James Nathanielsz Chief Executive Officer Propanc Health Group
How Will It Impact America’s Second Biggest Killer?
Healthcare remains one of the biggest issues on the Trump administration’s agenda. It’s no wonder: Healthcare spending heads toward 20% of GDP; inefficiencies at the FDA are accused of stymieing innovation; Obamacare is perceived as being mired in red tape and inefficient, while 45% of the population—133 million Americans—have at least one chronic disease.
The key question on everyone’s mind is whether President Trump’s reformist agenda can lead to fundamental improvements in healthcare?
For instance, cancer is the second biggest killer of all Americans next to heart disease—however, it could be the one disease to see the most transformation in this generation. According to the American Cancer Society, about 1.7 million new cancer cases are forecast to be diagnosed, with over 600,000 Americans expected to die from cancer this year alone. Meanwhile, direct medical costs related to cancer treatment reached a whopping $87.8 billion in 2014.
So, where should the Trump administration start? Appointing a new head of the FDA with strong ties to Wall Street and the pharmaceutical industry with an appetite for reduction of regulatory red tape was a good first step. Scott Gottlieb, M.D., appears to have the energy and experience within the sector to introduce the reform that the Trump administration needs to overcome the current FDA tag of being “slow and burdensome,” provided he survives his confirmation hearings and avoids stumbling over his conflicts of interest via his ties with Big Pharma.
This first key post by the Trump administration will be critical in revamping the FDA and a pillar in helping the Trump administration establish a revamped national healthcare system. To provide better healthcare, including better treatments for cancer, when patients need it and at a price they can afford, three immediate practical measures must be taken:
- Refocusing the FDA rather than restructuring
- Re-evaluating existing ways to improve access to new treatments, such as compassionate use, rather than driving down evidentiary standards
- Evaluating the economics of new cancer drugs, not just overhauling the rules for speeding up generic approvals to bring down the costs
Refocusing the FDA
Many argue that the current healthcare system is flawed. Patients are put at risk because they are denied access to unapproved, but effective drugs. Many small biotechs can’t fund most Phase II or III trials and therefore give up ownership to Big Pharma, because the cost to bring a new medicine to market ranges from $1 to $4 billion, which drives up the price of drugs for patients.
However, rather than restructuring the entire FDA, tackling specific issues within the drug-approval process could make a significant impact. For example, the evaluation of immuno-oncology drugs by a specialized unit would lead to a better understanding and acceptance within the industry, because the effects of these drugs are not as rapid as conventional treatment approaches. Instead, their effects are characterized by a cellular immune response, followed by potential changes in tumor burden or patient survival.
In the last few years, a new set of criteria has been developed called the immune-related response criteria (IrRC) which helps to classify survival outcomes for patients that are comparable to conventional responses, identified using RECIST (response evaluation criteria in solid tumors). It arose out of observations that immuno-oncology drugs would fail in clinical trials that measured responses using the WHO or RECIST Criteria, because these criteria could not account for the time gap in many patients between initial treatment and the apparent action of the immune system to reduce tumor burden.
With Dr. Gottlieb and the FDA leading the way in accepting these new criteria for drug approvals and altering the hurdles for evidence around efficacy, it may lead to broader acceptance within the healthcare sector; thus, contributing to the next wave of approved cancer drugs.
Furthermore, the FDA already has processes in place to speed up the development process. Fast track, breakthrough, and orphan drug designations are examples of areas where the regulatory body can simply refocus their efforts to reduce the time for evaluations and speed up the approval process. From experience, an initial response to an orphan drug designation application can take up to 180 days for the FDA to evaluate. Even then, the deadline is non-committal.
For life-threatening illnesses like cancer, orphan drug designation has the potential to fast track development and commercialization of the next breakthrough drug. For emerging companies, achieving orphan drug designation has the potential to unlock significant value and assist with crucial fund raising efforts needed for future trials. Better leveraging these approval processes would enable the FDA to create significant, tangible benefits to the healthcare industry without requiring a total restructuring.
Improving Access to Treatments
There are significant moral and ethical hurdles to supplying unapproved medicines which do not have safety and efficacy profiles established. Simply lowering the barriers to drug approval creates added risks for patients. However, the existing frameworks, such as compassionate use, could be restructured to reduce regulatory and legal burdens on the drug development companies, in exchange for greater access to patients deemed suitable for the experimental treatment.
As long as the patient, their healthcare practitioner, and families accept responsibility for receiving an experimental treatment, on the basis that fundamental obligations are met by the drug developer for a preapproved product, patient access to treatment can improve and they can receive the experimental treatment, without the threat of litigation. Compassionate use, as its name implies, can only be implemented on a basis of trust and duty, whilst recognizing that companies and the people who run them want to assist the very patients for whom they are developing their drug.
Economics of Cancer Drugs
Today, the sensitivity surrounding the pricing of drugs, especially cancer drugs, which arguably only marginally extend survival in some cases, remains extremely high. Despite scepticism and paranoia regarding pharma companies and pricing, pharmaceutical executives are acutely aware that reducing their drug prices is becoming progressively more important for a sustainable business model. Right now, the advancement of science and the engineering of highly synthesized and targeted molecules has become increasingly difficult and expensive. Consequently, many argue that speeding up the generic drug approval process will increase competition and ultimately lead to more generic treatments on the market for patients.
However, this does not impact new drug approvals, where high barriers to development mean high prices remain. Despite the biopharma industry’s efforts to avoid pricing regulation, the Trump administration and Dr. Gottlieb need to introduce a pricing cap to make healthcare affordable and accessible to Americans, or risk catastrophic consequences such as limited access to cancer treatment among the vulnerable patients who need it most, but cannot afford it. One step toward placing a cap on pricing is to use existing players within the healthcare space who are already measuring pricing sensitivities. This model has been operating in the U.K. for several years, where the National Institute for Health and Care Excellence (NICE), a non-departmental government body, evaluates the efficacy and economic benefits of a drug before approving its use, and has the power to implement pricing caps where necessary.
In America, some states are passing legislation requiring drug companies to justify major price increases, and to calculate the financial effect on insurance premiums. Vermont passed the first legislation in June last year and California appears to be following suit. However, a Federal policy shift from the Trump administration is what’s needed to make this uniform, less cumbersome; but still utilizing the insurance payers to establish a pricing justification mechanism, which facilitates an open market between drug company suppliers and insurance payers, whilst creating mandatory pricing controls overseen by the Federal regulatory framework supporting the sector. Genuinely innovative breakthrough products that extend lives, minimize hospital stays, and reduce the overall burden to the healthcare system will continue to achieve significant profitability over the “me too” products, which are currently crippling the healthcare system.
Whether the Trump administration can cure cancer will be answered over time. The sector will continue to pursue novel and inventive approaches to improve survival, but this must not be at the expense of patient safety. New and improved standards of care need to be supported by a rigorous, yet flexible regulatory framework. Practical measures can be taken to fast track new products, which could see cancer becoming more of a chronic illness than a life-ending one. But these new approaches need broader acceptance within the very sector looking to support cancer patients. Someone with a balanced view and strong leadership is required to support innovation and potential breakthroughs. It is possible; but whether Dr. Gottlieb and the Trump administration will achieve their goals to reform America’s healthcare system will be interesting to observe.
James Nathanielsz (email@example.com), is CEO of Propanc Health Group.