ROI for Patient-Centric Drug Development

By now there are few individuals remaining in the clinical research enterprise who are not familiar with the concept of patient-centric drug development and the many tactics and initiatives to support it. The mission is to engage patients and the healthcare community as partners and to engender in them a sense of connection and ownership in the success of new medical treatments. There are four core principles that frame the growing arsenal of patient centric initiatives:

Relevance: Patient-centric research targets unmet medical needs identified in collaboration with, and based on input from patients and their healthcare support network.

Pragmatism: Patient-centric research agendas and clinical trial designs recognize and accommodate real-life patient and healthcare community needs and experiences.

Feasibility: Patient-centric clinical trials minimize the burden of study participation and are supported by initiatives that improve convenience.

Interactivity/Participation: Patient-centric research planning and execution amplifies and responds to the patient’s voice, receives support from the patient community, and gives the patient community an opportunity to be actively involved and respected partners throughout the research process.

Kenneth A. Getz

Through adherence to these core principles, patient-centric drug development strives to minimize non-critical research activity and support the sharing of precompetitive information and drug development risk among a broader community of external partners (e.g., academic and basic research groups, co-development sponsors, development operations alliances, and patient advocacy groups). 

Clinical research professionals look to conduct not only great science, but also more feasible clinical trials that enhance study volunteer participation experiences and reduce the burden of participation. In this approach, contract research organizations (CROs), investigative sites, healthcare providers, and payers each play important roles as R&D partners and supporters helping to ensure that the four core principles are upheld.

During the past 24 months, a large and growing number of organizations have turned their attention to planning and piloting initiatives that touch all areas of the drug development continuum of activity. Regulatory agencies and quasi-regulatory groups such as the Clinical Trials Transformation Initiative (CTTI) and the Patient-Centered Outcomes Research Institute (PCORI) have been involved in soliciting patient input to define meaningful study endpoints and in establishing broad guidelines for patient engagement respectively. 

A growing number of pharmaceutical and biotechnology companies are beginning to solicit patient input into specific study designs through the use of patient advisory boards, one-on-one interviews, crowdsourcing, and social media. Sponsors and CROs are evaluating and piloting approaches designed to make study participation more convenient, including the use of telemedicine, home nursing networks, direct-to-patient platforms, and the use of wearable technologies. Sponsors are also routinely disseminating lay language non-technical clinical trial results summaries to volunteers at the completion of clinical studies.

Possible ROI measures

As clinical teams begin implementing patient-centric initiatives across multiple studies, it will be beneficial to centrally monitor and coordinate activity. Doing so will promote greater awareness and coordination within the organization and will better assist in communicating lessons learned from earlier implementations and from peer companies. This central function or mechanism, with the assistance of internal patient-centricity champions, can also play a key role in deriving consensus metrics to evaluate the impact of various initiatives on organizational and study-level processes and performance.

Return on investment (ROI) expectations need to take a reasonably long-term view. Clinical teams and their collaborative partners need time to gain experience with patient-centric initiatives, learn from mistakes, and continuously refine their use. As such, benchmark measures from multiple representative studies should be gathered two to three years pre- and post-implementation of initiatives. Stakeholders have been using a number of key implementation and ROI metrics to measure three broad areas: reach; patient/study volunteer feedback; and performance.

Reach measures

Reach measures are typically quantitative. Their primary purpose is to assess the extent of adoption and usage within organizations and the number of patients and study volunteers who have participated in a given initiative. Internal reach measures include:

• The total number of initiatives being piloted or implemented within a particular time frame.

• The number of studies for which a specific initiative has been piloted or implemented.

• The total number of initiatives in planning stages within a particular time frame.

• The total number of studies for which a specific initiative is planned within a particular time frame.

• The total number of studies for which all patient centric initiatives have been completed within a particular time frame.

• The total number of studies for which a specific initiative has been completed within a particular time frame.

External reach measures include:

• The total number of patients/study volunteers reached by all patient-centric initiatives within a particular time frame.

• The total number of patients/study volunteers reached by a specific initiative across all studies in which it was implemented within a particular time frame.

• The total number of patients/study volunteers reached by each specific initiative in a given study.

