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Children's Health & Safety Association

Issue 43: July 2018

Saturday, 30 August 2014 11:50

Pilot Project Launched for Orphan Drugs

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August 6, 2014 – The Government of Canada has launched a pilot project targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs.

Orphan drugs are used to treat life threatening, seriously debilitating and chronic conditions that affect a very small number of patients - typically less than 5 in 10,000 people.

"No one has a better understanding of what it means to have a rare disease than the patients who are affected by them. The input of these patient volunteers is absolutely invaluable and our government has committed to making their experiences and perspectives an important addition to our scientific approach to drug assessments for rare diseases," says The Honourable Rona Ambrose, Minister of Health.

In October of 2012, it was announced that Health Canada started the development of an Orphan Drug Framework to stimulate innovation and research into new treatments for rare diseases and encourage patient participation. The Pilot Project will simulate how input from patients will be gathered and incorporated into the drug submission review process once the Orphan Drug Framework is in effect.

Approximately 50% of the people affected by rare diseases are children.

Drug manufacturers, Hoffmann-La Roche Limited and Hyperion Therapeutics, Inc. have agreed to participate in the pilot with two of their drug review submissions. The review of Hoffmann-La Roche's medication ‘obinutuzumab’ for the treatment of chronic lymphocytic leukaemia (CLL) will be used for the pilot.

Hyperion Therapeutics Inc.'s review is for Ravicti ® (glycerol phenylbutyrate) Oral Liquid, which is used for the treatment of urea cycle disorders. Hoffmann-La Roche and Hyperion's participation will serve as models for how patient input may inform drug authorization decisions.

Lorenzo Biondi, Vice President of Medical & Regulatory Affairs for Hoffmann-La Roche Limited said, "Roche recognizes that patients and patient organizations are playing an increasingly important role in drug review and health technology assessment and bring valuable experience, perspective and insight to the process. As an organization committed to patients, Roche is pleased to participate in this Health Canada pilot program that is seeking patient input within the regulatory review process."

pilot-004Health Canada will be asking patient advocacy groups including the Canadian Organization for Rare Disorders to assist in engaging specific Canadian patients affected by CLL or urea cycle disorders to participate in the project. The patient feedback provided during the pilot will assist Health Canada in assessing and refining its approach to gathering patient input. This will ensure that patient perspectives are considered in future orphan drug authorizations.

Patients will be asked to comment on the following:

  • how the disease affects their ability to manage their day-to-day lives;
  • what treatments are currently available (if any);
  • what therapeutic benefits are most important to them; and
  • their risk tolerance for any new treatments.

pilot-007When the Orphan Drug Framework is fully implemented, Canadian patients will be invited to participate in future orphan drug authorizations through Health Canada's website. Patients with CLL or urea cycle disorders, their current or former caregivers or healthcare professionals, and people with first-hand knowledge of CLL or urea cycle disorders are encouraged to contact Health Canada by email if they wish to participate at: This email address is being protected from spambots. You need JavaScript enabled to view it. .

"Kudos to Health Canada for recognizing the value of the direct patient voice in the drug review process, for designing a user-friendly template that will encourage patient engagement, and for piloting this in advance of the orphan drug regulations. The Canadian Organization for Rare Disorders is pleased to have had the opportunity to collaborate on this important initiative and to promote this in the patient community," said Durhane Wong-Rieger, President of Canadian Organization for Rare Disorders.

New rules and approaches to the development, evaluation and approval of these drugs are needed to address the fact that the small size of the patient population makes it scientifically difficult and most often commercially impractical for drug companies to develop and market orphan drugs.

A key focus of this new approach will be on international information sharing and collaboration for the development and regulation of orphan drugs. Enabling Canadian scientists and regulators to participate with trusted global counterparts will make better use of scarce resources and benefit Canadian patients.

pilot-008The new framework will maintain evidence requirements based on clinical trials and will be supported by greater information sharing amongst international partners who are committed to pooling scarce resources for maximum benefit. Once authorized, drugs will continue to be closely monitored for effectiveness and safety while in use.

Facts about Rare Diseases

  • 350 million people globally are fighting rare diseases.
  • Approximately 50% of the people affected by rare diseases are children.
  • 30% of children with a rare disease will not live to see their 5th Birthday.
  • Rare diseases are responsible for 35% of deaths during the first year of life.
  • International health organizations estimate the number of rate diseases at over 7,000 with 80% linked to genetic factors.
  • In the United States, a condition is considered rare if it affects fewer than 200,000 people. In the United Kingdom, a disease is considered rare if it affects fewer than 50,000 citizens.  
  • According to the Kakkis EveryLife Foundation , 95% of rare diseases have not one single FDA approved drug treatment.
  • According to the National Institutes of Health Office of Rare Disease Research , approximately 6% of the inquiries made to the Genetic and Rare Disease Information Center

Resources

Orphanet Journal of Rare Diseases

Children with Rare Diseases Will Receive Access to New Drugs & Treatment but at What COST

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