PFDD Program and the Evolving Role of Patients in Healthcare, Drug Development and Access

The Affordable Care Act brought increased patient access to healthcare, but with the greater access comes greater responsibility for shouldering the burden of out-of-pocket costs.   As these costs can be substantial, the role of patients in healthcare decisions – from drug development to drug access – is expected to evolve and grow.

Long recognizing the value of patient insights in drug development, FDA has made great efforts to integrate patient perspectives into its benefit risk framework for a number of disease areas through the Patient-Focused Drug Development (PFDD) program, an initiative of the Prescription Drug User Fee Act (PDUFA) V of 2012. One of the common points patients shared at the PFDD workshops held over the past two years is the feeling that their “chief complaints” or needs are not being factored into drug development plans, including measures of a drug’s benefit in clinical trials.

To get an inside read on why this may be occurring, and what can be done to correct it, we spoke with representatives from the Obesity Action Coalition, the IBD Support Foundation and the Arthritis Foundation, all of whom are actively engaged in giving a voice to patients in their communities and improving access to optimal care. They shared their perspectives on how the role of patients has evolved in the past decade and what can be done in the future to better factor patients’ feedback into drug development and access planning.

Ted Kyle, RPh, MBA is Chairman of the Obesity Action Coalition (OAC). OAC is a national non-profit organization dedicated to giving a voice to the individual affected by the disease of obesity and helping individuals along their journey toward better health through education, advocacy and support.

FDA and industry are making great efforts to gain the patient community’s guidance in drug development. How have you seen the role of patients evolve in the drug development process over the past decade?

I have seen patients become involved more and more actively in the drug development process over the past ten years. It’s a trend that had its beginning is the 1980s with the health crisis of AIDS. A whole community found its voice when an inflexible drug development and approval process threatened its survival. Over the last ten years, I’ve seen a similar, though less dramatic change in the development and approval of drugs for the treatment of obesity.

In 2008, an entire decade had passed without FDA approving a single new drug for obesity treatment and, instead, withdrawing one of them from the market. Patients, their advocates, and leading researchers reached out to FDA to express concern about innovation for treating obesity being shut down. The result was that FDA began to account for the unmet medical need for obesity treatment options. Four new drugs and three medical devices for treating obesity have been approved as a result. Research organizations are renewing their commitment to obesity treatment innovation.

Patients currently do have a platform for sharing their insights at FDA Advisory Committee meeting open public hearings. Has the impact of testimony at these meetings changed?

Ten years ago in public hearings for new obesity treatments, patient voices were rarely heard or taken seriously. In hearings today, patient perspectives are given full consideration by advisory panels and often cited in the panelists reasoning for their votes. This shift in perspective has flowed from a much larger trend toward more patient-centered medical care.

Marci Reiss, LCSW is Founder and President of the IBD Support Foundation (IBDSF), a non-profit healthcare foundation whose mission is to improve the quality of life for individuals with Crohn’s Disease and Ulcerative Colitis through psychosocial support, education and research.

Patients believe their “chief complaints” are not be factored explicitly into drug development plans. Why do you think this is happening?

The current state of the medical world doesn’t provide sufficient opportunities for the patient’s voice to be heard. As a result, stakeholders are viewing disease treatment from their own perspectives, and not sufficiently from the patient perspective. For example, in the area of Inflammatory Bowel Disease, physicians and researchers may be looking at treatment from a reduced inflammation perspective, the pharmaceutical industry may be looking at treatment from a business perspective, and payers are likely looking at it from a cost perspective. There are few avenues to enable the true patient perspective to be heard. And the patient perspective may be far more multi-dimensional than the other perspectives.

So how can we do a better job of translating patient feedback into research-ready information?

As clinicians, our goals must include helping patients successfully incorporate illness into their lives so that they can realize their potential and achieve their hopes and dreams. To do this, we need to take into consideration how a patient’s personality, family, employment circumstances, finances, culture and religion influence their ability to manage their condition. This requires looking at the patient’s needs from a number of perspectives. Industry should consider these multiple perspectives in their drug development planning as they could help identify the various needs of patients.

Sandie Preiss is Vice President of Advocacy and Access at the Arthritis Foundation. The Arthritis Foundation is the leading nonprofit organization dedicated to the prevention, control and cure of arthritis in the United States.

As patients take on more of the burden of healthcare costs, how can they get more of a share of voice in access decisions that are being made?

With almost fifty-five percent of America insured by employers, there is a huge opportunity for education with employers. Drugs are replacing devices and surgeries, and because of this, pharmaceutical budgets are going up while medical budgets are going down. But people’s thinking hasn’t caught up with this shift.

So as employers are another audience that could benefit from patient feedback?

Yes. Not all of employers may be looking at the total cost of a patient’s experience. Rather, we hear they often look only at their drug budget, separate from their other medical expenses and don’t take into account the avoided costs and the fact that with these medication employees often have lower absenteeism.   Patients and industry will both benefit from educating employers on the long-term benefits of early treatment to prevent progression of diseases like Rheumatoid Arthritis.


Eileen Crowley brings more than 15 years of experience as a communications and advocacy specialist to clients in the pharmaceutical, medical device, and public health arenas. Eileen works with 3D clients to engage with professional and patient groups and to leverage that support throughout the product lifecycle, including FDA advisory committee meetings and grassroots disease education programs. Connect with Eileen on LinkedIn.