White Paper

Placing The Patient First In Clinical Trials

Placing The Patient First In Clinical Trials

The Industry Imperative
The biopharmaceutical industry continues to seek new ways to make clinical trials more efficient and improve success rates. We've progressed with innovations such as quality by design (QbD), risk-based monitoring (RBM) and adaptive clinical trials. What's still lacking in the standard clinical trial model is putting the patient first.

Now the industry must become more patient-centric to survive. A recent survey of 427 clinical trial professionals reports that for 75% of companies, putting the patient at the heart of the clinical trial is the top priority for their organization moving forward. Their greatest challenges include speed of enrollment (42%), narrowing patient eligibility criteria (23%) and patient retention (20%).1

With study protocols being more demanding than ever, there is a growing burden on patients in terms of their time and comfort level. While trial time, spend and complexity has grown, patient needs are not being met. For too long, patients have had to adapt their lives to the increasingly rigorous requirements of the clinical trial. Patricia Larrabee, CEO of Rochester Clinical Research and keynote speaker at a recent event, said that clinical investigators need to accommodate patient needs and understand that patients are volunteers who can choose to walk away at any time, and they may be irreplaceable.2

Today biopharmaceutical companies are transforming the focus of clinical trials, not viewing patients as subjects for generating data, but as informed collaborators whose participation is core to the overall success of trials.3 The industry is increasingly adopting a patient-centric drug development model, focusing more on patients and addressing their needs and convenience.

This article discusses ways to adopt patient-centered drug development, changing the corporate culture and trial design, understanding patient needs and incorporating them into trials, and leveraging technology to conduct remote patient-centric clinical trials.

Adopting a Patient-centered Corporate Culture
Adopting a patient-centered clinical trial design involves embedding a patient focus into the organization's culture. This may require a new business model, which calls for defining the purpose of a patient focus and expected behaviors, as well as fully committed patient-centered leadership, hiring people with shared values, setting up reinforcing systems that are patient-centric by design, and measuring the companywide cultural change.4

Shaping a new culture by focusing on the patient can contribute to satisfied patients, improved patient outcomes and increased profitability. Direction and commitment for a cultural change must come from the top, with purpose-driven leadership. One company, UCB Pharma, restructured its organization, introducing new executive positions to represent each patient value unit in major therapeutic areas and trimming nonpatientfocused sections. Restructuring allowed employees to delve deeper into the specific needs of patients, speaking directly to them to understand their holistic experience. Processes should be designed with the right amount of human effort and technology, while enlisting patients in self-providing care.4

As for measuring results, employee retention, relationships with customers, referrals, and productivity are crucial metrics that can be measured. Key performance indicators (KPIs) — such as patient adherence, feedback from physicians, the patient experience and outcomes — are other significant metrics to measure patient-centric outcomes.

KPI data can be analyzed and measured using TIBCO Spotfire®, the life science industry's de facto end user interface of choice for the strategic assessment of clinical data. The foundational use of Spotfire as the common visual interface across life science organizations remains strong, based on its welldesigned, highly intuitive user interface and ease in creating active information portals.5

Leading Signs of Patient-Controlled Clinical Trials6
The following are the top ten signs that next-generation patient-centric clinical trials are going mainstream:

  1. Most patients can undergo entire clinical trials from their home.
  2. Digital health is mainstreaming. Tools such as smart phones, smart sensors, and wearables eliminate data entry for patients. Mobile platforms collect information about physical activity, sleep, heart rate, temperature, and more to submit to the trial investigator..
  3. Patient electronic consent will soon be the norm.
  4. Patient recruitment will go online in the next five years
  5. Data privacy rules are being recalibrated.
  6. Social media angst is gradually being overcome.
  7. Research integrity remains uncompromised. We will need software systems that understand the integrity of the process so trials remain objective, unbiased and uncompromised.
  8. Regulatory support is challenging, but agencies are showing signs of supporting change.
  9. Favorable trial economics will overcome most objections.
  10. Behavioral analytics will be included in clinical trials for better patient engagement, faster recruitment, increased compliance and reduced patient attrition.

