October-November 2016:

  • Collaborate with relevant patient groups and manufacturers on identified need for a voice for the ultra rare community;
  • Develop Haystack Project, The Voice of the Ultra Rare entity and infrastructure;
  • Conduct needs and priorities assessment with interested members;
  • Develop initial goals and priorities with interested members;
  • Facilitate the consensus-building process toward a unified set of goals and priorities; and
  • Assess and communicate to members the potential trade-offs and negotiation strategies for activities identified as high priority or time-sensitive.

October–December 2016:

  • Develop advocacy strategy on the Hill and with relevant regulatory agencies;
  • Draft, or facilitate the drafting of, any white papers, position papers, talking points, and other advocacy materials;
  • Create grassroots initiatives through patient organizations, specialty societies and other relevant ultra-orphan drug stakeholders on behalf of the Haystack Project, The Voice of Ultra Rare; and
  • Develop an effective messaging strategy to promote recognition of ultra-orphan drugs and biologicals as a distinct, unique, and vulnerable subset of orphan products;
  • Explore existing incentives for R&D and commercialization of orphan products, their value, and the potential risk of their reduction, limitation, or elimination;
  • Assess the adequacy of existing incentives for ultra-orphan drugs and biologicals, and identify opportunities to “level the playing field” for individuals with ultra-rare disorders for which there are no FDA-approved treatment options;

Q1 2017:

  • Execute advocacy strategy;
  • Deploy messaging and other materials developed for the Haystack Project;
  • Initiate grassroots initiatives; and
  • Identify champions and assess opposition.

Q2 2017:

  • Assess progress toward initial goals, and evaluate emerging priorities, risks, and/or opportunities;
  • Assess and address gaps in patient advocacy partnerships; and
  • Identify risks and opportunities in upcoming CMS annual regulatory proposals, craft and execute strategic initiatives.

Q2-3 2017:

  • Continue to evaluate and pivot as socialization of a differentiated ultra-orphan reality develops and succeeds.
  • Evolve “asks” in response to emerging vehicles.
  • Kick off Hill meetings
  • Kick off stakeholder meetings

Q3 2017:

  • Website developed and launched at members’ request
  • Facebook page developed and launched at members’ request
  • Kick off of weekly calls