Key takeaways

Compared to shorter acting therapies, long-acting therapies might be overlooked and hold potential for patients, healthcare systems and society:

  • Enhanced Patient Outcomes: Long-acting (LA) therapies have potential to improve treatment adherence and clinical outcomes by reducing dosing frequency, thereby lowering treatment burden and minimising symptom relapses for chronic conditions. They also may improve patients’ quality of life, enabling greater patient choice and satisfaction, and addressing stigma.
  • Healthcare System Efficiency: LA therapies have the potential to alleviate healthcare system pressures by reducing the frequency of patient visits, freeing up resources, and lowering overall healthcare expenditures through improved disease management. However, some LA therapies in certain healthcare settings and disease areas may need more frequent visits to allow for HCP administration.
  • Broader Societal Impact: LA therapies can help to increase productivity by minimising disruptions for patients and caregivers. Some LA therapies can also be a part of strategies to enhance patient choice, equity, help mitigate antimicrobial resistance through sustained drug concentrations and may have a lower environmental footprint compared to shorter- acting alternatives.

Realising the potential value of long-acting therapies is currently hindered by a narrow view of value in health technology assessment (HTA) and variability across HTA agencies. HTA value frameworks risk under-recognising the broader value of LA therapies. The potential for benefits in key areas such as patient choice, productivity gains, and environmental impact are often overlooked. Countries with more flexible HTA frameworks, like Canada and the UK, are better positioned to capture these broader value elements than others like Germany and France.

Stakeholder collaboration can help to achieve the necessary policy changes. To account for broader potential of LA therapies, HTA frameworks need to adapt by incorporating broader value elements and leveraging real-world evidence. This will incentivise innovation, ensure equitable patient access, and support sustainable investments in transformative treatments for chronic disease management.

These therapies are particularly effective for chronic conditions, which require long-term treatment. In this report, we primarily focus on injectable therapies that have durations of action and administration frequencies of up to six months, acknowledging for some therapies it can be even more.

This report aims to identify the potential value of LA therapies for patients, physicians, healthcare systems, society, and the economy. Furthermore, it investigates whether the potential value of LA therapies is considered in HTA and payer decision-making. We conducted a targeted literature review and analysed four LA therapy product case studies in HTA decision making.

The full value of LA therapies

This report shows that LA therapies have the potential to offer a variety of benefits for patients, health systems, and society more broadly.
Patient-level effects:

  • Effectiveness: LA formulations have the potential to reduce treatment burden and reduce symptom relapses compared to daily oral therapies, for example for conditions like schizophrenia or osteoporosis. When integrated into clinical practice, LA therapies can also improve patient adherence by reducing dosing frequency and lead to better clinical outcomes. However, reduced dosing frequency might not lead to improved adherence in all cases and the prolonged treatment effect of LA therapies may extend or lead to different side effects compared to short-acting therapies.
  • Quality of life (QoL): LA therapies potentially improve the QoL of patients, including health and functional status, as well as enhance the quality of patient’s social and occupational lives. For some conditions l, this value element may also capture the impact on stigma experienced by patients affected by the condition, reducing stigma-associated treatment burden and the risk of disclosure, and daily medication reminders.
  • Patient-centeredness and choice: Less frequent dosing from LA therapies may align with patient preferences and lifestyle, enhancing patient satisfaction or sense of control over their condition. Nevertheless, in the cases where LA therapies also increase frequency of routine interactions with healthcare professionals as part of treatment administration, may improve the health and well-being of patients, by identifying any additional health needs and enabling intervention in a timely manner.

Healthcare system impact:

  • Cost savings: Long-acting therapies may contribute to overall cost savings through improved adherence and reduced disease incidence, which in turn lowers long-term healthcare expenditures.
  • Health system capacity: With improved disease management and reducing dosing frequency, LA therapies can help alleviate pressure on healthcare system resources. The extent to which this benefit is realised may vary by therapeutic area, depending on existing treatment schedules and service delivery models.

Societal and economic impact:

  • Increased productivity: Patients and caregivers can benefit from fewer treatment-related disruptions to productivity, but this may also depend on the frequency, type of administration and location of the administration site.
  • Equity improvements: LA therapies can help addressing medical needs for patient populations facing treatment challenges (e.g. nonadherence or discontinuation).
  • Prevention of antimicrobial resistance: LA antimicrobial therapies may preclude the emergence of antimicrobial resistance by maintaining more effective plasma drug concentrations over extended periods, reducing dosing requirements and lowering the risk of missed doses.
  • Environmental impact: some LA therapies may be the “greener” choice compared to current treatment options when considering a reduction on waste or production.

HTA considerations

Results from product case studies demonstrate that in certain therapy areas, despite the potential wide-ranging impact, the broader value of LA therapies is currently under-recognised in health technology assessment (HTA) and reimbursement decisions. Analysis across ten global HTA bodies reveals significant variability in the extent to which and how elements of broader value relevant to LA therapies are considered. While clinical effectiveness, patient quality of life, and healthcare cost reductions are commonly assessed, elements like productivity, patient choice, equity, and environmental effects are often overlooked. Notably, countries that use broader, more flexible value frameworks as part of their HTA and those that are open to considering real-world evidence, like Canada and the UK, incorporate these broader elements more frequently than markets focused strictly on additional clinical benefit, such as Germany and France.

Conclusion

Compared to shorter acting therapies, LA therapies hold great potential to transform chronic disease management, delivering value across multiple dimensions. Realising LA therapies’ full potential requires adapting HTA frameworks to reflect their comprehensive benefits, enabling equitable access for patients and incentivising further innovation. This strategic approach will ensure optimal healthcare outcomes in the short term and allow for sustainable ongoing investment in these treatments in the future.

Recommendations

Stakeholders should collaborate and involve patients and their healthcare professionals to ensure that the development and implementation of LA therapies align with patient needs, health system improvements, and societal or economic goals.

  • The research community should prioritise generating real-world evidence on LA therapies’ impacts and advance methodologies that quantify societal and environmental benefits.
  • Innovators should incorporate robust evidence generation of broader value elements into clinical development strategies.
  • Policy makers and payer decision-makers should expand value frameworks used in HTA to integrate broader value dimensions, fostering a comprehensive evaluation of the full value of LA (and other) therapies. Furthermore, this will help to foster a patient-centric approach where the choice and preferences of patients, caregivers, and healthcare professionals are acknowledged.

This report, Understanding the Full Value of Long-Acting Therapies: less is more?, was commissioned and funded by GSK.