Rare lipid disorders are often ignored, and patients might have little access to accurate diagnosis and effective treatments, particularly emerging therapies. There are many emerging or recently accessible treatments, including bio-drugs, which are effective and safe for rare or severe dyslipidemia.
Once marketed, novel therapies proven to be effective and safe through clinical trials are often expensive and their use is limited by payers claiming lack of sufficient strong and validated data on the natural history of the disease and of benefit from these novel therapies. In low- and middle-income countries, because of the cost of these novel therapies, reimbursement is essentially not possible.
Number of known rare diseases
Of rare diseases are genetic
Affected by rare diseases worldwide
As rare lipid disorders involve small numbers, it is difficult to generate trial evidence. It is therefore necessary to make use of collective efforts to document the clinical expression of rare lipid disorders and to ensure access to accurate diagnosis and effective, safe, and affordable therapies. To reach this goal it is necessary to design and implement comprehensive and creative strategies for access to diagnosis and treatment, regardless of disparities and inequalities.
SMASH (System and Molecular Approaches of Severe Hyperlipidemias) is an international network, a philanthropic initiative, of researchers, clinicians, health professionals, patients, patient organizations, interested individuals, innovators, sponsors and stakeholders aimed at facilitating access to accurate diagnosis and innovative treatments for rare and severe lipid disorders.
Ensure the proper development of this collaborative network to document rare or severe lipid disorders and provide patient access to accurate diagnosis and optimal treatments, including emerging therapies for all who need it, regardless of their living environment, mobility and economic status, in developed countries, remote regions and emerging economies.
We aim to improve the global understanding of the issues, causes and consequences of rare lipid disorders and promote access to accurate diagnosis and optimal personalized treatments for patients.
SMASH is committed to bridging the gap in healthcare disparities. We understand the challenges faced by patients with rare or severe, refractory lipid disorders, especially those lacking mobility or living in remote areas or in regions with underdeveloped healthcare systems. Our mission is to ensure that effective diagnosis and treatment is not a privilege but a right accessible to all, regardless of geographic or economic barriers.
The rarity and geographical dispersion of patients with rare lipid disorders pose significant challenges in documenting the natural history of diseases and developing new therapies. SMASH’s network serves as a vital link, connecting patients with the medical and research community, innovators and stakeholders and facilitating access to a crucial pool of patients and health professionals around the world, not only in developed countries or in urban academic centers.
In the complex landscape of rare lipid disorders, shared knowledge is power. SMASH recognizes the critical need for healthcare professionals, innovators and stakeholders to have trans-national access to shared data and information on diagnoses and cutting-edge research. We are dedicated to fostering collaboration and open exchange of information across borders, empowering professionals with the knowledge needed for groundbreaking advancements in treatment and care.
With the increasing pressure on health services and governmental institutions, the pace of developing innovative approaches for better accessibility and affordability of personalized diagnosis and treatments can be hindered. SMASH stands at the forefront, advocating for and driving the development of innovative solutions that can navigate and overcome these systemic challenges, ensuring that progress in the treatment of rare lipid disorders is not stalled by external pressures.
