The Medical System Pulled Out all the Stops for COVID. Can it do the Same for Rare Diseases?

Pfizer’s Regional President for Rare Disease, Reda Guiha, recently offered his opinion to The Parliament magazine about the urgency of what he refers to as the ‘rare disease crisis.’ Pres. Guiha said the urgency that propelled vaccines out to the public within one year should also be applicable to rare diseases.

At issue are the thirty million rare disease patients across Europe. Then there are millions in the rest of the world with no hope of a cure, no relief of symptoms, and in so many cases, no definitive diagnosis.

Pfizer dominated headlines for a year as it responded to the pandemic. Pres. Guiha would like to see that same scientific expertise along with the same collaboration of the private/public sectors applied to rare diseases. He adds that there is an urgent need for new regulations and innovative policies.

A Look At The Rare Disease Crisis

Some of the challenges that currently exist for the rare disease community are:

  • Lack of available treatment
  • Minimal accessibility to quality care
  • Research is hampered due to the inability to gain sufficient patient data
  • Patients may visit at least seven doctors and wait an average of five years for an accurate diagnosis
  • Of the seven thousand rare diseases, eighty percent are genetic disorders and fifty percent affect children
  • Patients often present symptoms of more common conditions hampering a definitive diagnosis

COVID-19 Changed the Landscape

Pres. Guiha told The Parliament that prior to the pandemic, a ten-year period for product development had been tolerated. That involves starting in the lab, regulatory submissions, pricing, and finally reaching the patient.

COVID-19 brought about a major change by reducing the ten-year development period to one year. According to Pres. Guiha, this was accomplished without compromising patient safety.

He stated that the concept of how to respond to urgent patient needs has been forever altered. That it should be possible to apply these experiences to the treatment of rare diseases.

Just as with COVID-19, there is limited data about rare diseases and hence there is a need to follow the new standards set by the public and private sectors in addressing the pandemic.

Lessons From the Pandemic

According to a recent survey, eighty-three percent of rare disease patients lost access to care during the pandemic. The need to help this underserved community is evident.

Pres. Guiha explained that Pfizer’s one-year timeline for the development of its COVID-19 vaccine was made possible only because of the unprecedented cooperation of health officials and regulators. Their cooperation expedited the review of the clinical trial protocol and critical scientific advice.

Another critical component was the collection of evidence confirming the efficacy of COVID-19 vaccines. Pres. Guiha expressed his opinion that new partnerships and industries will be necessary to gather this evidence. They will be especially necessary when entering a new era of gene therapy.

Looking Forward

Pres. Guiha foresees a future where the same urgency that was created by the COVID-19 pandemic will then address the needs of the rare disease community.

The pandemic has set a new standard.

Rose Duesterwald

Rose Duesterwald

Rose became acquainted with Patient Worthy after her husband was diagnosed with Acute Myeloid Leukemia four years ago. He was treated with a methylating agent While he was being treated with a hypomethylating agent, Rose researched investigational drugs being developed to treat relapsed/refractory AML.

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