Over the last half-century, the productivity of the drug development industry has been in steep decline. Pharmaceutical companies are spending more money on the development of drug therapies, but fewer molecules are making it to market. But despite the long-term lag in drug innovation, a surge in the development of technology is opening possibilities for improvement of care. According to Ray Dorsey, MD, David M. Levy Professor of Neurology and Director of CHET at the University of Rochester Medical Center, the wider adoption of increasingly refined technology may lead to the development of sensitive outcome measures, particularly in Parkinson’s disease, which would not only improve research but healthcare delivery, as well. In fact, the goal of developing these improved outcomes measures is to replace the “currently categorical, subjective, and largely insensitive in-clinic outcome measures,” says Dr. Dorsey.

Ahead, Dr. Dorsey shares insights on the important role technology—specifically smartphone apps—will play in evolving spectrum of research and care in Parkinson’s disease.

Healthcare and Technology: A Strained Relationship

In Parkinson’s disease, as with many other disease states, scientists and researchers lack strong sensitivity outcome measures. The standard outcome measure that’s been used to approve multiple drugs is the Unified Parkinson’s Disease Rating Scale (UPDRS), or, more recently, the Movement Disorder Society Unified Parkinson’s Disease rating Scale (MDS-UPDRS). “The MDS-UPDRS calls for us to rate bradykinesia based on someone’s ability to tap their thumb and index fingers as quickly as they can 10 times, and we score it from zero to four using a categorical scale,” says Dr. Dorsey. “In 2016 we would like to have better means of measuring a disease than that.”

 

Enter technology. Over the last several years, the rapid development of various tools and platforms has raised the possibility of enhanced care. However, that possibility has largely remained speculative and conceptual. According to Dr. Dorsey, a certain amount of reluctance to embrace change has long plagued the healthcare industry, particularly regarding the integration of technology. “There’s been a lack of willingness and practice in adopting technological advances to improve care and research,” Dr. Dorsey explains. As to the question of why the healthcare industry has been slow to adopt technology, the uncoupling of rewards from that investment is a major contributor, Dr. Dorsey suggests. “There are many ways to improve health to benefit patients, but currently that’s not economically rewarding,” he offers. In fairness, many technologies that have been introduced have not been validated or convincingly demonstrated. Nevertheless, we can never know the utility of a particular system unless it is investigated, which many parties are unwilling to do. “Politically powerful organizations tend not to be the earliest adopters of technology, and in general there is a great deal of reluctance to try new things.”

More recently, however, the rising cost of health services appears to have fostered a greater willingness to revisit the potential of technology at the levels of both care and research. And according to Dr. Dorsey, many avenues are currently being investigated, specifically in the realm of improving sensitive outcomes measures in Parkinson’s disease. “Smart phones, wearable sensors, and a whole range of tools and technologies may give us better outcome measures so that we are able to detect change more readily,” he notes.

One advantage of investigating the benefit of technology in research and care is reduced cost. “We can test the benefits of technology with smaller sample sizes and fewer numbers of participants, which is extremely important in slowly progressing neurodegenerative conditions,” says Dr. Dorsey. Large-scale, long-duration, expensive, and risky studies are common across the field of medicine, particularly in conditions such as Parkinson’s disease. “The largest Parkinson’s clinical trial ever was a phase 3 trial and was stopped prematurely due to futility. Specifically, a signal thought to have been present in phase 2 did not materialize in phase 3,” Dr. Dorsey explains. “Before conducting such large studies, it is important to have a pretty good sense that a signal explored in phase 2 is real and worth pursuing.” Having better outcome measures, he believes, can give people more confidence about whether signals are worth pursuing.

Due to fast rate of technological innovation, pharmaceutical companies are poised to invest increasing amounts of money to develop better outcome measures, according to Dr. Dorsey. “It’s strongly in their interest to have better outcomes measures that can more sensitively and objectively measure whether a drug works or doesn’t work. And when they can do that with small number of individuals in faster time, interests are aligned for investment and progress,” he says.

About the mPower App and the Study

The mPower app launched in March 2015 through Apple’s ResearchKit, which helps doctors and scientists quickly gather data for medical research on an ongoing basis using iPhone apps. It is the result of a collaboration between Sage Bionetworks and the Robert Wood Johnson Foundation.

Unlike traditional studies, mPower participants are able to choose who to share their data with. Sharing options include only those researchers associated with mPower, or qualified researchers worldwide. So far, over 75 percent of the more than 12,000 mPower participants chose to share their data broadly with researchers.

The app uses various unique data points to produce better understandings of how Parkinson’s disease affects individuals. For example, to measure dexterity, participants complete a speed tapping exercise on their iPhone’s touchscreen. To evaluate speech, participants use their iPhone’s microphone to record themselves pronouncing a vowel—saying “Aaaaah”—for 10 seconds.

The app also allows participants to track when each task is completed alongside the time they take their medication, to help determine the effects of that medicine on their symptoms. Participants also complete regular surveys, rating the severity of their symptoms and what they think makes them better or worse.

SmartPhones and Parkinson’s Disease

Given how much physicians have yet to learn about Parkinson’s disease and how it afflicts individual patients, modern technology can help identify patterns and answer questions that current models cannot adequately answer. “We still do not know the answers to fundamental questions—like the proportion of a day that a patient has tremor or the frequency of falls or near falls—because we haven’t had the tools to measure them,” says Dr. Dorsey. “The current model to assess people in clinical care is to have them come to an artificial environment and be seen by a clinician outside their home environment two to four times a year for 15 to 30 minutes,” he continues. In Parkinson’s disease, patients are often asked to complete surveys to determine the presence or absence of dyskinesias at home through the use of a diary every 30 minutes, which is not particularly reliable.

