• Biosimilars
  • Drug Development/R&D
  • All Topics
OHE OHE
Newsletter SignupSubscribe

News & Insights
  • News
  • Events
  • Insights
  • Bulletin
  • News
  • Events
  • Insights
  • Bulletin

News & Insights

  • News
  • Events
  • Insights
  • Bulletin
Newsletter SignupSubscribe
  • News
  • Events
  • Insights
  • Bulletin

Close
OHE OHE
  • Research & Publications
  • News & Insights
  • Education
  • Innovation Policy Prize
  • Events
  • About Us
  • OHE Experts
  • Contact Us
Newsletter SignupSubscribe

Research & Publications

All Publications

Filter by:
  • Antimicrobial Resistance (AMR)
  • Biosimilars
  • Cell and Gene Therapies
  • Chronic Diseases
  • Combination Therapies
  • COVID-19 Research
  • Digital Health
  • Drug Development/R&D
  • Emerging Markets
  • EQ-5D and PROMs
  • Health Care Systems
  • Health Data and Statistics
  • Health Technology Assessment
  • Precision Medicine
  • Real World Evidence
  • Use of Medicines
  • Value-Based Pricing
  • Vaccine Research
  • Economics of Innovation
  • Measuring and Valuing Outcomes
  • Policy, Organisation and Incentives in Health Systems
  • Value, Affordability and Decision Making

News & Insights

  • News
  • Events
  • Insights
  • Bulletin

Education

  • Education Hub
  • OHE Graduate School
  • EVIA Programme

Innovation Policy Prize

  • The Prize Fund
  • 2022 Prize Fund

Latest Research & Publications

Proposal for a General Outcome-based Value Attribution Framework for Combination Therapies

CombTher_Adobe_photoguns_portrait
Read more
© photoguns
  • Digital Health

Navigating the Landscape of Digital Health – United Kingdom

Healthcare_Adobe_elenabsl
Read more

2021 OHE Annual Report to the Charity Commission

charityreport_lina-trochez-unsplash_landscape
Read more
© Lina Trochez/Unsplash

Supporting the Era of Green Pharmaceuticals in the UK

Sustainability_AdobeStock_270582392_landscape
Read more

Quality of life and wellbeing in individuals with experience of fertility problems and assisted reproductive techniques

Quality of life assisted reproduction Cover
Read more
  • Cell and Gene Therapies
  • Value, Affordability, and…

Health Technology Assessment of Gene Therapies: Are Our Methods Fit for Purpose?

gene_therapies_national-cancer-institute-unsplash_landscape
Read more
© NCI/Unsplash
  • Drug Development/R&D
  • Economics of Innovation
  • Health Policy and Regulation

Limitations of CBO’s Simulation Model of New Drug Development as a Tool for Policymakers

CBO-US_mayer-tawfik-K4Ckc0AxgDI-unsplash_landscape
Read more
© Mayer Tawfik/Unsplash
  • Measuring and Valuing Outcomes

When Generic Measures Fail to Reflect What Matters to Patients: Three Case Studies

PROMS_unsplash_National Cancer Institute_landscape
Read more
© NCI/Unsplash
Close
OHE
  • All Publications

    Filter by:
    • Antimicrobial Resistance (AMR)
    • Biosimilars
    • Cell and Gene Therapies
    • Chronic Diseases
    • Combination Therapies
    • COVID-19 Research
    • Digital Health
    • Drug Development/R&D
    • Emerging Markets
    • EQ-5D and PROMs
    • Health Care Systems
    • Health Data and Statistics
    • Health Technology Assessment
    • Precision Medicine
    • Real World Evidence
    • Use of Medicines
    • Value-Based Pricing
    • Vaccine Research
    • Economics of Innovation
    • Measuring and Valuing Outcomes
    • Policy, Organisation and Incentives in Health Systems
    • Value, Affordability and Decision Making
    • News
    • Events
    • Insights
    • Bulletin
    • Education Hub
    • OHE Graduate School
    • EVIA Programme
    • The Prize Fund
    • 2022 Prize Fund
  • Events
  • About Us
  • OHE Experts
  • Contact Us
Newsletter SignupSubscribe
Back
  • News
11 min read 23rd August 2011

Recent Statistics on Orphan Approvals in Scotland and England

In October 2009, the OHE published research that compared access to orphan medicinal products (OMPs) in selected European countries. At the request of the UK Orphan Medicines Industry Group [1], OHE recently updated to May 2011 some of the data…

Share:
  •  Twitter
  •  LinkedIn
  •  Facebook
  • has-icon Email

In October 2009, the OHE published research that compared access to orphan medicinal products (OMPs) in selected European countries. At the request of the UK Orphan Medicines Industry Group [1], OHE recently updated to May 2011 some of the data included in that research.

In October 2009, the OHE published research that compared access to orphan medicinal products (OMPs) in selected European countries.  At the request of the UK Orphan Medicines Industry Group [1], OHE recently updated to May 2011 some of the data included in that research.  Using the same methodology, this new analysis focuses on decisions taken on orphan drugs by the Scottish Medicines Consortium (SMC) and the National Institute for Health and Clinical Excellence (NICE).  Included are the 74 orphan indications approved by the European Medicines Agency (EMA) up to May 2011.

Decisions by the SMC

The SMC has issued 55 decisions for the 74 orphan indications.  Because of resubmissions and reviews for some, a total of 69 decisions have been published.

