Pharmaceutical Market Europe • January 2024 • 8-9

NEWS

New terms for UK medicines scheme send ‘mixed message’ to life sciences industry

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The Department of Health and Social Care has published new terms for the UK’s Statutory Scheme for branded medicines.

The terms follow a warning from over 20 life sciences leaders against plans to change the scheme, stating that they are ‘unworkable’ and could be ‘highly damaging’ to UK life sciences and NHS patient access to medicines.

The publication also follows the announcement of a new Voluntary Scheme for Pricing, Access and Growth – a negotiated alternative scheme for companies to opt into over the Statutory Scheme.

The new scheme will require companies to pay a rebate on the sales of branded medicines to the NHS of 21.9% in 2024, 24% in 2025 and 26.8% in 2026.

Despite rebate rates being lower than 2023 (27.5%), they remain significantly higher than the historical average of 10.6% prior to 2023.

In comparison to other similar mechanisms operating in other countries, the UK’s statutory rebate rate is significantly higher, with rebate rates of 12% in Germany, 7.5% in Spain and 8.25% in Ireland.

Although a majority of 102 respondents have rejected the plans, the government has continued to move forward with the Statutory Scheme but will revoke one of the most controversial proposals in the scheme, the Life Cycle Adjustment.


FDA establishes advisory committee for genetic metabolic disease treatments

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The US Food and Drug Administration (FDA) has announced the establishment of a new advisory committee to evaluate potential treatments for genetic metabolic diseases.

The Genetic Metabolic Diseases Advisory Committee will advise the FDA on products used for the diagnosis, prevention and treatment of genetic metabolic diseases under the Division of Rare Diseases and Medical Genetics, established in 2020.

Genetic metabolic diseases are conditions that disrupt an individual’s metabolism, which is the mechanism responsible for transferring food into energy and removing toxins from the body.

Most of these types of diseases are rare, including Hurler syndrome, Gaucher disease and Tay-Sachs disease.

The committee will provide FDA-independent, knowledgeable advice and recommendations on technical, scientific and policy issues in relation to medical products for these diseases.

With nine voting members, including a chairperson, the committee will include experts in multiple areas of metabolic genetics such as carbohydrate disorders, amino acid disorders and organic acidaemias.

Experts in the management of inborn errors of metabolism, small population trial design, translational science, paediatrics, epidemiology or statistics and related specialties will also be involved.

All individuals nominated as scientific members of the committee must be technically qualified experts in their relevant fields and have experience interpreting complex data.


EMA and HMAs publish workplan to guide use of AI in medicines regulation

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The European Medicines Agency (EMA) and the Heads of Medicines Agencies (HMAs) have published an artificial intelligence (AI) workplan to guide the use of AI in medicines regulation until 2028.

The workplan aims to set out a collaborative and coordinated approach to maximise the benefits of AI to stakeholders while managing the risks.

Pharmaceutical companies are increasingly using AI-powered tools in research, development and to monitor medicines.

Prepared under the joint HMA-EMA Big Data Steering Group (BDSG), the Multi-annual AI workplan 2023-2028 will help the European Medicines Regulatory Network (EMRN) utilise AI for personal productivity, automate processes and systems, increase insights into data and support more robust decision-making to benefit public and animal health.

Additionally, the workplan will ensure that the EMRN will continue to benefit from AI in medicines regulation.

Focusing on four key dimensions: guidance, policy and product support; AI tools and technology; collaboration and training; and experimentation, the work plan addresses the new opportunities and challenges of starting to use and develop AI tools.

The BDSG will continue to regularly update the AI workplan and regulators, medicine developers, academics, patient organisations and other parties will be updated throughout the implementation of the plan.


Study identifies new amyloid-forming protein linked to early-onset dementia

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A study led by the Medical Research Council’s (MRC) Laboratory of Molecular Biology (LMB) has identified a new protein as a potential therapeutic target for frontotemporal dementia, a type of early-onset dementia.

Published in Nature, MRC LMB researchers successfully pinpointed the aggregated structures of the TAF15 protein using cutting-edge cryo-electron microscopy to study the brains of four patients who had this type of dementia.

The FUS protein is already known to be associated with frontotemporal dementia. However, researchers were surprised to identify the TAF15 protein in the four brains.

This type of early-onset dementia can be seen in patients with motor neurone disease (MND).

Researchers studied the brains of two patients who had signs of both diseases and identified the same aggregated structure of TAF15 in brain regions associated with MND.

The identification of these proteins and the basic structures of these filaments in this form of early-onset dementia could lead to the development of early diagnostic tests and drugs to combat their formation.

The study was funded by the MRC, along with Alzheimer’s Research UK, the US National Institute of Health, the Alzheimer’s Society, the Association for Frontotemporal Degeneration, the Swiss National Science Foundation and the Levehulme Trust.


CAR-T therapy shown to improve symptoms of autoimmune diseases

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Results from a study led by the University Hospital Erlangen in Germany have revealed that CAR-T therapy significantly improved the symptoms of autoimmune diseases or made them go away entirely.

First conducted in 2021, previous findings from five patients with systematic lupus erythematosus (SLE) showed promising results after they all went into remission.

Researchers tested whether CD19 CAR-T cells achieved a deeper reset of B cells, which trigger autoimmune diseases, eradicate autoimmunity and achieve lasting drug-free remission in autoantibody-dependent AID.

The study enrolled a further eight patients living with SLE, four with systemic sclerosis and three with inflammatory myositis (IIM), who were no longer responding to multiple standard treatments.

Within a week of receiving CAR-T infusions, researchers found that the patients’ B cells were eliminated from the blood and then reoccurred in most patients within a few months.

Additionally, all 15 patients were able to stop taking the immune-suppressive drugs to reduce the symptoms of their disease and all three IIM patients went into remission after three months, substantially reducing the activity of their disease.

Researchers have said that further research is required, with a larger group of patients, to determine whether the CAR-T therapy will have long-term benefits for patients living with chronic autoimmune diseases.


Stem cell therapy trial shows promising results for treating progressive MS

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Scientists at the University of Cambridge, University of Milan Bicocca and Hospital Casa Solievo della Sofferenza in Italy have shared promising results from an early-stage stem cell therapy trial in progressive multiple sclerosis (MS).

In a study published in Cell Stem Cell, scientists successfully injected neural stem cells directly into the brains of 15 patients with secondary MS, recruited from two hospitals in Italy.

The stem cells were taken from the brain tissue cells of a single, miscarried foetal donor, and after a 12-month period, researchers observed that no treatment-related deaths or serious adverse events had occurred and that side effects were temporary or reversible.

Additionally, none of the patients involved in the study showed an increased disability or worsening of symptoms, or symptoms that suggested a relapse and worsening of cognitive function.

After assessing volume changes in the brain tissue associated with MS in a subgroup of patients, researchers found that larger doses of injected stem cells led to smaller reductions in brain volume over time.

They also discovered that changes in the fluid around the brain and blood over time were linked to how the brain produced fatty acids. This was connected to how well the treatment worked and how the disease developed.

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