Involve Patients In Product Development … From The Beginning

By Liisa Eisenlohr & Gurjit Singh Bansel

In the drive to bring new therapies to market, life sciences companies justifiably focus on efficacy and safety data as the foundation for gaining regulatory approval. Increasingly, however, regulatory bodies and the healthcare industry are recognizing the importance of input from patients throughout the development of medical products.

The narrow focus on clinical study endpoints that demonstrate superiority of one medical product over another as required by regulatory bodies may not be of primary importance to patients. Furthermore, companies developing medical products have historically relied on consultations with healthcare professional KOLs who provide input from a clinical perspective but in most cases do not cope with the disease or condition personally as patients do. As a result, life sciences companies — usually inadvertently — have overlooked or disregarded the real-world situations and experiences of intended patients.

Consequently, what probably seemed logical to clinicians and easy enough during clinical trials often leaves patients and caregivers fumbling, confused, and frustrated once a product is on the market. Imagine taking a large pill when it’s painful or difficult to swallow or attempting to press down and inhale at the same time to use your inhaler when you can barely breathe and your hands are shaking. Those are just a couple of examples of what pharmacists, nurses, and others close to patients see firsthand, as patients struggle to take full advantage of the medical innovations offered to them.

This “lack of practicality” conundrum offers a sizable — and seizable — opportunity for astute medical device, biotechnology, and pharmaceutical companies to factor patient-centricity into the equation and add “ease of use” as another differentiation point to their value proposition. Furthermore, those companies that step up sooner rather than later can get ahead of what’s already coming, as regulatory bodies take measures to ensure companies obtain input from patients throughout the product’s life cycle, and nonprofit patient advocacy organizations pursue their missions to guide and arm patients with resources and support.

REGULATORY TRENDS

The FDA, EMA, and the U.K.’s Medicines and Healthcare Products Regulatory Agency (MHRA) each have published guidance on incorporating the voice of the patient in the development of medical products. Although the current focus of regulatory bodies is primarily on drugs, patient engagement also is relevant for medical device manufacturers. So far, these measures are not legally binding, but they do point to a shifting landscape.

“Patients have direct experience in living with a disease,” EMA guidance states. “They have firsthand knowledge of the impact of the disease on their lives and on how they feel and function. They bring a unique and valuable perspective to drug development, one that cannot be provided by clinical, scientific, legal, and other experts. It is important for health authorities and for drug developers to incorporate the patient’s perspective, beginning early in drug development.”

In 2016, the FDA passed the 21st Century Cures Act, which built on initiatives to incorporate patient perspectives into the development of drugs, biological products, and devices. It also aimed to speed the development and review of novel medical products and to modernize clinical trial designs, including the use of real-world evidence and clinical outcome assessments. The goal of this and follow-on legislation is to understand how the product performs in patients with varying conditions of severity, comorbidities, ages, and other characteristics.

The FDA’s draft guidance of October 2019 on “Patient-Focused Drug Development: Methods to Identify What Is Important to Patients” provides manufacturers with methodologies for patient-focused drug development. These methodologies also can be applied to medical device development.

The FDA also has begun creating patient-focused drug development (PFDD) guidance to advance the use of systematic approaches to collect and use robust and meaningful patient and caregiver input to inform medical product development and regulatory decision making. From April 2013 through June 2021, the agency convened 30 PFDD meetings on specific disease areas that were attended by a cross section of stakeholders, including patients, advocates, researchers, drug developers, and healthcare professionals. The goal of these sessions was to listen to patients’ perspectives on their disease, symptoms, and treatment options. Through the meetings, the FDA learned that many patients want to be as active as possible in the work to develop and evaluate new treatments. The agency also actively participates in PFDD meetings led by patient advocacy organizations focusing on a broad variety of disease areas.

More recently, the EMA finalized its program in January 2021 after completing a pilot project to involve patients directly in the assessment of the benefits and risks of medicines in its Committee for Medicinal Products for Human Use (CHMP). Published in 2017, the initial report concluded patients should continue to be invited to oral explanations when their input could be valuable to the assessment of a new or on-market medicine under review. Since then, through the International Council for Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use (ICH), the EMA has published guidelines for key areas where incorporating the patient perspective could improve the quality, relevance, safety, and efficiency of drug development and inform regulatory decision making.

