Doctors are evaluated on patient throughput, not the depth of their engagement. Pharmaceutical and medical device companies create and perfect new products for patients but often neglect their preferences, stated needs, and lived experiences in recruitment and development efforts.

Real-world evidence (RWE), a promising frontier in health settings from innovation to care decisions, is poised to change this dynamic. If we are open to acting upon it, it can become the basis for centering efforts directly on the patient experience.

As a father and caregiver, I’ve walked this journey firsthand. My child’s health challenges have brought me face-to-face with the limitations of a system that often measures success in drugs prescribed rather than resolutions achieved. I’ve seen the toll that rushed consultations and fragmented care take on families searching for answers. It is a stark reminder that even as the science of medicine advances, the art of listening lags.

Recently, we visited a doctor’s office to consult about our young adult’s rare disease. We hired a patient advocate to bridge the chasm between clinical expertise and lived experience. They translate medical jargon, connect patients with appropriate specialists, and, most importantly, validate the patient’s voice.

Listening to the advocate prompted me to ask a critical question about this complex clinical case: “What is the underlying cause of the symptoms?”When our non-medically trained patient advocate suggested surgery to address the root causes of our child’s GI problems, one doctor we consulted, who came from a noted teaching hospital, confronted us.

“Who are you going to believe,” he asked. A patient or me?”  Ultimately, the patient advocate, who consulted with other patients and their physicians, was spot-on. The operation took place; the outcome was outstanding. That is the power of RWE as a catalyst for objective conversation.

Real-World Evidence: A Catalyst for Change

The Food and Drug Administration (FDA) defines RWE as “the clinical evidence about the usage and potential benefits or risks of a medical product derived from analysis of real-world data (RWD).” These data include electronic health records, insurance claims, and patient-generated information from mobile health apps and wearable devices. Unlike randomized controlled trials, which too often exclude diverse populations, RWE provides a more comprehensive view of how treatments perform in real-world settings.

Physicians face immense pressure to see more patients in less time. Emergency physicians, for example, are expected to see 2.4 to 3.3 patients per hour, while family doctors might handle 3.3 patients or more per hour in high-volume settings. These metrics may boost productivity, yet at the same time, often come at the expense of meaningful patient interactions. The result? Missed opportunities to understand the full scope of a patient’s experience and even the true nature of the problem.

Empowering Patients and Data

One possible example of how RWE can prioritize the patient’s voice is StuffThatWorks, a platform founded by Yael Elish, one of the founders and executives of the consumer navigational company WAZE. Like WAZE, StuffThatWorks uses AI-powered crowdsourcing to connect individuals with similar diagnoses, enabling them to share experiences and generate actionable insights into treatments and outcomes. It’s a patient-led approach that democratizes data collection, making it richer and more representative. For sponsors of medical trials, the platform offers the chance to harness a massive customer focus group, gaining access to the voices of millions of people struggling with health concerns ranging from treatment to anxiety around illness to the cost burden of sickness.

StuffThatWorks – collaborating with companies and patient organizations, recently began collaborating with The Marfan Foundation, highlighting the potential to engage non-profits in leveraging RWE to benefit global disease communities. Similarly,  The StuffThatWorks partnership with Advanced Clinical, a major research services and clinical resourcing organization, allows project teams to engage with patients directly through surveys and focus groups, enhancing patient participation in clinical research. These initiatives demonstrate the transformative power of intentionally centering patient experiences in healthcare innovation.

More and more, leading academic hospitals such as Weil Cornell Medical Center’s Genetics and Personalized Cancer Prevention Department in New York City are recognizing that care extends beyond cutting-edge medicine and outstanding physicians—it calls for experts who have been on frontlines—as patients and counselors.

“I feel fortunate to bring my real-world experience as a patient to my work as a provider,” says Emily Epstein, LMSW, Genetic Social Worker/Clinical Behavioral Health Care Specialist at Weill Cornell. “This deep understanding of the patient journey inspired me to create a role where I can offer the very support I once advocated for, rooted in true empathy and knowledge. Real-world patient experience is essential to transforming healthcare, infusing it with humanity, compassion, and meaningful change—ensuring we honor and serve both the science of medicine and humanity at its heart.”

