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Parent Project Muscular Dystrophy Submits Results of Patient-Centered Benefit-Risk Assessment Study in Duchenne & Becker to FDA

HACKENSACK, N.J., Oct. 22, 2015 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) is pleased to submit the results of their most recent patient-centered benefit-risk assessment (PCBR) study to the U.S. Food & Drug Administration (FDA) today. The assessment was done with research partners at the Johns Hopkins Bloomberg School of Public Health. PCBR assessment is an increasingly-favored approach of incorporating patient preference information into regulatory decision-making. PPMD has been a leader in the rare disease community, using PCBR assessment to capture the priorities and preferences of the Duchenne and Becker muscular dystrophy (Duchenne and Becker) community. This study, which was sponsored by Santhera Pharmaceuticals, aimed to evaluate patient and caregiver preferences regarding pulmonary therapies for Duchenne and Becker.

In June 2014, PPMD drafted and submitted the first-ever patient advocacy-initiated guidance for a rare disease to the FDA to help accelerate development and review of potential therapies for Duchenne. One of the recommendations in the guidance was to create partnerships between patient groups and industry to study benefit-risk preferences. PPMD President Pat Furlong said that Santhera Pharmaceuticals, a Swiss specialty pharmaceutical company focusing on the development and marketing of innovative pharmaceutical products for the treatment of mitochondrial and neuromuscular diseases, was one of the first companies to come forward.

"Santhera stepped up immediately to work with PPMD on this first industry-supported benefit/risk study for patients with Duchenne or Becker. Santhera sponsored PPMD to conduct the study, and PPMD supported the participation of a health economics team at JHSPH as study collaborators. We are grateful to have such passionate partners and we believe that this study will do a great deal to help expand the understanding of regulators as they look at risk tolerance in Duchenne and Becker," says Furlong.

About the PPMD Benefit/Risk Study in Duchenne & Becker Patients

The PPMD survey was developed through a community engaged approach, incorporating feedback from individuals with Duchenne, parents, clinicians, and the sponsor. The study variables are based on data from Santhera's phase III clinical trial of Raxone®/Catena® (idebenone) in delaying the loss of respiratory function in patients with Duchenne not using concomitant glucocorticoid steroids.

Using Best-Worst Scaling (BWS) and a community engaged approach, the study comprised four activities:

  • In Activity 1: Prioritization of Treatment Targets, we assessed priorities for treatments that address various Duchenne/Becker signs and symptoms that are not directly related to skeletal muscle function.
  • In Activity 2: Preferences for Pulmonary Therapies we assessed preferences for potential pulmonary therapies for Duchenne and Becker muscular dystrophy.
  • The Activity 3: Intentions to Use we validated and extended the findings of activity 2. After each treatment profile in activity 2, participants were asked whether they would choose the treatment for themselves (or for their child).
  • Finally, Activity 4: Perceptions of Benefits and Harms consisted of a set of questions where participants indicated their perception of an expanded set of benefit, risk, and burden attributes.

One hundred thirty-three individuals participated in all of the activities in the study. Caregiver respondents were primarily biological mothers (76%). The mean age of caregivers was 46.8 years. The mean age of teenage participants was 15.5 years and the mean age of the adults with Duchenne or Becker was 30.7 years.  Most affected individuals had a diagnosis of Duchenne (85%).

Activity 1 Key Points:

  • Respondents prioritized treatments that address cardiovascular and pulmonary benefits. The greatest priority is for treatments that address weaker heart pumping. Treatments that address lung infections and cough strength were rated as the second and third most prioritized, respectively.
  • Stratified samples reveal that caregivers and patients had similar priorities.

Activity 2 Key Points:

  • Participants demonstrated greatest preference for maintaining cough strength. Cough strength maintained for 10 additional years is the attribute with the highest preference score and cough strength maintained for 2 additional years is the attribute with the second highest preference score. Respondents viewed "no benefit" to cough strength and lung infections as a disutility.
  • Greater importance reported towards the benefits relative to the risks demonstrates favorable benefit-risk profile, such that people are likely to accept the risk in exchange for the benefit.