However, the development and increased prominence of smartphones could very well change how clinicians understand Parkinson’s disease. “We now have tools—smartphone apps—which allow us assess people in their home environment at high frequencies, sometimes continuously, and enable us to get a better sense of the degree of variability in people’s symptoms within the course of the day,” Dr. Dorsey explains. “This allows us to better understand how Parkinson’s disease fluctuates over time and helps us to potentially identify factors that we didn’t even consider previously, to see if they impact a person’s condition.”

Last month, the non-profit research group, Sage Bionetworks, unveiled data from its novel study examining the utility of the mPower app, which captured the everyday experiences of thousands of individuals with Parkinson’s disease. The dataset consists of millions of data points collected on a nearly continuous basis through iPhone sensors. These data points include capacities affected by Parkinson’s disease, including dexterity, balance and gait, memory, and certain vocal characteristics (see sidebar above for more information about the app).

According to Dr. Dorsey, the study itself and its results are significant for a number of reasons. “Beyond the immense number of participants that independently signed up for the study, the early data suggest that we can differentiate individuals with Parkinson’s disease versus those without. We can also detect pharmacological response to treatment, mainly to levodopa,” says Dr. Dorsey. Additionally, he observes, many participants submitted feedback, such as suggestions on how to improve the app and what makes their disease better or worse. “Some factors that seem to impact Parkinson’s disease are known, like exercise and medication, but there are some we didn’t know about, like weather, that now warrant further investigation.”

Not only are researchers gaining insights about Parkinson’s disease from participants that they’ve never been previously able to get, but Dr. Dorsey also emphasizes that studies like this represent a low-cost way to harness widely used technology to conduct essential research. He further believes that these initial results are likely only the beginning of many more developments to come, which may include better versions of the app. “ Future versions of the mPower app will incorporate participant suggestions and have greater functionality while hopefully offering greater engagement and value for participation.” He also points out that pharmaceutical companies such as Roche have publicly indicated that they have used earlier versions in early-stage drug development, indicating an even wider application of this technology. Although Dr. Dorsey points out the results of the mPower study need to go through the peer review process, the preliminary findings are nonetheless extremely encouraging, especially in light of the robust patient feedback and drug development efforts to include the technology.

In terms of next steps, Dr. Dorsey is hopeful that tools such as the mPower app may be used to advance the care of individuals with Parkinson’s disease. “Any tool that can provide greater information about a disease to help guide care while also helping individuals better manage their own condition and control their own destiny is a great tool,” he says. Given the ubiquitous presence of smart phones and relatively low cost of apps for both patients and researchers, Dr. Dorsey feels that technological tools such as this can also broaden access to care. “We’re now at a point where we can press a button and participate in research. Hopefully that means patients can soon press a button and start getting connected to care, which could have a huge impact for individuals with limited mobility, are driving impaired, or have overburdened caregivers.” He also suggest that these tools may potentially have even greater applications outside the US where access to care is more limited.

Political Will and the Future of Telemedicine

Outside the realm of smartphones, technology in healthcare appears to be gaining increased traction. One example of this is telemedicine. Currently, 28 states have telemedicine parity laws, with one more having a partial parity law, and an additional eight have proposed legislation, according to American Telemedicine Association (www.americantelemed.org). “That’s political will being exercised by patients and stakeholders,” he says. Lagging behind, though, is Medicare, says Dr. Dorsey. “In 2012, the federal government spent $5 million on telehealth, which amounts to less than 0.001 percent of total budget,” he notes. This is reflected in the disproportionate levels of Medicare reimbursement. “Right now, Medicare provides $150 for me to see a patient in a hospital-based clinic, $75 to see a patient in a community-based clinic closer to the patient, and zero dollars to see patients virtually in their homes.” More acts of political will are needed to change this, Dr. Dorsey explains. “The people to change this aren’t physicians, per se, but Medicare beneficiaries who have been paying into the program for the last half-century that’s designed to explicitly increase access at a time when older Americans didn’t have such access.”

Technology and the Future of Care

As we move toward the future in which the role of technology is greatly increased in both the research and delivery of medical therapy, all clinicians and researchers should reflect on how these technologies can tangibly improve the way care is designed and delivered, according to Dr. Dorsey. “Right now, we ask people with neurological disorders, many of whom have impaired mobility and overburdened caregivers, and who may be traveling from rural and suburban areas, to come to urban centers for care to see us for a maximum of 30 minutes three to four times per year,” says Dr. Dorsey. “In terms of a neurological care delivery system, it would be hard to come up with something much worse.”

However, technologies such as the mPower app along with the concurrent development of telemedicine systems offer a distinct vision of more liberated model for the delivery of care. “Physicians have about 15 to 20 minutes to spend with patients, therefore it is incumbent on those developing the technologies to make these systems useful to patients and clinicians to help improve care.” Given the speed at which these technologies have been developed, investigated, and improved, there is hope that the future is bright for Parkinson’s care and research, according to Dr. Dorsey.

In fact, the wider use of technology may allow clinicians and patients to see the realization of a more ideal vision of the practice of medicine. “The fundamental aim of medicine is to reach all those in need, but throughout history we have seen that the care patients receive is largely dictated by who they are and where they live,” notes Dr. Dorsey. “Increasingly, however, we have tools and technologies to enable patients to receive optimal care no matter who they are and where they live, and it allows us to reach all of those in need.” n