Chief Patient Officers

Several pharmaceutical and clinical research companies have established Chief Patient Officer (CPO) roles, recognizing the importance of patient engagement. Parexel, for example, appointed Stacy Hurt as a CPO to lead patient advocacy efforts. Hurt says, “Parexel’s priority focus is on providing patient-centered solutions that enhance the clinical trial experience and make participation more accessible to everyone.”  A health professional who is both a cancer survivor and a caregiver for her son, Hurt brings a patient’s perspective to the work.  Companies like Merck, Pfizer, Sanofi, and Astellas have also created similar roles, signaling a broader industry commitment toward integrating patient insights into drug development.

The National Health Council emphasizes that as RWE becomes a cornerstone of health decision-making, the experience and needs of patients must be prioritized, not addressed as an afterthought. Integrating RWE into the health system can enhance treatment efficacy, safety, and patient satisfaction. However, this requires more than technological innovation; it demands a cultural shift within the healthcare ecosystem.

Now What?

Reflecting on my child’s medical journey and the rise of patient advocacy communities that began to take form during the HIV epidemic and that patient community’s vocal demand to be included in conversations about drug development and news flow, it’s clear that the journey toward patient-centered care is still at an awkward early stage.

Platforms like StuffThatWorks offer a glimpse into what’s possible when prioritizing patient voices. By fostering communities where individuals can share their experiences, these tools generate valuable data and empower patients to take an active role in their care.

Let us commit to creating a health system where every patient’s story is heard, valued, and acted upon. By doing so, we honor the science of medicine and humanity at its heart.

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Along that difficult road is support – a place of respite from the complex environment innovators face – the community of HITLAB, the Health Innovation Technology Lab.  HITLAB is a leading academic research institution focused on improving global healthcare through innovation and technology and housed within Columbia University in New York City and Chaired by Stan Kachnowski, Ph.D. MPA, HITLAB has become a haven for health, technology, and design champions to find common ground.

“The HITLAB Innovation Summit is a dynamic platform where healthcare leaders, entrepreneurs, and innovators come together to forge groundbreaking solutions,” reflects Dr. Kachnowski.  “It fosters collaboration, sparks creativity, and drives healthcare transformation through the power of technology and innovation.”

HITLAB academic roots at Columbia set a tone for research and training.  New York City has emerged as a hub for digital health innovation. It is home to more and more startups drawing on the resources of the nation’s financial capital, a close connection to academic medical centers and talent. The Big Apple – like Bean Town Boston – is a strategic epicenter to explore how emerging technologies such as augmented intelligence (AI) and virtual reality (VR) find a meeting point with telehealth and remote patient monitoring through rigorous research, analysis, and design thinking.

Centered Around Cutting-Edge Solutions

At its annual HITLAB Innovators Summit 2023, conversations center around cutting-edge solutions for the digital health community to overcome social determinants of health and unite disparate hospitals’ information systems. The gathering is a digital health sector roadmap to advance health access, address challenges, and improve patient outcomes.

Attendees of this intimate Summit that combines keynotes, panel conversations and scheduling networking are fans!

“The HITLAB Innovation Summit is a game-changer for the healthcare industry. The convergence of technology, innovation, and expertise creates an electric environment that fuels groundbreaking ideas and fosters meaningful collaborations,” says John Anderson, chief technology officer HealthTech Innovations.

While other like-forums have evolved into co-counseling sessions on how to survive EHRs and the heavy life to onboard the 500-pound EPIC gorilla, attendees at HITLAB also learn about “EPIC envy” – where small and community health centers that don’t have the resources to afford this state-of-the-art software find alternative paths tapping into technologies to piece together other ways to track patient care and outcomes.

Also, in hearing from major biopharmaceutical companies such as Merck, Novo Nordisk, and others, digital health innovators can reflect on the importance of ample science to shift the conversation from hype to hope and fact-based cautious optimism.  In turn, big pharma share openly on the main stage how science alone – absent of technology – can fall short in meeting patient information, adherence and care needs. 

As in previous years, the HITLAB Innovation Summit 2023 brings together experts across the health innovation ecosystem, including clinical trials, pharma, private equity, public health, engineering, design, and data science. The diverse community is seeking solutions that integrate health tech – digital therapeutics, health information, and smart devices meaningfully. The gathering’s success lies in bridging the gap between patients, payers, product innovators, policymakers and providers. It enables these communities to connect and accelerate practical solutions that can be implemented in real-world settings.

