Vicki Anastasi, VP and Global Head—Medical Device and Diagnostics Research & Real World Evidence Late Phase Research, ICON04.03.19
The foundation of healthcare is shifting from a provider-based to a patient-centric—or value-based—model. With aging populations, better-informed patients, and emerging technologies, the responsibilities and roles of every stakeholder in this industry is changing. In a value-based system, healthcare providers are responsible for not only helping patients live longer, healthier lives, but are also tasked with mitigating costs and ensuring patient safety. This is due, in part, to global regulatory agencies implementing major reforms that are intended to streamline efficiency and enhance regulatory controls.
As the healthcare landscape continues to take shape, the medical device and pharmaceutical industries must change conventional approaches to trial design, and commit to maximizing efficiency and ensuring success of their products.1 In response to this need, convergent development of medical devices and drugs has gained in popularity, as it enables each industry to attain new manufacturing, clinical, and regulatory knowledge by forming partnerships with those who have already acquired this expertise. In doing so, convergent development allows drug and device manufacturers to bring treatments to patients more quickly and efficiently.
In the following article, I present how, by focusing on the patient in a value-based system, convergent development provides new opportunities for drug and device manufacturers to form meaningful partnerships, and why CROs are uniquely positioned to foster the growth of these relationships.
Capturing Real-World Patient Outcomes in Real Time Is Critical
As device manufacturers begin to center clinical trial design around the patient, it is influencing trial conduct and data collection. Now, with patient-centered trials gaining in popularity, regulatory agencies are recognizing the need for better guidelines to support new ways to collect trial data.
For example, in early 2018, the U.S. Food and Drug Administration (FDA) presented final guidance on the use of real-world evidence (RWE) to enhance regulatory decision-making for medical devices. The FDA now recognizes that more traditional clinical trial designs sometimes measure data that do not clearly tie to actual patient improvement. Further, the guidance encourages the use of real-world data (RWD) under the right circumstances. Similarly, by 2020, the EU will have rolled out more detailed mandates around data collection in trials.2
Fortunately, RWD has the potential to deliver more effective evidence than traditional methodologies.2 RWD captured from mobile health technologies, or mHealth, can provide a more reliable real-time source of data than depending on self-reported information, and can be used to collect broader data points to support a wider range of RWE. Also, mHealth data can generate information from across large patient populations, as trials that include mHealth often require fewer patient visits.
Additionally, clinical trial participants who use wearables, apps, and other mHealth technologies typically adopt longer-term behaviors that lead to more promising real-world outcomes. These technologies allow clinicians to measure health variables and feedback in real time, without relying on patients to self-report or commit to regular site check-ins. For instance, data collected from wearable sensors allow for continuous monitoring of status for more customized care. This helps to reduce hospital visits by alerting and responding to adverse events in real time. The data collected also add R&D value, as it can detect early efficacy.
Another benefit of these technologies is how they lessen the patient burden, promoting and sustaining engagement. Uses of these technologies include home monitoring of chronic conditions after surgery, telemedicine assessment, and app-based self-assessment tools. Collectively, these technologies can help to ensure patients adhere to the necessary protocols, increasing the likelihood that respective treatments will have a positive impact, and ultimately, increasing a trial’s likelihood of success.
The First Step is Encouraging Patient Adoption of mHealth
Patient adoption of mHealth technology has paved the way for better integration of clinical trials into treatment protocols. In fact, smartphone penetration has reached 77 percent in the U.S. and the UK, and 44 percent worldwide.2 Since innovation of mHealth and other digital health technologies now occurs on a regular basis, with new wearables and apps appearing daily, patients are becoming increasingly comfortable with using these in a clinical setting.
Despite this adoption, there remains an opportunity to enhance patient trust in health apps used in clinical trials. Currently, only 32 percent of patients trust pharmaceutical apps, and only 30 percent are willing to share information with a pharmaceutical company.1 In contrast, 76 percent of patients place their trust in personal health management apps.1 Therefore, pharmaceutical or medical device companies can borrow from these apps’ designs and interaction practices to develop apps that patients will place more confidence in.
CROs have long recognized that adopting wearables, apps, and other mobile technologies facilitate more advantageous trial outcomes for patients and product owners. CROs can provide guidance on integrating mHealth technology to enhance a trial’s design, and support patient and clinician adoption to ensure correct usage of these technologies. CROs can also aid in the creation of trials designed with clearly defined measurements for patient improvement. They can work to incorporate the usage of RWD and collect meaningful evidence for presentation to regulatory bodies. Ultimately, a partnership with a CRO can ensure the right treatments reach the market and patients at a faster, more cost-effective pace.
