It’s Not the Same for Patients With Rare Disease
What would you do? Your child has a rare, progressively debilitating, and ultimately deadly disease–and you just learned that a new treatment has been developed that may provide a cure. A cure–what you and your family have always hoped for. Then you learn the bad news. There is a 20% chance that your child will die immediately as a result of the treatment. Should you take this chance, or should you choose instead to focus on making certain that he lives out the last 5 to 6 years of his life with the best possible quality of life?
Certainly it’s a decision that no parent ever expects to have to make. And there is no right answer. But these types of discussions are front and center at the Global Genes Annual Summit. Connexion Healthcare’s Rare Disease Center of Excellence attended this year’s conference on September 11 and 12 in Huntingdon Beach, California, as we do each year as a corporate alliance member.
Risk-benefit analysis is an assessment made regularly in drug development and regulatory review, but it’s also an important component of the discussions that take place among physicians and patients and their caregivers. Although the concept itself is fairly straightforward, risk-benefit assessments are often difficult to perform due to the varying perspectives of regulators, clinicians, payers, patients, and caregivers, to name just a few of those involved. This is particularly true in the case of rare and progressive diseases. When receiving a specific treatment may be a matter of life and death, what risks are acceptable, especially when there is no other available therapeutic option? If the FDA applies the same filters in orphan drug approval as it does for prevalent chronic diseases, it runs the risk of denying or delaying a therapeutic option for patients who may believe that, for them, the benefits outweigh the risks.
Although the FDA has been moving in a direction to include patients and advocates in their decision-making process, it is still not enough. In meeting the needs of rare disease patients, we are not talking about the difference between population health and individual health; rather, it is a question of the ability of some patient groups to willingly accept risks that might seem unacceptable to those in the general population. Patients with rare diseases are entitled to new approaches to decision making from the FDA, approaches that are as unique as their rare disease. This approach should include patient views on risk tolerance.
As a member of Global Genes corporate alliance, Connexion Healthcare has a seat at the table where these discussions are taking place.
For assistance on getting your orphan product to market, please contact our Rare Disease Center of Excellence.