Drug approval is a remarkably sensitive subject. Pressure from patients – and some doctors – will always be for better treatments for poorly served conditions. On the other hand, the health, financial and reputational impact of adverse side effects can be catastrophic – particularly for those same patients.
Regulation is key and the United States’s Food and Drug Administration (FDA) is the benchmark internationally. Approval by the FDA will secure a drug’s international reputation and market prospects – and not just because the US is the biggest market for medicines.
That is why a decision by the agency to call for a federal investigation of its own process that led to the approval of a new drug for Alzheimer's disease is so notable.
In a letter to the US department of health and human services' independent office of the inspector general, the FDA's acting commissioner, Dr Janet Woodcock, acknowledged the scrutiny the agency has faced about the approval process for the drug, which is known as Aduhelm.
She pointed to interactions between representatives from the drug's developer, Biogen, and the agency, saying some "may have occurred outside of the formal correspondence process".
She noted that the review should look at whether any of the communication between the agency’s staff and Biogen’s representatives violated FDA rules.
The FDA has already narrowed the terms of its approval for this drug.
The regulator green-lighted Aduhelm, which now has a €47,250 annual price tag in the US, last month over the strong objections of its own independent advisory panel – convened specifically to make recommendations on new drugs.
Resigned
Ten of the 11 independent members of the panel said the clinical trial data could not be considered as evidence of its effectiveness in treating Alzheimer’s. Three resigned following the FDA approval.
FDA panels conduct their business in public, offering greater transparency than is available elsewhere, including in Europe. As often groundless concerns about Covid-19 vaccine shave shown, such transparency is critical.
Ireland’s 64,000 Alzheimer’s patients – among about 35 million worldwide – are a valuable market for a company with an effective cure. What they really do not need is a therapy with unexplained question marks. And nor does the pharma sector, given the money invested in getting a drug to the point of FDA approval.
