Save Babies Through Screening Foundation UK

Save Babies Through Screening Foundation UK Trustees Annual Report 2015/2016Contents Statement from Executive Chair…………...... 21. Reference and Administration Details...... 42. Structure, Governance and Management...... 53. Objects and Activities...... 84. Achievements and Performance ...... 105. Declaration…………………...... 13Save Babies UK, Annual Report 2015-16 1 Statement from Pat Roberts, Executive ChairOn behalf of the Board of Trustees I am please to be able to report on the important work we have been undertaking during 2015-16. Early in the year Trustees came together to discuss and agree how we secured the future of Save Babies UK and transitioned our volunteer organisation to one with the capacity to grow and develop further. From this, ongoing work includes succession planning for a future Chair of the Board of Trustees, improving our support services to families, enhancing the position of our charity to attract appropriate grant funding and examining options for enhancing our capacity on delivering work around newborn screening. These present us with significant challenges which we are approaching with enthusiasm, however realistically we anticipate this will be a 3-5 year plan. During the year we were pleased to welcome Andrew Gibson as a Trustee.Throughout the year we have continued to support families affected by Krabbe disease. Although it is a rare condition, each year we lose too many children to this terrible disorder. Whilst we have made important progress in the level of support that we offer families, nevertheless we recognise that not all families are finding our organisation or are not finding us quickly enough. We have continued to work on this in both improving our website and initiatives on developing new information. However it remains a focus for the coming year. We are a charity run entirely by volunteers with a relatively small annual income. I would like to offer my appreciation to our Trustees, our families, our fundraisers and our colleagues in the health professions and other charities. Without all your hard work, your support and your advice we would not be able to give support to families and drive forward our important programme of work. I would particularly wish to express my appreciation to Mrs Janet Mills, one of our founding Trustees who retired from the Board in November 2015. Janet was our Treasurer since we formed in 2008 and was instrumental in ensuring robust accounting, including introducing our IT accounting system. On behalf of the Board of Trustees I sincerely thank everyone who has supported our work throughout the year.Save Babies UK, Annual Report 2015-16 2 What is Newborn Screening?Newborn Screening can enable the early detection of some rare disorders at birth and allow immediate treatment. Early diagnosis at birth is done by taking a small sample of blood, generally by a midwife and between 5-8 days of birth. This is known as the ‘heel prick’ test. This is a tried and tested method and does no harm to the infant. The sample is then screened in a Newborn Screening laboratory to detect potentially severe or rare disorders. Early detection at birth and the ability to give immediate treatment to the child before any symptoms appear can prevent physical disability, neurological disability and death. In the United Kingdom we now screen for 9 disorders at birth whilst many other countries screen for many more.What is Krabbe Disease?Krabbe (pronounced Krab-ay) disease (also called globoid cell leukodystrophy) is a rare inherited genetic disorder of the nervous system. The disorder can appear soon after birth in the ‘Infantile’ form or in older children or adults in the 'Juvenile' or 'Late Onset' form. Most patients have the infantile form. Affected infants appear normal at birth but soon develop neurological signs. There is progressive mental and motor deterioration often leading to deafness and blindness. The life expectancy of children with infantile Krabbe Disease is generally up to 2 years of age.Save Babies UK, Annual Report 2015-16 3 Trustees Annual Report: 1 April 2015 – 31 March 2016Report of the Trustees for year ended 31 March 2016.1. Reference and Administration DetailsCharity Name: Save Babies Through Screening Foundation UK (Save Babies UK)Registered Charity Number, England & Wales: 1133080 Registered Charity Number, Scotland: SC042501Registered Address: 22 Bransdale Avenue, Guiseley, Leeds, LS20 8QATelephone: 01943 878770Website: www.savebabiesuk.orgBoard of TrusteesPat Roberts Chair Melanie McPherson Vice-Chair Janet Mills Treasurer (Retired November 2015) Ros Grimwood Treasurer (Treasurer from November 2015) Sam Hainsworth Secretary Andrew Gibson (Appointed from February 2016) Sarah Dudko (Retired November 2015) Lindsey Parnaby (Appointed from August 2016)BankersYorkshire Bank, 17 High Street, Yeadon, Leeds, LS19 7SPIndependent ExaminerLee McCardie (CIMA) 49 Sandhill Oval, Alwoodley, Leeds, LS17 8EFSave Babies UK, Annual Report 2015-16 4 2. Structure, Governance and ManagementSave Babies Through Screening Foundation UK (Save Babies UK) is a non-profit making, charitable organisation, run entirely by volunteers. Our mission is to improve and save the