Pharmacy

The FDA generally approves drugs faster than its Canadian and European counterparts, according to a study published in this week’s edition of NEJM.

The researchers say these results refute criticisms that the drug approval process in the US is slow and that agencies in other countries tend to approve new therapies first.

“The perception that the FDA is too slow implies that sick patients are waiting unnecessarily for regulators to complete their review of new drug applications,” said lead study author Nicholas Downing, a medical student at Yale University.

He and his colleagues decided to conduct this study because there have been no recent comparisons of the FDA’s review speed with that of agencies in other countries.

So the researchers reviewed drug approval decisions made by the FDA, Health Canada, and the European Medicines Agency (EMA) between 2001 and 2010. The team said they chose Health Canada and the EMA as comparisons because these agencies face similar pressures to approve new drugs quickly while ensuring they don’t put patients at risk.

The investigators studied each regulator’s database of drug approvals to identify novel therapeutics, as well as the timing of key regulatory events. They then calculated each agency’s review speed.

The median total time to review a new drug application was 322 days at the FDA, 366 days at the EMA, and 393 days at Health Canada.

“Among the subsample of drugs approved for all 3 regulators, the FDA’s reviews were over 3 months faster than those of the EMA or Health Canada,” Downing said. “The total review time at the FDA was faster than EMA, despite the FDA’s far higher proportion of applications requiring multiple regulatory reviews.”

The researchers also found that, during the review period, the FDA approved 225 new drugs, the EMA approved 186, and Health Canada approved 99. Additionally, of the therapies that have been approved by all 3 agencies, most drugs were first approved in the US.

“[W]e found that 64% of medicines approved in both the US and in Europe were approved for US patients first,” Downing said. “And 86% of medicines approved in both the US and Canada were also approved first in the US.”

Downing and his colleagues noted that this study has 2 key limitations. First, the researchers didn’t account for drugs that were ultimately rejected, as the regulatory agencies don’t release review times for drugs that are never approved. However, the team also pointed out that the FDA approves more than 80% of its applications, so the exclusion may not have made much of an impact.

Secondly, the study included only new molecular entities and original biologic agents. In order to get a more accurate reading on the regulatory review process, research would need to evaluate the review of generic drugs, reformulated drugs, combination therapies, and medical devices.

The FDA generally approves drugs faster than its Canadian and European counterparts, according to a study published in this week’s edition of NEJM.

The researchers say these results refute criticisms that the drug approval process in the US is slow and that agencies in other countries tend to approve new therapies first.

“The perception that the FDA is too slow implies that sick patients are waiting unnecessarily for regulators to complete their review of new drug applications,” said lead study author Nicholas Downing, a medical student at Yale University.

He and his colleagues decided to conduct this study because there have been no recent comparisons of the FDA’s review speed with that of agencies in other countries.

So the researchers reviewed drug approval decisions made by the FDA, Health Canada, and the European Medicines Agency (EMA) between 2001 and 2010. The team said they chose Health Canada and the EMA as comparisons because these agencies face similar pressures to approve new drugs quickly while ensuring they don’t put patients at risk.

The investigators studied each regulator’s database of drug approvals to identify novel therapeutics, as well as the timing of key regulatory events. They then calculated each agency’s review speed.

The median total time to review a new drug application was 322 days at the FDA, 366 days at the EMA, and 393 days at Health Canada.

“Among the subsample of drugs approved for all 3 regulators, the FDA’s reviews were over 3 months faster than those of the EMA or Health Canada,” Downing said. “The total review time at the FDA was faster than EMA, despite the FDA’s far higher proportion of applications requiring multiple regulatory reviews.”

The researchers also found that, during the review period, the FDA approved 225 new drugs, the EMA approved 186, and Health Canada approved 99. Additionally, of the therapies that have been approved by all 3 agencies, most drugs were first approved in the US.

“[W]e found that 64% of medicines approved in both the US and in Europe were approved for US patients first,” Downing said. “And 86% of medicines approved in both the US and Canada were also approved first in the US.”

