Open letter to prime minister David Cameron and health secretary Andrew Lansley

  title={Open letter to prime minister David Cameron and health secretary Andrew Lansley},
  author={David Joseph Nicholl and David Hilton-Jones and Jacqueline A Palace and Sam Richmond and Sara Finlayson and John B. Winer and Andrew Weir and Paul Maddison and Nick Fletcher and Jon Sussman and Nicky Silver and John Nixon and Dimitri Michael Kullmann and Nick Embleton and David Beeson and Maria Elena Farrugia and Marguerite Hill and C. Mcdermott and Gareth Llewelyn and James Leonard and Michael Morris},
  journal={BMJ : British Medical Journal},
Neurologists and paediatricians call for action on “massive” rises in the prices of orphan drugs 
Viridian Pharma replies
  • T. Rockley
  • Medicine
    BMJ : British Medical Journal
  • 2010
In their open letter Nicholl and colleagues say that caffeine treatment for apnoea of prematurity has been subject to “massive” price rises in …
Too early to question effectiveness of Dutch system
The study by Evers et al is the first to show a higher mortality among births that started in primary care compared with secondary care.1 We have concerns about the methods used. Firstly, although
Ultra orphan drugs: the NHS model for managing extremely rare diseases
The concept of ultra orphan disease grew out of the English system for planning and organising health services, but is likely to be of increasing interest to planners and funders in other health systems struggling to make decisions about very high cost therapies for very rare disease.
Regulation is flawed
  • C. Counsell
  • Political Science
    BMJ : British Medical Journal
  • 2010
Something is fundamentally wrong with a drug regulatory system that makes it increasingly difficult to do independent pragmatic trials of widely used but unproven drugs yet licenses a ridiculously
BioMarin Europe replies
There are inaccuracies in the open letter of Nicholl and colleagues that cannot be substantiated by evidence since 3,4-DAP has not undergone systematic safety and toxicology testing nor does it have appropriate safety monitoring and pharmacovigilance systems in place to capture, analyse, and report such data.
Rare diseases and orphan drugs.
This paper illustrates mainly the European initiatives and will discuss the problematic and controversial aspects surrounding orphan drugs, as well as activities and measures adopted in Italy.
Contratos de riesgo compartido, ¿con medicamentos huérfanos?
If thoroughly managed, RSA may reasonably contribute to value-based pricing of OD, improve their pharmacovigilance, knowledge about their comparative effectiveness, and to reducing uncertainty and its consequences on patients, industry, payers and clinicians.
Estimating the budget impact of orphan medicines in Europe: 2010 - 2020
Although European orphan drug legislation has led to an increase in the number of approved orphan drugs, the growth in cost, as a proportion of total pharmaceutical expenditure, is likely to plateau over the next decade as orphan growth rates converge on those in the broader pharmaceutical market.
Amifampridine tablets for the treatment of Lambert-Eaton myasthenic syndrome
  • R. Mantegazza
  • Medicine, Biology
    Expert review of clinical pharmacology
  • 2019
The present review is focused on the use of Amifampridine Phosphate to treat LEMS patients due to its efficacy, safety and reliable GMP formulation.
Paying for the Orphan Drug System: break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?
The authors propose the development of a new assessment system based on several evaluation criteria, which would serve as a tool for Member State governments to evaluate each new orphan drug at the time of pricing and reimbursement.


Pricing of orphan drugs