Soleno Therapeutics, the pharmaceutical company behind the newly FDA-approved hyperphagia treatment, VYKAT XR, is conducting a groundbreaking ‘Burden of Illness Study.’ This study aims to provide vital evidence illustrating the medical, emotional, and financial impact of Prader-Willi Syndrome (PWS) on patients, their families, and the healthcare system.
Why do we need this study?
The global PWS community celebrated the FDA’s approval of VYKAT XR, marking an exciting milestone in the treatment of hyperphagia. With this approval, other countries, including the UK, are now taking steps to make the drug accessible to their PWS patients.
In the UK, approval by the MHRA (Medicines and Healthcare products Regulatory Agency) is the first step. The MHRA’s review focuses on the scientific evidence—safety, effectiveness, and functionality of the drug. However, for the NHS to provide this treatment, it must be clear that the drug is both essential and worth the cost. Medicines often come with high price tags, but so do the extensive costs of managing PWS. This study aims to provide evidence to show this when making the relevant applications.
What is the cost of PWS without treatments?
PWS influences family quality of life, emotional and physical well-being, social relationships, and day-to-day interactions. However, in addition to these social and emotional tolls, the financial burden of PWS can be significant. Beyond the direct healthcare costs of PWS such as time with professionals, tests, procedures etc, there are also indirect expenses like travel to appointments, medical equipment, lost earnings for caregivers, home adaptations for food security or mobility, and adult care costs. This study will be evidence of this and therefore present a full picture of the challenges faced by families and the healthcare system alike.
In the case of the UK, this is what we need to show when seeking approval from NICE for England and the other relevant bodies for the Home Countries. They are being asked to provide (which essentially means pay for) a drug, so they also need to be presented with the costs faced by families, the state and the healthcare system without this drug to measure the financial justifications of making this medication available to PWS Patients in the UK.
How the Study Works
The study involves two questionnaires—one for healthcare professionals and another for patients and caregivers. The healthcare professionals’ questionnaire focuses on direct medical costs, such as hospital stays, procedures, and treatments. Meanwhile, the caregiver questionnaire explores other expenses, including travel costs, loss of earnings, care expenses, home modifications, and more.
The study spans the USA, Germany, France, Italy, and the UK, with a select number of participants in each country. In the UK, 12 clinicians were chosen to each recruit 4-6 patients to participate. Once patients consent, clinicians complete their portion of the study while caregivers fill out their questionnaire at home—online, at their convenience, and in about 30 minutes.
Timeline and What You Can Do
Clinicians were approached in early February, and data collection is expected to conclude by late April or early May. The results will be analysed and compiled into a draft paper later in the year for publication in peer-reviewed journals.
How can you help? While clinicians for this study have already been selected, it’s worthwhile to ask your PWS specialist if they are involved and, if so, encourage their participation. If you or your loved one is approached to join the study, we strongly encourage you to consider taking part. Your participation could contribute to a body of evidence that will strengthen the case for greater access to essential treatments.
Why This Study Matters
This study plays a pivotal role in the journey to make VYKAT XR accessible to patients outside of the USA, including here in the UK.
Participating in the study is not just an opportunity to contribute to research—it’s a chance to champion access to essential care and improve lives.
We look forward to sharing the findings of this study and celebrating the progress it enables for PWS patients and families.