Patient/study volunteer feedback

Primarily qualitative in nature, several organizations have added a few quantitative feedback measures into the mix. Their primary purposes are to assess subjective reports of satisfaction; sense of involvement in a given study or in association with a specific initiative; and the perceived relevance of specific clinical trials. Quantitative measures have sought to demonstrate how well patient/study volunteer participation is facilitating change. Patient/study volunteer feedback measures include:

• Ratings (e.g., "Very, "Somewhat," "Not Very," "Not at All") of overall satisfaction among participants in a particular study.

• Aggregate ratings (e.g., "Very," "Somewhat," "Not Very," "Not at All") of overall volunteer satisfaction across multiple studies in which patient-centric initiatives have been implemented.

• Comparison of pre- and post ratings of volunteer satisfaction between multiple studies that did, and did not, implement patient-centric initiatives.

• Ratings (e.g., "Very," "Somewhat," "Not Very," "Not at All") of the impact that specific patient centric initiatives had on study volunteer attitudes about aspects of the participation process (e.g., ease of understanding the informed consent form; convenience of study visits and procedures) compared with those studies that have not implemented initiatives.

• Ratings of perceived relevance or importance of studies that have, and have not solicited patient input into study design, to understanding disease conditions and addressing unmet medical needs.

• Total number of protocol design and schedule of assessment changes that have been made as a result of patient/study volunteer feedback.

• Total number of changes that have been made to clinical trial communications as a result of patient/study volunteer feedback.

Performance

Performance measures are quantitative and they largely compare studies that do, and do not, include use of patient-centric initiatives. Many organizations are using consensus metric definitions developed and compiled by the Metrics Champion Consortium (www.metricschampion.org). The primary objectives of these measures are to gather hard outcomes data demonstrating improvements in feasibility and convenience. Performance metrics include:

• Screen failure rates.

• Number of procedures per visit.

• Number of protocol amendments.

• Number of missed visits.

• Number of study volunteers who drop out prematurely for reasons other than. adverse/serious adverse reactions.

• Number of study volunteers completing participation as a percentage of those who enrolled.

Areas of resistance

Some professionals are already discounting patient centricity as simply a "buzz-worthy" label for practices that certain professionals have long embraced. There is some basis for this view. However, the major difference is that this is the first time where broad consensus has been reached among stakeholders throughout the clinical research enterprise and where there is widely shared motivation to change.

Some have raised concern that the patient-centricity movement will result in clinical research professionals incrementally adding new practices and solutions onto already overburdened and demanding scientific and operating processes. In order for patient centricity to succeed, clinical research professionals will need to challenge, rethink, and redo certain conventional processes using the four-core principles as a guide. 

Critics fear that the patient-centricity movement is inviting too many cooks. Some professionals are reluctant to invite yet another voice to the table for fear that patients, healthcare payers, and providers will be hard to accommodate and will introduce more complexity. Others hold the traditional view that physicians and scientists know what is best for their patients and study subjects. Sponsors and CROs will need to set expectations among all stakeholders that all input is valued but that scientific integrity and safe and ethical principles must be met. Sponsors and CROs will also need to establish new processes that enable them to solicit and prioritize ideas and input.

A number of clinical research professionals fear that patient centricity is pushing them to discard traditional practices, including the use of blinded, randomized controlled clinical trials. This fear is particularly pronounced in the absence of regulatory guidance on acceptable and encouraged patient-centric practices. If patient centricity is to successfully transform R&D, it must be widely adopted and integrated into all aspects of R&D while preserving the strengths and value of traditional approaches.

Several stakeholders have raised concerns that involved and engaged patients will want to see their clinical trials succeed and, as a result, these patients will bias the study findings. The gravitas of conducting medical research to treat serious and life-threatening illnesses, and deep study volunteer motivation to search for real answers about disease and how to treat it are too strong to allow this effect to occur. Patients and their healthcare support network want accessible, safe, and effective treatments for themselves and future patients. They will not compromise on scientific integrity. They will demand the highest quality research results based on clinical and patient-reported outcomes.

It is still in the early days and there is limited data demonstrating the extent and impact of patient-centric initiatives. As more is learned, select initiatives will take hold and many will not. At a minimum, the patient-centricity movement is inspiring the drug development enterprise to challenge and transform the traditional drug development paradigm by putting the patient at its core.

Kenneth A. Getz, MBA, is the Director of Sponsored Research at the Tufts CSDD and Chairman of CISCRP, both in Boston, MA, e-mail: kenneth.getz@tufts.edu.