and lifelong medications.

The May 26, 2016 paper titled, “Clustering of Five Health-Related Behaviors for Chronic Disease Prevention Among Adults, United States, 2013”, captures a cluster of five health-related behaviors for chronic disease prevention as – never smoking, getting regular physical activity, consuming no alcohol or only moderate amounts, maintaining a normal body weight, and obtaining daily sufficient sleep. This article was published by the Centers of disease Control and Prevention of the United States.

Preventing chronic diseases with ‘digiceuticals’:

The April 7, 2017 article captioned, “Can ‘Digital Therapeutics’ Be as Good as Drugs?”, published by the MIT Technology Review, dwells on this area. The paper indicates an emerging belief among technology geeks that ‘digiceuticals’, or digital drugs will become ‘the third phase’ of medicine for many disease conditions, being the successor to the chemical and protein drugs that we have today, but without the billion-dollar cost of bringing one such drug to market. The core idea behind this new concept is to develop software that can improve a person’s health as much as a drug can, but without the same cost and side-effects, the author says.

An innovative new class of medicine:

The term digital therapeutics or ‘digiceuticals’, as many calls these, is considered as an innovative new class of medicine that gives participants access to the world’s most effective behavior therapies, enhanced with smart digital technology, and delivered directly to their front door. These can be used both as a replacing medicine, and also for enhancing efficacy of a medical treatment, as a situation would necessitate.…Continue reading…

A Sine Qua Non to Pharma Success in Digitized World

A Sine Qua Non to Pharma Success in Digitized World

A wind of change is now blowing at an accelerated speed – encompassing virtually anything, across the world, including India, with a varying degree, though. It leaves a profound impact on the day to day lives of many, including almost free access to a plethora of information of any kind available in the cyberspace. The way we express ourselves – connect with others – meet our various needs and requirements – make hassle-free financial transactions – increasing transparency – containing corruption, besides scores of others.

Fast evolving digital technology is predominantly catalyzing this paradigm shift. Its weighty impact can also be felt across the global business world, sparing virtually none. Digitally enabled recent GST implementation process in India is just one such example.

Newer technology driven transformation process of overall business ecosystem is sending a strong signal to all concerned to shape up – coming out of their respective comfort zones of the old paradigm, and embracing the new one. Squarely facing this challenge of change is equally critical even to one of the most conservative, tradition bound, and well-regulated pharma industry. It’s rather an absolute necessity for pharma, as virtually all its stakeholders, including the patients and governments, have already started stepping on to the digitized world. The fundamental choice is, therefore, between shaping-up and shipping-out.

In this article, I shall argue on this critical need, based on several recent, pertinent and contemporary research findings on this fascinating space.

Indian CEOs take:

The 20th CEO Survey of 2017 titled, “Being Fit for Growth”, conducted by PwC

reveals that the term ‘digital’ evokes both excitement and a sense of apprehension among CEOs, both globally and locally. The following are some interesting findings involving the Indian CEOs, as captured in this survey:

• 38 percent observed that over the past 5 years alone, disruptive technological innovations have had a significant impact on competition within their respective industries.

• 47 percent believe that in the next 5 years, disruptive technological innovations will have a significant impact on competition in their industry.

• 77 are concerned about the speed of technological change.

• 76 percent expressed concerns about rapidly changing customer behavior.

• 77 percent mentioned the need to create differentiation in their products and offerings by managing data better. 

Its relevance in pharma:

The relevance of taking this wind of change in stride and embracing it fast, is beyond any reasonable doubt today. The 2017 report of EY, titled ‘Reinventing pharma sales and marketing through digital in India,’ also reaffirms: ‘Digital will play an ever-increasing role in this era of profound transformations, characterized by increasingly informed patients/physicians, new range of customers and new disruptive entrants. To stay relevant, pharma companies need to adopt a nimbler approach and make data the currency of marketing.’.”.

The urgency:

A sense of urgency for this change has also been epitomized in the same report, as it underscores that digital disruption has demolished 52 percent of Fortune 500 companies, since 2000. The study further reiterates: “The pace of transformation has increased, competition has intensified and business models have been profoundly disrupted. This shift is happening at breakneck speed across industries, and pharma can no longer be an exception. Customers have already embraced technological changes, through their many digital touch points, and pharma must look toward digital to re-imagine the customer experience.”

Just changing manual processes to digital won’t suffice:

This is exactly what is mostly happening today in pharma. Concerned employees, in general, are also receiving training inputs accordingly. Vindicating this point, a recent study reiterates that just changing manual processes to digital won’t suffice, any longer. Delivering greater value to the stakeholders continuously through digitization of business is the name of the game.…Continue reading….