Pricing and reimbursement of orphan drugs: the need for more transparency

Steven Simoens
Orphanet Journal of Rare Diseases 2011, 6:42doi:10.1186/1750-1172-6-42
Published: 17 June 2011

OPEN ACCES

Abstract (provisional)

Pricing and reimbursement of orphan drugs are an issue of high priority for policy makers, legislators, health care professionals, industry leaders, academics and patients. This study aims to conduct a literature review to provide insight into the drivers of orphan drug pricing and reimbursement. Although orphan drug pricing follows the same economic logic as drug pricing in general, the monopolistic power of orphan drugs results in high prices: a) orphan drugs benefit from a period of marketing exclusivity; b) few alternative health technologies are available; c) third-party payers and patients have limited negotiating power; d) manufacturers attempt to maximise orphan drug prices within the constraints of domestic pricing and reimbursement policies; and e) substantial R&D costs need to be recouped from a small number of patients. Although these conditions apply to some orphan drugs, they do not apply to all orphan drugs. Indeed, the small number of patients treated with an orphan drug and the limited economic viability of orphan drugs can be questioned in a number of cases. Additionally, manufacturers have an incentive to game the system by artificially creating monopolistic market conditions. Given their high price for an often modest effectiveness, orphan drugs are unlikely to provide value for money. However, additional criteria are used to inform reimbursement decisions in some countries. These criteria may include: the seriousness of the disease; the availability of other therapies to treat the disease; and the cost to the patient if the medicine is not reimbursed. Therefore, the maximum cost per unit of outcome that a health care payer is willing to pay for a drug could be set higher for orphan drugs to which society attaches a high social value. There is a need for a transparent and evidence-based approach towards orphan drug pricing and reimbursement. Such an approach should be targeted at demonstrating the relative effectiveness, cost-effectiveness and economic viability of orphan drugs with a view to informing pricing and reimbursement decisions.

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Neurodegeneration in friedreich's ataxia is associated with a mixed activation pattern of the brain. A fMRI study.

Hum Brain Mapp. 2011 Jun 14. doi: 10.1002/hbm.21319. [Epub ahead of print]

Ginestroni A, Diciotti S, Cecchi P, Pesaresi I, Tessa C, Giannelli M, Nave RD, Salvatore E, Salvi F, Dotti MT, Piacentini S, Soricelli A, Cosottini M, De Stefano N, Mascalchi M.
Department of Clinical Physiopathology, Radiodiagnostic Section, University of Florence, Florence, Italy.

Keywords: Friedreich's ataxia (FRDA), neurodegeneration, spinal cord, brain, MR Imaging (fMRI), left primary sensory-motor cortex, right cerebellum, left thalamus, right dorsolateral prefrontal cortex, globus pallidus, anterior cingulum, globus pallidus, regional neuronal damage, compensatory significance.

Friedreich’s ataxia variants I154F and W155R diminish frataxin-based activation of the iron-sulfur cluster assembly complex

Biochemistry, Just Accepted Manuscript, DOI: 10.1021/bi200666h
Publication Date (Web): June 14, 2011

Chi-Lin Tsai , Jennifer Bridwell-Rabb , and David P. Barondeau

Keywords: Friedreich’s ataxia (FRDA), frataxin (Fxn), GAA expansion, missense mutation, FRDA I154F, W155R variants, Nfs1, Isd11, Isu2, Fe-S cluster assembly machine, Fxn triggers sulfur transfer from Nfs1 to Isu.

New project: Identification of the E3 ligase that ubiquitinates frataxin

Principal researcher: Dr Roberto Testi, Department of Experimental Medicine,
University of Rome, ‘Tor Vergata,’ Italy

Keywods: frataxin, ubiquitin-proteasome system, ubiquitination, E3 ligase.

