Innovating in a Value-Based World

Published on: 
Pharmaceutical Technology, Pharmaceutical Technology-05-02-2017, Volume 41, Issue 5
Pages: 16-23

Value-based medicine is putting patients at the center of pharmaceutical R&D and forcing the industry to move from treatment to prevention.

As the $3.4-trillion US healthcare industry adopts value-based practices to help control runaway costs and address imbalances in access, physicians and pharmaceutical companies find themselves in a similar situation: their performance will be judged, and payment based, on how well patients respond to their care or their products (see Sidebar).

This responsibility may frighten some doctors and pharmaceutical company executives, especially given the number of patients who refuse to comply with orders or take their medicine. Noncompliance accounts for $290 billion in medical costs each year, according to the New England Healthcare Institute (1).

Others, however, see the new focus on results as a way to reach out to or even retrain patients, reduce waste and costs, and regain the public’s trust. “We want to be rewarded for the tangible outcomes that our products provide patients, not for simply selling pills,” wrote Novartis CEO Joe Jimenez in a November 2016 editorial in 
Forbes (2).

Jimenez then went on to define any drug or treatment’s value based on the following metrics:

  • Improved survival rates (e.g., for oncology treatments)

  • Improved overall patient response (e.g., in clinical trials)

  • Better patient experience (e.g., reduced side effects and improved quality of life)

  • Impact on overall healthcare (e.g., whether it reduces the need for doctor’s office visits)

  • Value to society (e.g., reducing the economic impact of lost workdays, etc.).

Just as hospitals are moving away from individual to “bundled” services, pharmaceuticals will no longer be viewed as discrete products, but integrated treatment packages. Many of these will look like ordinary pills, capsules, or vials, but their formulations may incorporate precision medicine features (e.g., be designed for a specific patient population) and they are sure to be sold with one or all of the following: a smart phone app, companion monitoring and/or dosing device, or sensor.

In other cases, tomorrow’s pharmaceuticals won’t even be recognizable dosage forms, but combination treatments that could involve devices designed to generate electrical impulses or optical waves. An increasing number of them are likely to be therapies derived from the patient’s own cells.

Patient-focused development

More manufacturers are starting to design these products to help ensure, first, that patients take the treatment, but, second, to communicate with patients and help them manage dosages, remain engaged with healthcare providers, or stay in clinical trials.

Eventually, drug manufacturers will need to go farther. “Pharma must shift its focus from treating diseases to preventing them,” says Dr. Bertalan Mesko, the Medical Futurist, a physician who envisions the impact of digital technology on the future of healthcare. “Pharmaceutical companies will need to change their whole approach to the business,” he says.

The biggest change that pharmaceutical companies are grappling with is the fact that physicians and patients are becoming equal partners in treatment. “There is no hierarchy anymore,” says Mesko. “Digital health means that data should be available to physicians and patients at the same time and that both are in an equal partnership.”


Today, it’s easy to find patients (or their parents or family members) running patient action groups, corporations or foundations dedicated to rare diseases. Empowered by access to information and, via social media, introduced to others facing the same issues and experts treating or developing cures, patients will no longer tolerate physicians dictating treatment regimens from on high.

Similarly, pharma companies can’t simply sell them a bottle of pills and leave it at that. “Patients want to take an active part, not only in their own treatment but in helping companies produce better drugs for them,” says Mesko. “Producing drugs is just not enough anymore. How effectively pharmaceutical manufacturers respond to this change will determine whether they can even survive, says Mesko (3).

Competing with technology

A real concern, he says, is that, in the future, more patients may start up health-tech ventures or turn to tech-based treatments including apps, monitoring and dosing devices, virtual reality, and 3-D printing on their own, bypassing pharma, regulators, and even healthcare providers.

As he points out, this has already happened in the past, when some parents of children with Type 1 diabetes put together their own, unregulated monitoring and dosing devices to handle challenging dosing issues that device and drug manufacturers were not addressing rapidly enough (4). “Even though we want patients to be equal partners in the decisionmaking process, we do not want them to bypass the regulatory process and try to help solve their medical issues themselves, with startups in their garages.  We want to help push FDA forward for companies that are working on innovations,” says Mesko.