The guidance goals go beyond patient preference for the final product to include endpoints relevant to the broader patient pool. The preference of the method by which a treatment is measured can differ dramatically. As an example, R&D teams currently consider extended life expectancy as a crucial endpoint for oncology drugs. However, as shown in a recent review of 30 studies, cancer patients — particularly elderly ones — generally prioritize quality of life over length of life. Clinicians are, therefore, encouraged to consider each patient’s preferences regarding their health and the consequences of the treatment itself when determining the best treatment course.

CONSIDER PARTNERING WITH PATIENT ADVOCACY GROUPS

By rethinking their approach, manufacturers can help ensure that patients can more easily access treatments, for example, by obtaining input from patient advocacy organizations during the reimbursement negotiation process. In a recent high-profile case in the U.K. in which discussions took a wrong turn, price negotiations deadlocked between a pharma company with a life-saving therapy and the National Institute for Health and Care Excellence (NICE). A prominent patient advocacy group was persistent in raising the patient voice to the government and pivotal in securing a deal through which ultimately nationwide access was granted with no cap on patient numbers.

This illustrates why companies should consider ways to partner with patient advocacy organizations through all stages of product development. Of note, NICE and several health technology assessment authorities in Europe, including Germany’s Gemeinsamer Bundesausschuss (G-BA) and France’s National Authority for Health (HAS), are now actively seeking and leveraging patient input for reimbursement questions, particularly for high-value drugs such as cell and gene therapies.

THE IMPORTANCE OF MEDICAL AFFAIRS TEAMS

Patient advocacy organizations are nonprofit entities that educate, advocate for, and provide support services to patients and caregivers, including the establishment of best practices for improving overall patient experience and quality outcomes. Patient advocacy organizations are also increasingly partnering with the life sciences industry to help ensure patients are involved in the medical product development and commercialization process. In this capacity, advocacy organizations help companies gain patient perspectives throughout the product life cycle, spanning clinical trials, regulatory approval, adherence strategy design, creation of support materials and programs, public relations, and community outreach efforts, as well as collecting real-world data.

Medical affairs teams are the ideal function within a life sciences company to interact with patients, caregivers, and patient advocacy organizations and consistently elevate the patient voice throughout the medical product life cycle. They also are best positioned to engage with patients and patient groups to bring findings back into the business and ensure relevant data are being generated. By working closely with patient advocacy groups and, where permitted, patients themselves, life sciences companies can add a ready and informed base of real-world KOLs to the consultation pool. To this regard, medical affairs teams need to collaborate with counterparts in:

1. clinical development to design clinical trials that make patient participation easier and capture data relevant to patients

2. commercial functions that provide patient support services

3. the health economics and outcomes research (HEOR) team.

Another important component of advocacy aims to motivate manufacturers to create suitable plain language documents for patients and caregivers to understand complicated clinical trial data and product information, including clinical research results, package inserts, and use cases. The intention is to build trust among patients and the public, improve patient-provider communication, and provide patients with easier access to validated information. This initiative also aims to reduce the risk of patients accessing biased, poor-quality, or inaccurate data, such as through internet searches. Companies also are encouraged to test their direct-to-consumer marketing campaigns with patient groups to help ensure the materials are sensitive, clear, and understandable.

SHIFTING WITH THE TIMES

With increased access to information curated for them, patients have become more educated and proactive in the development of their treatment strategies and therapies. Especially as the healthcare industries in the U.S. and Europe continue to emphasize the triple aim of improving the individual’s care experience, improving population health, and reducing per capita costs of care, life sciences companies should seriously consider how they can help ensure that patients can be informed and prepared.

Initiated primarily in the U.S. and Europe, the focus on patient empowerment is rippling into other regions of the world and gaining momentum. In other words, patient centricity is not just a fad — it is a growing movement that companies need to participate in to stay competitive. For example, although patient engagement in medical-product development is still at a very early stage in Japan, in a new model that is slowly being adopted, patients and researchers are beginning to collaborate to develop therapies that address rare and difficult to treat diseases, such as Alzheimer’s.

In practical terms, following guidance from regulatory bodies and joining the calls for action from advocacy organizations often require more of a mind shift than a significant investment in resources. By incorporating patient centricity early and throughout the product life cycle, life sciences companies will help themselves keep pace and meet the real-world needs of patients.

LIISA EISENLOHR is an associate director at Guidehouse within the Life Sciences Medical Affairs Experts Community with more than 20 years of experience within the life sciences industry.

GURJIT SINGH BANSEL is a pharmacist and senior consultant at Guidehouse within the Life Sciences Medical Affairs Experts Community with more than 10 years of experience as a practicing pharmacist.