|The National MPS Society is pleased to bring a special opportunity to our supporters who develop and administer clinical trials.|
This year, at the WORLDSymposium, a panel of experts in neurocognition held a special session to discuss the urgent need to develop and implement precise metrics for quantitating change in children with lysosomal and other neurological diseases. “Precision Metrics for Cognitive and Adaptive Measures in Clinical Trials” featured an explanation and rationale for using growth scale values (GSVs) to measure longitudinal cognitive and adaptive changes in early onset and severe neurological diseases. These scores are available for tests that are widely used in clinical trials such as the Bayley and the Vineland.
We know this topic is important to your work. To facilitate a wider dissemination of the symposium we are hosting a live follow-up Q&A session with the panel on March 17, Noon, EST. Registering for this webinar will allow you access to the symposium recordings.
Clinical trial development could not happen without the strong partnership between academia and industry. To support this collaboration, there is no cost to attend or to access the recordings.
Click to register, and please feel free to share with others on your team who may be interested.
Terri Klein, MPA
President & CEO
National MPS Society
If you are interested but can not attend the live webinar, register and we will send you a link to the recording.
SAVE THE DATE! — Join us June 19-20 and 26-27 for our 35th Annual Family Conference! Taking place virtually, this conference will focus around the theme of remaining connected and the overlap within diagnoses, but highlight and recognize the uniqueness of each through individualized sessions for each syndrome type. The conference features clinicians, scientists, researchers, FDA representatives, advocacy, family support, and much more. More information here, registration information coming soon!
The National MPS Society USA has posted recommendations for patients and families concerned about COVID-19.
The Board of Trustees of the Society for Mucopolysaccharide Diseases in the United Kingdom (the “MPS Society”) has appointed Bob Stevens as its new CEO. Mr. Stevens was already working with Christine Lavery prior to her unexpected death, on the task of becoming prepared to lead the MPS Society after her retirement. Additionally, he has been a Trustee of The MPS Society for over 10 years. Mr. Stevens has two sons who have MPS II.
In making this appointment, Paul Moody, Chairman of the Board of Trustees of the MPS Society, wrote: “The Board of Trustees have now acted decisively and quickly in implementing not only what we believe were Christine’s wishes, but also the unanimous wishes of the board of trustees.” Mr. Stevens is also the CEO of the Society’s “MPS Commercial,” its unit that works to help the pharmaceutical industry successfully perform clinical trials for the MPS diseases. MPS Commercial is a wholly-owned, not-for-profit subsidiary of the Society, whose social objectives are to invest any profits into the MPS community for the purposes of education, enhancing needs-led advocacy support, quality-of-life research and scientific research.
Mrs. Christine Lavery MBE, a founder and the Chief Executive of the Society for Mucopolysaccharide Diseases (MPS Society) in the UK, died on Tuesday, December 19, 2017, following a brief illness. The MPS Society stated, “Christine has tirelessly championed the MPS Society from its very conception in the early 1980s up until her untimely death. She was a formidable lady who cared passionately for every MPS Society member, past and present. Her efforts saw her work with patients, families and professionals all over the world as she dedicated her life to improving the knowledge, advocacy support and clinical outlook for patients with MPS. We are all deeply saddened at the tragic loss of her life.” The MPS Society has announced the Christine Lavery Memorial Day at Childhood Wood, Sherwood Pines, Nottinghamshire, UK, on Saturday, June 30, 2018. To receive more details about this as they emerge, register for updates.
Mrs. Lavery was awarded an MBE for her services to metabolic diseases by HM Queen Elizabeth II in the New Year’s Honors List for 2002. At the 2006 International Symposium on Mucopolysaccharide and Related Diseases she received a ‘lifetime award’ from the International MPS Community. WORLDSymposium™ awarded Christine Lavery the 2017 Patient Advocate Leader (PAL) Award.
Mrs. Lavery was the mother of Simon Lavery, a child who had MPS II. There are some brief biographical highlights of Christine Lavery’s life at the Brains for Brain Foundation site, under the paragraph-heading “Christine Lavery Short Biography.” Prior to serving the lysosomal disease community, Mrs. Lavery volunteered for Save the Children and the International Year of the Child. That short biography reveals a lifetime of focused dedication to improving the lives of those with special needs. She is deeply missed throughout the world.
Christine Lavery, MBE, Group Chief Executive of the MPS Society in the United Kingdom, has sounded the alarm about the National Institute for Health and Care Excellence’s recently-announced plan to change the arrangements for evaluating and funding drugs and other healthcare technologies assessed through NICE’s Highly Specialized Technologies (“HST”) appraisal. From April 1, 2017, NICE plans on the introduction of a £100,000 quality-adjusted life year (“QALY”) threshold for medicines evaluated via NICE’s HST program, which assesses treatments for ultra-rare diseases.
The MPS Society in the UK has responded to this plan by pointing out that this threshold will effectively stop the flow of new medicines reaching patients with ultra-rare and complex diseases. Many treatments for ultra-rare conditions that are currently funded by NHS England have costs per QALY of more than £500,000, including the three medicines that have been approved by NICE’s HST process to date. The MPS Society states that QALY thresholds are not appropriate for evaluating medicines for ultra-rare diseases, due to the small patient populations, and often limited data.
Christine Lavery said, “Being born with an ultra-rare disease, a disease affecting less than 110 people in England, is not a lifestyle choice; it is no one’s fault; it happens albeit very rarely; it happened to my son. At that time, there was no treatment and Simon died aged 7 years. I can only imagine now how it might be, to be faced with a child with an ultra-rare disease who could be treated with a highly-specialized medicine, but is denied treatment on cost grounds. The pain for the family of seeing their child condemned to death by Andrew Dillon, Chief Executive of NICE; Simon Stevens, Chief Executive of NHS England; and the UK Government, is unimaginable. Let us also be clear to Members of Parliament, many of the babies and children who will be affected by this catastrophic decision are ‘yet to be born or diagnosed’ members of your constituencies.”
MPS Society Chairman Paul Moody said, “A decision by NICE and NHS England to implement this new policy . . . will affect the most vulnerable in UK society, and confirms that children and young adults with ultra-rare diseases going forward are economic pawns in a failing NHS, and cheaper dead than alive. The UK government now needs to act at lightning speed and reverse this initial NICE & NHS England policy, not just in the context of patients with ultra-rare diseases, but also that of the life sciences industry, who will see no incentive to investing in the UK market if their innovative medicines and technologies have no prospect of reaching the patient.”