Dual reality of ATMPs: transformative value & access hurdles

Dual reality of ATMPs: transformative value & access hurdles

Advanced Therapy Medicinal Products (ATMPs) can deliver ground-breaking benefits to patients, their caregivers and society, by targeting the underlying causes of diseases.¹ These innovative treatments could offer new opportunities for diseases with notable lack of effective treatments¹ and potentially revolutionize therapeutic strategies.

Nevertheless, a primary challenge associated with these transformative treatments is affordability.¹ Significant upfront costs must be paid for these one-off, potentially curative treatments, whereas patient benefits are only realized in the future.¹ This can directly impact the sustainability of the budgets of healthcare systems, especially with the ever-increasing number of ATMPs being developed/launched.¹ This affordability concern, paired with the uncertainty surrounding long-term clinical effect and risks, fundamentally explain the difficulties pharmaceutical companies face to have their ATMPs reimbursed by healthcare systems.¹

The case of Zolgensma in Germany highlights some of the hurdles that even highly promising ATMPs must navigate.^2,3

Zolgensma is a one-off gene therapy for the treatment of spinal muscular atrophy (SMA). Exceeding the turnover limit (50 million Euro at the time) triggered a full benefit assessment by the G-BA, which determined that an additional benefit is not proven in any of the four defined patient subgroups.

In one of these subgroups (patients with 5q SMA type 1), the pharmaceutical company presented comparisons of individual arms from different studies between Zolgensma and nusinersen (i.e., the G-BA-determined appropriate comparator therapy, ACT), but these were deemed unsuitable for the benefit assessment due to large uncertainties and significant differences in the considered patient populations. Regarding the other patient subgroups, the pharmaceutical company did not present any data for the assessment of the additional benefit of Zolgensma compared to the ACT or did not compare the identified data.

Still, it was recognized that Zolgensma may represent a relevant therapeutic option, given the available evidence on its medical benefit, the disease severity and the scientific-medical society statements on the current reality of care for all patient subgroups. However, a requirement for routine practice data collection and evaluations was put in place, to allow for a better comparison of Zolgensma with physician’s choice of therapy, taking into account nusinersen and risdiplam. This data would be sourced from registers, primarily the SMArtCARE register, and would subsequently work towards the final evaluations for the purpose of the renewed benefit assessment.

Unlocking successful market access for ATMPs

Navigating the space of ATMPs and turning promising treatments into successful market access cases is no easy task. Below are some critical steps for this journey and we can support you at each one.

Engage as early as possible with the relevant regulatory authorities, HTA bodies & pricing committees to receive critical scientific & payer advice. This is key to align on their expectations on asset’s positioning in the treatment pathway, eligible patient population, trial design (including endpoints, comparators) and evidence package (clinical, HEOR, RWE). Payer research can also offer valuable insights here, providing indirect HTA insights through the expertise of former payers.

• Identify appropriate clinical comparators & pricing benchmarks

• Draw learnings and parallels from previous HTA assessments

• Strategically plan to cover the evidence generation requirements across the product life cycle. These can work towards future HTA reassessments and novel payment models (e.g., outcomes-based agreements)

• Explore pre-marketing authorisation access opportunities, such as early-access and named-patient programmes¹

• Be prepared for innovative payment models, as part of the broader patient access plan¹; review data collection systems & infrastructure in scope markets¹

• Build a comprehensive and compelling value story, capturing the holistic value of the ATMP, including any direct/indirect benefits and savings to patients, families, caregivers, healthcare systems and society. Engage with KOLs to validate and further strengthen your narrative

Ultimately, a proactive and tactical approach is key. We are here to help you reduce commercial risk and unlock the full potential of your ATMP