Patient-centered drug delivery

For the past few years, manufacturers have begun to focus on engaging patients and making it easier for them to comply with physicians’ prescription orders for any new drug (e.g., via more convenient dosing or delivery). This focus is reflected, for example, in formulations such as controlled release and orally disintegrating tablets, improved autoinjector systems and inhalers, as well as biopharmaceutical formulations that don’t require infusion or that can be taken less frequently.

Companion diagnostics are also being developed for more pharmaceuticals. In 2006, there were only five drug-companion diagnostic combinations on the market, but by 2012 the number had grown to 63 (5). Roche continues to work on drug and companion diagnostics packages, and 60% of its target drug candidates are being developed with a companion device (6).

But devices are also going beyond simply measuring the level of a marker in the body to dynamic real-time measurement, dosing, and control. Research is progressing in disease areas that are difficult to dose correctly, especially in children, notably asthma and Type 1 diabetes.  In the United Kingdom, clinical trials are currently underway evaluating a Bluetooth-enabled smart inhaler that would allow healthcare givers to observe patient’s dosage and technique (7).

In 2016, FDA approved Medtronic’s first “bionic pancreas” for diabetes (8), designed to measure blood levels of glucose continuously, then control delivery of insulin when levels get too high or too low. Too much insulin can lead to hypoglycemia, which can result in patient death, especially at night.

In advanced clinical trials is BetaBionic’s i-Let, named for part of the organ whose function it mimics, another bionic pancreas (9) developed by the father of a boy with Type 1 diabetes. It uses advanced algorithms and sensors to monitor glucose levels and administer insulin when glucose levels get too high and glucagon, which was recently processed in a more stable form by Xeris Pharmaceuticals (10), when they get too low.

M-health and virtual reality

Mobile health apps, whether developed in house or with outside partners, have become a given at most large pharma companies today, to engage patients and healthcare providers and promote the communication that can be so crucial to optimizing patient outcomes. Sanofi, Johnson & Johnson (J&J), Merck, and GlaxoSmithKline (GSK) have developed the best known apps. Approaching this from the tech side are companies such as Monsenso, which offers apps to help patients deal with mental disorders ranging from bipolar to schizophrenia.

GSK, Boheringer Ingelheim, and Novartis are working with another m-health provider, Propeller, which is developing a smart inhaler via an add-on and a smart phone app that would help patients treat asthma (11).

But some tech companies have also been developing apps that might compete with pharmaceutical treatments. They might not, on their own, be useful in treating cancer or diabetes, but could ameliorate symptoms of some mental conditions such as anxiety, or help patients deal with “lifestyle” conditions, e.g.,  Sleepio, Big Health’s digital treatment for insomnia (12). In the UK, government agencies, and even the National Institute for Health and Care Excellence, have started to track and rank m-health apps (13, 14).

Tech companies are also developing augmented and virtual reality to help treat conditions ranging from post-traumatic stress disorder (15) to agoraphobia and schizophrenia (16). Pharma companies are working with virtual reality in the diabetes area with NovoNordisk’s Diabetes Voyager, which uses Samsung’s Oculus Rift technology and the xBox to simulate what diabetes is and what it does (17). Other companies have developed virtual reality tools to give caregivers and healthcare providers an idea of what patients go through. Examples are J&J’s Virtual Hallucination (18), which simulates the schizophrenic’s experience, and Merck’s MS Inside Out (19), recently completed with Studio Lidell, which offers interactive insights into multiple sclerosis. An example of augmented reality is AstraZeneca’s Fit2Me digital coach for diabetes (20).

If patient communication is crucial in a value-based environment, so is reducing the cost and timelines for clinical trials and drug development. Pharmaceutical manufacturers are collaborating with universities and research companies to leverage advances in science and technology that will allow them to filter through noise and solve problems much faster and more efficiently.



