Clinical trial success through patient engagement
The world of clinical development is changing. The rapid pace of scientific & technological advances, the rise of personalised medicine and patient engagement, escalating R&D costs, and increased scrutiny from regulators and payers looking to reign in healthcare costs are all driving disruption. Added to this, the patient is more informed and connected technologically and socially than ever before. No longer happy to be subservient in their relationship with healthcare, patients are asking for recognition, respect and consultation on what matters to them. Whether industry is ready, willing or not, patient advocates are looking for a seat at the table with funders, regulators and the therapeutics industry, some of which are now welcoming this extra input to help guide their processes and decision-making.
Patient-centricity, patient engagement, patient involvement, patient experience – these topics are increasingly discussed at meetings and conferences. But, there is still confusion about what it all means, why and how to go about it, and its return on investment.
In 2016, Astra Zenecaa worked with patients to create a definition (BOX 1) of patient centricity and principles (BOX 2) to help inform how they and other organisations could think about the value and purpose of patient engagement throughout the therapeutics lifecycle.
Box 1: Patient centricity:
Putting the patient first in an open and sustained engagement of the patient to respectfully and compassionately achieve the best experience and outcome for the person and their family.
Box 2: Principles of importance to patients and caregivers (in no particular order):
- The company helps make sure that the people who need medicines have access to them.
- The company communicates transparent and unbiased information on your disease, treatment options and available resources with care and compassion.
- The company provides easy-to-undersand and convenient information in plain language because they understand that ‘words matter’.
- The company helps you gain affordable access to their medications.
- The company equips you to make informed choices about your healthcare and your treatment options.
- The company listens and responds to your feedback with respect humility.
- The company partners with you to innovate and measure impact and outcomes that are important to you.
- The company provides access to support programmes and resources to help you improve quality of life.
- Everything the company does begins with an understanding of your needs and experiences.
- The company helps empower you to help other patients and their families.
Patient engagement activities might seem a luxury in a resource-constrained environment. But so is spending time and money developing products not needed or unlikely to be approved/paid for, or protocols that don’t interest patients and clinicians, can’t recruit or retain participants, and don’t deliver the data regulators and payers are increasingly looking for. In the future, those companies that do not engage and partner with their target population early will be at a disadvantage in a world where regulators, payers and investors will be looking for evidence of the need for and value of an innovation to the patient population.
Arguing that something as touchy-feely as patient engagement will provide a return on investment can be challenging. In 2017, the Clinical Trials Transformation Initiative (CTTI)’s Patient Groups and Clinical Trials Project set out to make it easier, publishing a quantitative method for calculating its financial value. They found that engagement activities with the potential to avoid protocol amendments and/or improve enrollment, adherence, and retention may add considerable financial value, worth for example hundreds of millions of dollars, and 1-2 years off the development lifecycle in the pre-phase 2 example they worked through.
So to break it down, here are our top 3 reasons you might want to consider committing to patient engagement early in your development lifecycle:
1. Obtain fundeding/investment and working smart
In a crowded market of risky ideas, how do you make your potential medical innovation rise above others in the eyes of investors, funders and potential partners? Being able to demonstrate you understand your target market and have partnered with them to refine and develop your innovation will help reduce investor/partner concerns about whether the product has a market, and is likely to gain regulatory and payer acceptance. It will also ensure your own organisation can be smart about which assets deserve the limited financial, human and time resources it has available.
2. Recruitment & Retention
Recruitment delays hit 80% of projects, resulting in cash-burn, protocol amendments, and in some cases, trial failure. Proactively identifying and addressing potential recruitment and retention issues up front will help de-risk your project and increase its chance of completing. Engaging your target patients during the design phase can help make sure the trial addresses the questions that matter to patients (thereby increasing their interest to participate) and identify practical issues that may limit recruitment, such as eligibility, recruitment methods, visit burden, documentation issues, language used, participant expenses, transport and site/trial access issues, and retention strategies for long and/or burdensome trials. It can improve the credibility of your trials with potential participants, to be able to share that patients were involved in its development.
3. Product approval, reimbursement and use
Patients present data in way that provides life context to scientific results. As such, they can be very powerful allies in presenting the case for approval and reimbursement of a medical innovation, as well as effective disseminators of information amongst other patients and their clinicians for it use. You will create far more invested and vocal patient allies and advocates if you involve and partner with them early to develop solutions you hope will help them.
Southern Star Research is committed to supporting the long term success of its clients and their products, as a path to helping patients live longer, better lives. Talk to us today about how we can help you build long-term, trusted relationships with the patients you aim to serve, and achieve the product success you want.
References (Click on the links below):
b. Levitan B, Getz K et al. Assessing the Financial Value of Patient Engagement – A Quantitative Approach from CTTI’s Patient Groups and Clinical Trials Project. Ther Innov Regul Sci 2018; 52(2): 220–229.