Presenters are Category Leaders

Presenters are bold and blunt. From Bill Taranto, President, Merck Global Health Initiative Fund, to Dr. David Berger, CEO, SUNY Downstate Medical Center, to David Klein, CEO and Co-founder, Click Therapeutics, to Tim McCarthy, Head of Digital Sciences & Translational Imaging, Pfizer and representatives from public health, venture capital finance, legal and regulatory; the three-day gathering offers a bolus of category knowledge.

“The time is now for this space – a revolution in medicine is about to happen that will shift care to center around patients,” emphasizes Click Founder and CEO David Klein. 

Klein was among the key voices discussing integrating therapeutic choices and that physicians would look more holistically in treating patients with drugs, digital therapeutics and behavioral guidance – focusing on people care satisfaction.  Digital therapeutics must model its efforts akin to biotechnology driven by evidence-based clinical outcomes looking beyond the 510K regulatory milestone for approval.

Actionable Insights

The insights shared will guide business planning decisions among newcomers and veterans to the category.  Among the noted attendees at this year’s HITLAB Innovation Summit is Amir Kishon, Ph.D., CEO of Twig Health. Kishon was among the early pioneers in the sector.

“Healthcare as a regulated industry is somewhat apprehensive about innovation. It is in its DNA. Digital health is challenging this predisposition and the question I ask myself is when this industry will tip to appreciate that innovation is a friend. We are getting there,” notes Kishon.

The program conducts studies and clinical trials that set out the scientific methodology to assess digital health interventions’ efficacy, usability, and safety.  It’s now acknowledged that Food and Drug Administration 510K approval to market for digital applications and therapies is a necessary regulatory threshold; the barrier to success is convincing science as a solid foundation for access that requires payer and provider support and a clear pathway to reimbursement.

Bottomline – it’s about reimbursement and ease of use. In the session What’s Needed for Sustainable Adoption of Digital Health American Medical Association Vice President for Digital Health Strategy, Meg Baron, offered very clear insight into how digital health enterprises can better integrate into the physician’s workflow – the “return on health” to show value.

Beyond the Summit, HITLAB promotes entrepreneurship and innovation in the digital health space. HITLAB is multi-faceted – professional development, accelerator and crowd-sources community for startups and early-stage companies, helping them navigate the complex healthcare landscape, access funding opportunities, and refine their product or service offerings.

In incubating promising ideas, HITLAB fuels the growth of the digital health ecosystem, fostering the development of transformative solutions that address unmet needs and drive positive change. Put the fall meeting on your calendars!

The post HITLAB Innovation June Summit is a Power Hit for Digital Health Change Agents appeared first on Medika Life.

But what happens when goods and services are priced beyond the reach of the average person? Pandemic-fueled unemployment and inflation, and looming electricity, food and water shortages caused by climate emergencies, have provided an unsettling reminder that the basic things many people depend on to live can become unaffordable.

For most Americans and their employers, however, one of the single greatest threats to prosperity – even survival – is the uncontrolled cost of healthcare. In the past 20 years, the cost of the average family insurance premium has increased twice as fast each year as the annual rate of wage growth. In real terms, the average American family in 2002 paid $7,954 for health insurance but saw that figure skyrocket to $22,463 last year. Within a couple of decades, the cost to insure a family could be three to four times higher if nothing changes.

To put perspective to that calculation, assuming wage growth remains the same, it will require one parent to work full time just to cover the cost of protecting their family’s health. For many American households, this already is the case. Left unchecked, healthcare costs will significantly constrain economic growth as employers pass on costs to consumers and curtail investments, while governments at every level see a decrease in tax revenues that would fund a range of essential programs.

This bleak outlook should force a reckoning for the global pharmaceutical industry. While health companies must remain profitable to continue to fuel innovation, maintaining business as usual cannot come at the expense of the very customers who depend on functional healthcare to prosper. Such a conundrum poses a unique opportunity for pharma companies to live up to the credo of truly putting patients first by redefining how they lead on areas that are fundamental to strengthening health access and affordability for the long term. Among the many ways to achieve this outcome, some areas stand out:

Fostering greater cross-industry collaboration

Contrary to the views of some policymakers, the solution to averting the worst-case scenario of pricing most Americans out of affordable healthcare is not to increase competition. A better path may be to create greater incentives for collaboration between industry peers and government agencies through all stages of the value chain, from early discovery through manufacturing to patient administration.

