In this consensus Statement the InterAcademy Partnership (IAP) seeks to raise awareness of two main priorities:
- To use advances in research and development as rapidly as possible, safely and equitably, to provide new routes to patient benefit.
- To support medical claims by robust and replicable evidence so that patients and the public are not misled.
The focus of this IAP Statement is on unmet medical needs: stem cells are described as a case study with many of our conclusions relevant more broadly for regenerative medicine. Although stem cell therapy is well-established in only a limited number of clinical indications, there is active research and development in many more. However, enthusiasm about the clinical potential has led to a disconnect between expectations and the realities of translating advances in technology into clinical practice. In many countries, there are two main problems.
First, unscrupulous private clinics offer unregulated therapies promising much, but using poorly characterised products with little scientific basis or evidence for efficacy, with safety concerns unresolved. Second, premature regulatory authority approval and commercialisation based on some, but insufficient, scientific rationale and clinical evidence. Accelerated access is a vital tool for patient benefit but researchers must not cut corners.
In order to strengthen the frameworks for research and innovation and patient protection, IAP has identified priority actions for: engaging with patients, the public and policy makers; ethical assessment; pre-clinical and clinical research procedures; regulatory authorisation and options for facilitating access to new medicines; and noted the particular relevance of these actions also in the response to COVID-19.
IAP concludes that:
- For the present, regenerative medicine should concentrate on serious medical conditions and be judged by rigorous consideration of the potential risks versus the benefits.
- For many potential applications, more evidence is needed on product quality, safety and efficacy.
- Good science must be promoted at every step, from fundamental research through to clinical trials and the translation to practice.
- Proportionate and harmonised regulatory authority actions should be based on robust and replicable science, ethically informed by science across the disciplines. Unregulated provision of unproven regenerative medicine must be deterred.
- Researchers must follow guidelines on responsible science, and teaching on regenerative medicine should be part of the curriculum for health professionals. Clinicians must be bound by both professional guidelines and community standards of medical practice.
- In putting patient interests first, the scientific and medical communities have a responsibility to provide reliable information and ensure that decisions are evidence-based. Therefore, to deliver clinical benefits equitably, a coordinated strategy must encompass better science, better funding, better governance and better public and patient engagement.