1-20 of 159 results for subject:"Orphan drugs"
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To ask His Majesty's Government what assessment they have made of the impact of the 2025 Highly Specialised Technologies routing criteria updates on the number of orphan medicines being diverted to the Standard Technology Appraisal (STA) pathway; and what steps they are taking to ensure that medicines for populations of...
To ask His Majesty's Government what assessment they have made of the impact of the 2025 Highly Specialised Technologies routing criteria updates on the number of orphan medicines being diverted to the Standard Technology Appraisal (STA) pathway; and what steps they are taking to ensure that medicines for populations of...
The National Institute for Health and Care Excellence (NICE) highly specialised technologies programme (HST) is reserved for the evaluation of a small number of medicines licensed for the treatment of very rare, very severe diseases. Decisions on whether medicines are routed to the HST programme are taken against a set of published criteria that were updated in April 2025 following public and stakeholder engagement. The purpose of the new criteria is not to change the number or nature of the topics evaluated through the HST programme, but to ensure that the criteria are sufficiently clear and predictable for companies and patient groups and are aligned to the HST vision.
The Government has recently announced a number of pilots and projects following the United States and Untied Kingdom partnership on pharmaceuticals that will shape a commercial environment that actively encourages innovation and improves patient access. As part of that work, NICE will review the approach to valuing rare disease medicines through its Health Technology Assessment Innovation Laboratory. This research will consider both technology appraisal and highly specialised technologies routes. The review will assess a range of both radical and incremental options to improve the current framework.
To ask His Majesty's Government what assessment they have made of the UK's fall in ranking from fifth to eleventh between 2020 and 2025 in the EFPIA Waiting to Access Innovative Therapies Indicator for orphan medicine availability; and what steps they are taking to ensure the UK remains a priority...
To ask His Majesty's Government what assessment they have made of the UK's fall in ranking from fifth to eleventh between 2020 and 2025 in the EFPIA Waiting to Access Innovative Therapies Indicator for orphan medicine availability; and what steps they are taking to ensure the UK remains a priority...
No specific assessment has been made. The Government recognises how important it is that patients with rare diseases are able to benefit from access to effective new medicines. The National Institute for Health and Care Excellence (NICE) has a strong track record in supporting access to new medicines for patients with rare diseases with 89% of the rare disease medicines that it has appraised since March 2024 recommended for some or all of the eligible patient population.
Through the Life Sciences Sector Plan and the 10-Year Health Plan, we are taking steps to make the United Kingdom a faster place to approve and adopt new medicines. This includes wider use of real-world evidence, more flexible commercial deals, faster NICE guidance, and a new joint NICE and Medicines and Healthcare products Regulatory Agency pathway to shorten the time from approval to National Health Service use.
We recognise that there is more to do to improve and accelerate access to new medicines for NHS patients and the steps that we are taking as part of the pharmaceuticals partnership with the United States are already resulting in medicines, including medicines for patients with rare diseases, becoming available to NHS patients that may not otherwise have been recommended.
To ask His Majesty's Government what assessment they have made of the barriers to patient access for orphan medicines that do not meet the population size criteria for the Highly Specialised Technologies pathway.
To ask His Majesty's Government what assessment they have made of the barriers to patient access for orphan medicines that do not meet the population size criteria for the Highly Specialised Technologies pathway.
To ask His Majesty's Government what assessment they have made of the effectiveness of the Standard Technology Appraisal (STA) pathway, in particular the STA methodology’s capacity to capture the clinical and societal value of orphan medicines which do not meet the population criteria for the Highly Specialised Technologies pathway.
To ask His Majesty's Government what assessment they have made of the effectiveness of the Standard Technology Appraisal (STA) pathway, in particular the STA methodology’s capacity to capture the clinical and societal value of orphan medicines which do not meet the population criteria for the Highly Specialised Technologies pathway.
To ask the Secretary of State for Health and Social Care, whether the review of orphan medicines regulation required by the Rare Cancers Act 2026 will consider the full pathway from regulatory authorisation to NHS patient access, including evidence generation, health technology assessment and uptake of treatments for rare cancers.
To ask the Secretary of State for Health and Social Care, whether the review of orphan medicines regulation required by the Rare Cancers Act 2026 will consider the full pathway from regulatory authorisation to NHS patient access, including evidence generation, health technology assessment and uptake of treatments for rare cancers.
