PFD report

Ryleigh Hillcoat - Bee · Prevention of Future Deaths report

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Issued 12 Jul 2024•Blackpool and the Fylde

Report record

Published report and response evidence

This page connects the concerns raised in this report with statements found in recipients’ published responses. A link shows a clear evidence connection; it does not assign responsibility.

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Concerns
2

Raised in this report

Recipients
1

Named on the report

Responses found
1

Of 1 recipient

Stated actions
2

Described in responses

Recipients and published responses

Source document

Full report text

This is the full text from the original published report.

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Concerns and recipient responses

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Report evidence summary

Concerns raised2

  1. Lack of awareness and recognition of rhabdomyolysis among paediatricians in general paediatric departments
  2. Limited guidance on rhabdomyolysis in young children
Responses linked to these concerns

Each statement is shown once, even when linked to more than one concern.

Actions described in response An action is something a recipient says it has done, is doing, or plans to do in response to a concern raised.1

  1. Action

    Work with the Genomics Education Programme and Medics for Rare Diseases on solutions addressing limited rare-disease guidance, workforce awareness, education and training.

    Stated by Department of Health and Social CareStated plannedThe respondent said that this action was planned when they made their response on 30 July 2024.

Respondent positions A position is what a recipient says about a concern when it does not describe a specific action.3

  1. Position

    Existing paediatric curricula are considered sufficient; paediatricians need awareness of serious illness and referral rather than complete rhabdomyolysis expertise.

    Stated by Department of Health and Social CareExisting arrangements considered sufficientThe respondent said that existing arrangements were sufficient, so no further action was needed.

Source evidence

How this individual concern was interpreted

PFD Monitor created a concise, searchable interpretation from the report wording shown below. Response links show a clear evidence connection; they do not assign responsibility.

PFD Monitor interpretation

Lack of awareness and recognition of rhabdomyolysis among paediatricians in general paediatric departments

Wider context from the report

“• Ryleigh died from a rare condition, but one known to affect young children. There is a clear lack of awareness of the condition amongst paediatricians in general paediatric departments. • In my view there is a strong likelihood that previous cases of rhabdomyolysis in young children have been missed. • What guidance is available appears to be very limited. • In the event other young children attend a general paediatric department in the future for reasons connected to rhabdomyolysis, there is a concern the condition will go unrecognized and with fatal consequences. ”

Is this part of a recurring concern?

No recurring-concern membership is currently published.

Open source report

Source evidence

How this individual concern was interpreted

PFD Monitor created a concise, searchable interpretation from the report wording shown below. Response links show a clear evidence connection; they do not assign responsibility.

PFD Monitor interpretation

Limited guidance on rhabdomyolysis in young children

Wider context from the report

“• Ryleigh died from a rare condition, but one known to affect young children. There is a clear lack of awareness of the condition amongst paediatricians in general paediatric departments. • In my view there is a strong likelihood that previous cases of rhabdomyolysis in young children have been missed. • What guidance is available appears to be very limited. • In the event other young children attend a general paediatric department in the future for reasons connected to rhabdomyolysis, there is a concern the condition will go unrecognized and with fatal consequences. ”

Is this part of a recurring concern?

No recurring-concern membership is currently published.

Open source report

Source evidence

How this respondent action was interpreted

PFD Monitor created a concise, searchable interpretation from the published response wording shown below.

PFD Monitor interpretation

Work with the Genomics Education Programme and Medics for Rare Diseases on solutions addressing limited rare-disease guidance, workforce awareness, education and training.

Verbatim wording from the response

“With respect to the specific concerns raised in the report, where guidance and workforce awareness appear to be limited and further education and training is required, the GEP will contribute by working with the Department and in collaboration with Medics for Rare Diseases (M4RD) on a number of solutions aligned to some of the actions above.”

Source location

Response from DHSC
Page 4 · response
Published 30 July 2024

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Source evidence

How this respondent position was interpreted

PFD Monitor created a concise, searchable interpretation from the published response wording shown below.

PFD Monitor interpretation

Existing paediatric curricula are considered sufficient; paediatricians need awareness of serious illness and referral rather than complete rhabdomyolysis expertise.

Verbatim wording from the response

“to NHSE by RCPCH. The information provided by RCPCH clarifies that in line with the GMC expectations of curricula and 'excellence by design', they do not have a list of specific conditions included. Their curriculum is an outcomes based one with high level generic learning outcomes which focus on professional and clinical behaviours and capabilities.”

Source location

Response from DHSC
Page 3 · response
Published 30 July 2024

Open published response

Source evidence

How this respondent position was interpreted

PFD Monitor created a concise, searchable interpretation from the published response wording shown below.

PFD Monitor interpretation

Existing specialist guidance is considered sufficient because NICE cannot extend it beyond the available evidence.

Verbatim wording from the response

“Officials engaged NICE with respect to your concern around a lack of guidance on rare diseases. NICE state that they do not usually produce guidance on the management of rare diseases as there is usually limited high quality evidence available on which to base guidance on. Therefore, guidance for rare conditions is usually developed by groups of interested clinicians or specialist societies and by consensus. NICE advise that specialist guidance already exists for the conditions in question, and, with limited evidence, they would not be able to go beyond that existing guidance.”

Source location

Response from DHSC
Page 2 · response
Published 30 July 2024

Open published response

Source evidence

How this respondent position was interpreted

PFD Monitor created a concise, searchable interpretation from the published response wording shown below.

PFD Monitor interpretation

Limited high-quality evidence prevents NICE from going beyond existing specialist guidance for these rare conditions.

Verbatim wording from the response

“Officials engaged NICE with respect to your concern around a lack of guidance on rare diseases. NICE state that they do not usually produce guidance on the management of rare diseases as there is usually limited high quality evidence available on which to base guidance on. Therefore, guidance for rare conditions is usually developed by groups of interested clinicians or specialist societies and by consensus. NICE advise that specialist guidance already exists for the conditions in question, and, with limited evidence, they would not be able to go beyond that existing guidance.”

Source location

Response from DHSC
Page 2 · response
Published 30 July 2024

Open published response

Other statements in published responses

These actions and other statements could not be clearly connected to one concern in this report.

Recipient-stated actions An action is something a recipient says it has done, is doing, or plans to do in response to a concern raised.1

  1. 1

    Publish three action plans implementing the UK Rare Diseases Framework priorities, with named owners, desired outcomes and progress reporting arrangements.

    Stated by Department of Health and Social CareStated completedThe respondent said that this action was complete when they made their response on 30 July 2024.

Source evidence

How this respondent action was interpreted

PFD Monitor created a concise, searchable interpretation from the published response wording shown below.

PFD Monitor interpretation

Publish three action plans implementing the UK Rare Diseases Framework priorities, with named owners, desired outcomes and progress reporting arrangements.

Verbatim wording from the response

“I recognise that people living with rare diseases such as LIPIN-1 deficiency face numerous challenges and more can be done to prevent such deaths in the future. You might be aware that in England, we have now published three action plans setting out how we will address the four priorities of the UK Rare Diseases Framework (the Framework). To ensure delivery”

Source location

Response from DHSC
Page 1 · response
Published 30 July 2024

Open published response
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Information checked against the published report and official responses · Data reviewed 7 Sep 2026 · About data quality and limitations

Official responses located
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Data last updated 7 September 2026