Dr. Michelle McMurry-Heath, president and CEO of the Biotechnology Innovation Organization (BIO), released the following statement in response to the Centers for Medicare and Medicaid Services’ (CMS) proposed National Coverage Determination (NCD) to restrict coverage for drugs used to treat patients suffering from Alzheimer’s:
“The announcement by CMS sets a dangerous and far-reaching precedent for patients struggling with Alzheimer’s and other difficult to treat diseases. Limiting coverage only to patients enrolled in government-mandated randomized clinical trials means that most of those suffering from Alzheimer’s would be denied access to an approved medicine simply because of where they live or where they go for care.
“The CMS proposal also would require post-approval randomized controlled trials for all future drugs in this class, regardless of the type and volume of evidence demonstrated during FDA-required trials and regardless of whether future drugs are approved during an accelerated pathway or through traditional channels. In essence, through this coverage announcement, CMS is pronouncing its disagreement with FDA’s gold-standard, science-based, safety and efficacy standards. Surely the first time such a conflict has arisen and now a dangerous rebuke of FDA’s scientific autonomy and decision making.
“In the coming weeks, we hope that CMS will reevaluate its proposal in light of the fact that it would restrict access for the most vulnerable patients and adversely impact the future investment needed to develop new medicines to treat patients suffering from Alzheimer’s.
“Most Medicare beneficiaries that meet FDA-approval criteria would not be able to access the drug under this proposal. There also is the issue that many enrolled in randomized trials won’t even have access to the drug, since they will be receiving the placebo. In addition, under this proposal, coverage wouldn’t even begin to be available until a trial is approved and enrolling patients, likely a year or more after any coverage decision is final.
“The reality is that there has only been one approved treatment for Alzheimer’s in the last twenty years. Clinical development success in this space has been extremely difficult. Since 2008, nearly 90 clinical-stage disease-modifying programs have been suspended.
“Despite the challenges and complexity of this disease, our companies continue to dedicate themselves to developing new treatments. Today there are approximately 70 clinical-stage programs with disease-modifying potential in Alzheimer’s disease. These programs are attempting to stop, prevent, or slow the progression of Alzheimer’s disease using a variety of novel strategies. Small biotech companies account for more than three-fourths of these clinical programs.
“The Accelerated Approval Pathway is often the only mechanism for approving effective therapies to address critical unmet patient need in challenging and serious disease states. Drugs granted accelerated approval must meet the same statutory standards of evidence for safety and effectiveness as those granted traditional approval.
“The proposal released today by CMS, as currently drafted, would severely impact future investment and research going towards treating patient with Alzheimer’s, and its impact could extend well beyond just this class of medicines. It would negate the purpose of the FDA’s accelerated approval pathway by impeding, instead of supporting, access to therapies while confirmatory studies are conducted. The accelerated approval pathway encourages scientific and medical advancement and innovation by leveraging the use of surrogate or intermediate clinical endpoints that are reasonably likely to predict clinical benefit. The approval of the first HIV/AIDS drug based on the use of surrogate endpoints, prolonged and saved the lives of millions of patients that did not have any other therapeutic options. Hence, this pathway underscores that a chronic illness does not have to be a death sentence.
“We look forward to working with CMS to address these concerns and ensuring that Medicare beneficiaries have appropriate access.”