Convergent Development Drives Efficiency
While a greater focus on the patient has become a high priority for healthcare professionals, healthcare costs remain an important concern. In the U.S., for example, health spending is projected to grow at an average rate of 5.8 percent from 2012 to 2022, which is 1 percent faster than the expected average annual GDP growth (19.9 percent of GDP by 2022), according to a Center for Medicare and Medicaid Services report.3
Moreover, with the average price of bringing a low-to-moderate 510(k) product from concept to market costing $31 million, it is crucial that cost-reducing trial strategies are considered when designing a new trial.4
Strategic alliances, or convergent development partnerships, between drug and device manufacturers can streamline efficiencies while still providing acceptable treatment options to patients. Convergent development partnerships enable each industry to attain new manufacturing, clinical, and regulatory knowledge by forming partnerships with those who have already acquired this expertise.
For example, a major component of convergent development are combination treatment approaches—which unite two or more different types of regulated drugs or medical devices to enhance patient outcomes—and their supporting trials. These approaches provide a chance for each industry to leverage a mutually beneficial partnership to help ensure their respective products stay on or are approved for market. Combination treatments also elevate or reveal new capabilities of each individual product. Since combination trials often leverage evidence from previous trials, these combination treatments can reach patients more quickly and have the potential to offer more comprehensive evidence of efficacy.
CROs are working to facilitate these combination partnerships across industries, patients, and healthcare providers. By fostering connections with clinicians and sites to ensure safety, CROs are strengthening a combination trial’s ability to retain patients. Additionally, CROs are building relationships with providers and physicians to spread awareness of trial options to eligible patients.
CROs Can Foster Relationships and Help Streamline Convergent Development
Since CROs are positioned to apply broader healthcare trends and practices to help implement patient-centric trials, they can serve as a bridge between the trial, pharmaceutical company, medical device manufacturer, and patient to streamline convergent development.
CROs have a wide network of physicians who work to assist combination trial partners in engaging with other physicians and in developing relationships that will give trials better access to the right patient populations. Establishing reliable patient relationships supports a greater likelihood of continued patient participation during a combination trial. Additionally, a CRO can apply a value-based healthcare model to cultivate trust between patients, providers, and payers. For example, CROs can enable transparent conversations in forums designed to produce effective solutions that help define and measure trial outcomes in ways that benefit all stakeholders.
Furthermore, partnering with a CRO can also improve cost and operating efficiencies, and streamline existing business models—without compromising quality.3 Since many devices are paid under value-based contracts that reward cost and clinical quality performance over periods of 90 days to one year, it is easy to overlook cost and clinical returns that may play out over decades. A CRO could help develop forward-thinking contracts for combination trials that more reliably guarantee value-based reimbursement.
And, lastly, forming a strategic partnership can allow for systematic savings by leveraging supply-chain and distribution efficiencies, and separating sales activities from service delivery.3 A CRO can set up purchasing processes that position medical device manufacturers to invest in bundled product purchases, for example, at a lower cost than purchasing individual items from multiple suppliers.
Harnessing the Opportunities of Convergent Development
As the costs of developing drugs and devices continue to rise, manufacturers are confronting pricing pressures and an increasing need to continuously demonstrate the value of their products in real-world populations. These market forces, in addition to new regulatory guidances, are driving more companies to converge to invest in new capabilities with the goal of harnessing RWE for better clinical trial outcomes.
While the global shift toward focusing on the patient and the accompanying changes can seem intimidating, value-based healthcare offers great opportunities for drug and device developers to streamline their trials. By harnessing mHealth technologies and implementing combination treatment partnerships, shorter trials are more feasible now than they were in the past. Streamlining development can facilitate patient-centric participation and attract the right patients to the right trials for optimal outcomes.
Because CROs can enable trial designs that are cutting edge, flexible, and cost-effective, they can be leveraged by industry leaders to support all aspects of cost-reducing, patient-centric combination trials. Moreover, CROs are uniquely positioned to foster meaningful relationships between clinicians, and the pharmaceutical and medical device industries to enable the formation of stronger healthcare partnerships.
