Orphan Drugs for Rare Diseases Gain Popularity With Pharmaceutical Companies – NYTimes.com

Esta estrategia comercial se ve en la práctica con la judicialización de las demandas de pacientes por acceso a estos medicamentos en cumplimiento del indiscutible derecho a la salud. Sin embargo, debería haber un amplio debate social al respecto, ya que en algunas situaciones esto puede generar un aumento de la inequidad del sistema (ver Ferraz en Health&Human Rights. Como se puede ver en el artículo del NYT abajo, las farmacéuticas sostienen e impulsan las asociaciones de pacientes.

The right to health in the courts of Brazil: Worsening health inequities?

Octavio Luiz Motta Ferraz

Orphan Drugs for Rare Diseases Gain Popularity With Pharmaceutical Companies – NYTimes.com.

Making ‘Every Patient Counts’ a Business Imperative

BEDMINSTER, N.J. — Drug companies are fond of saying that every patient counts, but in the world of orphan diseases, entire business plans are built around the idea.

That is why Andrew E. Jablonski, a 26-year-old from Lincoln, Neb., was a guest of honor this month at NPS Pharmaceuticals, a small New Jersey company that is about to begin selling Gattex, its only approved drug. Mr. Jablonski, who was born missing most of his intestines and has a condition known as short bowel syndrome, is central to the company’s mission to win over the tiny pool of patients — fewer than 5,000 nationwide — that the drug is intended to treat.

Over the last two years, NPS has carefully tended its relationship with Mr. Jablonski, helping to finance his nonprofit, the Short Bowel Syndrome Foundation, and flying company leaders to visit him in Lincoln. On Jan. 15, he met with the chief executive and strategized with marketing employees about how to promote Gattex to his social network of 1,000 patients and caregivers.

“It’s a nice, close relationship,” Mr. Jablonski said. “I’m an asset to them, as they are an asset to me.”

NPS hopes that Gattex will ultimately bring in hundreds of millions of dollars in annual sales and help establish the company in the increasingly hot orphan drug market, where companies can charge premium prices to treat overlooked diseases with little or no competition. NPS expects to charge about $300,000 a year for Gattex, a breathtaking price tag that is nonetheless on par with other ultra-orphan drugs on the market.

Development of these drugs is increasingly attractive to pharmaceutical companies, which are searching for new sources of revenue as sales of more traditional, mass-market drugs have been lost to generic competition. The orphan drug market was worth more than $50 billion in 2011 and turns out blockbusters at the same rate as the broader industry, according to a recent Thomson Reuters analysis. Pfizer opened a rare disease unit in 2010, and in 2011, Sanofi acquired the biotech company Genzyme, considered one of the pioneers of the orphan drug industry.

As orphan drugs have become increasingly popular, companies have honed their marketing techniques, knowing that they cannot afford to lose a single patient. Following in the footsteps of drug makers like Vertex, which makes the cystic fibrosis drug Kalydeco, NPS has formed close bonds with advocates like Mr. Jablonski and hired so-called patient care coordinators who will do everything from making sure that Gattex is delivered to patients’ doorsteps to writing appeal letters if an insurer refuses to pay for it.



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