Expedited Specialty Drug Approvals From Multiple Points of View


While the job of the FDA is to protect the public from ineffective drugs, patients in need of life-saving treatments push for accelerated approval of specialty drugs.

As a society, we have come to expect a medical solution for whatever ails us. A diagnosis seems to always be followed up by an option or two to consider.

If the initial options are not to your liking, there is always the internet to seek additional ideas, such as enrollment in a clinical trial, a non-traditional medicine plan, or a drug therapy offered outside the United States. As a society, we feel that it is our right to have options, and to receive the best cutting edge treatments that science can offer.

The FDA drug approval process is not perfect, but it does keep the American public safe from ineffective products or counterfeit products coming to the market. In September 2016, the FDA granted accelerated approval for a drug that was based upon a trial of 12 patients that seemed to circumvent the requirement to demonstrate a clear clinical outcome.

The result of this decision created a buzz of controversy, concern, and commentary. The accelerated approval pathway was created for drugs that treat serious, life-threatening diseases that have limited or no current treatment options.

This pathway permits approval based upon well-controlled studies focused on a surrogate endpoint that leads to a predictable clinical benefit. The clinical benefit needs to be reportable as an improvement in patient health and/or functions or length of survival.

The accelerated pathway offers access to promising therapies while the manufacturer continues to conduct additional trials to verify the outcomes in a larger subset of patients. A pharmacist might question such a small trial with minimal data being able to gain accelerated approval.

That the FDA, known for delaying approval for minor fixes to a medical guide, can somehow approve a 12-person study with limited outcomes data, seems illogical. The available drug information about side effects, drug interactions, and how to monitor appropriate dosing and clinical impact would be a challenge for the health care team to reference or extrapolate from a trial of 12 patients.

Anyone involved with the health care of a patient living with an incurable disease wants to do all they can to make a difference for that person and enhance their quality of life. To dispense a drug with no known clinical benefit is difficult for many pharmacists, and seems to be against the pharmacist’s oath.

A specialty pharmacist would be an appropriate resource to include in the distribution and data collection needed once the accelerated approval product comes to market, but it does not mean the drug is guaranteed to deliver results.

When a drug is designated as a specialty drug, it is expected to command a high price and to deliver incredible results. The risk of accelerated approval is the withdrawal of a drug from the market if it does not maintain or enhance the initial outcome claims that lead to its approval.

Any business student with an interest in health care would demand a specific clinical result from a medical treatment, including a high-cost drug therapy, before covering the costs. It is a known fact that the US health care system cannot sustain the rising costs of care, and the change in reimbursement from fee-for-service to outcomes-based payment schedules is soon to be a reality.

Health care is a business and there will be conflict each time the FDA approves a drug with limited outcomes data, a high price tag, and the expectation the drug will be paid for under a medical or prescription benefit plan. Insurance companies and health plans will evaluate and strictly control which high-cost products will be reimbursed, as patient improvement and outcomes will be required for reimbursement.

The idea of a novel drug gaining accelerated approval from the FDA and not being a covered expense under medical or prescription benefits due to a lack of efficacy and outcomes data will be surprising to many Americans. Any drug approved with questionable results data is at risk of being exempt from formularies, resulting in a difficult drug launch and minimal market capture. It is not realistic to expect anyone to pay for something that does not work.

If you followed the approval of Sarepta’s product, Exondys 51 (Eteplirsen) injection, there was plenty of pressure applied to the FDA from the families of children with Duchenne Muscular Dystrophy (DMD) and lawmakers to escalate the approval process. As a parent of a sick child, you would do anything to make your child healthy, knowing there is a potential new drug to treat a disease with no other options would be enough to demand the drug be fast-tracked through trials to get to market.

The patient voice is strong, and when a group with a common bond joins together, they demand attention. In the early years of research and development of HIV/AIDS treatments, the voices of social and political activists forced changes that resulted in HIV drugs getting through trials and up for approval in less time. If it had not been for the voices of the activists, many more people would have suffered the effects of the virus and developed AIDS.

The demands of the public, whether parents, activists, or lawmakers is heard, but it may not always be appropriate to lower the standards of a program just to quiet a vocal group. It is not clear as to why the FDA approved a drug with such limited information, using the example of Exondys 51, but it does offer a lot to think about and which argument speaks the loudest to you.

If you had to pick a side of this argument and defend your pharmacist position, would you align with the FDA’s decision to approve or would you delay approval until additional data was available? What if you ran a self-insured business with a limited budget to cover the medication needs of 100 people, how do you spend the money?

Do you spend a large portion of your budget on 1 patient for a drug with little to no evidence of being effective to improve the patient’s condition? What if your child was diagnosed with an untreatable condition that suddenly had a potential treatment with a small chance of improving your child’s quality of life, where would you stand?

My position on this topic? I find being a pharmacist rewarding, fulfilling, and challenging and I always want the best outcome for every patient and to trust what I am dispensing is safe and effective.

As a person intrigued by the business of medicine and the innovation of health care, it is fascinating to work through the challenges presented by the accelerated approval process. As a parent who went through testing with my son to determine if he had muscular dystrophy, I will say the love for my child outweighs any other position presented.

I would have been standing in front of the FDA offices asking for accelerated approval if it meant slowing the disease’s progression or improvement. Even the slimmest hope for improvement is worth the battle.

My son is healthy today; the results were negative for any form of muscular dystrophy. I do not know what it means to be the parent of a child with DMD, but I share in the hope for a game changing treatment.

About the Author

Jill Schachte earned her BS in Pharmacy from Duquesne University and her Masters of Science in Pharmacy Business Administration (MSPBA) from the University of Pittsburgh. Jill has spent the past 20 years working in specialty pharmacy, starting as a clinical pharmacist at Stadtlanders Pharmacy and working in a variety of management roles in specialty pharmacy operations for CVS Health. Jill’s current role is on the CVS Specialty Professional Practice team, with a focus on accreditation and compliance for all the specialty pharmacy locations within CVS Health.

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