Accelerated Approvals Benefit Companies Over Patients: Bloomberg
(Thursday, May 18, 2023) As Sarepta looks towards another controversial FDA approval for a new treatment for DMD, the accelerated approval pathway is once again being debated for its merits and abuses. Without major regulatory changes, there is not much that the FDA can do or want to do, while patients potentially get hurt both physically and financially. A scathing Bloomberg review of the regulatory pathways shows that while companies like Sarepta reap billions in profit with such approvals, patients end up with unproven and many times ineffective drugs. Accelerated approval pathway was created in the early 1990’s to help manage the AIDS epidemic. For the first 25 years of its existence, the accelerated pathway led to a few approvals each year but in the last 5 years, this pathway has become a cash cow for the developers of unproven and risky therapies, according to the Bloomberg investigation. Of the 300 products that were approved by the FDA using this pathway, about 150 approval decisions happened in the last 6 years. Once approved, the companies immediately cash in billions in sales leading to huge profits but delay the post-marketing studies they promised to the FDA at the time of approval. There are both practical and financial reasons for such delays. In about 20% of the completed post-market studies, the drugs approved under the accelerated pathway was later found to be ineffective or had unacceptable safety issues leading to the accelerated approval being withdrawn. In most cases, these withdrawals happened many years after the initial approval, during which time the manufacturer kept selling the drug making huge profits. Upon withdrawal there is no legal mechanism for the patients or payors to claim refunds, so the money made is for the manufacturers to keep. In the case of Sarepta, the company did not start its post-marketing study for almost 4 years after the initial approval and expects to complete that study about 8 years from approval. There is scant evidence to date that Sarepta’s treatments are clinically effective. The company touts its high sale numbers but does not comment on the improvement of patients’ lives. Oft used phrase, “saving patients lives” is abused ad libitum. For the company with a revenue from the sales of these drugs at about $1 billion each year, this translates to about $8 billion over eight years. The company got 2 other drugs approved subsequently under accelerated approval and have its fourth treatment, this time a gene therapy, lined up for FDA approval using the same strategy, assuring years of huge profits for the company while its drugs remain unproven. Its latest gene therapy is projected to bring in about $3 billion in annual revenue on its own to the company. Sarepta is just one of the many companies using accelerated approval as a business model. While there have been several success stories of the accelerated program, the recent abusers of the pathway have created a strong public opinion against this pathway. Companies use emotional arguments with the patients, when scientific justifications are weak. This week Sarepta once again used patient advocates to tilt the FDA Advisory Committee vote in its favor, exactly as it did the first time in 2016. This strategy has been used by other companies, most recently by Amylyx for its ALS therapy. The regulatory process urgently needs to be updated. In Europe, accelerated approvals are still rare. Even when approved, the approval is for a one-year period at a time, with the extension conditional to the company presenting ongoing evidence of clinical benefits. This incentivizes the manufacturers to expedite post-market studies. So, unlike the US where the manufacturers can game the system to delay post-market studies while making profits from sales, in Europe, these same manufacturers work hard to meet their clinical commitments. It should be noted that most of these controversial approvals were unsuccessful in getting approval in the European Union in the first place. There has been a strong debate to create a similar conditional approval here in the US as well. But the extremely strong industry lobby would not support it and the patient support for such measures is hard to get as patients are desperate for any hope, no matter how feeble. In such an environment, no such commonsense changes are expected in the laws, at least in the near future. Companies like Sarepta have nothing to worry about, for many years to come. AUTHOR
Dr. Mukesh Kumar Founder & CEO, FDAMap Email: mkumar@fdamap.com Linkedin: Mukesh Kumar, PhD, RAC Instagram: mukeshkumarrac Twitter: @FDA_MAP Youtube: MukeshKumarFDAMap |
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