Any Threat To Current Commercial Model Of ‘Gene Therapy’?

Any Threat To Current Commercial Model Of ‘Gene Therapy’?

Wish All My Readers A Very Happy, Healthy, Peaceful and Prosperous 2020

One of the most complex areas in disease management, is the ailments related to genetic disorders. As these were incurable, over the last four decades, medical researchers are engaged in understanding the complex and intricate process to modify human DNA, using viruses for treatment. This painstaking initiative led to the evolution of ‘gene therapy’ which, according to Mayo Clinic, ‘involves altering the genes inside human body’s cells in an effort to treat or stop the disease.’ In that process, ‘gene therapy’ replaces a faulty gene or adds a new gene, to cure a disease or improve the human body’s ability to safely and effectively treat dreaded ailments, such as cancer, cystic fibrosis, heart disease, diabetes, hemophilia and AIDS, it further added.

Several studies, e.g., one titled ‘Gene therapy on the move,’ published in the EMBO Molecular Medicine highlighted, the first gene therapy clinical trials were initiated more than two decades ago. However, initially many of these were impeded by the occurrence of severe side effects in a few treated patients. Nevertheless, over a period of time, ‘highly efficient gene targeting strategies and site-directed gene editing technologies have been developed and applied clinically.’ With over hundreds of clinical trials to date, gene therapy has moved from a vision to clinical reality – offering a powerful treatment option for the correction of monogenic disorders.

It is believed that in the new millennium, ‘gene therapy’ has emerged as one of biotech’s momentous success stories for curing many genetic disorders, which were once considered incurable. But, the cost of ‘gene therapy’ treatment is indeed jaw-dropping – ranging ‘from about US$ 500,000 to US$ 1.5m. And for treatment over a lifetime, some drugs can cost as much as US$ 750,000 in the first year, followed by US$ 375,000 a year after that – for life.

Since, I have already deliberated on ‘gene therapy’ price and associated moral dilemma that it causes, in this article, I shall focus on different concerns that could pose a threat to its ongoing commercial model. Nevertheless, let’s start with the current scenario on ‘gene therapy,’ for better understanding of the issue.

The current scenario:

According to McKinsey & Company’s October 2019 article - ‘Gene therapy coming of age’ - till 2019, the primary focus in development of ‘gene therapy’ has been on monogenic rare diseases with all currently approved therapeutics falling into this category. It is worth noting, rare diseases tend to have clear genomic targets, as well as, high unmet need in a very small patient population, who have generally been under-served by other, more traditional, therapeutic modalities (including monoclonal antibodies)—making them ideal targets for gene therapies.

More than 150 investigational new drug applications were filed for gene therapy in 2018 alone. With this in mind, McKinsey & Company expects this market to grow significantly, with ten to 20 cell and gene therapy approvals per year over the next five years.

Major ‘gene therapy’ launched:

If one takes a broad look at the ‘gene therapy’ treatments launched so far, which I have compiled from different sources, it may appear as follows.…continue reading…

Is Pharma Industry A Late Learner, Always?

Is Pharma Industry A Late Learner, Always?

Several upcoming concepts in the pharmaceutical industry are becoming buzzwords today. But, most of these were recommended by stalwarts several decades ago. Interestingly, the prevailing scenario is no different, even related to wide-scale adoption of a number of cutting-edge technologies, to squarely face the ongoing challenge of changing market dynamics. Various studies point out that other industries are making transformative use of these – to be on the same page with their customers, much faster.

Pharma is considered to be a late entrant in the digital space, too. It’s still not quite clear to many, the extent by which ‘Digitalization’ is transforming the way pharma industry functions – aiming at unleashing huge opportunities for value creation – from supply chain to manufacturing – right up to creating a unique customer experience. As this subject was well deliberated in the August 2016 article on McKinsey Digital, I am not going to delve into that area today.