To ask His Majesty's Government, further to the Written Answer by Baroness Merron on 11 March (HL15195), what assessment they have made of the suitability of conventional cost-effectiveness assessments for medicines where structural evidence constraints arising from small patient populations cannot be resolved.
To ask His Majesty's Government, further to the Written Answer by Baroness Merron on 11 March (HL15195), what assessment they have made of the suitability of conventional cost-effectiveness assessments for medicines where structural evidence constraints arising from small patient populations cannot be resolved.
The National Institute for Health and Care Excellence (NICE) is the independent body that makes recommendations for the National Health Service on whether new medicines should be routinely funded by the NHS based on an assessment of clinical and cost effectiveness. NICE’s methods have been proven to be suitable for evaluating rare disease medicines where companies engage with the process and set prices for medicines that fairly reflect their clinical benefits.
NICE can recommend promising new non-cancer medicines for use through the Innovative Medicines Fund (IMF) where there is clinical uncertainty that may be resolved through the collection of real-world evidence. The IMF provides time limited access to innovative non-cancer medicines while further evidence is generated to address uncertainties identified by the NICE appraisal committee. Following a managed access period of up to five years, NICE reviews the updated evidence to determine whether the medicine should be routinely commissioned by the NHS.
The NICE methods manual allows its independent committees to apply flexibilities in circumstances where evidence generation is particularly difficult, for example in rare disease, technologies for use predominantly in children, and innovative and complex technologies. In these specific circumstances, the committee may be able to make recommendations accepting a higher degree of uncertainty. The committee will consider how the nature of the condition or technology affects the ability to generate high-quality evidence before applying greater flexibility.
To ask His Majesty's Government, further to the Written Answer by Baroness Merron on 11 March (HL15195), what assessment they have made of the suitability of conventional cost-effectiveness assessments for medicines where structural evidence constraints arising from small patient populations cannot be resolved.
To ask His Majesty's Government, further to the Written Answer by Baroness Merron on 11 March (HL15195), what assessment they have made of the suitability of conventional cost-effectiveness assessments for medicines where structural evidence constraints arising from small patient populations cannot be resolved.
To ask His Majesty's Government what assessment they have made of the finding from the report by the European Federation of Pharmaceutical Industries and Associations, Patients W.A.I.T. Indicator 2024 Survey, published in May 2025, that only 50 per cent of European Medicines Agency approved non-oncology orphan medicines were reimbursed and made...
To ask His Majesty's Government what assessment they have made of the finding from the report by the European Federation of Pharmaceutical Industries and Associations, Patients W.A.I.T. Indicator 2024 Survey, published in May 2025, that only 50 per cent of European Medicines Agency approved non-oncology orphan medicines were reimbursed and made...
The Government recognises how important it is that patients with rare diseases can benefit from access to effective new medicines.
The National Institute for Health and Care Excellence (NICE) is able to recommend the vast majority of medicines it evaluates for use in the National Health Service, including medicines for the treatment of rare diseases. The recently announced increase to the cost-effectiveness threshold will, alongside measures announced in the Life Sciences Sector Plan, increase both the speed and breadth of patient access to innovative medicines.
To ask His Majesty's Government what assessment they have made of the recommendation in the report by the UK BioIndustry Association From innovation to impact: unlocking patient access to innovative rare disease medicines, published on 4 December 2025, to develop a separate evaluation pathway for innovative orphan medicines where a...
To ask His Majesty's Government what assessment they have made of the recommendation in the report by the UK BioIndustry Association From innovation to impact: unlocking patient access to innovative rare disease medicines, published on 4 December 2025, to develop a separate evaluation pathway for innovative orphan medicines where a...
There are no plans to introduce a separate evaluation process for orphan medicines. Most medicines, including orphan medicines, are assessed through the National Institute for Health and Care Excellence’s (NICE) standard technology appraisal programme, with a small number of treatments for very rare and severe conditions considered through the highly specialised technologies programme, which applies a higher cost-effectiveness threshold.
NICE’s methods are suitable for evaluating rare disease medicines where prices are set fairly. Approval rates for rare disease medicines are in line with overall NICE recommendations, and between April 2024 and April 2025 NICE recommended all 15 rare disease medicines assessed through the standard programme.
We are also investing approximately 25% more in innovative treatments through an increase to NICE’s cost-effectiveness threshold and changes to how health benefits are valued. This will support access to medicines delivering significant health benefits, including for rare diseases, that may previously have been declined on cost-effectiveness grounds.
Lords second reading. Agreed to on question. Bill committed to a Committee of the Whole House.