Downing and his colleagues noted that this study has 2 key limitations. First, the researchers didn’t account for drugs that were ultimately rejected, as the regulatory agencies don’t release review times for drugs that are never approved. However, the team also pointed out that the FDA approves more than 80% of its applications, so the exclusion may not have made much of an impact.

Secondly, the study included only new molecular entities and original biologic agents. In order to get a more accurate reading on the regulatory review process, research would need to evaluate the review of generic drugs, reformulated drugs, combination therapies, and medical devices.

The FDA generally approves drugs faster than its Canadian and European counterparts, according to a study published in this week’s edition of NEJM.

The researchers say these results refute criticisms that the drug approval process in the US is slow and that agencies in other countries tend to approve new therapies first.

“The perception that the FDA is too slow implies that sick patients are waiting unnecessarily for regulators to complete their review of new drug applications,” said lead study author Nicholas Downing, a medical student at Yale University.

He and his colleagues decided to conduct this study because there have been no recent comparisons of the FDA’s review speed with that of agencies in other countries.

So the researchers reviewed drug approval decisions made by the FDA, Health Canada, and the European Medicines Agency (EMA) between 2001 and 2010. The team said they chose Health Canada and the EMA as comparisons because these agencies face similar pressures to approve new drugs quickly while ensuring they don’t put patients at risk.

The investigators studied each regulator’s database of drug approvals to identify novel therapeutics, as well as the timing of key regulatory events. They then calculated each agency’s review speed.

The median total time to review a new drug application was 322 days at the FDA, 366 days at the EMA, and 393 days at Health Canada.

“Among the subsample of drugs approved for all 3 regulators, the FDA’s reviews were over 3 months faster than those of the EMA or Health Canada,” Downing said. “The total review time at the FDA was faster than EMA, despite the FDA’s far higher proportion of applications requiring multiple regulatory reviews.”

The researchers also found that, during the review period, the FDA approved 225 new drugs, the EMA approved 186, and Health Canada approved 99. Additionally, of the therapies that have been approved by all 3 agencies, most drugs were first approved in the US.

“[W]e found that 64% of medicines approved in both the US and in Europe were approved for US patients first,” Downing said. “And 86% of medicines approved in both the US and Canada were also approved first in the US.”

Downing and his colleagues noted that this study has 2 key limitations. First, the researchers didn’t account for drugs that were ultimately rejected, as the regulatory agencies don’t release review times for drugs that are never approved. However, the team also pointed out that the FDA approves more than 80% of its applications, so the exclusion may not have made much of an impact.

Secondly, the study included only new molecular entities and original biologic agents. In order to get a more accurate reading on the regulatory review process, research would need to evaluate the review of generic drugs, reformulated drugs, combination therapies, and medical devices.

The US Food and Drug Administration (FDA) has granted accelerated approval for eculizumab (Soliris) to treat patients with atypical hemolytic uremic syndrome (aHUS).

This rare and chronic disease can lead to renal failure and is associated with an increased risk of death and stroke. It accounts for 5% to 10% of all cases of hemolytic uremic syndrome and disproportionately affects children.

Eculizumab is a targeted therapy that works by inhibiting proteins that play a role in aHUS. The FDA granted accelerated approval for eculizumab based on data suggesting the drug likely confers a clinical benefit for patients with aHUS.

The FDA’s Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint that is thought to predict clinical benefit. The makers of eculizumab, Alexion Pharmaceuticals, are still required to conduct research to confirm the anticipated clinical benefit.

If this research indicates that eculizumab does provide a clinical benefit, the FDA will grant traditional approval for the drug. If research suggests eculizumab does not provide a clinical benefit, the FDA has regulatory procedures in place that could lead to removing the drug from the market.

There are no other FDA-approved treatments for aHUS. The safety and efficacy of the current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well-controlled trials.

Researchers have examined the safety and efficacy of eculizumab in 2 single-arm trials of 37 adult and adolescent patients with aHUS and 1 retrospective study of 19 pediatric and 11 adult patients with aHUS.
 