What Is a Patient-Centered Clinical Trial?
Today patients are typically well-informed about their condition and available treatments. They are technology-driven, with easy access to information about treatment options through the Internet and social media. A recent survey showed that 72% of U.S. Internet users look online for health information, and 85% of online Europeans use the Internet for health and prescription drug information.7 Patients are also more engaged with their healthcare providers, and communicate with other patients about disease experiences and treatments.

The pharma industry is leveraging the change, utilizing technology to better understand and communicate with patients. Clinical trial teams are realizing the benefits that come from involving the patient in the clinical trial development process, including better written protocols, higher recruitment and retention rates, and enabling protocol updating.

Three areas of focus for patient-centric clinical research are patient retention and engagement, improved data quality and collection methods, and enhancing the human aspect of the clinical research experience. This means seeing patients as people, not subjects, and considering their daily living in trial design.

Understanding Patients via Social Listening8
Patient-centric clinical trials begin by understanding patients. One good source of patient information is social media platforms, where patients share valuable information. By tapping these platforms, investigators can use social listening to access newly discovered data that can lead to effective and efficient patient recruitment and retention campaigns. In addition to using mobile health technologies and social networks, patients and investigators use remote trial components such as patient diaries, ePROs, eConsent, sensors, and RBM.

Here are some key benefits of using social media to learn about patients:

  • Learn about the lifestyle and condition of patients, which helps with study protocol development and recruiting.
  • Understand what the competition is doing and how people perceive a competitive approved drug.
  • Online conversations can lead to key market countries selection for study deployment. Look for a high volume of conversation and identify areas with a positive outlook on clinical research.
  • Develop key messaging based on patient insights.
  • Develop a media strategy for recruitment.
  • Monitor study activity. Continue listening to understand what patients say about their experience and how this might impact retention.
  • Identify and report adverse events.

Case Study: Using Social Media to Drive Patient Centricity
UCB, a multinational biopharmaceutical manufacturing company, sought a way to use social media responsibly to engage directly with patients. UCB focuses on products to address immunology-related disorders and central nervous system disorders, such as epilepsy.

In 2006, UCB launched an epilepsy advocacy website, enabling people with epilepsy to share their stories online. CB recognized that people with epilepsy don't know enough about their condition, and are generally isolated physically and socially. UCB's CEO and senior management team felt this situation could be addressed with social and digital channels, enabling the company to meet its mission of being patient-centric.

In 2012, the CEO hired two social media experts and soon the Epilepsy Advocate Facebook page was launched. They formed a cross-functional team and educated members on social media, built a standard operating process, and managed all content and interactions with patients and advocates on Facebook. They also enrolled the medical, legal and regulatory teams as advocates. In 2014, the Facebook page hosted its first live chat where patients asked questions of advocates in real time. The chat engaged more than 2,000 Facebook users and was very successful. The team is extending the Epilepsy Advocate to new channels, such as mobile.

Some keys to the success of UCB's social media efforts are CEO commitment to patient centricity, a willingness to allow the program to grow without considering ROI, involving everyone from the beginning, and enlightening business partners.

The Role of Technology7
Remote data capture is enabling centralized control and monitoring of clinical trials. By leveraging technology, centrally controlled remote clinical trials are possible for many trials, enabling patients to remain in the comfort of their homes, rather than regularly traveling to a trial site, with less disruption of their lifestyle. A remote patient-centric trial involves recruitment through disease-specific social network platforms, electronic health records and payer databases, and digital tools enhancing physician referrals.

Other digital components of a remote trial include electronic informed consent, electronic data capture (EDC) at the source — electronic patient-reported outcome (ePRO), bring your own device (BYOD), and sensors. ePROs and eDiaries offer enhanced data integrity, accuracy and quality. However, they involve device leasing costs and patients need to carry another device. BYOD enables patients to use their own smart phone with an app for ePRO. Wearables and sensors, such as activity and sleep monitors, continuously report clinical data. Wearable include devices such as smart phones and watches, fitness trackers and Bluetooth technologies.

RBM monitors patient activities remotely, performing risk assessment and identifying critical data early. Risks can be remotely measured for study conduct, data integrity, patient protection, and treatment adherence. Telemedicine is also available for remote consultation with an investigator.