lives of children by working to prevent disabilities or early death resulting from diseases that are both detectable and treatable at birth through Newborn Screening. We are also working to advance the health and wellbeing of children suffering from Krabbe Leukodystrophy and other genetically inherited rare diseases in the UK. We aim to promote the advancement of research and treatments for these diseases whilst providing support for families and children diagnosed with Krabbe disease.Save Babies through Screening Foundation UK is a charitable trust that is managed by a Board of Trustees operating through a Trust Deed which was enacted on 7 th November 2009. Save Babies UK became a registered charity in England and Wales in December 2009. We received Charitable Trust status in Scotland in August 2011.Structure and GovernanceSave Babies UK is a volunteer-run charity led by the Board of Trustees. Strategy is set and decisions are taken by the Board of Trustees.The Trustees held two full Board meetings this year, in May and November 2015. In addition we held 2 further meetings to specifically focus on securing the future of the organisation and plans for the next 3-5 years. We were fortunate to have the services of two facilitators from other larger charitable organisations to assist us with this task. The Trustees have also held a programme of regular teleconferences throughout the year in order to progress our work. Save Babies UK is the Chair of the UK Patient Advocacy for Newborn Screening (PANS) working group, which supports our objective on advancing the programme of Newborn Screening in the UK. This is a collaboration of Patient Advocate Charities including Climb (Children Living with Inherited Metabolic Diseases), The MPS Society (Society for Mucopolysaccharide Diseases), the Association for Glycogen Storage Disease (AGSD UK) and ALD Life. We have a scientific, midwifery and other advisors as members. Funds for PANS activities are administrated by Save Babies UK and managed as reserved funds. Save Babies UK is a registered stakeholder with the Public Health England, UK National Screening Committee.Save Babies UK is also a member of the UK Lysosomal Storage Disorder (LSD) Collaborative. The collaborative includes The MPS Society, The Gauchers Association, The Niemann Pick Disease Group UK, AGSD UK, the Batten Disease Family Association and Tay Sachs Association. This collaborative works to ensure that specialised services for all LSD Patients across the UK are in place, are relevant, robust and that new and existing services meet the needs and requirements of patients. The Chair of the Board of Trustees, Pat Roberts has been a patient and public voice (PPV) member of the NHS England, Clinical Reference Group for Inherited Metabolic Disorders (CRG IMD) for 3 years. Pat has not reapplied for this position from MarchSave Babies UK, Annual Report 2015-16 5 2016 following reorganisation of CRGs. This is to enable her to concentrate on succession planning, however Save Babies UK remains a stakeholder of this group.The charity formally registered with the Information Commissioner's Office from 26th January 2016 as part of the Trustees review of our obligations, requirements and policy under the Data Protection Act 1998.Appointment of TrusteesTrustees are appointed to the Board of Trustees on the basis of skills, knowledge and experience needed to run the charity successfully. All trustees give their time voluntarily and receive no remuneration or other benefits.Further Trustees can be appointed by the Board of Trustees as required. Prospective Trustees will be asked to provide information regarding their skills and suitability for the role of a Trustee and to declare any conflicts of interest. Appointment of a Trustee is dependent on a DBS check. Training and induction will be provided for any new Trustees appointed, covering on the work of the charity, the role and responsibilities of a Trustee and Save Babies’ governing documents.RiskThe Trustees manage an Assessment of Risk document and this is reviewed annually at a minimum. In addition, individual risk assessments are undertaken for each event that is held. Where appropriate, systems or procedures have been established to manage the risks that the charity faces. The critical risks identified within the 2015/16 year included:  Insufficient income raised to deliver the activities within the business plan.  Insufficient people resource to deliver the activities within the business plan.  SBUK does not provide families with the appropriate level of support at the point of contact, on an ongoing basis and at a time of bereavement.  