From The Editor | May 10, 2016

Johnson & Johnson Puts Focus On Patient Voice And Compassionate Use In Trials

Ed Miseta

By Ed Miseta, Chief Editor, Clinical Leader
Follow Me On Twitter @EdClinical

Johnson & Johnson Puts Focus On Patient Voice And Compassionate Use In Trials

While Big Pharma discusses the importance of patient centricity and putting patients at the center of all drug discovery efforts, many still wonder if a lot of the buzz isn’t simply lip service simply masking business as usual. After all, we can spend hours discussing the importance of patients. But at the end of the day, if the patient experience is unchanged, it’s all for naught.

Janssen is one company putting programs in place to make sure the patient voice is heard at all levels of the drug development process. Beverly Harrison, Senior Director, Patient Support, Janssen Pharmaceutical Companies of Johnson & Johnson has played a pivotal role in the process. One area in which her company is leading change in the industry is in the area of compassionate use. Last year Johnson & Johnson announced a pilot program that outsourced the compassionate use decision-making process to an independent review panel convened by the New York University (NYU) School of Medicine.  

“As a leader in the research and development of promising new treatments, Johnson & Johnson recognizes that patients who have not responded to currently available treatment options or who are not eligible for clinical trials will have a heightened and emotionally-charged interest in securing early access to companies’ investigational medications,” she says. “At Janssen, we strive to provide access to investigational medicines in response to requests from patients with serious, life-threatening diseases. We always seek to advance our investigational medicines through the required clinical trials approval processes to obtain regulatory approvals as quickly as possible. This is to ensure all appropriate patients can receive the medicine when available for marketing. But we also recognize that some seriously ill patients may seek compassionate use when they are unable to enroll in clinical trials or expanded access programs.”

When evaluating individual requests for compassionate use, Janssen strives to follow three important ethical principles:

  • That all patient requests are treated fairly and equally
  • That the company does not undermine its ability to conduct thorough scientific studies to understand the potential risks and benefits of any investigational medication. These studies provide the information needed to seek approval from government health authorities and bring new medicines to all patients who need them.
  • That they are not putting patients at risk of unnecessary harm.

The first-of-its-kind compassionate use trial program was launched in May 2015 to review requests made to Janssen. This innovative program was established to address the complex and emotional challenges associated with compassionate access to medicines through a transparent, ethical and consistent process. As part of this new collaboration, the NYU School of Medicine established and leads the Compassionate Use Advisory Committee (CompAC), a group of internationally recognized medical experts, bioethicists and patient representatives to independently review compassionate use requests.

A JAMA ‘Viewpoint’ piece published online on February 11th conveys our experience with this new approach and the successful use of the Compassionate Use Advisory Committee, or CompAC,” states Harrison. “This model offers the health care community and the public a new framework to consider in addressing the complex and often emotional challenges associated with compassionate access.”

Clinical Trials And Data Transparency

Many companies listen to the voice of patients when designing trials, but care must still be taken to make sure other voices do not take priority. A program in place at Johnson & Johnson seeks to more effectively bring the patient voice into the trial design process. The program, called “Patient Voice in Trial Design,” seeks to make the patient voice on any trial just as loud and significant as the many other voices that have always helped guide trial design, such as physicians, researchers, and key opinion leaders.

Another program is iSTEP (integrated Smart Trial & Engagement Platform). This initiative builds on the foundation of Janssen eMeds project, and aims to develop a platform that uses smart technologies to transform how clinical supplies are managed, with the goal of increasing operational efficiency as well as patient engagement and compliance. The key modules of iSTEP/eMeds platform include eKit, eLabel, eCommunications and eAdherence. CenterWatch published an article in November 2013 highlighting Janssen's efforts, via the eMeds project, toward a future with significantly improved patient medication compliance, faster delivery of dosing changes, better verification, and ease of use.

Harrison states, “In order to create transparency around Janssen clinical trials, we set a new standard for data sharing in 2014 through a unique agreement with the Yale Open Access (YODA) Project to provide increased access to our pharmaceutical clinical trial data with the following principles: (1) safeguarding the privacy of patients (2) respecting the integrity of national regulatory systems (3) maintaining incentives for investment in biomedical research.”

YODA serves as an independent body, comprised of pharmaceutical and medical device experts. It reviews research proposals and makes final decisions regarding all requests from outside researchers who are not engaged in a research collaboration with Johnson & Johnson operating companies, but would like to independently access clinical trial data from them, including clinical study reports (CSRs) and de-identified participant level data. Through this agreement, the Johnson & Johnson commits to sharing clinical trial data, including participant level data, from its portfolio of products with qualified researchers, physicians and investigators. 

In 2015, this commitment was extended to include medical device clinical trial data. Johnson & Johnson is the first integrated healthcare company to work through an independent third party to review all requests and approve access to clinical data across its portfolio of products.

Engage Employees And CROs

No effort to better engage and support patients can be successful without the buy-in of all employees involved in the process. With an eye toward supporting its employees, Janssen has initiated efforts and programs to provide guidance around implementing patient centric efforts. One example is the deployment of a new guideline, or “playbook,” to guide all compound teams through capturing and incorporating the patient journey into drug development at its earliest stages.

“In doing so, we are improving the clinical trial experience for those who selflessly provide their time and energy to participating in a Janssen clinical trial from end to end,” notes Harrison. “Further, we have established a global infrastructure to fluidly interface with advocacy organizations to ensure accountability to our patient partners.”

With most Big Pharma companies, studies are not always conducted in-house. When a clinical trial is outsourced to a CRO, that CRO facilitates a lot of the interaction with sites and patients. When a CRO is involved, they can also support the effort for greater patient centricity, often through protocol design and bringing patient perspectives to the table.    

Johnson & Johnson has partnered with all types of CROs for this purpose, from the small, specialty-service providers to large, full-service CROs. More recently the company has also partnered with a small, niche CRO that specializes in market research, including a “product design” approach that involves testing prototype patient-facing tools in a simulated trial setting, as well as anonymous “exit surveys” to assess trial participant satisfaction. Harrison notes that via a CRO, her company conducted a comprehensive market-research study where it interviewed numerous former trial participants living with the same conditions for which novel therapies were being developed.   

“Our large, full-service CRO partners are critical in helping us deploy patient-centric programs,” she adds. “A recent success story is our “Patient Data Access” initiative, whereby we offer patients the ability to access their routine lab results from the trial via a secure patient portal. The first trial within which this service was offered was conducted by a major CRO. Being quite a novel concept, the CRO site management team worked hard to explain the program and its benefits to each site individually. The Patient Data Access initiative paves the way for additional engagement activities, such as incorporating the voice of former trial participants in future research endeavors.”