The Covid-19 pandemic illustrated how collaboration can deliver speed and scale during an emergency. But despite the active involvement of the National Institutes of Health (NIH) in sponsoring early research for the most successful vaccine candidates and heavy government intervention to waive or reduce the costs of vaccination in many countries, the success of intra-industry collaboration has been muted by headlines of windfall profits from vaccine sales.

While many pharmaceutical and biotechnology companies already participate in the NIH’s Accelerating Medicines Partnership (AMP), more can be done to use this program as a launch pad for cooperation among industry leaders. With greater funding for the AMP and a commitment by participating companies to leverage the partnership to deliver cost-effective therapeutics, we could see a groundswell of pioneering products that provide lifesaving benefits without bankrupting patients. Such a push would support specific companies in their efforts to strengthen reputation among a variety of audiences.

Using digital tools to deliver increased transparency

From the rapid growth of electronic health records to the increase in clinical trial management solutions, information technology has been fully integrated into nearly every aspect of the pharmaceutical industry. But how can the array of digital tools be used to benefit patients?

As the health industry faces ever greater scrutiny over soaring costs, leading companies could forge a different approach by leveraging the power of their IT and storytelling capabilities to provide patients, advocacy groups and policymakers with important details on the drug delivery process. By bringing R&D to life and engaging patients in the various facets of drug development, companies may be able to spark a new dialog with informed audiences about realistic ways to lower the costs of care and focus on therapeutics with the greatest potential for public benefit without sacrificing the profits that are necessary to support medical breakthroughs.

Achieving health access equity by innovating for all

More than two decades ago, Merck’s CEO at the time, Dr. Roy Vagelos, took the bold step of restricting the company’s drug prices to match inflation. Under his leadership, Merck made efforts to price its drugs according to the economic benefits to patients. Years later, Allergan CEO Brent Saunders announced a social contract to adhere to single-digit percentage increases no more than once a year.

Few would argue that Merck or Allergan set themselves up for failure by staking patient-first positions on cost management and drug pricing. In fact, Merck’s stock price rose 22% annually during the Vagelos years. The moves likely strengthened employee morale and public perception, while serving as reputational insurance for each company against future issues.

These examples – and others – show that there is a middle path to developing innovative products with patient access and affordability at the center. This is the foundation to supporting a level field in healthcare, where patients enjoy equitable care and the health ecosystem becomes more accessible and sustainable.

It’s easy for health leaders to look at the growing crisis and kick the can down the road. That’s the playbook oil companies employed when confronted with clear evidence that their actions were causing global warming and fueling a public health crisis. Alternatively, they can recognize the moral imperative to move quickly and stake out bold pipeline, communications and pricing strategies that put cost sustainability and equitable access to care squarely in the center of everything they do. It’s worked before; it can work again.

The post Reckoning or Opportunity? appeared first on Medika Life.

It is critical that this oversight be completely divested of any interests within the pharmaceutical industry, beyond the reach of lobbyists and political influence. Not, you’ll readily agree, an easy ask in the world we currently occupy. Two things need to occur, preferably globally, to ensure our survival as a species is not compromised by the never-ending quest for profits and industry glory. We need to create the oversight and pharma needs to be forced into legal compliance.

And these things need to happen soon!

Where does the danger lie?

Contrary to the populist nonsense circulating on social media and right-wing conspiracy websites, mRNA vaccines don’t pose a risk to our DNA. At least not yet. The current slew of mRNA vaccines is unable to engage the mechanisms required to alter our DNA. It’s a complex topic which I’ve tried to simplify in another article you can reference here if it’s of interest.

That isn’t to say, however, that all future development of mRNA technology will obey the same delivery paths or mechanisms of action, but for now, the mRNA vaccines, in their current forms, cannot interact with your DNA. You can happily get vaccinated without the risk of growing a tail.

The real danger currently lies with another branch of pharma that is going to be at the forefront of new medicines for a while to come as we rage an ongoing war against our most deadly enemy. The virus.

I am, of course referring to antivirals. drugs that are designed to deliver their payloads to and engage with our genetic machinery. While these medicines, take for instance the antivirals developed for AIDS, can be hugely effective and lifesaving, they pose a very real risk of interfering negatively with our genetic code, or DNA. Not all antivirals have this ability, but many do and many newer drugs undoubtedly will.