Lords second reading. Agreed to on question. Bill committed to a Committee of the Whole House.
My Lords, it is a great honour to be opening the debate on the Rare Cancers Bill today. I thank Dr Scott Arthur, the Member of Parliament for Edinburgh South West, who, when he drew number six in the Private Members’
Bill ballot in autumn 2024, chose this crucially important...
My Lords, it is a great honour to be opening the debate on the Rare Cancers Bill today. I thank Dr Scott Arthur, the Member of Parliament for Edinburgh South West, who, when he drew number six in the Private Members’
Bill ballot in autumn 2024, chose this crucially important...
My Lords, it would be rare for any noble Lord in this House not toknow someone affected by cancer. In the UK, nearly 500 people die from cancer every day. Those figures are not abstract, they represent personal lives: families, friendships and futures cut short. I commend the noble Baroness,...
My Lords, it would be rare for any noble Lord in this House not toknow someone affected by cancer. In the UK, nearly 500 people die from cancer every day. Those figures are not abstract, they represent personal lives: families, friendships and futures cut short. I commend the noble Baroness,...
I thank the sponsors of this Bill, Dr Scott Arthur and the noble Baroness, Lady Elliott of Whitburn Bay, for bringing it to this House. Noble Lords must excuse me if I am a bit emotional after listening to the stories, because I too have experienced grief recently due to...
I thank the sponsors of this Bill, Dr Scott Arthur and the noble Baroness, Lady Elliott of Whitburn Bay, for bringing it to this House. Noble Lords must excuse me if I am a bit emotional after listening to the stories, because I too have experienced grief recently due to...
My Lords, before beginning my speech, I draw attention to my entry in the register of interests, particularly my involvement with Cambridge University Health Partners, Newmarket Strategy and Health Data Research UK, and in particular as a patron and adviser of the Tessa Jowell Brain Cancer Mission, of which more...
My Lords, before beginning my speech, I draw attention to my entry in the register of interests, particularly my involvement with Cambridge University Health Partners, Newmarket Strategy and Health Data Research UK, and in particular as a patron and adviser of the Tessa Jowell Brain Cancer Mission, of which more...
My Lords, may I take one minute of the House’s time? I commend what was just said by the noble Lord, whom I was pleased to work with many years ago, when he was the Minister. I am very pleased that he recalled the last contribution that my long-standing friend,...
My Lords, may I take one minute of the House’s time? I commend what was just said by the noble Lord, whom I was pleased to work with many years ago, when he was the Minister. I am very pleased that he recalled the last contribution that my long-standing friend,...
My apologies.
10.40 am
My apologies.
10.40 am
My Lords, I am sure that, not only in this House but across the country, people are grateful to Scott Arthur MP and the noble Baroness, Lady Elliott of Whitburn Bay, for introducing this incredibly important Bill.
I declare that my working life has been in palliative care. I have looked...
My Lords, I am sure that, not only in this House but across the country, people are grateful to Scott Arthur MP and the noble Baroness, Lady Elliott of Whitburn Bay, for introducing this incredibly important Bill.
I declare that my working life has been in palliative care. I have looked...
My Lords, it is a great pleasure to follow the noble Baroness, Lady Finlay, who always speaks with authority on these issues. I particularly agree with her and the noble Lord, Lord Patel, on the timescales; anything that can be done to compress them would be very welcome.
I associate myself...
My Lords, it is a great pleasure to follow the noble Baroness, Lady Finlay, who always speaks with authority on these issues. I particularly agree with her and the noble Lord, Lord Patel, on the timescales; anything that can be done to compress them would be very welcome.
I associate myself...
My Lords, I am absolutely delighted to take part in this Second Reading debate. Although we are only a third of the way through the speakers’ list, this has already been a very moving—as well as extremely well-informed, interesting and expert—debate. Perhaps it shows the House of Lords at its...
My Lords, I am absolutely delighted to take part in this Second Reading debate. Although we are only a third of the way through the speakers’ list, this has already been a very moving—as well as extremely well-informed, interesting and expert—debate. Perhaps it shows the House of Lords at its...
My Lords, it is a great pleasure to follow the noble Baroness in her contribution this morning. This short but very important Bill was brought from the Commons in July last year. Many of us feel we have waited a very long time for it to appear on the Floor...
My Lords, it is a great pleasure to follow the noble Baroness in her contribution this morning. This short but very important Bill was brought from the Commons in July last year. Many of us feel we have waited a very long time for it to appear on the Floor...