With so many biosimilars in the U.S. pipeline, the federal government has had its hands full in recent months managing legislation and expectations from patient groups, health organizations, pharmaceutical companies and advocates. While debates rage on and in-fighting has severed several previously harmonious relationships, the fall is expected to bring some big changes to the biosimilar world. To better understand what September and beyond hold for the space, I sat down with Dr. Sarfaraz Niazi, a biosimilar expert and advisor on several biologics-based bills introduced in the House and Senate.
Nicole F. Roberts: Dr. Niazi, you have been a pioneer in the biosimilar space, not only as an inventor but as an advocate and now a policy advisor. Seeing things from so many sides, can you briefly share your motivation and tell us about the evolution of the field?
Sarfaraz Niazi: The story goes back a few decades. It comes from the realization that the future of humanity will depend on biological drugs, essentially those we produce in our own body’s pharmacy that is open 24/7. We’ve been able to find treatments for diseases that were considered untreatable. And I am projecting that within a couple of decades, we will no longer have anyone dying of cancer.
Roberts: That’s a bold statement.
Niazi: You see, I began working on biosimilars long before the concept arose in the early 90s, first with the U.N. entity in Italy, leading to technology that I gave to several countries. In early 2000 I established the first biosimilars company in Chicago, raised $500 Million, and secured several FDA approvals. So I’ve been around the system since the beginning.
It’s important to understand that biological drugs are chemicals but with a variable structure, as they are produced in a biological entity. And that means billions of dollars to get a new molecule approved. And so, they remain unaffordable. Ironically, the cost of manufacturing is very affordable. But, when biosimilars are treated as new drugs they cost millions. The estimate from McKinsey remains at $100-300 million for each biosimilar. This is not acceptable. So, I’ve begun educating the regulatory agencies on how to rely on science.
There are four significant changes in the FDA guidelines I’ve worked on, and another in the works.
– First, a guideline for analytical testing that the FDA withdrew once I filed a citizen petition to show that it made no sense.
– Second, came the acceptance by the FDA that for those molecules with pharmacodynamic markers tested in healthy subjects, there is no need for testing patients, which adds 70% cost of development.
– Third, removing “animal toxicology” from the testing requirement of biosimilars since these drugs are just added nutrition to animals. They said this could not be done since it was part of the legislation. So, I published my thesis in Science, took it to Congress, and convinced them to amend the legislation. It was approved with a 100% vote, and came out as the FDA Modernization Act 2.0.
– Fourth, is an attempt to remove “interchangeable” biosimilars. All biosimilars should be interchangeable. I used the same tactics and published scientific papers proving this does not make sense. People agreed, and now there is a bill in the Senate to amend the BPCIA.
And now comes the most significant change—no efficacy testing in patients. After dozens of scientific papers and talks, the FDA has agreed to call a meeting to decide. Once this barrier is removed, the doors to biosimilars will open wide across the globe.
Roberts: That’s a long list of changes. Can you describe the impact of biosimilars cost for patients?
Niazi: More than 70% of new products are filed as biologics. So ultimately, these products will represent most treatments. And all of these medicines are lifesaving, thus making them affordable is an ethical call. I am confident that soon these drugs will become the same as the chemical generic drugs and we will not be talking about their affordability. But, there are significant hurdles in the U.S. Most important is the distribution system, like PBMs.
Roberts: You said right now you’re dealing with two bills in the Senate, one for removing animal toxicology and the other concerning interchangeability. Are you able to share your experiences working with legislators?
Niazi: The animal toxicology bill was approved as an amendment signed by President Biden in late 2022. The experience of dealing with the Senate was interesting. For example, by placing the emphasis on the rationality of testing rather than as an act to prevent cruelty to animals worked well. We got 100% vote in favor. The other for interchangeability did not go very far in 2022. Republicans introduced the bill. But, I have been able to get Democrats to join. Senator Lujan in particular, who had also initiated the animal toxicology bill.
Until now, I never realized oftentimes bills have to be initiated several times. To help this one, I published a peer-reviewed, scientific paper that analyzed every conceivable objection. This article and my letter will be given to all Senators and Representatives. But I learned the real challenge is to explain a technical and mathematical issue to non-experts.
Roberts: Earlier you mentioned you’ve pushed to remove clinical efficacy testing of biosimilars. But there are a lot of people who believe that the proof required to demonstrate safety and efficacy for patients – is patients. How do you dispute that, and do you think the FDA will go along with it?
Niazi: This is a formidable barrier, historically and intuitively. Clinicians like to see data from the patient population, and I have no issue with that. Here we run into a scientific argument that is difficult to present because of the mindset of stakeholders. In simple language, proving that two things are different, after proving they are not different, is almost impossible. None of the studies conducted failed because they cannot fail. So, why are we doing these studies;? It is merely a check list to satisfy the popular belief. No more.
I have to admit, this was the most unpopular effort as even those who believe in science had second thoughts, including FDA reviewers. But finally, the FDA has called a meeting for September 2023 to deal with this issue. I have a pretty good idea of how this meeting will end. The FDA will agree with me. This will work because recently, I was able to get such waivers for antibodies for the first time, with the same argument. After September, I hope developers will also challenge the FDA and get out of paying for testing in patients. However, this is not popular with big pharma, they want to keep costs high. And then, it will be up to more competent biosimilar developers to take the lead.
Roberts: You have talked about and written extensively on the affordability of biological drugs as the most critical issue today. Why is there so much discord between manufacturing costs and the market cost, and who or what is responsible for keeping drugs so expensive in the US, despite patents and exclusivity rights expiring?
Niazi: A new biological drug costs a couple of billion dollars to get approval. When I was working with President Obama, before he got into office, we wrote a draft of the BPCIA and included seven years of exclusivity for originators to recover their investment. If it were not for the Affordable Care Act, the BPCIA would not have made it. It was buried somewhere, and most didn’t read it. Anyway, the bill passed, and when everyone realized what seven years meant for patents and earnings, all hell broke loose. In the end, we landed at a compromise of 12 years.
But what you have to understand is that regardless of who is paying, it all adds to someone’s bottom line, and since then the challenges have come from everywhere. During the Trump administration, CMS reimbursed but would not question pricing. So I started a vigil when President Biden came in with Vice President Harris. During their time the Inflation Reduction Act allowed CMS to reduce the price of biological drugs. Although after the Senate passed the bill, it stopped in the House. And surprisingly, associations that claim to support the biosimilar industry created aggressive marketing campaigns about how it will destroy biosimilars, which is wrong.
Another current challenge to reduce costs is taking PBMs to task. They really are destroying everything. But, I have a tool in the toolbox—another paper I am working on now. I wrote a long letter to President Biden telling him that biotechnology needs to be stirred up in the U.S. Six months later, he signed an order to fund innovation in the space.
Roberts: Is this vigil, as you call it, why you give all of your inventions to the public domain? You own many U.S. patents on technology and new drugs. Why don’t you choose to capitalize on those?
Niazi: My experience teaches me that we should fight for our beliefs and not give up. My mentality is many should receive the fruits if an invention is fruitful. With over 100 patents in many fields, from biotechnology to defense systems to automobile designs, I have enjoyed being identified as someone to bring up a creative idea. To help others do the same, I have secured hundreds of patents for scientists across the world, even paying myself for the filing fees. The idea is to innovate. I withdrew my global filings. Besides, who would have the money and time to watch out for the usurpers? In one country that used many of my environment control patents, I was recognized with the highest civil award, but more than that I received confirmation that it has helped reduce pollution.
Roberts: Maybe that’s your next area of expertise and political influence.
Niazi: Maybe it is.