Not only our DNA is at risk here. Antivirals work by mutating the DNA of the virus they’re designed to attack. The risk exists that resulting mutations can create drug-resistant new strains or variants of a virus, and some experts believe insufficient data exist on drugs like Merck’s new antiviral to combat Covid, Molnupiravir, to allow the drug to be released into the market. The UK has recently approved the drug for use.

Currently, we have to accept the assurances of the pharmaceutical companies, the same companies that develop these drugs, that the treatments are, in fact, safe.

Read that sentence again. There is no world in which that statement makes logical, sane sense or fosters an environment of trust and transparency. We don’t self-police well and if you don’t understand why then drop by your local police precinct and ask them to explain it to you. We simply cannot be trusted, and when the stakes are as high as they are in the world of big pharma, even independent bodies struggle to maintain any autonomy.

What about Federal and Government bodies

Doesn’t the FDA look out for our safety, you ask? Technically, yes, however their admirable mission statements and what actually occurs in practice, are two totally different realities. Woefully understaffed departments, political pressure, pharma lobbyists, and staff, many of whom owe their training and loyalty to large pharma companies, having served as interns or former employees, oversee clinical trials and approval processes, all contributing to potential breakdowns in safety protocols.

The recent scandal involving a whistleblower and the falsification and fabrication of results from a company engaged in submitting Covid vaccine data highlights the problem. Complaints around possible breaches in protocols designed to protect the public are often simply ignored. Lack of resources and manpower are usually assigned the blame and this instance was no exception.

Despite this, systems like the FDA still offer the public a certain level of protection and if they fail, as callous as it may sound, a worst-case scenario results in a few hundred deaths. Until now, that is. Treatments that can affect our DNA work differently. They operate silently and can take years, even decades before we are faced with the consequences. Genetic mutations may be passed on to descendants rather than affecting the patient directly.

Without wanting to sound melodramatic, the biggest challenge we will face in the coming decades will be ensuring the integrity of our human DNA. The reason is simple.

Antiviral medications work. There are very few effective mechanisms to combat viruses and antivirals are one of our best hopes to interfere with the virus’s interaction with our cells. Now, mRNA technology has also entered the fray, and will, undoubtedly stray into the world of DNA before long. It’s a sensible place to go if you’re looking to cure all that ails humanity.

While targetted DNA treatments may benefit the patient of the future, we need to ensure now that in our haste to produce profits, we don’t throw the baby out with the bathwater. Oversight is required.

If not governments, where do we turn?

Does the World Health Organization (WHO) still hold enough credibility globally to be entrusted with a task like this? It’s a difficult question to answer. They botched the Covid response spectacularly, erring on the side of cautious statements when they could have closed the whole planet down in days, buying us time to contain the virus.

They failed to address flawed policies on aerosolized particles when presented with new evidence that contradicted 50-year-old flawed science, evidence that affected how the virus was being distributed. The list goes on, and yet, what other option are we left with?

Only the WHO is placed in a position to engage globally with all pharmaceutical companies. New treatments in development or undergoing clinical trials that target our DNA or may, in any remote fashion, risk contaminating our DNA, would require full WHO approval prior to having a license granted for taking the treatment to market.

It’s by no means a clean or perfect solution, but it would offer a starting point. A place that we could look to and say with confidence, we are doing something to ensure we don’t accidentally self-destruct as a species. A hugely embarrassing and eminently avoidable scenario, but only if we take action to avoid it.

Don’t blame the Tech

Our levels of technology are expanding exponentially, allowing us a deeper and more complex understanding of diseases and viruses on a cellular level. Nanotechnology enables us to deliver microscopic particles to cells, that even five years ago, were only pipe dreams, dreamt by molecular biologists with a vision of what the future might hold. The wonders that await us in the next decade could very well change the face of medicine forever.

Eradication of cancer and other genetic conditions are now real possibilities thanks to technologies like mRNA treatments. Antivirals may very well finally achieve the unthinkable, a world free of influenza. It’s heady times for the industry, exciting times and times for great fortunes to be made, gambled, and lost. Therein lies the biggest danger.

It is not technology, in and of itself, that poses the risk, but rather what we choose to do with it, and given our history as a species, we know we cannot leave that to chance.

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