Ad Comms Update: Defining Unmet Medical Need

After yesterday’s news regarding the Ad Comms for both BioMarin and Sarepta, the community continues to ask questions. PPMD has been working around the clock to get some answers and will continue to share information with you as it becomes available. One topic of discussion is the question of ‘unmet need’ – if one therapy is approved does that hurt the potential of the other therapy because there is no longer the urgency of unmet need?


It is difficult not to spend time and energy speculating and imaging various scenarios, but I think it’s important to keep in mind the facts…what we do know. In a number of rare diseases, there are multiple therapies (for instance, enzyme replacement therapies in Goucher and Pompe). And there are other cases.


One decision for a sub-population of individuals does not in any way mean that the 'need' has been met. Both drugs (and all drugs in development for Duchenne) meet the standard set for a rare disease indication. The tools of Accelerated Approval, Breakthrough Designation, etc., are just that, tools/mechanisms for approval. Each company will distinguish their drug, one from the other. We have seen this publicly with discussions around dystrophin, fiber type, safety, and efficacy.


FDA must review each application based on respective merits/data, without consideration of other products, for approval assessment. In recent competing Hep C submissions, when the first NDA got approved, the second in line did have their “breakthrough designation” revoked (since it was no longer an unmet need) but that did not influence whether the drug could be approved. 


The Definition of Unmet Medical Need:

source: Guidance for Industry (published May, 2014)


"Unmet Medical Need

An unmet medical need is a condition whose treatment or diagnosis is not addressed adequately by available therapy.   An unmet medical need includes an immediate need for a defined population (i.e., to treat a serious condition with no or limited treatment) or a longer-term need for society (e.g., to address the development of resistance to antibacterial drugs).

  1. When There Is No Therapy Available
    • If there is no available therapy for the serius condition, there is clearly an unmet medical need.
  2. When There is Available Therapy
    • When available therapy exists for a condition, a new treatment generally would be considered to address and unmet medical need if the treatment:
    • Has an effect on a serious outcome of the condition that is not known to be influenced by available therapy (e.g., progressive disability or disease progression when the available therapy has shown an effect on symptoms, but has not showed an effect on progressive disability or disease progression.
    • Has an improved effect on a serious outcome(s) of the condition compared with available therapy (e.g., superiority of the new drug to available therapy, when either used alone or in combination with available therapy (i.e., as demonstrated in an add-on study)
    • Has an effect on a serious outcome of the condition in patients who are unable to tolerate or failed to respond to available therapy
    • Can be used effectively with other critical agents that cannot be combined with available therapy
    • Provides efficacy comparable to those of available therapy, while (1) avoiding serious toxicity that occurs with available therapy, (2) avoiding less serious toxicity that is common and causes discontinuation of treatment of a serious condition or (3) reducing the potential for harmful drug interactions
    • Provides safety and efficacy comparable to those of available therapy but has a documented benefit, such as compliance, that is expected to lead to an improvement in serious outcomes
    • Addresses an emerging or anticipated public health need, such as drug shortage

In some disease settings, a drug that is not shown to provide a direct efficacy or safety advantage over available therapy may nonetheless  provide an advantage that would be of sufficient public health to qualify as meeting an unmet medical need."

I think the real issue may be downstream in terms of choice, access, and reimbursement. Issues PPMD is working tirelessly to address. Not only we will be following up with the FDA for answers on yesterday’s decision to switch the Ad Comm dates, but we are working with both BioMarin and Sarepta to help ensure success at their Advisory Committee meetings. And we are actively working on legislative and policy solutions to address the issues of access and reimbursement because we are confident that drug approvals are in the near future for this community.


The heavy lifting is just getting underway and this community must unite to ensure success. PPMD looks forward to working alongside each of you as we get one step closer to the day we end Duchenne.

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