Activity 3 Key Points:

  • Respondents almost always chose to accept a treatment with a strong benefit and little risk and to accept a treatment with a strong benefit and high risk more than two-thirds of the time.
  • The probability of acceptance for the treatment profile with mixed benefit (cough highest benefit; infection moderate) and risk (diarrhea worst; blood draw moderate) was 85%.

Activity 4 Key Points:

  • The large majority of parents and patients perceive the three benefits (general pulmonary, maintain cough strength, and fewer lung infections) as important. Perceptions of burden displayed more variation, though the majority of respondents reported low perceived burden for each of the three attributes (pill burden, blood tests, and diarrhea).

Study Implications:
Overall, using multiple approaches, the study demonstrates that patients and caregivers value pulmonary benefits and are willing to accept risks and burden to achieve those benefits. The study shows relatively little difference between patients and caregivers in their treatment priorities and risk tolerance.

Our participants were willing to accept an increased risk for diarrhea, even a high risk of 50%, plus accept blood monitoring twice a year to maintain their current level of cough strength for up to 10 additional years. Our participants perceive pulmonary benefits as important, regardless of whether they are defined globally (i.e., slowing pulmonary decline) or as meaningful pulmonary outcomes. Though participants do not discount treatment risk and burden, the importance of potential benefits are rated significantly higher than the side effects and burden. The majority of patients and caregivers intend to use a drug with the profile most similar to idebenone.

Finally, this study provides an additional set of evidence to support the use of stated preference methodologies that are applied through a highly collaborative, patient/caregiver engaged approach that benefits from disease community leadership and health economic expertise—in this case, with support from trial sponsor willing to incorporate the preferences of patients and caregivers into their regulatory approaches.

Holly Peay, PhD, a lead researcher on this study, believes this study will be extremely useful for the FDA, "Based on this study, PPMD and collaborators from Johns Hopkins are hopeful the FDA will have a better understanding of preferences of Duchenne patients and caregivers in regards to a specific pulmonary therapeutic target. Especially when faced with low drug-associated risk and few side effects, we hope that this data will inform a permissive response by the agency in light of data showing patient and caregiver prioritization of pulmonary benefits."

About Duchenne and Becker muscular dystrophy 
Duchenne muscular dystrophy is the most common fatal genetic disorder diagnosed in childhood, affecting approximately 1 in every 3,500 live male births (about 20,000 new cases each year worldwide). Because the Duchenne gene is found on the X-chromosome, it primarily affects boys; however, it occurs across all races and cultures. Duchenne results in progressive loss of strength and is caused by a mutation in the gene that encodes for dystrophin. Because dystrophin is absent, the muscle cells are easily damaged. The progressive muscle weakness leads to serious medical problems, particularly issues relating to the heart and lungs. People with Duchenne typically live into their late twenties.

Becker muscular dystrophy, which is less severe than Duchenne, occurs when dystrophin is manufactured, but not in the normal form or amount.

About Parent Project Muscular Dystrophy (PPMD)
Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne Muscular Dystrophy – their mission is to end Duchenne. PPMD invests deeply in treatments for this generation of people affected by Duchenne and in research that will benefit future generations. They advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. They demand optimal care, and strengthen, unite and educate the global Duchenne community. Everything PPMD does – and everything they have done since their founding in 1994 – helps people with Duchenne live longer, stronger lives. For more information, visit

About Santhera
Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases. Santhera's lead product Raxone® is authorized in the European Union for the treatment of Leber's Hereditary Optic Neuropathy (LHON). Santhera develops Raxone®/Catena® in two additional indications, Duchenne Muscular Dystrophy (DMD) and primary progressive Multiple Sclerosis (ppMS), and omigapil for Congenital Muscular Dystrophy (CMD), all areas of high unmet medical need. For further information, please visit the Company's website

Raxone® and Catena® are trademarks of Santhera Pharmaceuticals.

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SOURCE Parent Project Muscular Dystrophy
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