Therefore, the question that comes up: Is pharma industry, in general, a late learner – always, to be in sync with its contemporary customers? For exploring this point, I shall focus mainly on four areas of current hypes in the pharma business, namely - ‘patient empowerment’, ‘patient-centricity’, ‘customer experience’ and ‘E-Patients’.

In this article, I shall dwell on this subject, ferreting out some critical recent findings on the relevance of these not so recent concepts in today’s perspective. Let me start by diving deep into the time capsule.

How old are these concepts?

Industry watchers may know that these are not new concepts, in any way. The relevance of ‘patient empowerment’, ‘patient-centricity’, ‘customer experience’ and ‘E-Patients’ in the drug industry has not unfolded today, neither are these new ideas. The American medical doctor - Thomas William ”Tom” Ferguson (July 8, 1943 – April 14, 2006) was an early advocate for ‘patient empowerment’.

Since 1975: “He urged patients to educate themselves and share knowledge with one another and urged doctors to collaborate with patients rather than command them. Predicting the Internet’s potential for disseminating medical information long before it became a familiar conduit, he was an early proponent of its use, terming laymen who did so – ‘E-Patients‘.”  

Technology follows a concept and not vice versa:

With ‘E-Patient’ terminology, Dr. Thomas Ferguson talked about empowered, engaged, equipped and enabled patients. I reckon, even after close to 45 years, most of the drug industry, is still not quite there – ‘Digitalization’ initiatives notwithstanding. This is because, technology follows a concept and not vice versa.

Why it’s so?

I reckon, this is primarily because, many stakeholders often don’t pay much importance to a critical fact, which is…continue reading…

Drug Pricing: Why Justify On R&D Cost Rather Than Precise ‘Customer Value’?

Drug Pricing: Why Justify On R&D Cost Rather Than Precise ‘Customer Value’?

While looking around, it won’t be difficult to spot many types of steep-priced highly innovative products, where high costs aren’t justified by high R&D expenditure, but for unique ‘customer value’ offerings. Many consumers evaluate those and decide to settle for one, instead of opting for cheaper variants – delivering the basic customer requirements in that product class or category. Although, both pharma and electronic goods belong to high tech-based knowledge industries, similar examples are in plenty of the latter, but hardly any in pharma.

Agreed that pharma is a highly regulated industry, unlike electronic goods. But so are banks, financial services, airlines, telecommunication, among many others. Interestingly, all these industries are building great brands without talking about their investment costs in R&D, while doing so.

In this article, I shall focus on – despite facing a formidable headwind, mostly for the same, pharma industry, in general, continue to lack in two critical areas of brand building. But, before doing that let me quote from some recent research papers wondering, how is this situation continuing unchanged, despite all concerned being aware of it.

Two opposing views:

Just to recap, let me put below, two diametrically opposing views that continue to clash with each one, since long:

• New and innovative drug costs being excessive, globally, lowering their prices will not harm the progress of innovation.
• Drug industry argues, any restriction of free pricing of innovative drugs, will seriously jeopardize innovation of newer medicines and treatments.

So much of divergence in the views of two key partners within the industry, can’t just continue any longer, without a serious intervention of governments across the world, including the United States.

Pharma does want to talk about ‘Cost & Value of Medicines’. But…

It’s not that pharma doesn’t want to talk about ‘Value of Medicines,’ but not, apparently, to create an ‘emotional connect’ with its stakeholders, including the patients. It appears, more as a general justification for the high cost of new drugs. For example, a pharma trade association’s communication, after acknowledging ‘that many are struggling to access the medicine they need,’ says upfront: ‘Discussions about costs are important.’ It follows a series of much-repeated common justifications, which are no- brainer, such as:

• Medicines Help Patients Avoid Expensive Hospital Services,
• Developing New Treatments and Cures is a Complex and Risky Undertaking,
• Medicines are Transforming the Treatment of Devastating Diseases.

