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Theravance Biopharma Releases Clinical Trial Findings on Symptomatic nOH Treatment

Theravance Biopharma Releases Clinical Trial Findings on Symptomatic nOH Treatment

Does Ampreloxetine Help People with Symptomatic Neurogenic Orthostatic Hypotension Feel Better?

A Phase 2 Study to Investigate Ampreloxetine in the Treatment of Symptomatic Neurogenic Orthostatic Hypotension (nOH)

Background

  • Researchers wanted to learn if an investigational medication (ampreloxetine) could help adults diagnosed with symptomatic neurogenic orthostatic hypotension (symptomatic nOH)
  • When people living with symptomatic nOH stand, or sit up quickly, they may experience symptoms such as lightheadedness, dizziness, or the feeling that they are about to black out
  • The purpose of this study was to learn whether ampreloxetine is able to improve blood pressure and lessen symptoms in people living with symptomatic nOH, and to assess its safety and tolerability
  • Ampreloxetine is not currently approved

What is symptomatic nOH?

  • In symptomatic nOH, the nerves that control blood pressure do not work adequately
  • When people with symptomatic nOH sit up or stand up, blood pressure falls, with symptoms of dizziness, lightheadedness, and feeling faint
  • Symptomatic nOH occurs in people with multiple system atrophy, Parkinson’s disease, and pure autonomic failure

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Rare Disease Day 2018: The Importance of Funding and Research

Rare Disease Day 2018: The Importance of Funding and Research

Rare diseases are a daily battle for some 30 million Americans. One in 10 people in the country are fighting an illness that is shrouded in mystery. The only way to alleviate the unknown – and the burden felt by patients- is through increased research.

Patients with rare diseases have tremendous unmet needs, including but not limited to misdiagnosis. It is common for patients to wait years for an accurate diagnosis, roughly 4.8 years. Unfortunately, even after receiving an accurate diagnosis, 95 percent of rare diseases have no treatment options or cures. The purpose of Rare Disease Day is to increase awareness within the general public, and decision makers, to improve options for those suffering.

Rare Disease Day is celebrated in February each year. The month, rare for it’s odd number of days, is the perfect time to highlight the roughly 7,000 rare diseases. Rare diseases, like multiple system atrophy and mesothelioma cancer, affect only a limited number of people which often leads to less funding allocation. Diseases that impact a greater portion of the general public are more likely to receive funding because there is a greater profit margin.

Limited funds impact the medications, treatment options, and clinical trials available to patients. The prevalence of a disease should not dictate the level of care a patient receives or how greatly the out of pocket costs are felt.

According to a survey conducted by the Rare Disease Impact Report of both the United States and United Kingdom payers – those who finance healthcare within a country’s health system – treatment for a rare disease is relatively more expensive and costs are rising quickly compared to more common diseases. For example, the estimated annual cost for a patient with Hemophilia, a rare blood disorder, is $131,111. Over a lifetime that equates to 8.7 million dollars.

Funding allocation is complicated for rare diseases due to segmentation – each rare disease is unique and requires specialized attention. Eighty percent of rare diseases have identified genetic origins, others are the result of infections, allergies and environmental causes, or are degenerative and proliferative.

The key to advancements for all the Americans suffering from a rare disease is collaboration between researchers and the ability to have cross over medication and treatments. Keytruda, a drug originally used for advanced stage melanoma, is a an example of what is possible if funding is dedicated to enabling necessary research. The medication has been proven a viable treatment for pleural mesothelioma. Keytruda was an effective therapy in approximately 76 percent of the treated pleural mesothelioma patients, which represents a promising result and an improvement in prognosis. Among the 25 patients who received Keytruda, 28 percent had some shrinkage of tumors, while 48 percent of the treated patients experienced stable disease or no increase in extent of tumors.

A similar outcome for multiple system atrophy would be life changing for the about 14,000 people living with MSA in the U.S. There is currently no specific course of treatment for those with MSA – patients are often treated with medication created for Parkinson’s disease. Unfortunately that medication is not very effective – in many cases there is no response.

It is hoped that through the use of the orphan drugs a more specific treatment approach can be achieved. In 2016 there was $450 billion in drug spending – 60 percent of that was non-orphan traditional drugs, one third on non-orphan specialty medication and only 7.9 percent of the spending was on approved orphan drugs.

As scientific advancements are continually achieved it is expected that the number of recognized rare diseases will grow – as the number of diseases increases so does the necessity of precision medicine through the use of orphan drugs.

In an effort to raise funds and awareness the National Organization of Rare Disorders (NORD) is hosting a 7,000 mile rare movement. The campaign challenges advocates to join together and seek pledges to walk, run, or bike 7,000 miles collectively throughout the month of February. For those with multiple system atrophy and patients with all types of rare diseases research funding cannot come soon enough. On February 28 #ShowYourRare to show you care!