Health Canada considers changes to orphan drug approvals

After years of lobbying by patient groups, Health Canada is preparing to change the way drugs are approved to accommodate rare diseases and the “orphan drugs” needed to treat them (Montreal Gazette).

Later in October, Health Canada will launch consultations on broad drug-regulation reforms and, for the first time, how orphan drugs should be treated differently and incorporated into the regulatory framework will be part of those conversations.  The department hasn’t committed to implementing a formal orphan drug policy but the changes that will eventually be made as part of wider modernizations to Health Canada rules are considered major progress and a departure from previous positions.

There are thousands of rare disorders that affect only small groups of people, but altogether, an estimated one in 12 Canadians has a rare condition and many people are undiagnosed.

Unlike other countries, Canada doesn’t have a standard definition of rare diseases, let alone a national plan to deal with them. The United States has had an orphan drug policy since 1983 and the European Union has also implemented changes to their regulations to accommodate orphan drugs.

Canada needs to follow their lead, say groups such as CORD and BIOTECanada, because the absence of a policy means patients and researchers here are at a disadvantage.

The potential revisions could first include defining a rare disease as a condition that affects one in fewer than 2,000 people, and beyond that, may change the way drugs for rare diseases are developed, approved and brought to the market.

If the American and European models are followed, there will be more incentives for drug companies to pour money into research and development — a challenge considering the customer base for orphan drugs is so small. Those incentives could include more tax credits and funding grants for research, extending patent protection, and reducing drug-approval application fees or dropping them altogether.

Clinical-trial protocols might also be modified to accommodate the challenges associated with testing drugs for rare diseases, said David Lee, head of the Health Canada branch that is in charge of updating the regulations.

Lee said Health Canada is carefully studying how other countries handle orphan drugs with an aim to see what could be implemented here.

After the consultation period, which will last several months, draft regulations will be proposed for overhauling the drug-regulation system.

New US legislation seeks R&D incentives for rare childhood diseases

New bipartisan legislation was introduced in the US Senate last week which seeks to encourage innovative R&D by drugmakers aimed at treating rare and neglected pediatric diseases. (source PharmaTimes)

The Creating Hope Act of 2010 builds on existing law to increase incentives for the development of treatments for disabling and deadly diseases, with a focus on rare conditions that may otherwise fail to attract sufficient R&D funding.

“Seven thousand known rare or orphan diseases afflict nearly 30 million Americans – approximately 50% of whom are children,” said Democrat Senator Sherrod Brown, who is co-sponsoring the bill with Republican Sam Brownback and Democrat Al Franken.

Under this law, companies which develop new drugs and biologics for neglected tropical diseases are eligible for a “priority review voucher” entitling them to expedited review of another drug produced by that manufacturer. Because this voucher can be used to expedite the marketing of a “blockbuster” or “me-too” drug, it provides a strong financial incentive for the development of treatments for otherwise neglected diseases.

The proposed new legislation would improve upon this incentive not only by increasing the commercial value of the priority review voucher by making it transferable, but by expanding priority review voucher eligibility to include rare pediatric diseases.

New research unit boosts hope for patients with rare diseases

On Monday, Pfizer announced the creation of a new research unit which will specifically focus on the treatment of orphan diseases — those that affect fewer than 200,000 patients worldwide.

Of the more than 6000 diseases classified as orphan diseases, fewer than ten percent have therapies that directly address the underlying disease.  Pfizer stated that they intend to work closely with patient advocacy groups as it develops and advances the unit’s research strategy.

The new unit got the thumbs-up from National Organization for Rare Disorders in the USA. Its chief executive, Peter Saltonstall, noted that “30 million Americans, 30 million Europeans and millions more around the world have rare diseases” and applauded the development of new treatments “for this medically-underserved population.”

Increase in innovation, access to orphan drugs

The number of orphan drug designations in the US more than doubled in the last decade, growing from 208 during 2000-02 to 425 in 2006-08, says a new study.

Since the US Orphan Drug Act of 1983 was signed into law, more than 2,000 products in development have been designated as orphan drugs, while the Food and Drug Administration (FDA) has granted market approval to 350 drugs and biologics, according to the study, which was conducted by the Tufts Center for the Study of Drug Development (CSDD).

Orphan drugs are products developed for a rare disease or condition affecting fewer than 200,000 people in the US, while in the European Union (EU) they are defined as treatments for diseases or conditions affecting five people out of every 10,000, or fewer.

The National Institutes of Health (NIH) estimates that 25 million Americans have a rare disease.

Malaria drugs may help fight lupus

Drugs used to treat malaria may be useful for patients with lupus, a chronic debilitating “autoimmune” disease, according to according to a new report published in the journal Arthritis and Rheumatism.

“The data presented, taken in conjunction with the data from the published literature, suggest that antimalarials should be used in all lupus patients regardless of their disease manifestation or disease duration,” the authors concluded.

Pons-Estel and his team studied nearly 1,500 patients with lupus from 9 countries.  About 12 percent of the patients who did not use the drugs died during the follow-up period, compared to about 4 percent of those who did.  The difference was even higher for patients who used the drugs for more than two years.  After the team accounted for various factors, using antimalarial drugs appeared to reduce the risk of death during the study by almost 40 percent.

Lupus is a chronic disease in which the immune system confuses its own healthy tissues with foreign tissues and sometimes attacks both. The condition can manifest as a skin rash or arthritis and may lead to damage to the kidneys, heart, lungs and brain to varying degrees. The disorder disproportionately affects women.