Patient-Centered Clinical Trials USA 2015

Oct 19, 2015 - Oct 20, 2015, Philadelphia

Boost Clinical Trial Productivity – Collaborate with Patients at the Heart of your Research.

Establishing a Culture of Safety

We investigate the crucial link between a culture of safety and patient-centricity.

Sonal Singh, M.D., M.P.H, Assistant Professor of Medicine, Health Policy and Management, and International Health

In the past, data falsification, unpopular pricing policies, and safety scares have damaged the reputations of some large drug development companies and the pharmaceutical industry in general. Clearly, a global circle of trust needs to be established between pharmaceutical companies, government health industry regulators, researchers, and most importantly, patients.

In order to achieve this, the pharmaceutical industry is embracing the concept of patient-centricity. This means that the industry intends to meld Corporate Social Responsibility (CSR) programs with patient-centred outcomes research. If this aim is to succeed, however, it is vital to inculcate a culture of safety. While companies need to prove to investors that their bottom line is sound, they also have a duty to the public to provide safe and effective medication. Various CSR programs led by pharmaceutical companies are tackling this dichotomy by supplying drugs at reduced cost, as well as providing health education to needy populations.

CSR programs for all

Although the concept of CSR lacks a universally accepted definition, the motivation behind its burgeoning popularity in the industry has been driven by higher and sometimes unrealistic expectations from both governments and society. In order to implement CSR programs that are both beneficial and appropriate, companies are looking at patient-centricity to supply insight about the communities they serve.

Novo Nordisk, for example, has made a commitment to spearhead the fight against diabetes with their Cities Changing Diabetes (CCD) initiative, the aim of which is to inspire municipalities worldwide to collaborate. Cities can then share their findings and work together to determine the particular social and cultural risk factors that are contributing to the huge increases in obesity and concomitant Type 2 diabetes problems in their cities, as well as through public-private collaboration, and work on diabetes prevention and control.

By recruiting residents who speak the local dialect, CSR programs can ensure that patients are aware of the safety factors involved in participating in clinical trials as well as how to manage their treatments effectively. Mobile technology is increasingly being used to assist patients by reminding them which pills to take and when to take them. An example of this is“Life-txt,” where health professionals can send their patients regular messages such as, “Take two of the red pills (name of medication) half an hour before breakfast,” or, “Take 1 white pill (name of pill) after supper. Do not combine with alcohol and do not drive after taking this pill.” Research has indicated that around 80% of what a health professional tells a patient is forgotten within an hour or so of leaving the clinic, so such strategies appear a vital move forwards in creating a safety culture.

Reducing costs

Certain drug trials which have garnered significant funding, are carried out at the expense of others, causing people to question the rationale behind such choices. An example of this is studies approved for diabetes mellitus 1 at the expense of diabetes mellitus 2. Globally, there are far more people who need drugs and treatments to help with type 2 diabetes. The cost of certain drugs has previously been criticized. There have also been concerns about adverse side-effects being discovered after trials have been successfully concluded and the drug released for sale.

Sonal Singh and Yoon Loke, in their paper on ‘Drug safety assessment in clinical trials: methodological challenges and opportunities,’ observe that, “Clinical trials are carried out to establish the therapeutic benefit for the patient in order for the drug or treatment to receive the required regulatory approval. Because clinical trials are time-bound and costly, this can pose a challenge to establishing long time cause and effect and safety signals.”

Focusing on the patient

Anne Beal, M.D. M.P.H., was Deputy Executive Director at the Patient Centered Outcomes Research Institute (PCORI), the largest institute of its kind in the US. Her task was to ensure the priorities of patients were reflected in the research portfolio for the institute. She has since joined Sanofi as Chief Patient Officer, a newly created position, to ensure the perspectives of patients and caregivers are considered right through the process of R&D to the on-market availability of Sanofi’s healthcare offerings.

Anne Beal, Chief Patient Officer, Sanofi

Beal’s presentation at Barcelona 2015 focused on patient-centric strategy. The strategy involves three core principles: the first comprises using patient input to understand their needs in order to provide appropriate solutions; the second, to engage with patients and stakeholders such as caregivers and health professionals to ensure enhancement of quality of life and improved outcomes for patients, and third, to involve the pharmaceutical company’s employees in creating a patient-centric culture and engaging community involvement.

Embedding a culture of safety

Absence of evidence is not evidence of safety. We need to design studies that LOOK for safety concerns and report them".

Sonal Singh, M.D., M.P.H, Assistant Professor of Medicine, Health Policy and Management, and International Health, sees safety culture being embedded into patient-centricity through, “incorporating patient input into a shared decision-making process.”

Asked about safety concerns that have arisen from CSR programs, Singh said, “In various cases they may prioritize physician input and institutional priorities over patient participation in decisions. Saving money and improving on quality metrics is not necessarily aligned with patient wishes.”

Singh sees the way forward in re-establishing public trust through improved safety measures as being to,“Ensure public participation, not just nominal participation. There are well-designed methods for doing so.” Regarding safety measures during clinical trials, Singh mentions that, “Absence of evidence is not evidence of safety. We need to design studies that LOOK for safety concerns and report them.”

Adverse drug effects may vary from mildly symptomatic to life-threatening. Timing is important – the event may occur soon after taking the medication or years later when the medication may be associated with a long-term risk of cancer. Spontaneous reporting by patients should be encouraged. Rather than those that conduct the clinical trials determining what they should look for in randomized controlled trials, instead let patient-centricity drive the safety features by collecting patient feedback on any adverse effects they may have experienced, and then corroborating with the results from other patients to establish reliability.

“Randomized controlled trials are the principal means of establishing the efficacy of drugs. However, pre-marketing trials are limited in size and duration and exclude high-risk populations. They have limited statistical power to detect rare but potentially serious adverse events in real-world patients,” report Singh and Loke in their paper ‘Drug safety assessment in clinical trials: methodological challenges and opportunities.’

Linking CSR, patient-centricity and safety culture

Safety in the production facility is something all pharmaceutical companies take very seriously, but accidents do occur. It has happened that the wrong pills have been inserted into blister packs, leading to a costly recall when the error came to light. This type of safety lapse can occur if insufficient checks are in place. Imagine the devastating effects this could have on unwitting patients and consequently on a company’s reputation.  Aside from the above scenario, patients need to be aware of what medication has been prescribed, and that the correct medication is taken. This is where CSR programs involving education are useful in making patients aware and, to some extent, responsible for their own safety.

For more information on the presentation "New Approaches to Ensure Safety in Clinical Trials", see Patient Centric Clinical Trials Europe.

Patient-Centered Clinical Trials USA 2015

Oct 19, 2015 - Oct 20, 2015, Philadelphia

Boost Clinical Trial Productivity – Collaborate with Patients at the Heart of your Research.