Patients treated with eculizumab in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients who did not respond to plasma therapy.

Patients treated with eculizumab also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.

The most common side effects included hypertension, diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and leukopenia.

Eculizumab will continue to be available only through a restricted program. Prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.

Eculizumab is marketed as Soliris by Alexion Pharmaceuticals, located in Cheshire, Connecticut.

The US Food and Drug Administration (FDA) has granted accelerated approval for eculizumab (Soliris) to treat patients with atypical hemolytic uremic syndrome (aHUS).

This rare and chronic disease can lead to renal failure and is associated with an increased risk of death and stroke. It accounts for 5% to 10% of all cases of hemolytic uremic syndrome and disproportionately affects children.

Eculizumab is a targeted therapy that works by inhibiting proteins that play a role in aHUS. The FDA granted accelerated approval for eculizumab based on data suggesting the drug likely confers a clinical benefit for patients with aHUS.

The FDA’s Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint that is thought to predict clinical benefit. The makers of eculizumab, Alexion Pharmaceuticals, are still required to conduct research to confirm the anticipated clinical benefit.

If this research indicates that eculizumab does provide a clinical benefit, the FDA will grant traditional approval for the drug. If research suggests eculizumab does not provide a clinical benefit, the FDA has regulatory procedures in place that could lead to removing the drug from the market.

There are no other FDA-approved treatments for aHUS. The safety and efficacy of the current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well-controlled trials.

Researchers have examined the safety and efficacy of eculizumab in 2 single-arm trials of 37 adult and adolescent patients with aHUS and 1 retrospective study of 19 pediatric and 11 adult patients with aHUS.
 
Patients treated with eculizumab in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients who did not respond to plasma therapy.

Patients treated with eculizumab also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.

The most common side effects included hypertension, diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and leukopenia.

Eculizumab will continue to be available only through a restricted program. Prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.

Eculizumab is marketed as Soliris by Alexion Pharmaceuticals, located in Cheshire, Connecticut.

The US Food and Drug Administration (FDA) has granted accelerated approval for eculizumab (Soliris) to treat patients with atypical hemolytic uremic syndrome (aHUS).

This rare and chronic disease can lead to renal failure and is associated with an increased risk of death and stroke. It accounts for 5% to 10% of all cases of hemolytic uremic syndrome and disproportionately affects children.

Eculizumab is a targeted therapy that works by inhibiting proteins that play a role in aHUS. The FDA granted accelerated approval for eculizumab based on data suggesting the drug likely confers a clinical benefit for patients with aHUS.

The FDA’s Accelerated Approval Program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need based on a surrogate endpoint that is thought to predict clinical benefit. The makers of eculizumab, Alexion Pharmaceuticals, are still required to conduct research to confirm the anticipated clinical benefit.

If this research indicates that eculizumab does provide a clinical benefit, the FDA will grant traditional approval for the drug. If research suggests eculizumab does not provide a clinical benefit, the FDA has regulatory procedures in place that could lead to removing the drug from the market.

There are no other FDA-approved treatments for aHUS. The safety and efficacy of the current standard treatment, plasma therapy (plasma exchange or fresh frozen plasma infusion), have not been studied in well-controlled trials.

Researchers have examined the safety and efficacy of eculizumab in 2 single-arm trials of 37 adult and adolescent patients with aHUS and 1 retrospective study of 19 pediatric and 11 adult patients with aHUS.
 
Patients treated with eculizumab in these studies experienced a favorable improvement in kidney function, including elimination of the requirement for dialysis in several patients who did not respond to plasma therapy.

Patients treated with eculizumab also exhibited improvement in platelet counts and other blood parameters that correlate with aHUS disease activity.

The most common side effects included hypertension, diarrhea, headache, anemia, vomiting, nausea, upper respiratory and urinary tract infections, and leukopenia.

Eculizumab will continue to be available only through a restricted program. Prescribers must enroll in a registration program and provide a medication guide to patients who receive the drug.

Eculizumab is marketed as Soliris by Alexion Pharmaceuticals, located in Cheshire, Connecticut.