An end to trial by ordeal and a shift to patient-centricity
A new eyeforpharma survey “Patient Centricity, Technology and Data in Clinical Trials” finds that technology is helping to transform the clinical trials process in the face of growing costs and a fall-off in enrollments.
Introduction
Traditional pharma operating models have faced a perfect storm that has been raging for the past few years: the triple threats of intensifying competition, new technology and an increasingly empoweredcustomersearching for outcomes and value. Yet, all the while, the industry itself has appearedas if becalmed, unsure of how to respond.Nevertheless, although the storm may well continue to intensify, it has also brought with itthe very winds of change that have the potential to lead the industry out of the doldrums.
Patients – the industry’s ultimate customers – are potentiallythe big winners from the new course that the industry has embarked upon, thanks to their new seat at the healthcare tableof influence. Moreover, evidence-based medicine, developments in personalized medicine and digital health,as well as the means to interpret big data, all look set to change the dynamic and shine the spotlight ever more brightly on individuals and patient populations. We’ve truly arrived in the era of patient-centricity.
There is considerable potential for pharm to gain commercial insights by focusing on the patient along the whole length of the brand development lifecycle – from identifying potential market opportunities, through the R&D phases, and on into the commercial stages. Nowhere is this more evident than in the context of clinical trials, which in many cases have been suffering from significant cost inflation alongsidechronic and accelerating erosion of the number of patients willing to take part. Fewer physicians, too, are opting to engage in clinical research.
Equallytroubling is the trend for patients not tostay the course: the FDA reports that less than half of enrolled patients in its registered trials remain in the trial to the end, according to the PAREXEL Biopharmaceutical Statistical Sourcebook 2013/2014.
So, what can be done to modernize the trial process and to implement methodologiesthat are both initially more attractive topatients and HCPs and then capable of retainingparticipants? eyeforpharma conducted a survey among senior individuals with responsibility for or a special interest in clinical trials to understand more about how pharma is tackling the issue.
Download the survey results here.
Survey results
Patient centricity
Perhaps the key result from the survey is that the overwhelming majority of respondents said that their organization is currently focused on patient-centricity in the context of clinical trials. Asked whether they agreed with the statement “‘Putting the patient at the heart of the clinical trial’ has been named as the top priority for our organization going into 2015”, almost 75% agreed, while only 7.96% disagreed; 18.29% were in the “don’t know” category.
Patient centricity is seen as a key factor in improving both the efficiency and the focus of clinical trials, although it is also perceived as being so important to the new pharma operating and commercial model that 21.24% of respondents reported it as being a company-wide imperative. That said, a worrying 16.52% of respondents reported that “no one is spearheading the concept.”
Who is championing patient-centricity?
Further evidence of the significance of patient-centricity comes from the factthat the concept is being championed by the CEO or other board-level executives in 19.17% of organizations. Clinical operations (14.75%), the chief medical officer (7.67%), medical affairs (6.49%) and the clinical design or clinical development organization (5.60%) were also leading the patient-centricity charge, with initiatives also occasionally being spearheaded from within disease areas (3.83%). Less frequently, the patient-centricity driveis lead by other departments: data management/information management (2.65%) or the regulatory department (2.06%)
Key challenges
Respondents reported that pharma is currently facing a number of key challenges with respect to clinical trials. Top of the list is speed of trial enrollment(42.77%) followed by managing timelines(39.53%).Integrating newtechnologies into clinical trials procedures is also perceived as a challenge – by 34.51% of respondents – as is working internally across silos (25.66%). Further key challenges include: oversight of outsourced operations(23.60%), narrow patient eligibility criteria(23.30%), patient retention(20.06%),displaying R&D productivity(18.88%), protocol adherence (17.70%), site selection (16.52%),certification and standardization of investigators (12.09%), site activation (12.09%) and data triangulation (5.01%).
Other issues flagged up by respondents include: managing big data from multiple sources, implementing innovation quickly and “harmonized standards for ethics committees”. Regulations and bureaucracy and regulatory delays were also perceived to be problematic by some respondents.
Outsourcing clinical operations
Outsourcing clinical operations is seen by some as an inhibiting factor when it comes to engaging the patient. However, the question split respondents: 38.05%agreed that “the degree of outsourcing of clinical operations has become a barrier whentrying to engage the patient” but almost as many (33.63%) didn’t agree; 28.32% were not sure.
Risk-based monitoring
More than half of the respondents agreed (57.82%) that “risk-based monitoring will have amassive positive impact on clinical trialsoperations in the next two years”, while only 7.37% disagreed. However, approximately one third of respondents were unsure (34.81%).
Adaptive trial design
Meanwhile, adaptive trial design is also seen as an important development: 56.93% of respondents agreed with the statement: “Adaptive trial design will have a huge
impact on reducing clinical trial costs”; in contrast 12.39% did not agree. Almost a third (30.68%) were not sure of its implications. At the same time, 46.31% of respondents included adaptive trial design among the top three areas with the most potential to transform clinical trials.