As figure 1 shows, companies most often submitted full submissions for OMPs to the SMC.  Compared to non-orphan products, the proportion of non-submissions is slightly higher, perhaps because the expense of HTA may be prohibitive relative to the prospects for approval.  Abbreviated submissions were less frequent for OMPs, possibly because OMPs are more likely to be new molecules/active substances.

Figure 2 shows that rejection is more likely for orphan than for non-orphan indications. (Note that ‘not recommended’ includes products that were automatically rejected by SMC because the manufacturer did not make a submission to it.)  Of the total number of orphan rejections (43), 29% were due to non-submissions (12). For the remaining 31, the key reason for rejection stated in the SMC’s Detailed Advice Documents (DADs) was lack of economic evidence (‘economic case has not been demonstrated’); this means that the cost per QALY estimate was too high, or the model involved too much uncertainty, or no cost effectiveness model was provided. In 20 of these 43 rejected submissions, the clinical evidence was considered sufficient.  For the remaining 23 rejected submissions (more than half of the rejections), the level of clinical evidence available at the time of the review was considered by SMC to be inadequate.

Four of the decisions classified as ‘restricted’ in the DADs are effectively recommendations because the restriction coincides with the licence, i.e. the ‘restriction’ is that the medicine can be used only by a specialist in the relevant medical area.

To limit the analysis to one SMC decision per orphan indication, only the most recent submission is included in figure 3.  This shows approval was unrestricted for 10 orphan products and restricted for 14.  Thirty-one orphan products were not recommended.

As part of the submission, manufacturers supply details about the budgetary impact expected during the first and the fifth years after launch. These estimates may include the number of patients receiving treatment. Table 1 summarises statistics for patient numbers extracted from the 41 submissions that included this information.

Table 1. Range of reported patient numbers per indication (total indications = 41) Year 1 Year 5
Average across indications 29 43
Minimum 1 2
50 percentile 12 25
Maximum 179 179

NICE Decisions on OMPs[*]

NICE has assessed nine indications for orphan conditions:

  • azacitidine for mylelodyplastic synromes
  • imatinib for gastrointestinal stromal tumours and leukaemia
  • lenalidomide for multiple myeloma
  • sorafenib for RCC and hepatocellular cancer
  • temsirolimus for renal cell cancer (RCC)
  • trabectedin for ovarian cancer and soft tissue sarcoma

In four of the nine indications (temsirolimus and sorafenib, for both indications, and trabectedin for ovarian cancer), the overall decision was to not recommend use of the medicine. In three instances (imatinib, for both indications, and lenalidomide), the decision was to restrict use to a subgroup within the licensed indication. In two cases (trabectedin for soft tissue sarcoma and azacitidine), the treatment was recommended for use subject to a Patient Access Scheme.

Two NICE Technology Appraisals for other products were initiated, but not completed either because the NHS had already decided on treatment protocols (pulmonary arterial hypertension) or the drug (nilotinib) was considered in tandem with other treatments.

[*]This section was updated with new data on 9 January 2012.

Contact Martina Garau at the OHE with questions or for additional information.


[1] The Orphan Medicines Industry Group (OMIG) consists of orphan drug manufacturers that are members of the Association of the British Pharmaceutical Industry (ABPI).

 

  • Health Data and Statistics
  • Economics of Innovation
  • Innovation

Related News

Prize event
  • News
  • January 2023

Professor Aidan Hollis wins first £40,000 OHE Policy Innovation Prize

Read more
  • News
  • October 2020

Opportunities to Increase Efficiency in Healthcare

Read more
  • News
  • September 2020

Cornerstones of ‘Fair’ Drug Coverage

Read more
  • News
  • September 2020

Establishing a Reasonable Price for an Orphan Drug

Read more
footer_ohe_logo

Leading intellectual authority on global health economics

Sign Up for the OHE News Bulletin

Newsletter SignupStart Sign Up

Research & Publications

News & Insights

Innovation Policy Prize

Education

Events

About Us

OHE Experts

Contact Us

Sign Up for the OHE News Bulletin

Newsletter SignupStart Sign Up

The Office of Health Economics (OHE) is a company limited by guarantee registered in England and Wales (registered number 09848965) and its registered office is at 2nd Floor Goldings House, Hay’s Galleria, 2 Hay’s Lane, London, SE1 2HB.

Terms & Conditions

Privacy Policy

Cookies Policy

© 2023 Website Design

An error has occurred, please try again later.An error has occurred, please try again later.

We are using cookies to give you the best experience on our website.

You can find out more about which cookies we are using or switch them off in settings.

 Twitter
 Facebook
 LinkedIn
 Copy
 Email
Powered by  GDPR Cookie Compliance
Privacy Overview

This website uses cookies so that we can provide you with the best user experience possible. Cookie information is stored in your browser and performs functions such as recognising you when you return to our website and helping our team to understand which sections of the website you find most interesting and useful.

Strictly Necessary Cookies

Strictly Necessary Cookie should be enabled at all times so that we can save your preferences for cookie settings.

If you disable this cookie, we will not be able to save your preferences. This means that every time you visit this website you will need to enable or disable cookies again.

3rd Party Cookies

This website uses Google Analytics to collect anonymous information such as the number of visitors to the site, and the most popular pages.

Keeping this cookie enabled helps us to improve our website.

Please enable Strictly Necessary Cookies first so that we can save your preferences!