21st Century Cures Gets Closer To Revolutionizing The Pharma Industry
By Ed Miseta, Chief Editor, Clinical Leader
It’s not often you will hear me celebrating greater government involvement in the pharmaceutical industry. If you’re a regular reader of my articles, you know my background is in economics, and I tend to favor free markets over more central control. But when I first became aware of the 21st Century cures initiative last year, I felt it was something the industry needed. With the time and cost it currently takes to get a new drug from discovery to approval, anything the government can do to better facilitate that process should be welcomed with open arms. Because medicines affect lives, and the FDA regulates the development and approval of new drugs, there is a relationship between pharma and government that needs to be in place.
The pharma industry is certainly different from other industries that consumers interact with on a daily basis. If I don’t like Coke, I can drink Pepsi. If I don’t like game systems, or the cost of them, I can simply choose to do without them. But in that regard, pharma is different.
If you have an ailment that can kill you, purchasing a medicine to treat it is no longer an option or a luxury. It is a necessity. And if there is only one pharmaceutical company manufacturing that treatment/cure, you have to buy it from that supplier. In economics, we refer to that as a vertical, or perfectly inelastic, supply curve.
This is no doubt one of the reasons for the negative image that many consumers and consumer advocates have of the industry. For example, I recently read about a new television manufactured by Samsung. The ultra-high definition (U-HD) TV measures a whopping 110-inches diagonally and has four times the resolution (four times more pixels) of a standard high-definition TV. As of December 30th of last year, the company had received 10 orders for the television. The cost? $150,000.
When news of the offering was announced, I did not hear many complaints from consumers. After all, if you can’t afford it, you simply don’t buy it. Of course, if a top 10 pharma company discovered a cure for cancer and tried to sell it for $150,000, my guess is you would hear plenty of complaints. Pharma may have spent more in R&D than Samsung, but, in a matter of life and death, that fact becomes irrelevant.
Do We Need More Government?
One of the tasks we seem to place upon government is the health and well-being of citizens. Medicare, Medicaid, and now the passing of the Affordable Care Act are proof that some in government feel it’s their duty to provide medical care and coverage to constituents. We also have tax dollars that support various health initiatives, put forth by organizations such as the NIH and universities that are working to uncover cures. And let’s not forget that government, through its regulatory arm (FDA), already oversees the conduct of pharmaceutical discovery, testing, clinical trials, and manufacturing. If a patient needs a drug they can’t afford, or if someone needs to subsidize and oversee the discovery process, most would look no farther than the government.
All of which brings us to 21st Century Cures. This initiative may be new, and it will involve additional government involvement in the market. But if the end result is the strengthening of relationships and more timely and cost effective approval of medicines, it’s hard to see the initiative as a negative. Most pharma professionals I have spoken to are excited about the possibilities it holds.
Last week, the industry got its first look at what the initiative might entail. The U.S. Congress House Energy and Commerce Committee released a discussion draft related to the initiative. The 393-page draft gave a fairly detailed insight into the work our representatives and industry professionals have put into the project, and covers 10 months-worth of roundtable meetings, white papers, and public hearings. The first thing that jumps out at you is the cooperation that went into it. In the Nation’s capital, where little is getting accomplished due to gridlock, Democrats and Republicans seem to have miraculously come together to pave a pharma bridge to the future.
In a nutshell, 21st Century Cures will incorporate patient perspectives into the regulatory process, build the foundation for the future of medicine, streamline clinical trials, modernize product regulation, and most importantly for pharma and patients, accelerate the discovery, development, and delivery cycle for life saving medicines.
21st Century Cures – A Summary
The Committee draft is divided into five major sections, denoted Title I through V. The staff at Pennsylvania Bio reviewed the draft and prepared a summary, which you can see here. I will simply mention a few of the highlights.
Title I deals with putting patients first. Patient centric is a buzz word in the industry today, with pharma companies putting a greater emphasis on incorporating the patient’s voice into all aspects of drug discovery, and particularly clinical trials. It’s no surprise that 21st Century Cures also opted to put patients first, particularly those with unmet needs.
Various subtitles of this section address the approval of breakthrough therapies, the development of antibiotic drugs, priority review and accelerated approval of breakthrough devices, additional oversight of expanded access programs, streamlined review of data supporting the adding of indications, and additional market exclusivity for orphan products.
Title II focuses on building the foundation for 21st century medicine and putting requirements in place for the NIH. Subtitles in this section set guidance for combination products and require the Secretary of Health and Human Services to develop a plan to improve outcomes of patients with chronic disease. Several subtitles deal with the NIH. One would establish a program to help young scientists. Subtitle P would require NIH to support projects that are high risk but could lead to breakthroughs, while Subtitle L requires those receiving NIH grants to share their data.
Title III , which addresses modernizing clinical trials, would streamline the institutional Review Board (IRB) process, encourage broader use of Bayesian statistics and adaptive trial designs, and seek to have the NIH and FDA work with the EU and other stakeholders to establish a global pediatric clinical trial network.
Titles IV and V address issues related to accelerating discovery, development, and delivery of innovation at NIH, FDA, CDC, and CMS, as well as modernizing medical product regulation.
Although there are no guarantees that any of these ideas will get needed medicines to patients faster or at a lower cost, few would argue that the industry should continue to abide by dated doctrines that do not help developers, their partners, regulators, or patients. A lot has changed in the pharma industry in the last 50 years, and technology and science continue to evolve on a daily basis. It’s nice to see government recognize the changes and attempt to evolve the approval process in order to keep up with them.