References
Vicki Anastasi, vice president and global head of ICON’s Medical Device and Diagnostics Research and Real World Evidence Late Phase Research, has more than 25 years of experience in the clinical research industry. She guides pharmaceutical, biotechnology, and medical device companies in developing and implementing roadmaps for demonstrating drug and device value, safety, and effectiveness.
As the healthcare landscape continues to take shape, the medical device and pharmaceutical industries must change conventional approaches to trial design, and commit to maximizing efficiency and ensuring success of their products.1 In response to this need, convergent development of medical devices and drugs has gained in popularity, as it enables each industry to attain new manufacturing, clinical, and regulatory knowledge by forming partnerships with those who have already acquired this expertise. In doing so, convergent development allows drug and device manufacturers to bring treatments to patients more quickly and efficiently.
In the following article, I present how, by focusing on the patient in a value-based system, convergent development provides new opportunities for drug and device manufacturers to form meaningful partnerships, and why CROs are uniquely positioned to foster the growth of these relationships.
Capturing Real-World Patient Outcomes in Real Time Is Critical
As device manufacturers begin to center clinical trial design around the patient, it is influencing trial conduct and data collection. Now, with patient-centered trials gaining in popularity, regulatory agencies are recognizing the need for better guidelines to support new ways to collect trial data.
For example, in early 2018, the U.S. Food and Drug Administration (FDA) presented final guidance on the use of real-world evidence (RWE) to enhance regulatory decision-making for medical devices. The FDA now recognizes that more traditional clinical trial designs sometimes measure data that do not clearly tie to actual patient improvement. Further, the guidance encourages the use of real-world data (RWD) under the right circumstances. Similarly, by 2020, the EU will have rolled out more detailed mandates around data collection in trials.2
Fortunately, RWD has the potential to deliver more effective evidence than traditional methodologies.2 RWD captured from mobile health technologies, or mHealth, can provide a more reliable real-time source of data than depending on self-reported information, and can be used to collect broader data points to support a wider range of RWE. Also, mHealth data can generate information from across large patient populations, as trials that include mHealth often require fewer patient visits.
Additionally, clinical trial participants who use wearables, apps, and other mHealth technologies typically adopt longer-term behaviors that lead to more promising real-world outcomes. These technologies allow clinicians to measure health variables and feedback in real time, without relying on patients to self-report or commit to regular site check-ins. For instance, data collected from wearable sensors allow for continuous monitoring of status for more customized care. This helps to reduce hospital visits by alerting and responding to adverse events in real time. The data collected also add R&D value, as it can detect early efficacy.
Another benefit of these technologies is how they lessen the patient burden, promoting and sustaining engagement. Uses of these technologies include home monitoring of chronic conditions after surgery, telemedicine assessment, and app-based self-assessment tools. Collectively, these technologies can help to ensure patients adhere to the necessary protocols, increasing the likelihood that respective treatments will have a positive impact, and ultimately, increasing a trial’s likelihood of success.
The First Step is Encouraging Patient Adoption of mHealth
Patient adoption of mHealth technology has paved the way for better integration of clinical trials into treatment protocols. In fact, smartphone penetration has reached 77 percent in the U.S. and the UK, and 44 percent worldwide.2 Since innovation of mHealth and other digital health technologies now occurs on a regular basis, with new wearables and apps appearing daily, patients are becoming increasingly comfortable with using these in a clinical setting.
Despite this adoption, there remains an opportunity to enhance patient trust in health apps used in clinical trials. Currently, only 32 percent of patients trust pharmaceutical apps, and only 30 percent are willing to share information with a pharmaceutical company.1 In contrast, 76 percent of patients place their trust in personal health management apps.1 Therefore, pharmaceutical or medical device companies can borrow from these apps’ designs and interaction practices to develop apps that patients will place more confidence in.
CROs have long recognized that adopting wearables, apps, and other mobile technologies facilitate more advantageous trial outcomes for patients and product owners. CROs can provide guidance on integrating mHealth technology to enhance a trial’s design, and support patient and clinician adoption to ensure correct usage of these technologies. CROs can also aid in the creation of trials designed with clearly defined measurements for patient improvement. They can work to incorporate the usage of RWD and collect meaningful evidence for presentation to regulatory bodies. Ultimately, a partnership with a CRO can ensure the right treatments reach the market and patients at a faster, more cost-effective pace.
