A new dynamic is at play in the industry today. While U.S. drug development always included a patient-focused viewpoint, patients today have more knowledge and influence than ever before. Taking advantage of the extraordinary reach of online medical information, patients are aggressively participating in their own healthcare decisions by researching medications and treatment options. They are also gaining in influence as more people become part of the healthcare system as a result of the Affordable Care Act. While they will have greater access to new medications, patients will also face an increased cost burden for medicines. This dynamic will be increasingly challenging to decision makers (such as payers and providers) as the patient voice is a critical component of the benefit-risk calculations for medications. Industry must also redouble its focus on patient perspectives on issues such as defining value, maximum acceptable risk and minimum acceptable efficacy, particularly during new drug development.

To that end, the Food and Drug Administration (FDA) has embarked on a major effort called the Patient-Focused Drug Development (PFDD) initiative. It is a key provision of the Prescription Drug User Fee Act V designed to improve drug development and review. As part of this initiative, the FDA will take a systematic approach to conducting benefit-risk assessment for new drugs by considering disease severity and unmet medical needs. These factors will include analysis of condition, current treatment options, benefits, risk and risk management (e.g., REMS). One of the critical factors the FDA will consider is the patient perspective regarding the impact of a drug or disease.

Is Patient-Focused Drug Development New to the FDA?

Patient-centered drug development is not new but its market impact has gained importance. The FDA has included the patient perspective during FDA advisory committee meetings since 1991—and also during the review of new medical products in selected meetings with the FDA and product developers. It encourages and fosters sponsors to use patient-reported outcome measures in clinical trials, such as impact on quality of life or pain control, and in medical product development which may support labeling claims.

Today, the FDA PFDD initiative gathers patients’ perspectives earlier in the drug development process. The initiative relies on public workshops and a systematic approach, signaling that the FDA considers the patient perspective particularly important in the evolving healthcare space. It has been developed and executed in consultation with patient and consumer advocates, healthcare professionals, other public stakeholders and drug industry representatives. This collaboration with the patient community is vital to obtaining the patient’s perspective on disease severity and currently available treatments. The intent is to ensure a more thorough understanding of the severity of the treated condition and the adequacy of the existing treatment options.

The FDA solicited public comment from patients, patient advocates and advocacy groups, healthcare providers, caregivers, professional societies and academic experts, pharmaceutical companies and others prior to finalizing the list of disease areas.1 It is focusing on 20 different disease areas and will hold public meetings four times a year over a five-year period, examining a different disease area each time. The final selection criteria for disease areas are those:

  • That are chronic, symptomatic or affect functioning and activities of daily living;
  • For which aspects of the disease are not formally captured in clinical trials; and
  • For which there are currently no therapies or very few therapies, or available therapies do not directly affect how a patient feels or functions.
  • That reflect a range of severity, from diseases that are life-threatening to those that are mild and symptomatic;
  • That have a severe impact on identifiable subpopulations, such as children or the elderly; and
  • That represent a broad range in terms of size of the affected population, including common conditions experienced by large numbers of patients and rare diseases that affect much smaller patient populations.

The FDA also made clear that the final list of disease states does not represent the conditions that are of highest priority in its work. Rather, they are conditions where little is known about the burden of disease or where outcomes to measure benefits are lacking. Public meetings on selected disease areas began FY 2013 and are:

  • Chronic Fatigue Syndrome and Myalgic Encephalomyelitis (April 25)
  • Human Immunodeficiency Virus (HIV) (June 14)
  • Lung cancer (June 28)
  • Narcolepsy (September 24)

See the complete list for meeting planned for FY 2014 and FY 2015 below:

  • Alpha-1 antitrypsin deficiency
  • Breast cancer
  • Chronic Chagas disease
  • Female sexual dysfunction
  • Fibromyalgia
  • Hemophilia A, Hemophilia B, von Willebrand disease, and other heritable bleeding disorders
  • Idiopathic pulmonary fibrosis
  • Irritable bowel syndrome, gastroparesis and gastroesophageal reflux disease with persistent regurgitation symptoms on proton-pump inhibitors
  • Neurological manifestations of inborn errors of metabolism
  • Parkinson’s disease and Huntington’s disease
  • Pulmonary arterial hypertension
  • Sickle cell disease

Patient Viewpoints: FDA Listening Carefully

The FDA’s PFDD is currently gathering patient perspectives as a component of the analysis of the condition and current treatment options by asking the following questions:

  • What clinical manifestations of the disease have the greatest impact on you?
  • Are there other aspects of the disease that have a significant impact on your daily life (e.g., impaired mobility, sleep problems, etc.)?
  • How do the clinical manifestations change with disease progression?
  • How do the other aspects of the disease change with disease progression?
  • What is the current standard of care?
  • What therapies are being used to treat the condition (including approved and indicated therapies, drugs used off-label, and non-pharmacological therapies)
    • How effective are the existing therapies at treating the clinical manifestations of the disease?
    • How well do they mitigate other aspects of the disease?
    • How well tolerated are the existing therapies?
    • How does the effectiveness of approved therapies change with progression of the disease?
    • Does therapy effectiveness vary by patient sub-population?

The FDA is leading the industry with its PFDD initiative. However, other health authorities, such as the European Medicines Agency (EMA), are focusing on more systematic approaches to benefit-risk assessment.

Next Steps for Marketing Professionals

1. Stay abreast and follow the FDA PFDD proceedings in collaboration with your company’s regulatory affairs group.

2. Work with your colleagues dedicated to patient advocacy groups to help increase the awareness of this important FDA PFDD initiative. Organizations including the National Health Council assist patient organizations with tools to ensure their communications with the FDA regarding benefit-risk are comprehensive.

When considering the benefits and risks of medicines, patients provide a unique and valuable perspective. The FDA’s efforts to seek patient input into the assessment of the benefit-risk balance of new medicines should be applauded. Patients can identify areas of unmet medical need, provide critical perspective on the impact of a disease and its manifestations and uniquely inform the development of outcome measures meaningful to other patients and themselves. An active dialogue between patients and industry can result in greater transparency and aid in the development of new medicines.

The FDA will determine, in time, how it will leverage patient focus into the drug development and approval process as a result of these public meetings. The PFDD initiative may mark the beginning of introducing patient perspective earlier and earlier. This could include setting the research agenda; developing research questions to be answered in clinical development; selection of outcomes and comparators; and developing innovative medicines that provide value to a company’s stakeholders, including the patients themselves.


1. Federal Register Notice. Vol 78 No. 70/Thurs April 11, 2013

  • Cheryl Beal Anderson

    Dr. Cheryl Beal Anderson is the Senior Director, Regulatory Development Strategy and Registration at Lundbeck. She is a 20-year pharmaceutical and biotechnology industry regulatory affairs leader with technical and supervisory experience at Fortune 500 companies.


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