Data
Capturing and using data is set to make a big impact. For instance, 50.74% of respondents reported that their organization was planning to deploy electronic data capture (EDC) toenhance clinical trials.Moreover, 35.99% of respondents said that EDC is among the top three areas in which they or their organization were planning to invest the most resources.
Among othernew data technologies respondents or their organizations were planning to deploy were: clinical trial management systems (CTMS) (44.84%), electronic patient reported outcome (ePRO) (45.72%), leveraging of e-health and m-health applications (37.76%), interactive response technologies (IRT),(25.96%) and randomization and trial supply management (RTSM) (19.47%). Several respondents noted that systems such as EDC, CTMS and IRT were already implemented.
In terms of investing time and resources, respondents indicated that the following were among their top three priorities: risk-based monitoring strategies (35.40%),outsourcing of trial operations (30.68%), real-world data integration (27.43%), ePRO and leveraging of e-health and m-health applications (both 20.35%), adaptive trial designs (19.17%), non-interventional studies 15.63%, trial modeling and simulation (15.04%), telemonitoring and mhealth(14.16%), patient advisory boards (13.86%), patient advocacy boards (11.80%) and drug repurposing (7.67%).
Other areas of investment mentioned by respondents, of which several related to data, included: regulatory authority contacts, standardized digital identities, data visualization and analytics and permission to contact platforms.
What will transform clinical trials?
Asked to nominate the top three areas with the most potential to transformclinical trials, respondents highlighted adaptive trial design(46.31% – see above), followed by real-world data integration (40.41%). Other significant areas included: risk-based monitoring strategies(33.33%), ePRO (27.14%), leveraging of e-health and m-health applications (26.84%), trial modeling and simulation (21.24%), telemonitoring and mhealth(19.76%), EDC (19.47%), outsourcing of trial operations (12.98%), patient advisory boards and patient advocacy boards (both 12.09%),non-interventional studies (8.26%) and drug repurposing(7.67%).
Two-year outlook
Looking ahead, digital is seen as an important enabler of patient-centricity and is set to make an increasing impact over the next two years, especially in the context of the social web. Respondents are looking to web-based patient portals (50.15%), general interest social media such as Facebook and Twitter (42.18%) and dedicated disease-specific social networks (41.30%).
Pharma is also looking to partner with patient advocacy groups (41.59%), deploy physician referral tools (30.68%), leverage electronic health records (28.61%) and work with patient recruitment companies (17.11%). Pharmacy outreach (13.27%) and email outreach (5.31%) were seen as becoming more important by far fewer, as was the use of brochures and flyers(5.31%).
Conclusion
Following comprehensive primary research among dozens of senior figures working on clinical trial design and delivery in Europe, this survey global survey confirms that there has been a clear movement towards a more patient-centric model: almost 75% of respondents agreed that “putting the patient at the heart of the clinical trial” had been named as the top priority for their organization going into 2015.
Social media and other online communities are seen as an important component of this new focus on patient centricity, often in conjunction with patient advocacy groups.
At the same time, innovative methodologies, especially around collection and use of data, are set to transform the clinical trials process. The leaders have already implemented many of these new methodologies. Adaptive trial design, real-world data integration and risk-based monitoring strategies were the three initiatives deemed to have the most potential to transform the trial process.
In many way’s, these are uncharted waters and it won’t always be plain sailing. However, the fallout from the storm that disrupted pharma’s traditional operating model looks set to deliver a whole new era of patient-centricity.
Background to the research and methodology
The purpose of the survey was to find out where pharma companies and CROs are focusing their efforts, methodologies and investments in clinical trials. It followed initial research among a smaller sample of senior figures working on clinical trial design and delivery from across Europe. This had indicated there had been a significant shift towards patient centricity in clinical trials. More extensive globalresearch was designed to test that premise.
The organizations polled in the research were a self-selecting group that participated via an online survey: 47.49% were from pharma/biotech organizations; 12.98% from contract research organizations; 12.39% were consultants; 9.44% were from academia or engaged in academic research; and 7.96% described themselves as solution providers. The remainderwere drawn from the ranks of research sites, technology partners, medical device companies, charities, clinical trial support organizations, the wider healthcare sector, standards organizations, nutrition, the media and a range of government, advocacy and trade organizations.
Respondents were typically senior clinicians, academics or research-focused individuals within an organization – with roles such as clinical operations director, senior clinical scientist, chief medical science officer – as wellas patient advocates, CEOs, executives from the commercial side of the pharma business and strategists.
Respondents were asked 10 questions (beyond their contact details, type of organization they represented, and their role) as part of the survey entitled “Patient Centricity, Technology and Data in Clinical Trials”. There were a total of 342 respondents.
Download the survey results here.