The president's FDA commissioner could become a “Scrooge” for the industry

In the June 2017 issue of PharmaTimes, we wrote about president’s Trump nomination of Dr Scott Gottlieb as the next FDA commissioner. A physician who had deep links with Big Pharma, Dr Gottlieb was viewed by critics as being too biased to assume the role as the nation’s top drug regulator. However, he also felt that “the agency should demonstrate more flexibility in granting product licences” and “called for the faster approval of complex generics in order to compete with the higher-priced originals”; policies which echo Trump’s campaign rhetoric to bring down perceived high drug prices.

Since his appointment as the 23rd FDA commissioner in May 2017, has Dr Gottlieb fulfilled the above promises  and will these be a threat to big pharma’s current  modus operandi?

“They’re getting away with murder”

While Trump has never been the type to stay focused on any topic, a few issues have attracted his constant attention, including the Iranian nuclear deal, the US-China trade war and Big Pharma drug pricing. He has consistently referred to the industry as “getting away with murder”, accusing them of engaging in price gouging and anticompetitive practices.

In July this year, Pfizer reversed or postponed its drug hikes a day after Trump’s Twitter criticism that the company, amongst others, "should be ashamed for raising drug prices for no reason; taking advantage of the poor and others unable to defend themselves, while at the same time giving bargain-basement prices to other countries in Europe & elsewhere". However, it is not necessarily Trump that Big Pharma should be worried about but his FDA commissioner. While Trump pays very little attention to policy implementation, Dr Gottlieb has been laser-focused on cutting drug prices in the past year.

One of Dr Gottlieb’s biggest focus has been on increasing market access and the ease at which generic drugmakers can manufacture, import and sell drugs. A major stumbling block to achieving this is the significant and perceived obstructive tactics of Big Pharma.

Although some critics see Dr Gottlieb’s price-cutting proposals as inappropriate government interference, he sees no such contradiction. While he believes that free market forces and vibrant competitions are vital for lowering drug prices and increasing accessibility, he feels that it is vital that the FDA is able to reform the regulatory system, including speeding up approvals for generic alternatives, thus allowing competition to emerge in the first place.

A major accusation is that Big Pharma erects barriers to entry by preventing sales of their original drugs to generic drug-makers who need to buy between 2,000-5,000 doses to conduct bioequivalence studies. In the very first month of Dr Gottlieb’s appointment, the FDA set up a website to effectively name and shame companies who have failed to provide samples for bioequivalence testing. Fifty branded drugs appeared on the list initially, including well-known drugs like Roche’s Accutane (acne), Actelion’s Tracleer (PAH) and AZ’s Brilinta (anti-platelets). Manufacturers of original drugs were accused of using on-going risk mitigation plans as an excuse for not supplying the samples, or simply instructing their distributors not to sell their drugs to potential generics competitors.

While Big Pharma is a main target, Dr Gottlieb has also criticised pharmacy benefit managers (PBMs) and the control that the top three firms, which together account for over two-thirds of the market, has led to a lack of competition. Citing their role as middleman, he stated that PBMs are equally as responsible as big pharma for limiting choices and keeping drug prices high. PBMs often ignore cheaper generics and biosimilars as they don’t receive rebates that they would otherwise have collected from manufacturers of original medicines. As a result, despite the presence of cheaper options, patients are forced to purchase expensive medicines due to the above practice.

What has he achieved so far?

The FDA has been speeding up its drug approval process with 46 new drugs being approved in 2017, including therapies for cancers and uncommon diseases like Celgene’s Idhifa (leukaemia), Roche’s Ocrevus (multiple sclerosis) and Teva’s Austedo (Huntington’s). However, as many of these new treatments have similar mechanism of actions as those already on the market, it has generated concerns whether the new drugs are truly groundbreaking. With critics citing a study showing that between 2002 and 2014, newly-approved cancer drugs only prolonged life by a few short months, they have concerns that this will only worsen with more mediocre cancer drugs making their way into the market under Dr Gottlieb’s stewardship.

In response, Dr Gottlieb noted that in some patients, their lives have been prolonged for a much longer period than the studies indicated. Needless to say, for patients with cancer, even a few more months of life can be very significant for them and their families. Additionally, further research can be conducted to ascertain why these patients are able to survive longer than expected, thus leading to improvements in later versions of these drugs.

In an effort to increase the availability of biosimilars, Dr Gottlieb has recently floated a highly controversial idea - circumventing the need to conduct expensive and time-consuming 'bridging’ studies. Under a proposed arrangement, the FDA will allow generics companies to import less expensive biosimilars from the EU without the need to carry out studies to prove that the European biologics are similar to the American versions. He rightly pointed out that in many cases, both versions are manufactured in the same plants anyway. If the plans do proceed, it will address the fact that the US currently lags the EU in terms of approved biosimilars and their usage. Of course, there are many legal challenges and he has instructed the agency’s lawyers to look into viable solutions. Another consideration is whether the biosimilar manufacturers would want to go along and cannibalise the lucrative US markets with supplies designated for the EU; they may want to seek outright US product approvals for their own long-term gains.

Of course, it is still early days, and there is a long way to go to make drugs more affordable in the US. However, as Dr Gottlieb noted, none of his proposals will be a “silver bullet” on their own and will not provide instant answers for patients. But with radical changes, these individual policies will combine to create a more efficient system of drug approval and fairer accessibility to medicines, thus benefiting patients and the industry alike in the long run.

James Huang is a policy researcher and Stephen Huang is a pharmaceutical medicine consultant, both at SCP Medical