Successful succession planning to enable the Board of Trustees to secure the standing and long-term operation of Save Babies UK.Financial Review Our main source of income has been from fund raising activities and donations. We have received a small sponsorship grant from industry to take forward Newborn Screening activities with the PANS (Patient Advocacy for Newborn Screening) working group. Our main source of income has been from fund raising activities and donations. We have received a small sponsorship grant from industry to take forward Newborn Screening activities with the PANS (Patient Advocacy for Newborn Screening) working group.Our overall income in 2015/16 was £41,174 taking our total available funds to £78,877 (including £19,633 of restricted and designated funds). Our overall expenditure was £14,163 which includes our charitable activities, family support, cost of generating funds, insurance and administration costs.Save Babies UK, Annual Report 2015-16 6We designate £10,000 for operating costs (included in the designated amount referred to above). All other income is available to benefit the significant projects planned for 2016/17 and beyond including funding for research into Krabbe disease. The full accounts can be found appended to this annual report.Public Benefit StatementThe Board of Trustees confirm that they have complied with their duty to have due regard to the Charity Commission’s guidance on public benefit in exercising their powers and in planning activities for this and future years. The activities benefit children across the UK.  Through directly helping children and families affected by Krabbe Leukodystrophy through our many support services. Through raising awareness of the benefits of Newborn Screening and through supporting the research base for extending the Newborn Screening programme in the UK, we can support the early diagnosis and detection of disorders to prevent disability, and death in children. This area of our work covers all inherited metabolic disorders and is not confined to Krabbe disease. Through our continued support to Cambridge University School of Medicine in their collaborative programme of research with the end ambition of finding a therapy.  Through the specific joint initiative with the LSD Collaborative and the Students 4 Rare Diseases (S4RD) in educating medical students across the UK on rare diseases to aid better and earlier diagnosis for children. 3. Objects and Activities Our Objects Our Objects as set out in the Trust Deed are; To relieve sickness and preserve and protect the health of children suffering from Krabbe disease and Leukodystrophies and other genetically inherited diseases in the UK, in particular but not exclusively by funding research and promoting the extension of Newborn Screening; To preserve and protect the health of the families caring for children suffering from Krabbe disease and Leukodystrophies and other genetically inherited diseases in the UK by such means as the trustees may determine.Save Babies UK, Annual Report 2015-16 7 Our 2015/16 objectives and activities The objectives and main activities undertaken are as follows:1. To advance the programme of Newborn Screening in the UKThrough our chair of UK Patient Advocacy for Newborn Screening (PANS) working group we have:  Identified and agreed through PANS the diseases appropriate for advocacy and submission work.  Continued our role as key stakeholders to the UK National Screening Committee (UK NSC) in evaluating evidence and responding on disorders being considered for the newborn screening programme in the UK.  Progressed work with stakeholders to engage other patient organisations with an interest in Newborn Screening and with international stakeholders on comparative studies for the global Newborn Screening position.  Engaging with scientists at Manchester and at Great Ormond Street Hospital on proof of concept studies for NBS for Lysosomal Storage Disorders.Through Save Babies UK, additionally we have: Attended key conferences and events in order to raise awareness of Newborn Screening in the UK.  Supported other patient organisations in giving advice and guidance on the process of proposing new disorders onto the UK Newborn Screening programme.  Given the Keynote speech to pathologists at the Viapath Innovation Academy 2015, 'It's Diagnosis, Not Diagnostics'  Attended the first UK National Screening Committee Stakeholder conference in December 2015. This stakeholder engagement initiative had resulted from some of our recommendations on improving engagement with patient organisations to the House of Commons, Science and Technology Committee Inquiry into National Health Screening in the UK during 2014. Some quality and other improvements, e.g. improved guidance, annual call for newborn screening proposals have also been made as a result of our submission.  Continued to build links both in the UK and internationally to understand and develop the evidence base on Newborn Screening.  Save Babies UK continues as an active member of the International Society for Neonatal Screening.2. To provide support for children, and the families of children with Krabbe disease and other Leukodystrophies; Provided support services to families with children diagnosed with Krabbe Leukodystrophy. Save Babies UK, Annual Report 2015-16 8  Supporting families through our annual family conference held in September 2015. This enables families of those who have, or have had, children with Krabbe disease to have an opportunity of support from others who have been through the same experiences, to learn more about the disorder from health experts, to learn more about the work of our charity and to provide input, feedback and information to help us continue improving our support services.  