A congressional committee voted on Wednesday to make changes to a controversial program that was designed to spur development of new drugs for neglected tropical diseases. But the effort — in the form of an amendment to an unrelated bill — is still encountering criticism for not going far enough.
At issue are priority review vouchers, which can be awarded to a drug maker that wins regulatory approval of a treatment for certain tropical diseases. Companies can later redeem a voucher when seeking approval for yet another medicine that would be used to treat any illness. And the Food and Drug Administration must review the other drug in six months, instead of the standard 10 months.
The deal is akin to a grand bargain because public health is promoted by development of a medicine that may not otherwise become available, and a drug maker can profit because a faster review means an approved medicine could be sold sooner and generate revenue. Last August, a voucher sold for $350 million, which was a record price.
But critics say the program does not do enough to ensure drug makers develop needed salves. A chief complaint is that vouchers can be awarded to a company without having to invest in new R&D or even in a new medicine. An example occurred two years ago, when a recently formed company called Knight Therapeutics obtained the rights to a drug and then received a voucher from the FDA. But the drug was already available for many years in other countries. Knight then sold the voucher to Gilead Sciences (GILD) for $125 million.
So the US House Energy and Commerce Committee Wednesday voted to address such concerns. For instance, companies would not be awarded a voucher for drugs previously approved in other countries; they would have to provide more information about plans to launch their tropical disease medicines outside the United States; and they would be required to provide data on patient demand worldwide. The bill to which the amendment was attached goes to the House floor for a vote.
But some reactions to these proposed fixes was decidedly mixed.
“We are concerned that this may not close the loophole on novelty, as companies could win a voucher by simply registering a product with the FDA and doing Phase 3 clinical trials, but without necessarily having created a new (drug or vaccine) for neglected disease patients,” explained Jennifer Reid, a researcher in the Access to Medicines campaign at Doctors Without Borders.
The eligibility requirement linked to clinical trials, she added, sets a “lower bar that allows companies to win vouchers without necessarily having contributed something new.” She also pointed out that companies may be dissuaded from registering treatments in countries where a tropical disease is prevalent. This might delay making a drug or vaccine available to the people who need them the most.
Meanwhile, it remains unclear if the amendment would satisfy FDA officials, who have been critical of voucher programs. In March, for instance, the agency went on record criticizing the voucher program for rare pediatric drugs, in particular. FDA officials complained the added workload strains resources, despite special-use fees paid by drug makers for the reviews that come with a voucher.
The FDA has also anticipated that drug makers will redeem vouchers for medicines that would otherwise receive a standard 10-month review for illnesses that already have available treatments, such as diabetes or cholesterol. In effect, they worry that vouchers allow a company to purchase a priority review at the expense of other important public health goals.
The amendment “does not address the fundamental issue that FDA review time is not an economic commodity — these reviews exist to protect patients by ensuring that drugs are safe and effective, and they are already designed to prioritize the most important drugs, said Dr. Aaron Kesselheim, an associate professor of medicine at Harvard Medical School and a faculty member at Brigham and Women’s Hospital in Boston, who has studied the voucher programs.
“I think it’s problematic and potentially dangerous to use this crucial process as a lever to try to artificially create value for a for-profit company, even for an area like neglected diseases that desperately needs more attention,” he added.
We asked the FDA for comment and will update you accordingly.