Leveraging Artificial Intelligence

The single most important tool now available is artificial intelligence (AI), and powerful algorithms that allow patterns to emerge from huge volumes of data.  “AI can allow a company to do a year’s worth of research in a day,” says Mesko. “The results could take drug development to a whole new dimension,” he says.

More pharma companies are collaborating with AI specialists, who, in turn, have added simulation and the techniques of precision medicine to their toolkits. Merck, for example, is working with Atomwise, which uses in-silico models and deep learning algorithms to find new combinations of drugs that could treat existing disorders, or find new drugs from huge databases (21). The company did this recently, to find a treatment for Ebola.

Turbine is another research firm that uses simulation and artificial intelligence to help companies repurpose drugs and drug combinations and focus on likely candidates, using models to run millions of virtual trials on simulated cancer cells. The company plans to make personalized cures possible for every cancer patient by the next decade (22).

Pharma companies are also evaluating AI as a way to improve clinical research. One goal is targeting the best candidates for clinical trials so that testing is no longer random but focused on the patient groups that could potentially benefit most from the treatment. Oncompass, which focuses on the specific cancer tissues and types unique to each patient, sequences the DNA of tumor cells and then runs results through a database to determine the most relevant trials for patients with that type of tumor (23).

In-silico clinical trials, combining AI with simulation and modeling, won’t be that far off, Mesko says. “It’s nonsense to take a decade and billions of dollars to test a new drug on human beings, when thousands of people could be tested in days using in-silico methods and a supercomputer (e.g., IBM Watson) and human physiological models.”  In Europe, the Avicenna Support Action has developed a roadmap (24) for in-silico clinical trials in the future.

New patient-centered clinical trial models are already being seen. Sanofi, for example, is working with Science 37 on a digital clinical trial design for a siteless trial (25) that would use digital technology to communicate with and engage physicians and participants. Science 37 claims that the new approach could reduce the time required by 30% or more, potentially shaving two years off the time required for each trial.

Genomics has become far more accessible within the past few years, with the cost of genome sequencing moving from hundreds of thousands of dollars to $1000 and getting cheaper. “Now, companies have new tools and a chance to make meaningful therapies,” says Mesko.

As gene and cell therapies are developed, pharmaceutical companies are also entering into research projects involving gene-editing techniques. One of the most promising techniques is CRISPR, says Mesko, although it poses serious ethical questions, including concerns that there might be attempts to develop “designer zygotes” in the future.

CRISPR tests planned

At this point, it is being evaluated mainly to treat rare, genetically-based diseases. Bayer AG and CRISPR Therapeutics recently launched a venture that would evaluate its use in treating blood diseases and other conditions (26). China was the first to actually inject human patients with CRISPR-edited genes in clinical trials for cancer treatment (27), but in the United States, tests are expected to begin in 2017 to evaluate gene editing in cancer T-cell therapies and as an option for treating a rare form of blindness (28).

Some of tomorrow’s therapies will look like nothing we’ve seen to date. An example is electroceuticals, which use electricity from a tiny implanted device to change electrical impulses within the body. GSK has invested significantly in the technology (29), and plans to start clinical trials with external partners at some point in 2017 and internally in 2019.

Farther away from development is optogenetics, which applies light to neurons in the brain, activating or deactivating specific areas (30). Mesko believes that this research will be extremely important in developing treatments for neurological conditions in the future.

In the end, what will make effective innovation possible is better patient data. For the past few decades, physicians have been required to invest in and use electronic health record systems (EHRs). Patient data will be essential to developing evidence on best treatment practices and responses to specific therapies.

With value-based care, the pharmaceutical industry and US healthcare are on a highway that is still under construction. AI and advances in sensors, devices, and mobile health offer pharma the opportunity to keep its innovation alive and relevant.


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Article Details

Pharmaceutical Technology
Vol. 41, No. 5
Pages: 16–23


When referring to this article, please cite it as A. Shanley, “Innovating in a Value-Based World," Pharmaceutical Technology 41 (5) 2017.