In terms of succession planning, worked to secure and enhance the support and services we are able to offer families. We have commenced an initiative to secure services from a professionally trained families officer from a similar charity, with the intention that this will be piloted for an 18 month period from the Autumn/Winter of 2016.  As members of the UK LSD Collaborative we have contributed to work to enhance the services to families affected by LSDs. This has included the introduction of annual meetings with health professionals at the LSD specialist centres and children's hospitals in the UK.  By making Children’s hospitals and hospices aware of Save Babies UK as a supportive patient organization. Families with children diagnosed with Krabbe disease are more likely to be referred to us so that we can offer them help and support.  Through forging effective working relationships with scientists, clinicians and other health professionals both in the UK and worldwide, we are able to understand the position on research, clinical trials, diagnosis and treatment, in order to establish how this can benefit children in the UK by improved care and treatment.3. To promote and support research into Krabbe disease and other Leukodystrophies; Continued to engage with scientists, clinicians and other health professionals both in the UK and overseas on the current position worldwide and in the UK, with a view to determining what is further required for the UK.  Continued our feasibility work on the development of a Krabbe History Study and registry.  Continued membership of the UK LSD Collaborative Group and participation of the work within that group.  Through membership of the NHS England Clinical Reference Group for IMDs contribute to review and development of service specifications, quality dashboards and protocols for therapies. This has included work on the development of a new service specification for White Matter disorders.4. To raise awareness of the work and objectives of Save Babies UK and further develop as an organisation; Worked to expand awareness of Save Babies UK, Newborn Screening and Krabbe Leukodystrophy.  Sought opportunities to expand our volunteer base.  Continued to build our partnerships with other organisations and health professionals.Save Babies UK, Annual Report 2015-16 9 4. Achievements and PerformanceThis section expands on the activities that we have undertaken this year, with particular focus on what we have achieved through these activities. 1. To advance the programme of Newborn Screening in the UKSave Babies UK has a collaborative approach to all our activities, in order to increase the opportunities available to us and avoid duplication of activities. In collaboration with the British Inherited Metabolic Disease Group (BIMDG), organised a conference to examine and discuss the challenges to introducing new and appropriate disorders onto the UK newborn screening programme and to find ways of addressing any issues and barriers. This involved scientists, clinicians, patient organisations, industry and representatives of the UK National Screening Committee. Work has commenced on a recommendations paper to go to Ministers, DoH, MP's, members of Health Committees and others during the Autumn of 2016.Work has also commenced on work to raise the awareness of the importance of newborn screening to coincide with Rare Disease Day 2017.Through our advice and support role to other patient organisations who are submitting proposals to the UK National Screening Committee to have their particular disorders/s added to the newborn screening programme.As key stakeholders to the UK National Screening Committee, we submit comments, observations and evidence in response to the UK NSC reviews of proposed disorders.We continue to attend meetings and conferences to raise awareness of the importance of NBS. Other activities for Save Babies UK: We have continued to ensure we are properly informed by robust advice and guidance through networking and development of links with the heads of screening laboratories in the UK.2. To provide support for children, and the families of children with Krabbe disease and other Leukodystrophies Save Babies UK provides an information and support network for families with children diagnosed with Krabbe Leukodystrophy. This is a rare genetic disease and we believe there are three elements to the successful delivery of this service:Fast referral to the support provided by Save Babies UK: We have continued to develop our network with health professionals in Children’s hospitals across the UK to raise awareness of our organisation and the support we can provide. We now attend periodic face-to-face meetings with some hospitals. We remain members of Together for Short Lives to utilise resources to assist our families.Interactions with families being personal, well informed and responsive to the needs of the family: Our volunteers that provide this support have firsthand experience of Krabbe disease. Responding to the needs of families during the year, we have continued to build relationships throughout 2015-16 with those authorities andSave Babies UK, Annual Report 2015-16 10 organisations who are able to provide support, equipment and grants and now