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James Nathanielsz, CEO, Propanc Health Group Corporation
Curing cancer is not an impossibility anymore. Essential to any effort to achieve this will be the policies coming from President Trump’s administration, writes James Nathanielsz.
Curing cancer-the second biggest killer of all Americans next to heart disease-is not an impossibility anymore. Essential to any effort to achieve this will be the policies coming from President Trump’s administration.
Appointing Dr. Scott Gottlieb to head the FDA shows the administration understands the need to reduce regulatory red tape. To provide better treatments for cancer, when patients need it and at a price they can afford, three immediate practical measures must be taken:
1. Refocus the FDA
2. Rewrite the rules for compassionate use
3. Reassess the true costs of cancer drugs
Refocus the FDA
Currently, patients are put at risk because they are denied access to unapproved, but effective drugs. Many small biotechs can’t spend the $1 to $4 billion necessary to enter most Phase II or III trials and give up ownership to Big Pharma, which consequently drives up the price of drugs for patients.
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. Recently, a new set of criteria has been developed called the Immune-related Response Criteria, IrRC, which helps to classify survival outcomes for patients 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, contributing to the next wave of approved cancer drugs.
Further, the FDA already has processes in place to speed up the development process which needs to be implemented more often. Fast track, breakthrough and orphan drug designations are examples where the regulatory body can refocus efforts to reduce the time for evaluations and approvals. From Propanc’s 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 this 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.
Rewrite the rules for compassionate use
The compassion in compassionate use has been lost in translation over the years due to the greater threat of litigation.
There are significant moral and ethical hurdles to supplying unapproved medicines which do not have safety and efficacy profiles established. 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. In other words, a “use it at your own risk” policy, allowing for some responsibility from the manufacturer to do its utmost to provide a drug product that is categorized appropriately, and clearly communicate the development stage it is in, and the risks associated with the treatment, by the patients’ healthcare practitioners.
Reassess the true costs of cancer drugs
Drugs which extend patients’ lives continue to undergo significant debate as to their value versus the overall cost to the healthcare system. Recently, it was highlighted how the bulk of expensive cancer treatments were administered during the patients’ last three months of life. To complicate matters, these treatments might have unwanted side effects, which reduce a patient’s quality of life, taking away any meaningful benefit from the small life extension gained.
The Trump administration and Dr. Gottlieb need to introduce a pricing cap to make healthcare affordable and accessible for all Americans. One step toward this price cap is to use existing players within the healthcare space who are already measuring pricing sensitivities. This model is already operating in the UK, as the National Institute for Health and Care Excellence, a non-departmental government body, evaluating the efficacy and economic benefits of a drug before approving its use.
Despite industry concerns, I believe genuinely innovative breakthroughs which extend lives, minimize hospital stays and reduce overall burden to the healthcare system will continue to achieve significant profitability over “me too” products, which are currently crippling the healthcare system.
New and improved standards of cancer 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 become more of a chronic illness than a life-ending one.
James Nathanielsz is CEO, Propanc Health Group Corporation