Most therapeutic indications are suitable for remote trials. They are especially beneficial for chronic diseases such as COPD, diabetes and asthma, and rare and orphan diseases where patients are scattered over large geographies. They are more challenging for high-burden diseases (oncology, infectious diseases) and perceived high-risk conditions for patient safety (obstetrics, pediatrics).

Case Study: Speeding enrollment and boosting retention with a patientcentric clinical trial9
Science 37 was formed in 2014 to bring disruptive change to the inefficient clinical trial process by shifting to a patient-centric approach that could shorten patient enrollment timelines and boost retention. The startup company recently won an industry Best Practices award, announced at the 2016 SCOPE Summit, for its metasite model, a virtual site that speeds enrollment by allowing patients to participate in trials remotely through the company's Network Oriented Research Assistant (NORA) technology platform.

Science 37 physicians and researchers used the model in a Phase III study for a rare skin condition, which was an intense challenge, since less than 1/100 of 1% of the global population has the condition. The remote trial leveraged technology to remove the geographic barriers, unlock access, and improve the patient experience.

The NORA technology functions as an app accessible on patients' mobile phones. The technology enables communication with the research team, conducted through secure video chats and messaging. Patients can log into their study and have direct access to the investigator and study coordinator. The patient touches the photo of the coordinator, for example, which results in a call to that coordinator's cell phone 24 hours a day. Home-based visits are conducted by mobile nurses who use iPads to enter source data into a cloud-based database. Coordinators then enter those data remotely into an electronic data capture system.

In the first six months of the trial, the metasite was responsible for enrolling 30% of all enrolled patients, translating into an enrollment speed that was more than 20 times faster than the projected per site enrollment rate for the study. The study is actively recruiting at 60 global sites and hopes to achieve an enrollment of 124 patients, averaging two patients per site. So far, the company has achieved a 100% retention rate and approximately 200 patients have participated in studies using this approach.

Looking Ahead
Patient centricity is essential to improving the quality of development data and must become the standard method of conducting business and clinical trials in the future. The industry is undergoing a transformation, moving toward patient-centric drug development with increased remote clinical trials and a change in corporate culture. As with most changes in the industry, moving toward patient-centric drug development for pharma/biopharmaceutical companies will be gradual.

References

  1. Patient-centric clinical trials becoming a higher priority for biopharaceutical, CRO management. January 12, 2015. CenterWatch News Online. Accessible at: http://www.centerwatch.com/news-online/2015/01/12/patient-centric-clinical-trials-becoming-a-higher-priority-for-biopharmaceutical-cro-management/
  2. Larrabee P. Driving Ideas That Focus on Patient Centric Clinical Research. Fisher Clinical Services. Accessible at: http://www.fisherclinicalservices.com/driving-ideas-focus-patient-centric-clinical-research/
  3. Sharma NS. Patient centric approach for clinical trials: Current trend and new opportunities. Perspect Clin Res. 2015 July-Sep; 6(3);134-138.
  4. Davies N. Patient-Centred Culture by Design. eyeforpharma. Accessible at: https://www.ashfieldhealthcare.com/wp-content/uploads/2016/01/Patient-Centred-Culture-WhitePaper.pdf
  5. Louie AS. Familiar and User-Friendly: TIBCO Spotfire Owns the Front End in the Life Sciences. IDC Health Insights. Accessible at: http://spotfire.tibco.com/assets/blt8885136c56b51df6/idc.pdf
  6. Iyer G. Top 10 signs that next generation patient-controlled clinical trials are going mainsream. Accessible at: https://www.linkedin.com/pulse/top-10-signs-patient-centric-clinical-trials-going-mainstream-iyer
  7. Kadhim H. Remote Trials to Drive Remote Patient Centricity. SCOPE Summit 2016. Feb. 24, 2016.
  8. Understanding Patients Through Social Listening. SCOPE Summit 2016 Feb. 23, 2016.
  9. Neuer A. Technology for Patient-Centric Clinical Trials Wins for Science 37. 2016. Accessible at: http://www.clinicalinformaticsnews.com/2016/3/25/technology-for-patient-centric-clinical-trials-wins-Science-37.aspx