have improved information to hand to meet needs on a quicker timescale. A significant area of work during 2015-16 has been around our proposal to provide professional family support services, to families across the UK, under a Service Level Agreement with another patient organisation who we already work closely with and who already have professional, fully trained and paid employees. Significant discussions with both Boards of Trustees and a mutual agreement to this proposal has resulted in successful applications for pro bono legal work to support the development of the Service Level Agreement. We will be piloting this initiative for a period of 18 months. This has been a challenging undertaking in a number of areas. We are however optimistic that the service can commence in the late Autumn of 2016.Financial support where needed: We consider and, where possible, continue to provide financial support for specific items for children where this is required but not provided by the NHS or the Local Authority. With considerable success we have been able to direct families to other grant awarding bodies for the more expensive and more specialist equipment that we are unable to provide.3. To promote and support research into Krabbe disease and other Leukodystrophies for development of improved treatments for these diseasesBuilding on our previous support for the Krabbe research project at Cambridge University School of Medicine, we continue to raise funds for this programme. Current funding for the research project expires at the end of 2016 and we are trying to position ourselves to offer further financial assistance in 2017. We are only a small charity and relatively can only make a small contribution however this has benefited the programme in the past.Save Babies UK as a member of the UK Lysosomal Storage Disorder (LSD) Patient Organisation Collaborative. continues to work on behalf of LSD patients and their families in the UK. We continue to support the Students for Rare Diseases (S4RD) on the educational model on rare disease education for medical students and roll out of this model across the UK. A member of staff has been employed by the collaborative for a 2 year period from 2016 to support the S4RD initiative.We continue to explore the introduction of a history study and patient registry of Krabbe patients in the UK and families have expressed interest in participating in this. This involves the retention of the most sensitive information. We have spent valuable time during the year in reviewing and ensuring that the governance and our responsibilities to meet all Data Protection legislation and requirements has been reviewed are in place. We are developing new forms to support this. Data protection requirements are critical and cannot be rushed. This challenging area of work and required training is being carried forward into 2016-17.Through membership of the NHS England Clinical Reference Group for IMDs, contribute to the review and development of service specifications, quality dashboards and protocols for therapies. Save Babies UK, Annual Report 2015-16 11 4. To raise awareness of the work and aims of Save Babies UK and further develop as an organisation.Charity DevelopmentThe significant body of work that we have achieved this year has been delivered by a small team of volunteers, fundraisers and advisers, who give so selflessly of their professional skills and time. This year we have continued to build a network of volunteers and supporters. Raising AwarenessWe have raised awareness through the following channels; Twice yearly issues of our Newsletter.  Our website and social media channels.  Development of information materials for families and research/health professionals;  Providing information at our fundraising events and supporting our fundraisers.  Attendance at health and patient conferences;  Through collaborative relationships with other patient advocacy groups.FundraisingSave Babies UK is a charitable organisation run entirely by volunteers that receives no government funding or grants. The success of our work is dependent on the generosity of people in making donations and supporting our fundraising events. To support our objectives and activities through the year, Save Babies UK volunteers have held collections, sponsored events and fundraising events. We have also received charitable donations from individuals and organisations. We would like to thank all those who have given generously and have been involved in raising funds to allow the continuation of our work. We are grateful for the award of a small annual grant by Perkin Elmer Ltd. to support the administration costs of the UK PANS group on Newborn Screening.We are also grateful to Sobi in the UK for financially supporting our Newborn Screening conference in January 2016.Save Babies UK, Annual Report 2015-16 12 5. Declaration The trustees declare that they have approved the trustees’ report above.Signed on behalf of the Charity’s trusteesSignature Full Name PATRICIA ANN ROBERTSPosition CHAIR OF THE BOARD OF TRUSTEES Date 4th September 2016Save Babies UK, Annual Report 2015-16 13

Save Babies Through Screening Foundation UK

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