Market access update
The influence of the patient voice in regulatory and health technology assessment activities
In the second of her two-part series, Sarah Richard explores a patient-centric approach to market access.
Whether it be preferences, burden of disease, or other insights, regulators, health technology assessment (HTA) agencies, and public-private partnerships are recognizing the importance of systematically incorporating patient insights.
The FDA’s new guidance on gathering patient input is an example of the signals it is sending to manufacturers about more and better patient involvement.
HTA agencies are also broadening the scope of how they integrate patient perspectives and decision-making. At the time of writing, some countries, such as the UK and Germany, have had a well-established process for some time, while others, such as France and Italy, are developing a suitable framework to engage patients.
NICE, a forerunner for patient involvement, involves patients across its HTA and scientific advice work. In February 2019, NICE provided its first scientific advice on a manufacturer’s patient preference study design, sending a signal to industry about the importance of these data in HTA.
“The FDA and EMA are increasing the rate at which they involve patients and their committees, and are setting expectations for manufacturer engagement with patients.”
A lack of clarity over how patient input is used in decision-making processes can make it hard to demonstrate the influence of patients’ input on decision-making.
An example of influence by a patient advocacy group has been cited by NICE during the assessment of vedolizumab for ulcerative colitis. The patient group undertook a large survey that helped to highlight aspects of the condition around lost productivity and the impact on quality of life. The value of the treatment to particular subgroups was highlighted, especially in avoiding surgery and returning to work. The patient voice helped address areas of uncertainty in the original assessment and contributed to a favorable recommendation.
In other assessments, patient experts and advocacy groups have provided evidence needed to confirm what outcomes are appropriate for modeling, or highlighted additional social and economic impacts that influence what is considered a final decision. These impacts may be less obvious in helping to overturn a negative decision but they are impactful nonetheless.
More initiatives are driving a systematic role for the patient’s voice in market access.
The influence of patient advocacy is observable beyond their involvement in committee processes. Patients are using their increased exposure to regulatory and HTA activities to change policy, challenge industry and governments, and forge solutions to overcome the challenges in accessing high-cost treatments.
Access to dedicated resources and training to support patient advocates to get involved in drug development and HTA is contributing to this trend. The European Patients’ Academy on Therapeutic
Innovation (EUPATI) is an example of a successful program that is educating and training patient advocates to engage in drug development and market access.
Patient groups are also bringing industry together to find solutions to common market access challenges in particular disease areas, such as Project Hercules, a novel collaboration forged by the Duchenne UK patient group.
These examples illustrate a growing trend towards more expert and engaged patient groups, working with both industry and HTA agencies to find solutions around price and affordability.
Patients groups are holding industry to account for drug reimbursement hurdles.
Establishing long-term partnerships is the most likely way to successfully bring the patient’s voice into product development. However, effective collaboration is not always straightforward.
Ethical or legal boundaries or public perception may be a concern, particularly where there is a funding relationship or new drugs under review. There are clear guidelines for industry to help overcome this but the fear of perceived influence can still be a stumbling block.
Budgetary or organizational constraints within a company can get in the way of partnerships with advocacy groups being prioritized.
In some disease areas the scarcity of patients, competition for partnership, or a lack of established advocacy groups presents an additional challenge.
Recommendations could help manufacturers establish successful invaluable partnerships with patients.
Recommendations for mitigating these challenges include: engaging early and regularly throughout product development and not just at the point of launch, collaborating on projects that go beyond market access, following industry guidelines and principles, and getting internal alignment on the importance of investing in partnership with patients. All these could help manufacturers establish invaluable partnerships with patients.
The future of patient engagement is constantly being redefined but the direction is one of increased appreciation of the patient voice in both product development and assessment, and towards more systematic and sophisticated ways to incorporate it. This presents a challenge and an important opportunity for pharmaceutical manufacturers.
I would encourage manufacturers to engage with patient advocacy groups early on in product development, and understand how they can partner on both clinical trial design and broader evidence development projects to help set the scene for successful market access.
PRMA Consulting is an independent consultancy solving some of the most challenging pricing, reimbursement, and market access issues facing international pharmaceutical and biotechnology companies today.
We can offer the following support:
- devising patient engagement strategies and implementing programs
- developing a patient-centric market access strategy for your product or portfolio
- exploring patient engagement opportunities with advocates and representatives
- providing insights and recommendations for or pressure testing engagement projects and programs
For a confidential conversation about incorporating a patient perspective into your market access strategy, please get in touch.
Patient-centricity: a crucial approach for market access?