Sherman ku PhD working with friedreich's ataxia and induced pluripotent stem cells (iPSC)

Sigma bioblogs (3 Jun 2011)
Sherman Ku PhD interview
The Scripps Research Institute, Member of the Gottesfeld Lab

What is the focus of your research?
My research has focused on developing an induced pluripotent stem cell (iPSC) model of Friedreich’s ataxia (FRDA), which is ..... read more....

Edison Pharmaceuticals Announces Results of EPI-A0001 Phase 2A Double Blind Placebo Controlled 28-Day Clinical Trial in the Mitochondrial Disease- Friedreich's Ataxia

10th of June 2011

MOUNTAIN VIEW, Calif., June 10, 2011 /PRNewswire/ -- Edison Pharmaceuticals, Inc. announced today preliminary results obtained on a 28-day phase 2A clinical trial in Friedreich's ataxia.

EPI-A0001 did significantly improve neurological function as assessed by the Friedreich's Ataxia Rating Scale (FARS).

Read more

Emergency Use Protocol for EPI-743 in Acutely Ill Patients With Inherited Mitochondrial Respiratory Chain Disease Within 90 Days of End-of-Life Care

This study is currently recruiting participants.

First Received on June 7, 2011. Last Updated on June 9, 2011
Sponsor: Edison Pharmaceuticals Inc
Information provided by: Edison Pharmaceuticals Inc
ClinicalTrials.gov Identifier: NCT01370447


Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment

Transcranial Sonography Reveals Cerebellar, Nigral, and Forebrain Abnormalities in Friedreich’s Ataxia

Neurodegenerative Dis, (DOI: 10.1159/000327751)

Matthis Synofzik, Jana Godau, Tobias Lindig, Ludger Schöls, Daniela Berg
Department of Neurodegenerative Diseases, Hertie Institute for Clinical Brain Research, and German Research Center for Neurodegenerative Diseases, University of Tübingen, Tübingen, Germany

Keywords: Ataxia, Transcranial sonography, Friedreich’s ataxia, Imaging, Substantia nigra, Dentate nucleus, Iron, Cerebellum

Mortality in Friedreich Ataxia

Journal of the Neurological Sciences,Article in Press,
doi:10.1016/j.jns.2011.05.023

(J Neurol Sci. 2011 Aug 15;307(1-2):46-9. Epub 2011 Jun 8.)

Amy Y. Tsoua, Erin K. Paulsena, b, c, d, Sarah J. Lagedrosta, b, c, d, Susan L. Perlmane, Katherine D. Mathewsf, George R. Wilmotg, Bernard Ravinah, Arnulf H. Koeppeni, j and David R. Lyncha, b, c, d,

a Department of Neurology, University of Pennsylvania Medical School, United States
b Department of Pediatrics, University of Pennsylvania Medical School, United States
c Children's Hospital of Philadelphia, United States
d University of Pennsylvania Medical School, United States
e University of California Los Angeles, Los Angeles, CA, United States
f University of Iowa, Iowa City, IA, United States
g Emory University, Atlanta, GA, United States
h University of Rochester, Rochester, NY, United States
i VA Hospital, Albany, NY, United States

Keywords: Cerebellum; Sensory; Cardiomyopathy; Echocardiogram; Arrhythmia; Neurodegenerative disease

Repligen Reports Fourth Quarter ........ "RG2833 for Friedreich’s Ataxia"

9th of June 2011

RG2833 for Friedreich’s Ataxia
We are currently developing histone deacetylase class 1 inhibitors for the treatment of inherited neurodegenerative diseases such as Friedreich's ataxia. Friedreich's ataxia is caused by inadequate production of the protein frataxin which leads to degeneration of the nerves controlling muscle movements. Pending regulatory approval, we plan to initiate a Phase 1 study of RG2833 in Friedreich's ataxia patients in Europe by the end of the year. We are currently engaged in discussions with potential
pharmaceutical partners to evaluate whether partnering this program would increase the trajectory for global development and ultimately maximize the commercial potential of the asset.