Women in Rare

Rare diseases: women wait two years longer from the first symptoms to diagnosis

Diagnostic delays, gender inequalities, hidden costs: a White Paper quantifies, for the first time, the inequity surrounding rare diseases in Italia

Malattie rare: ritardo diagnostico, disuguaglianze di genere e costi invisibili per le donne

4' min read

Translated by AI
Versione italiana

4' min read

Translated by AI
Versione italiana

There is also a gender issue to be addressed in the context of rare diseases. Women with rare diseases wait on average two years longer than men to receive a diagnosis; in the south, one in two patients is forced to travel outside their region to receive a diagnosis; and 37.2 per cent of female carers give up work to look after a loved one. These figures come from the White Paper ‘From Rare to Recognised: Causes and impact of diagnostic delay on the lives of people with rare diseases and their carers’, a report that reveals the severe impact of rare diseases on women’s work, family life and economic opportunities.

The survey, presented to the Senate on the initiative of Senator Elena Murelli, was conducted by Censis and Altems, a spin-off of the Catholic University of the Sacred Heart in Rome, for Women in Rare – the think tank conceived and promoted by Alexion, AstraZeneca Rare Disease, in partnership with Uniamo, the Italian Federation for Rare Diseases, and in collaboration with the Onda Foundation and a multi-specialist and institutional scientific committee.

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The impact of a diagnosis on people’s lives

““This survey represents a unique contribution to research as it documents the time elapsed from the first symptoms to diagnosis and focuses on the profound social and economic repercussions that rare diseases have on the lives of people and their carers,” says Giorgio De Rita, Secretary-General of the Censis Foundation. The consequences of rare diseases affect a wide range of areas of life, from family relationships to work, leisure activities and socialising with others. In particular, the study highlights a number of critical issues, starting with the difficulties in interpreting the symptoms of these conditions, which result in longer timescales before a final diagnosis is reached. The findings of this research are a formidable tool to support the necessary collective effort to improve the healthcare and social infrastructure supporting people with rare diseases and their carers.”

The picture that emerges is that of a fragmented diagnostic pathway characterised by two critical phases. The first – from the onset of symptoms to the first medical consultation – takes an average of 2.5 years, but a gender difference is apparent: women wait on average almost 3 years, whilst men wait 1.4 years. This is followed by a second stage – from the first consultation to the definitive diagnosis – which takes an average of 3 years.

Inequalities are widening across the region

Diagnostic delays are not confined to the healthcare sector; on the contrary, they reshape entire life trajectories, erasing or diverting them, leading to career interruptions or even the complete abandonment of the search for employment. And for those who were in employment at the time of diagnosis, the impact has often been dramatic: 46.6 per cent were in fact forced to take early retirement.

The survey also documents a phenomenon that exacerbates the inequalities already evident in the diagnostic process. “The research shows that 31.6% of people with a rare disease were forced to travel outside their region to obtain a correct diagnosis. However, at a regional level, this healthcare mobility is not evenly distributed: whilst in the North and Centre almost one in three patients travels elsewhere, in the South and on the Islands the figure rises to 46 per cent – practically one in two people with a rare disease,” comments Eugenio Di Brino, Altems Advisory. Added to this is the socio-economic burden: 70% of patients come from families with an annual income of less than 35,000 euros, making the impact of the additional healthcare, care and social costs associated with rare diseases particularly onerous.”

Women in Rare: from data to action

With this study, Women in Rare consolidates its role as a think tank – a permanent forum where rigorous analysis is translated into concrete proposals for institutions and advocacy for people with rare diseases. This commitment does not end with the presentation of the results: all the findings have been compiled into a White Paper with the aim of promoting a number of recommendations and supporting institutions in translating these into concrete courses of action and policies, whilst monitoring, over time, the impact of these measures on the lives of people with rare diseases, with particular attention to gender and regional inequalities.

“The voices of people with rare diseases and their carers contained in this report are a call to the authorities to take action,” says Annalisa Scopinaro, president of Uniamo – Italian Federation for Rare Diseases. “We have highlighted inequalities that, until now, were invisible in official data. Delays in diagnosis are not just a clinical issue: they are a matter of equity and social justice. The women and the families who care for them deserve swift, transparent and fair pathways. We now have solid evidence to demand this forcefully.”

This research, therefore, could mark a turning point and present an opportunity to transform evidence into practical tools to support decision-making. “In recent years, Women in Rare has highlighted a critical situation affecting over two million women who, as patients and carers, face the impact of a rare disease every day,” says Anna Chiara Rossi, VP & General Manager Italy at Alexion, AstraZeneca Rare Disease. Today, it is essential to provide decision-makers with a clear understanding of the real-world implications of the diagnostic journey on quality of life and socio-economic outcomes, and to identify policy actions capable of bringing about tangible change. Gender emerges as a key factor throughout the patient journey and cannot be considered a secondary issue, but rather an essential lens through which to rethink how we diagnose, treat and protect people living with a rare disease. “Women in Rare aims to be a space where research translates into concrete commitment and recommendations become actions for change.”

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