Convergent Development Drives Efficiency
While a greater focus on the patient has become a high priority for healthcare professionals, healthcare costs remain an important concern. In the U.S., for example, health spending is projected to grow at an average rate of 5.8 percent from 2012 to 2022, which is 1 percent faster than the expected average annual GDP growth (19.9 percent of GDP by 2022), according to a Center for Medicare and Medicaid Services report.3
Moreover, with the average price of bringing a low-to-moderate 510(k) product from concept to market costing $31 million, it is crucial that cost-reducing trial strategies are considered when designing a new trial.4
Strategic alliances, or convergent development partnerships, between drug and device manufacturers can streamline efficiencies while still providing acceptable treatment options to patients. Convergent development partnerships enable each industry to attain new manufacturing, clinical, and regulatory knowledge by forming partnerships with those who have already acquired this expertise.
For example, a major component of convergent development are combination treatment approaches—which unite two or more different types of regulated drugs or medical devices to enhance patient outcomes—and their supporting trials. These approaches provide a chance for each industry to leverage a mutually beneficial partnership to help ensure their respective products stay on or are approved for market. Combination treatments also elevate or reveal new capabilities of each individual product. Since combination trials often leverage evidence from previous trials, these combination treatments can reach patients more quickly and have the potential to offer more comprehensive evidence of efficacy.
CROs are working to facilitate these combination partnerships across industries, patients, and healthcare providers. By fostering connections with clinicians and sites to ensure safety, CROs are strengthening a combination trial’s ability to retain patients. Additionally, CROs are building relationships with providers and physicians to spread awareness of trial options to eligible patients.
CROs Can Foster Relationships and Help Streamline Convergent Development
Since CROs are positioned to apply broader healthcare trends and practices to help implement patient-centric trials, they can serve as a bridge between the trial, pharmaceutical company, medical device manufacturer, and patient to streamline convergent development.
CROs have a wide network of physicians who work to assist combination trial partners in engaging with other physicians and in developing relationships that will give trials better access to the right patient populations. Establishing reliable patient relationships supports a greater likelihood of continued patient participation during a combination trial. Additionally, a CRO can apply a value-based healthcare model to cultivate trust between patients, providers, and payers. For example, CROs can enable transparent conversations in forums designed to produce effective solutions that help define and measure trial outcomes in ways that benefit all stakeholders.
Furthermore, partnering with a CRO can also improve cost and operating efficiencies, and streamline existing business models—without compromising quality.3 Since many devices are paid under value-based contracts that reward cost and clinical quality performance over periods of 90 days to one year, it is easy to overlook cost and clinical returns that may play out over decades. A CRO could help develop forward-thinking contracts for combination trials that more reliably guarantee value-based reimbursement.
And, lastly, forming a strategic partnership can allow for systematic savings by leveraging supply-chain and distribution efficiencies, and separating sales activities from service delivery.3 A CRO can set up purchasing processes that position medical device manufacturers to invest in bundled product purchases, for example, at a lower cost than purchasing individual items from multiple suppliers.
Harnessing the Opportunities of Convergent Development
As the costs of developing drugs and devices continue to rise, manufacturers are confronting pricing pressures and an increasing need to continuously demonstrate the value of their products in real-world populations. These market forces, in addition to new regulatory guidances, are driving more companies to converge to invest in new capabilities with the goal of harnessing RWE for better clinical trial outcomes.
While the global shift toward focusing on the patient and the accompanying changes can seem intimidating, value-based healthcare offers great opportunities for drug and device developers to streamline their trials. By harnessing mHealth technologies and implementing combination treatment partnerships, shorter trials are more feasible now than they were in the past. Streamlining development can facilitate patient-centric participation and attract the right patients to the right trials for optimal outcomes.
Because CROs can enable trial designs that are cutting edge, flexible, and cost-effective, they can be leveraged by industry leaders to support all aspects of cost-reducing, patient-centric combination trials. Moreover, CROs are uniquely positioned to foster meaningful relationships between clinicians, and the pharmaceutical and medical device industries to enable the formation of stronger healthcare partnerships.
References
- http://bit.ly/mpo190420 [PDF]
- http://bit.ly/mpo190421
- http://bit.ly/mpo190422 [PDF]
- http://bit.ly/mpo190423
Vicki Anastasi, vice president and global head of ICON’s Medical Device and Diagnostics Research and Real World Evidence Late Phase Research, has more than 25 years of experience in the clinical research industry. She guides pharmaceutical, biotechnology, and medical device companies in developing and implementing roadmaps for demonstrating drug and device value, safety, and effectiveness.
