Clinical development plan for regenerative therapy in heart failure

The emerging heart failure pandemic, afflicting nearly 30 million individuals worldwide, underscores the imposing burden of chronic disease on medical and public unmet needs.1 Tangible achievements in managing acute coronary syndrome have been in part offset by incipient organ failure in patients that survive the initial myocardial insult but ultimately develop an adverse course and require escalation of therapy.2 Heart failure is a leading indication for repeat hospitalizations across geographies, with poor survivorship halving affected patient populations within 5 years post-onset.3,4 Identification of actionable strategies to reverse and reduce myocardial injury is a recognized priority in order to avert progressive dysfunction and prevent organ decompensation.5 The increasingly vulnerable global elderly population necessitates radical advancements in combating heart failure beyond the current standards of care. Calls for accelerated discovery and development of new therapies have been issued.6 To address ‘real-life’ patient needs along with the societal quest for ‘health as value’, modern algorithms for clinical development of candidate technology incorporate multidisciplinary assessment by healthcare providers, developers, regulators, and payers.7 This evolving landscape heralds a shift in the process of development and authorization of novel therapies, from the traditional paradigm zoomed-in on therapeutics, to an increasingly holistic evaluation that integrates the whole patient within a healthcare regimen. Regenerative technologies exemplify an emerging class of disruptive innovations that are practice-transformative in nature aiming at normative organ restitution in the context of advancing whole-person care.8 Poised to achieve functional and structural repair, the prospect of regenerative medicine offers