TMC Pharma champions holistic approach to medical services in rare disease drug development
March 13, 2024 – Drug Discovery, Pharmaceutical – drug development, rare diseases, TMC Pharma
13 March 2024 – In a recent webinar, rare disease specialist and global clinical research organisation (CRO) TMC Pharma gathered esteemed speakers to share insights into the scope of medical services during a drug’s life cycle.
The panel consisted of Dr Marcelo Vaz (head of Medical Services, TMC), Dr Lucy Fulford-Smith (associate director of Medical Services, TMC) and Dr Naz Tadayon (senior medical director, TMC Associate).
These industry experts highlighted the specific challenges for rare disease therapeutic studies considering recent FDA guidance – with a particular focus on the role of medical services during the development phase and after marketing authorisation.
The role of medical services in the drug life cycle
There are several key differences in the medical service requirements of rare and ultra-rare disease trials that are often overlooked or misunderstood by drug developers — as outlined by Dr Vaz, a pulmonary and general medicine specialist.
‘Most of the differences come thanks to patient characteristics, disease heterogeneity and the very different drug development landscape we have in this area,’ he said. ‘Even key stakeholders such as regulatory agencies like the FDA have lately become concerned with bringing up discussions about how to adapt and adjust for this environment.’
Dr Tadayon, a distinguished physician with expertise in clinical development and pharmacovigilance, named small patient populations the first and most important challenge to rare disease clinical trials. The lack of patients makes it difficult to recruit participants and conduct larger-scale studies to achieve statistically significant results — compounding other obstacles such as limited scientific and medical knowledge, diagnosis and misdiagnosis and heterogeneity of disease presentation.
The question is, how can medical services and teams help overcome these unique challenges and support more successful clinical trials for rare disease treatments?
To breach the gap, Dr Tadayon suggested that continuous oversight, early-stage study design and access to the necessary medical expertise are critical to rare disease drug life cycles.
‘Effective rare disease research requires a multidisciplinary approach,’ she commented. ‘There’s a need to bring together specialists from different medical fields, and medical experts will play a crucial role in fostering these collaborations that will ensure the studies benefit from a broad range of perspectives.’
Building on Dr Tadayon’s presentation on the role of medical services during the development phase, Dr Fulford-Smith highlighted the need for medical services after marketing authorisation and the lesser-known areas in the post-marketing space for rare disease projects.
According to her experience as a pharmaceutical physician working across rare diseases, paediatrics and oncology, there’s a lack of cross-functional support at these touchpoints. As a result, she stressed the need for more tailored communications with regulators, HCPs and the public, as well as critical appraisal of safety and pharmacovigilance data and greater disease-specific understanding — led by specialist medical personnel.
‘What’s really important to remember is that physicians who have expertise in the rare disease space and the therapy areas associated with a lot of treatment of rare disease is that they can take some of the burden of knowledge off their colleagues,’ Dr Fulford-Smith concluded. ‘They can provide additional in-depth expertise to the regulatory teams, pharmacovigilance teams and medical information departments to support their expert work and take some of the load from them.’
It’s clear that thanks to the unique study designs, complex development landscape and lack of knowledge of disease evolution and safety profiles within rare disease trials, drug developers should demand wider and deeper medical involvement in all steps of drug development and maintenance.
With this in mind, CROs such as TMC are championing a more holistic approach to these projects that accounts for the intricacies of the rare disease landscape rather than applying the same processes used for prevalent disease trials — leading to more successful outcomes and better treatment options for patients worldwide.