But, the reality is,…continue reading…

On The Flip Side of Pharma Industry: A Saga of Perennial Contradictions

On The Flip Side of Pharma Industry: A Saga of Perennial Contradictions

Awesome contribution in the battle against multiple diseases, is obviously the primary facet of the pharma industry. However, on its flip side, one would witness a saga of numerous contradictions. Some of these exist perennially in well-protected opaque cocoons, regardless of what recent research data reveal. The consequences of which leaves a detrimental impact on the patient’s health interests, eventually turning into highly contentious issues, in the socio-political milieu of recent times.

While there are many such contradictions involving the pharma industry, this article will endeavor to understand just one inherent dispute. This is related to the impact of high R&D expenditure on drug prices. It assumes importance, especially at a time, when the world’s most influential pharma trade organization continues arguing in favor of the dictum – high new drug prices are driven by mind-boggling cost of drug innovation, as R&D spending keep shooting north. Incidentally, many others challenge this assertion backed by robust data, claiming it’s not so, actually.

Thus, the question that comes up, if high R&D cost prompts high drug prices, what happens when this major cost of new drug innovation comes down, as is, apparently, happening now. A proper resolution of this contradiction by ushering in transparency in this area, is important to safeguard a critical health interest of many patients. A recent research report, followed by several other important developments in this area, exposes this contradiction, probably more than ever before.  

Some recent reports revealing the contradictions:

To drive home the point of contradictions, I shall cite a few references below, from a pool of many others. For example, one such report of September 26, 2019 unfolded: ‘The cost to bring a new drug to market has decreased to under US$ 2Billion’. This was announced by Clarivate Analytics plc  while releasing the “2019 Centre for Medicines Research (CMR) International Pharmaceutical R&D Factbook.”

Interestingly, another article had sharply contradicted the above, presenting a different story altogether. Quoting the Tufts University Center for the Study of Drug Development, it highlighted that it costs US$ 2.6 billion growing at 8.5 percent annually. However, adding an estimate of post-approval R&D costs increases, the cost estimate to US$ 2870 million. Many estimated, it would take pharma companies more than 15 years of average sales to reach breakeven.

Curiously, a different research paper,…continue reading…

Honing Patient Outcomes With WHDs

Honing Patient Outcomes With WHDs

On November 01, 2019, San Francisco-based Fitbit, Inc. announced that it has entered into a definitive agreement to be acquired by Google LLC for approximately US$ 2.1 billion. Many believe, though, the value of Fitbit lies in the health data that its wearables capture for its large base of users.

According to the CEO of Fitbit, currently the Fitbit brand supports more than 28 million active users around the globe who rely on these wearable products ‘to live a healthier, more active life.’ With Google’s resources and global platform, Fitbit will be able to accelerate innovation in the wearables category, scale faster, and make health even more accessible to everyone, he added.

The article – ‘The Real Reason Google Is Buying Fitbit,’ published in the Time magazine on November 04, 2019 makes some interesting points, such as the following:

• The fast-growing healthcare tech space could be worth US$24 billion by 2020, says an estimate from Statista.
• Although, Google has been working on cardiovascular health, diabetes and more, it hasn’t been publicly pushing healthcare as a business proposition, just yet.
• Whereas, Google’s rivals, most notably Apple, have embraced healthcare as the next big battleground in the tech world, attracted by the promises of big profits for those who can help simplify a byzantine healthcare system.

Nonetheless, the Fitbit acquisition would facilitate Google’s entry into the Wearable Health Devices (WHDs) market in a big way, alongside other big players, such as, Apple and Samsung.

Driven by the most likely scenario of increasing usage and usefulness of WHDs, several pharma players are sniffing huge underlying commercial opportunity in this space, alongside being demonstrably patient-centric. Thus, my today’ article will deliberate whether or not WHDs will be able to offer a cutting edge to innovative drug marketers, by continually honing patient outcomes. Let me initiate this discussion by fathoming the importance of WHDs in the fast transforming digital world.

The importance of WHDs in the digital world:

‘Mary Meeker‘s 2019 Internet Trends Report’ highlights, about 51 percent of the global population is now connected to the internet, with the majority of users based in China, India and the United States.…continue reading…